National Analysts

When Oncologists Look at Oncology, They See the Same Dire Shortages Year After Year

noreply@blogger.com (Anonymous) — Thu, 6 Jun 2013 15:37:00 -0400

Survey Research at the Clinical Frontlines

In August and November 2011, we wrote here about the increase in oncology drug shortages and the implications for oncology patient care. We’d been moved by alarming media reports, including a New York Times Op-ed by Ezekiel J. Emanuel, MD, PhD, to poll oncologists about their personal experiences in the January 2012 wave of our ongoing survey initiative, “Oncologists Look At Oncology".

Nearly all of the 204 oncologists (95%) who participated in our 2012 survey had seen patients in the prior year (2011) who were unable to receive timely treatment due to drug shortages, with half (49%) encountering this problem on at least a monthly basis throughout the year. A full 85% said they’d had patients who were unable to receive the best therapy at all because of drug shortages. Those data were taken to Capitol Hill by the Community Oncology Alliance as part of a broader conversation that involved Congress and FDA. Some constructive solutions were proposed by health advocates and policy-makers while essentially nothing happened. We have become a society that is extraordinarily patient with the unacceptable.

Cue 2013, and another round of surveys.

This past week, the American Society of Clinical Oncology held its annual meeting in Chicago. At that meeting, Dr. Emanuel and Dr. Keerthi Gogineni reported the results of a new survey of oncologists which they and their colleagues at the University of Pennsylvania conducted in Fall/Winter of 2012-13. Sadly, they continued to find that 92% of oncologists say that their patients’ treatment has been affected by shortages and 83% have been unable at times to prescribe standard chemotherapy.

The Deplorable ‘New Normal’ Requires New Guidelines

Oncologists feel that they lack formal guidance about how to make allocation decisions and, therefore, that they must improvise their responses when drugs are unavailable. Oncologists have substituted scarce generic medications such as leucovorin with more expensive branded options, but the implications of swapping drugs involve far more than reduced cost-effectiveness. They can lead to reduced efficacy and increased morbidity.

In an interview with CBS Radio, Dr. Gogineni commented that, “These are substitutions for which we sometimes don’t have data about how that affects the outcome for a patient.” She went on to suggest that guidelines are needed to inform and standardize the decisions oncologists make in times of scarcity.

And indeed, a disheartening number of oncologists polled in our 2012 survey were already convinced that patient outcomes have already been affected by shortages. Almost half (48%) believed they had had patients whose tumors had recurred because of drug shortages and 40% had at least one patient whom they believe had died sooner. Opinion surveys of oncologists cannot and should not carry the weight of well-designed empirical assessment of patient outcomes, but oncologists are the first to see that these shortages have consequences, and we ignore the early warnings at patients’ peril.

Safeguarding Legacy Drugs While We Still Need Them

Oncologists have always prided themselves on practicing evidence-based medicine long before “evidence-based” had become a medical mantra. We should be indignant that patients are now forced to be treated with something other than the evidence-based standard of care simply because we have not found a way to ensure the availability of the best medicines. And we should despair to think that things have gone so far that there is now a call for new guidelines to compensate for loss of old but important drugs. Our emphasis on medical discovery should remain absolute and unqualified. But there is grim irony in the implication that forward progress toward newer, more expensive therapies continues to have as an unintended consequence the loss of cheaper therapies we already know to be critically important.

Celiac Disease and Gluten Allergies: Separating the Wheat-related Illness from the Fad

noreply@blogger.com (Unknown) — Thu, 14 Feb 2013 11:28:00 -0500

The Fashionable Demonology of Gluten…

A recent article in The New York Times on “gluten allergies” – as distinct from Celiac Disease – is an interesting reminder of the way health-fashionistas can propel a powerful food fad by driving a bandwagon fueled by scant data. Just a decade ago, few people had heard the word “celiac,” and only bakers talked about gluten. Today, many shun gluten in pursuit of vague, putative health improvements, inspired by celebrities and diet missionaries who set fashion trends of all types. People are forever looking to find, and rout, the single dietary culprit that stands between them and general well-being. (If only one could so easily pinpoint the chief obstacle to health, happiness and a submissive GI tract!)

…And the Indisputable Gravity of Celiac Disease

Real or not, widespread claims of gluten allergy can look, to the unsympathetic eye, a little like Munchausen Syndrome. I would have viewed those claims as just a curious cultural trend, were it not for my professional interest in the “real deal” – Celiac Disease, informally known as celiac sprue. Celiac Disease (CeD) is an auto-immune response to gluten: at once insidious and debilitatingly symptomatic, persistent and shape-shifting, life-altering and potentially lethal. It often eludes diagnosis for years due to the astonishing variety of symptoms, from hallmark GI side effects (not always present) to diffuse neurological symptoms; from malnutrition, anemia, and osteoporosis to infertility and miscarriage. Longer-term effects include T-cell lymphoma, the sort of cancer that can spring from chronic immunologic insult. Epidemiological estimates place incidence at roughly 1%, but the condition remains significantly underdiagnosed, even though documented incidence has been rising steadily with the availability of tTG antibody testing, approved in the US a decade ago.

Emerging from the Shadows…

CeD is certainly not a new disease but the medical community has been largely disengaged because the only management is “a gluten-free diet” (GDF), which means there is little to be accomplished through systematic follow-up. Diseases amenable only to lifestyle modification put patients squarely at the controls. And because CeD patients are often highly symptomatic, they also tend to be highly motivated. Many are remarkably knowledgeable and self-directed, often more expert than their physicians. Pioneering celiac crusaders and disease advocacy groups [NFCA, CDF, and CSA] have helped make the world safer for these patients – medically and psychologically – by creating public awareness, increasing access to information, spurring diagnosis, and legitimizing what was for so long a very lonely, neglected affliction. There are some excellent CeD centers around the country but, by and large, physicians in the community are just catching up.

…and Now Catching the Eye of Pharma

Fortunately, the pharmaceutical industry has also begun to pay serious attention. A Baltimore-based company called Alba Therapeutics is partnering with Teva to develop larazotide acetate, an oral peptide that regulates tight junctions and modulates cellular permeability, reducing gluten leakage into the gut.

Alba is currently enrolling patients in its Phase IIb trial at 74 sites around the country. Larazotide acetate is not an immune-modulator; successful trials would position it as an adjunct to diet, significantly enhancing symptom control and, by implication, reducing long-term risk. These patients need an additional backstop because gluten lurks everywhere and, as we know so well from diabetes and other lifestyle-mediated diseases, self-control is a famously imperfect health strategy. Even most of the disciplined patients continue to be symptomatic.

Let’s Not Blur the Picture

This brings us back to the starting point – the probability that many people now on gluten-free diets have nothing at all wrong with them that gluten abstinence can cure, although some are CeD family members who adopt gluten-free diets in an act of solidarity. (The disease is genetic, so people with close family members who’ve received a diagnosis should be screened, even if they are not symptomatic.) Gluten sensitivity remains speculative, despite a tidbit of clinical data and some anecdotal testimony by health professionals. The question is: Does all the attention paid to this pop-culture “wannabe” ultimately help or hinder the process by which CeD, a documented and serious disease, achieves mainstream medical awareness?

One can argue the case both ways. The concept of gluten allergies has put gluten on the dietary map, as well as on most food labels. A flourishing specialized food industry, which has gotten added impetus from the GFD dietary fad, provides CeD patients convenient access to a much larger and tastier array of products. If the fad wanes, the food industry may lose some interest.

On the other hand, we don’t want our received wisdom on gluten and health to come from starlet interviews in Marie Claire. All this misdirection risks blurring the boundaries between “allergies” and true CeD in ways that discourage formal diagnosis and invite casual self-care. CeD patients will always need to be at the helm of their disease -- well-educated and well-motivated to combine serious diet with pharmacology. If ever there was a case for DTC outreach, this is certainly it.

The Annual Physical: Maligned and Misunderstood

noreply@blogger.com (Anonymous) — Thu, 7 Jun 2012 10:07:00 -0400

In The New York Times' Sunday Review, Elisabeth Rosenthal mounted an attack on the cost-effectiveness of the annual physical, a ritual whose net value has actually been covertly questioned for decades by many health professionals, but whose sanctity has now become safe to challenge in very public view. What we’re seeing today is a seismic collision between two forces: our historic belief that deluxe healthcare, with no test or procedure spared, is an inalienable right, and our urgent need to rethink the value of every entitlement -- including the rituals of care we hold most sacred.

Forget the tricky pharmacoeconomic calculus around PSA tests and mammograms. There’s a larger picture to look at. Problems that require immediate intervention tend to be more acute and symptomatic -- and those emergent problems don’t necessarily present in accordance with the arbitrary timetable by which we set our medical appointments. Annual cycles have astronomical and cultural meaning for us, but the body doesn’t pay them much heed. Even civilians can understand that. And, in fact, the so-called annual physical is a bit of a misnomer anyway. Relatively healthy people and those who are healthcare-averse have check-ups far less often.

Rosenthal’s general perspective is sound but she conflates a few issues to make periodic “well check-ups” seem even less productive, or cost-effective, than they are. Executive physicals are surely capitalism at work, but ironically, the people who seek them out tend to be a very small minority of relatively healthy individuals. And CT scans for headaches or MRIs for lower back pain don’t tend to come about as a result of annual check-ups; they’re usually precipitated by symptoms and sick visits. We may spend way too much on medical tests for symptoms that, on average, are transitory and unimportant, but that problem can be solved by smarter protocols and more disciplined, effective MD-patient interactions. We don’t need to bash the physical to reduce unnecessary testing.

Actually, most of the testing done for annual physicals is relatively inexpensive and non-invasive blood work, and there are useful things to be learned from it, even in patients who feel quite well. The PSA, which can produce false-positives, should be accompanied by a DRE to palpate the prostate so net costs need not be unduly high. And as someone who has helped bring bisphosphonates to market (but doesn’t choose to treat her mild osteopenia with any of them), I count on a DEXA scan every few years to track the success of exercise, provide a platform for discussion about supplements, and give me greater clarity on my risk profile.

Here are some other things the annual physical does:

It creates a more coherent picture of each patient for his or her primary care physician. If properly spent, time in the annual physical fosters the sort of MD-patient relationship that’s bound to produce more cost-effective healthcare -- for instance, by preventing very expensive ER visits, or by promoting lifestyle change and modifying risk profiles at earlier points.
It is probably the most reliable way to diagnose and act on hypertension in patients who are otherwise healthy, in a medical culture that has clearly documented the long-term mortality benefits of increasingly tighter BP control.
It gives physicians and patients a regular forum for discussing trends in BP, cholesterol, and blood glucose -- and for finding trigger points that persuade recalcitrant patients to embrace more aggressive pharmacologic strategies once diet and wishful thinking have truly failed.
It allows physicians to monitor the side effects associated with all those life-extending therapies they prescribe -- drugs that we all have come to see as involving complex risk-benefit trade-offs requiring more vigilant post-market surveillance.

There are lots of ways to cut medical costs but it’s not clear that the annual physical is necessarily the greatest source of leverage -- or the right sacred cow to slaughter. We need to decide how to use those precious 45 minutes in better ways, perhaps. But we ought to work harder on figuring out how to keep insured patients out of the ER for non-emergencies than keeping them out of the PCP’s office for regular check-ups. Developing improved lines of communication (at the annual physical, perhaps?) will go a long way towards producing healthier patient behaviors, less impetuous use of testing, and more effective patient-clinician collaboration in the realm of both prevention and treatment.

FDA DTC and ROI: The simmering alphabet soup of pharmaceutical consumer outreach

noreply@blogger.com (Anonymous) — Tue, 1 May 2012 13:08:00 -0400

The New FDA: champion of DTC and of survey-guided policy input

Just a few weeks ago, the FDA closed its public comments period on a questionnaire for a survey it intends to conduct later this year among health care professionals. The goal of the survey is to gather insight on how consumer-targeted promotion ultimately impacts the quality of interactions and decisions in provider offices around the country. The instrument itself is still a work in progress, and no one will be surprised to hear that our firm made a number of friendly, civic-minded suggestions. It’s clear that FDA has become a patron of survey research for many purposes -- policy development as well as risk surveillance.

The very fact that this particular survey is on the docket speaks volumes about what many FDA-watchers have already concluded: that the agency has morphed from wary and grudging to comfortable and upbeat about the benefit of allowing pharma to converse with patients. After several decades of experience, the FDA seems ready to conclude that direct-to-consumer advertising (DTC) can do more than bring relevant therapies to broader awareness. It can also, through “fair balance,” arm patients with many of the pros and cons to promote more informed conversation with caregivers. Actually, my husband (an attorney, not a citizen of our pharma community) has often expressed astonishment that any of my clients pay to run ads on TV because he is utterly convinced that they make patients run fast in the other direction. As it happens, he is mistaken.

Without much fanfare, the FDA has also been soliciting perspectives from the industry about the impact of DTC advertising. But, as I noted earlier this year (in presenting some data at the Pharma Market Research Conference in Parsippany, NJ), the methods by which pharmaceutical companies set about measuring ROI have not been conducive to a rigorous assessment of DTC’s larger health impact -- and often not even conducive to a calculation of financial impact. Much of what we know is best described as statistical anecdote. It’s ad hoc, impressionistic, and circumstantial -- frequently conducted more in hopes of proving that DTC is a good investment rather than with intent to document the kinds of conversations and outcomes it stimulates.

For that reason, we believe the FDA is on the right track when it aims to have providers weigh in on whether DTC is advancing the cause of health care or creating obstacles. We also anticipate the FDA survey will ultimately support the conclusion that, more often than not, DTC (a) contributes to improved conversations and (b) probably promotes patient adherence and persistence more than it undermines it. I could say cynically that these FDA survey results are so far in the future that no one will ever recall this prediction if I happen to be wrong, but actually, we have some research evidence of our own to support the case. We’ll be sharing it in future blogs.

How much information is too much?

The FDA is also looking to chart the outer bounds of what consumers can effectively handle with respect to drug information and what can be done to make it easier for them to pull all the elements together in a coherent risk-benefit story. On that score, here’s another prediction, although the endpoints and the proof-points are far more elusive. The FDA will find that, beyond a certain point, more is not more. It doesn’t necessarily matter how it’s framed, how big the print, or even whether someone manages to translate esoteric side effects and statistical probabilities into fifth grade level. It’s never possible to supply complete context to a lay person (whatever the topic), so more information inevitably begs more questions. The goal of fair balance and full disclosure -- even brief summary -- is to arm patients with the right questions and prepare them to assimilate information from their providers, not to be autonomous decision-makers. Even in a clinical democracy, that level of self-determination is unrealistic.

Today’s stilted DTC monologue in the parlor versus tomorrow’s cross-talk in the great outdoors

The most important question the FDA is mulling is how social media ultimately fits within the broader information eco-system. The agency has been exposed to testimony that argues for the enormous importance of the web but has coyly left pharmaceutical companies to blaze that trail with no guidance other than conscience and broad regulatory principles. It’s easy to be sympathetic to both sides: pharmaceutical companies, who are loathe to pioneer, and regulators, who would rather take a reactive approach to oversight, learning empirically by watching companies muddle through.

Ultimately, though, if we read between the lines or squint at the horizon, we have to see that the FDA will soon expect a significant amount of interaction between patients and manufacturers in the open spaces of the worldwide web. The web is now the most important source of information about nearly every single thing we care to know about, so consumers need to be directed to well-curated company websites that are organized to deliver accurate, accessible, and meaningful information on all drugs, for those who seek it out.

Meeting full disclosure obligations by sending consumers from TV commercials to print ads in narrowly targeted publications doesn’t do the trick. If pharmaceutical companies are going to speak to consumers and patients, they will need to do it in the place more of them hang out, not in cubbyholes like Golf Digest.

Pharma-sponsored web sites must not be static and impermeable but neither can they follow the current social media model. Pharma companies need literally to invent a way of interacting with the patients they serve. The FDA is neither prepared nor equipped to do it for them. But we think they very much want it to happen.

Fitness versus Health: Marathons Test Not Just Endurance but Logic Too

noreply@blogger.com (Anonymous) — Wed, 28 Mar 2012 15:02:00 -0400

Safer on the sidelines?

The mortality toll of the 2011 Philadelphia Marathon was two runners out of approximately 25,000, a death rate that proponents of the event might represent as less hazardous than the risk of commuting the distance by car. And probably much safer than couch-bound obesity. Even when we take into account the many people who require first aid for dehydration or suffer orthopedic injuries, the hazards of marathon participation might strike many as relatively low. Life is risky business, after all, and testing human limits -- for pleasure or heroism -- often comes at a price.

Nonetheless, it is curious to observe how sanguine health experts and the public have become about tests of endurance that can result in needless death, and how little discussion we hear in the aftermath about the wisdom or health value of marathon participation. That’s because marathons -- once considered an “extreme sport” for the very few -- are now an icon of our fitness culture, attracting virtual hordes of runners in cities across the country, more and more of whom run the full distance each year.

Marathons are a spectator sport and a badge of fitness honor -- not to mention a source of mood elevation for those addicted to long-distance running. And for the cities that host them, they are also very much a business that creates dollars, press, and caché. When endurance events like marathons and triathlons are promoted by health celebrities and fitness missionaries like CNN’s Sanjay Gupta, they gain more credibility and many more converts.

Maybe not born to run long distance

However, the case for long-distance runs is not nearly so easy to make as, say, brisk walking, which is now widely acknowledged to be ideal exercise, beneficial even in relatively small doses. Running is punishing to the musculoskeletal system, and as we learn more about the “science” of running and athletic apparel, we are discovering that our expensive, high-tech footwear may be doing us more harm than good. The miles we run as a culture add up to a lot of chronic injury at a lot of medical expense and pain. And after every marathon, we are confronted with the logical implications of pushing anything to the limit. Such as when a 26-mile run unmasks fatal cardiovascular anomalies in a few apparently healthy people, or stresses normal kidneys beyond their limits. Short runs are healthier, plain and simple. Even the famous runner at Marathon who started it all fell dead on the ground when he reached Athens.

The modern paradox: marathons and morbid obesity

In a society of extremes, simultaneously plagued by obesity and infatuated with fitness, we have to ask ourselves whether we are doing our culture something of a disservice by conflating fitness with basic health. Fitness is big business and it has grown increasingly powerful as a cultural idea since the President’s Council on Fitness, Sports and Nutrition waged the first campaign to get us Baby Boomers out of our school desk chairs in the late 50s. (Little did anyone know how much we’d ultimately need the help!) But fitness is a cultural term owned by many people whose health credentials are not always clear, and by enterprises whose motives are based as much on commerce as science.

Although the fitness strivers among us are secretly hoping we can out-run death at the gym or track, we need to acknowledge that the correlation between this ill-defined thing called fitness and conventional health measures is actually ambiguous. Even statistics that vault the low death rate for marathon runners fail to note that the relevant comparison of their own risk of dying on another more leisurely day is roughly seven times lower.

Clearly, it’s important to be active and avoid excessive weight gain. Physical activity delivers significant health dividends and social ones as well. But the relentless pursuit of fitness -- particularly compulsive, punishing demonstrations of endurance -- is something the medical community might legitimately challenge among that fit minority of their patients who seem bent on running marathons.

Presumably, there is little motivation to do that because if you play the numbers, more people will die this year of lifelong inactivity than long-distance running. But maybe if we de-glorified marathons a little, and we adulated shorter walking events, then getting to the last mile-post would be easier for those among us who must set more realistic aspirations, and public health would be better served.

Oncologists Answer the Question: What is an Added Month of Life Worth?

noreply@blogger.com (Debbie Kossman) — Thu, 15 Mar 2012 10:27:00 -0400

We recently surveyed U.S. oncologists in order to gain their perspectives on the emerging changes and challenges in the cancer landscape. The survey results spotlight an increasing tension between oncologists’ overwhelming optimism about the state of advancing science and their deep pessimism about the affordability of care.

One particular line of survey questioning addressed oncologists’ views of the value of incremental survival in the context of advanced cancer. We asked a series of questions designed to assess whether oncologists believe a new therapy that improves survival would be “worth it” under a variety of different contextual scenarios. Holding as a constant the assumption that the baseline price of the next best alternative care would total $15,000, we systematically varied: (1) expected survival time with the next best alternative care (either two months or nine months); (2) incremental survival with the new therapy (from one month to 12 months); and (3) the total cost of the new therapy (from $40,000 to $120,000 -- in other words, an increase in $25,000 to $105,000 over the cost of the next best alternative).

There are two key findings from those questions. First, in order for a majority of oncologists to declare the new therapy “worth it” even at the lowest of the prices we tested, the therapy must deliver a median of three additional months of life. Second, for the majority to judge those three additional months of survival an acceptable value, the cost of each additional month must be no more than roughly $9,000 to $10,000, regardless of whether baseline survival is two months or nine months.

The price-value yardstick that oncologists are using seems to have been appreciated by Roche when it set Zelboraf’s (vemurafenib) price at $9,400 per month. In clinical trials, Zelboraf extended overall survival from about six to 10 months to about 16 months in the approximately 50 percent of metastatic melanoma patients whose tumors are positive for the BRAF V600E gene mutation. Zelboraf was introduced with a companion diagnostic to identify tumors with the relevant mutation. The cost for a patient who takes Zelboraf for six months is $56,400.

After 13 years with no new drug approvals for melanoma, 2011 saw the introduction not just of Zelboraf but also of Bristol-Myers Squibb’s Yervoy (ipilimumab), a targeted immunotherapy that has improved overall survival from 6.4 to 10.1 months in late-stage melanoma. The approved treatment course for Yervoy is one infusion every three weeks for a total of four infusions; the total cost is $120,000.

On its face, Yervoy’s price-value -- four months of incremental survival at a cost of $30,000 per month -- seems to falls short of what oncologists require in order to say that the incremental survival is worth the added cost. But the calculus in this case is not simply a matter of averages. A subset of patients -- about 11 percent -- had striking and potentially longer-lasting responses to Yervoy, although it is not yet possible to identify these patients in advance. A drug that benefits fewer patients for a potentially longer period of time might be able to command a higher price than a drug that works modestly well for many people.

We as a society are in the process of arriving at a framework for thinking about the value of life extension at a time when the model for cancer therapy is slowly shifting away from the mass market to the niche. The economics of these drugs are feasible only if society bears higher costs for a smaller subset of patients because manufacturers have to be able to reap the rewards of developing drugs that are, in some sense, personalized. And this economic perspective seems entirely consonant with our intuitive value system that recognizes that adding a year of life (rather than just a few months) is adding quality, not just quantity. In effect, the scale is not linear.

This is the new challenge of 21st century cancer care. It will be interesting to see how professional and public perceptions of price-value evolve as newer drugs bend the framework. With our survey on oncologists, we have looked at provider perspectives. In the future, we’ll discuss the perspectives of patients and payers on what will be one of most important commercial and ethical questions of the next two decades in medicine.

A Survey of Battlefields in the War on Cancer: Oncologists Reporting from the Front Lines

noreply@blogger.com (Debbie Kossman) — Tue, 31 Jan 2012 17:03:00 -0500

In 2008, we launched an oncology market surveillance program, “Oncologists Look at Oncology: Prognosis for U.S. Cancer Care.” We were moved to do this by what we were hearing from oncologists as we conducted in-depth interviews with them on behalf of our clients. Our clients in the field, who range from small startups to leading “Big Pharma” companies, shepherd innovative molecules from the bench to the clinic and, if successful, to the commercial marketplace. But over 35 years after the “War on Cancer” was famously declared by President Nixon in 1971, what we were hearing from oncologists on the front lines of cancer care was a sense of frustration, even despair.

A significant part of oncologists’ frustration at the time was financial. In 2006, Medicare and Medicaid reimbursement to physicians for in-office intravenous cancer medications was cut dramatically. Private practice oncologists had been earning 50 percent or more of their total personal income from profits on IV medication -- profits earned directly from reimbursement paid by public and private payers. The industry published fictional “average wholesale prices” (AWP) that set the reimbursement physicians would receive, but physicians were able to purchase drugs at prices substantially below AWP. Centers for Medicare and Medicaid Services’ updated reimbursement policy based on actual average selling prices rationalized reimbursement but changed the game for oncologists.

But loss of personal income wasn’t the only thing that seemed to have disheartened oncologists. They told us that they were sending more patients to hospitals for drug administration when coverage was uncertain -- even though patients generally feel more comfortable receiving treatment in familiar, less institutional treatment settings -- because private practices could no longer absorb unreimbursed drug costs. They told us that they were unable to treat patients with the best available care because of patients’ inability to afford requisite out-of-pocket co-payments. They told us that they did not see how providers or patients or payers could keep pace with the escalating costs of new cancer therapies. They told us that their colleagues were retiring and their children were pursuing specialties other than oncology because both financial compensation and lifestyle were better in procedure-oriented fields.

We decided that it was important to check in with oncologists periodically about their perspectives on cancer care, and to put some statistics around the sort of anecdotal laments we hear on a daily basis. Today, oncologists are very optimistic about our growing understanding of the biology of cancer and the ability of novel therapies to lengthen and improve quality of life in patients with cancer diagnoses. That’s good news. But oncologists doubt that we will be able -- individually or collectively -- to afford the innovations, and they also doubt there will be enough oncologists to care for patients.

In “The Emperor of All Maladies: A Biography of Cancer,” Pulitzer prize-winning author Siddhartha Mukherjee has written a powerful history of “the men and women who have waged a battle against cancer for four thousand years” and he describes the book as “a military history.” The rising cost of care and our strained capacity to pay for it has put us on the horns of a dilemma. If that dilemma does not oblige us to accept defeat in that war, it will force us to redefine the meaning of victory -- not just how long patients live, but also how appropriately and expensively they are cared for until they die.

Over the coming weeks, we will be discussing our survey data in greater detail and sharing how physicians and cancer patients are likely to rescale the terms of success in these best and worst times of scientific abundance and financial scarcity.

“Oncologists Look at Oncology: Prognosis for U.S. Cancer Care,” is an oncology market surveillance program launched by National Analysts Worldwide in 2008; each wave surveys 200 U.S. oncologists recruited from the Epocrates physician web panel.

So What if Statins Increase Diabetes? The Real Risk May be Patient Non-Adherence

noreply@blogger.com (Debbie Kossman) — Mon, 23 Jan 2012 12:27:00 -0500

Just when we thought we had clarity on statin safety …

Last week’s leading health headline was a startling new finding that statin use increases the risk for diabetes mellitus (DM) in post-menopausal women by 50 percent, from six percent to nine percent.

While the data source -- the Women’s Health Initiative -- is an observational rather than a prospective study, the finding persists in a variety of patient subgroups, and even after the authors adjusted for baseline patient characteristics. This makes it unlikely that the increase in DM rate is due to women’s pre-statin risks of developing DM. Since diabetes is probably the single most important cardiac risk factor -- often considered the equivalent of a first MI in certain predictive models -- this news was both unsettling and perplexing. How might physicians and their patients interpret and act on this new information?

We were curious, so last week we conducted a small survey of full-time, office-based U.S. primary care physicians (PCPs) from the Epocrates physician panel* to test our hunches. Seven out of 10 PCPs had heard the recent mass media news coverage and, as a point of interest, two-thirds of them found the coverage “sensational” rather than “balanced/informative” -- despite the fact that clinicians interviewed on NBC Nightly News emphasized that they expected to keep their patients on statins.

Certainly, the news got patients’ attention. As of our survey date, at least one-third of PCP offices had received inquiries from statin patients. Balanced coverage or not, this sort of news event raises alarms, and it’s impossible for patients to draw implications on their own. What should patients, in fact, make of it?

Actually, nothing's really changed ...

On average, a post-menopausal woman with elevated lipids is still better off taking a statin than not taking one. The authors of the study concluded that current ADA guidelines should not change because “statins significantly benefit vascular and secondary prevention … in diabetic populations with rates comparable with those without DM.” The conclusion remains: Net morbidity and mortality decline on a statin despite a 50 percent increase in diabetes diagnoses among a key population of statin users. We’re left to do the forensics on how and why statins raise blood glucose in some patients, but there is no urgent need to reassess the risk-benefits.

Presumably, many or most of the patients who made calls were encouraged to stay on statins. But many patients didn’t call, and even those reassured by their physicians, now or in the future, have one more concern nagging at them when they consider just how adherent they want to be with statin therapy.

What physicians are actually worried about

Our survey suggests that primary care physicians are worried less about these new findings than they are about how patients will react, since physicians have always struggled with the challenge of enforcing statin compliance. Six in 10 surveyed PCPs expressed concern that news of the results will erode patient compliance with statin therapy, and one-third anticipate difficulty keeping some patients on their statin medication altogether. In fact, some PCPs predict that defections in their practice may number in the hundreds. If true, this finding may have set cardiovascular prevention back a few paces, just when we might have imagined that statin phobia was a dwindling issue.

Keeping it real

So what’s to be done? Physicians will need to stick to the evidence and stick to their guns when patients challenge the safety and relevance of statin therapy. But now more than ever, they will also need to remind patients that statins can’t be seen as license to eat what they want, absolving them of all responsibility for self-discipline.

We’re also curious about another aspect of last week’s media flurry. Coverage of the study results was much more prominent on television than in print media. Morning shows and evening news coverage was full of discussions by anchors and physicians. On the other hand, The New York Times made no mention of the study at all, as far as we can find. It leads us to speculate that medical journalists who scan for new data concluded that attention to the study wasn’t worth the fuss. It also suggests that what physicians found so sensational about last week’s coverage may have been simply seeing it on the six o’clock news. TV seems more sensational than print, no matter how circumspect the coverage.

In any case, the newest study findings are one more reminder that drugs are complex tools with plenty of unanticipated effects (as if we needed it that reminder). And the statin link with diabetes is yet another item to add to the long list of issues that require open, fruitful dialogue between patients and physicians about the risk-benefits of any therapy. The good news here is that the big picture evidence is still clear on statins. Physicians remain fully convinced. Now, they just need to keep an eye out for waning conviction among their patients.

If you would like to receive a copy of the complete survey data, leave a comment below or email us for more information.

*Epocrates Honors Market Research program has the largest opted-in, verified physician panel in the industry with over 225,000 U.S.-based physicians, as well as 885,000 allied healthcare professionals.

The Obesity Epidemic: When Biology and Culture Conspire Against Us

noreply@blogger.com (Anonymous) — Fri, 6 Jan 2012 09:41:00 -0500

What We Eat or What We Are?

Tara Parker-Pope’s recent cover story in The New York Times Magazine, “The Fat Trap: Do You Have to be Superhuman to Lose Weight?” describes what is fast becoming canon: that modern culture has unmasked in our evolutionary biology a genetic propensity to defend against scarcity and starvation by retaining excess fat.

Research that documents weight plateaus for dieters, and biological differences between obese subjects and others is allowing us to assemble a picture of the human animal as metabolically hard-wired to conserve or regain weight -- especially after it has been reprogrammed by periods of obesity. Research also lends strong support to genetic differences in the degree to which individuals may be prone to obesity based on innate metabolism, food addictions, or other epigenetic factors.

Many frustrated dieters will find redemption in that thesis -- as, clearly, does Parker-Pope, whose article is unusually solipsistic for a card-carrying science writer. It is really a lamentation … which just goes to show how intractable and all-consuming this problem truly is for those of us who live in societies of plenty.

Dim Prospects for Cure

It turns out that when culture and biology team up, they have the potential to do great mischief together. Modern obesity and modern terrorism both demonstrate how aspects of our basic nature can be potentiated by forces of culture -- including both the Big Mac and the Internet -- to produce negative outcomes.

Under the broad heading of “lifestyle,” we see the simultaneous effects of many phenomena that all work to pack on the pounds: transportation and technology that keep us sedentary; an oversupply of cheap, addictive calories; and permissive attitudes concerning where, when, and how we eat. Among other factors, less often discussed but also quite relevant, are the impulse to use high-calorie food as compensation for social deprivations or economic inequities, and the peculiar elevation of food imagery in Western society to something very nearly pornographic. It turns out that the biochemistry of obesity is complex and still mysterious. Culture is too.

Unfortunately, we do not appear to be having much success manipulating biology right now, and future prospects seem no more promising. The pharmaceutical industry would love to see obesity addressed with a chronic therapy model -- and many patients probably would too -- but drugs have been remarkably unsuccessful in achieving safe, significant, and sustained weight loss, and the complexity of the problem (from a genetic or biochemical perspective) makes a magic bullet mechanism unlikely. In fact, it may be more plausible to look for ways to get dieters over the humps and hurdles intermittently than to medicate them chronically against the return of obesity.

Bariatric surgery is often offered up as a more effective alternative, and recent data suggest positive CV outcomes. But in a society where obesity is already commonplace, it seems reckless to endorse an approach that might make gastric bypass as ubiquitous as tonsillectomies used to be.

Fighting the Culture Wars

That leaves us with culture modification and behavior change -- an equally tall order but one with so many different levers to pull that the potential for meaningful effects might actually exceed the benefits of drug chemistry. The relative success of commercial programs like Weight Watchers suggests that dietary strategies may come closest to cracking the code when they parlay basic portion control, social reinforcement, and structured behavior in equal measure. This all passes for common sense, but in order for more of us to achieve greater mastery over our environment and our metabolism, we need some important things to happen:

As a society and as individuals, we have to acknowledge that, whatever the causes, obesity is a preventable illness, not a preordained destiny, and that it ultimately reflects an imbalance between calorie supply and demand.
We have to recognize that childhood obesity is exceptionally dangerous because, like certain kinds of drug exposure, it may reset biochemical mechanisms and create metabolic memories that are very hard to erase.
As a result, we need to take a much more aggressive approach to nutritional intervention in schools -- woefully difficult at a time when we struggle to teach children to read, much less to eat.
We have to give it even higher priority as a health goal and look to promote more frequent (often uncomfortable) conversations between physicians and their patients on the subject of weight -- not fewer.
Even acknowledging that the genetics of obesity are interesting and worthy of study, we need to dedicate adequate resources to studying what kinds of behavioral manipulations work best.
We need to direct an even greater amount of research toward nutrition and the way various foods tend to stoke or suppress the metabolism.
Armed with that kind of insight, we need to foster an approach that treats behavior modification programs as a reimbursable therapy, not just an option for motivated patients.

As to articles like Parker-Pope’s, people who struggle with weight should be treated compassionately and respectfully but it’s important not to spend too much time in the comforting arms of biological self-justification. We need to move on from there.

Updated Jan. 9, 2012

Plan B and Public Health: Morning-After Problems We Can’t Easily Fix

noreply@blogger.com (Anonymous) — Tue, 20 Dec 2011 09:19:00 -0500

Today I signed a petition to protest Kathleen Sebelius’ decision to override the recommendations of the FDA and the American Academy of Pediatrics by banning OTC availability of Teva’s Plan B morning-after contraceptive. I say that without intent to be provocative. I simply want to acknowledge right up front that I have no moral compunction about morning-after contraception -- although I tend, dispositionally, to prefer a pound of prevention to even an ounce of cure -- and I certainly don’t believe in subjugating science to religious principles. I was surprised at the Administration’s decision because it most assuredly wasn’t in the best interest of public health -- but neither do I know how to chalk up the political wins and losses on this for Obama, who was clearly willing to ignite a controlled burn on his left in order to avoid a firestorm on his right.

The petition I signed enjoined the President “not to put politics above science” -- a credo that sounds like the right slogan for the professional (and personal) world I inhabit. Still, I can’t help but note the irony that since all regulation of public health is inherently political, this distinction is forced and artificial. The FDA came down on what I happen to think is the right side of this issue -- public health and equal protection for those young teens who have, as a practical matter, limited access to all health resources. But the FDA, like all agencies charged with health policy oversight, is an instrument of politics. Public health is just a euphemism for politics. In fact, the whole public health sphere is becoming a house built by government and merely furnished by science.

I’m reminded of that fact when a client gets a thumbs-down decision on an NSAID because the FDA has decided to bar the gates to new branded (read expensive) NSAIDs that aren’t superior to what’s currently available.

I’m reminded of that fact whenever the FDA sets guidelines for how much life extension is required to justify a very costly drug. Or when I consider that routine PSA screening is now no longer deemed by some health authorities to be cost-effective, a term now used with more sanctimony and intellectual conviction than it probably deserves.

And I’m reminded of how health and politics cross-pollenate in even odder ways every time municipalities like the city of Philadelphia feel empowered to reach over the line and ban trans-fats, when personally I think they have better, healthier things to do. (Fix a playground or something.)

Let’s face it: religion is politics and, increasingly, health is politics. This problem is simply going to get worse as growing need and strained resources put law-makers in charge of health decisions. The rational among us had better get busy and work both sides of that equation in order to ensure that reasonable decisions get made.

Universal Health Insurance Coverage -- Really?

noreply@blogger.com (Debbie Kossman) — Thu, 15 Dec 2011 16:17:00 -0500

"Universal" vs. "Coverage"

Yesterday, CNN’s Tim Langmaid reported that Health and Human Services estimates that the percent of insured young people aged 19 to 25 rose from 64 percent to 73 percent. Based on census figures, that percentage change represents about 2.5 million young people. The increase is attributed by some to a provision of the health care reform legislation signed into law last year that allows young adults to remain on their parents’ insurance plans through age 26.

Today, however, Sara Rosenbaum writes in The New England Journal of Medicine about the Douglas cases for which the Supreme Court heard oral arguments in October. These consolidated cases are comprised of suits against California Medicaid for cutting reimbursement rates to the degree that, plaintiffs argued, beneficiaries’ access to care was seriously threatened.

The question before the Supreme Court is all about legal standing in the shadowy ground between federal legislative authority and the role of the states. Does the U.S. Constitution give private individuals the right to go to court to halt state actions that violate federal law and threaten immediate and irreparable harm? This will be a key issue in the years ahead as the federal government and the states get set to squabble over who gets the last legislative word in the realm of public health and safety.

The question for the rest of us is more pedestrian, but just as important. What is the value of universal health insurance if coverage is increasingly limited, in essence, a safety net full of holes?

Life in a leaky tent

An increasing number of physicians we speak to are telling us that patients -- even patients with private, employer-sponsored health insurance -- are not able to receive treatment because co-payment responsibilities exceed patients’ ability to pay.

An example? Our industry news in recent months has heralded the arrival of protease inhibitors against hepatitis C and their ability to increase sustained responses -- presumptive cures -- in more patients. But some patients -- employed, insured patients -- go untreated. Our therapeutic innovation is remarkable, but it is an option only for those with health insurance coverage. Increasingly that coverage is shot through with holes that threaten to make the concept of universal care illusive and theoretical.

Like many shared sacrifices of modern economic times, the burden associated with the paradox of more inclusive coverage that affords thinner protection will be felt by both the poor and the middle class. Over the next few months, we will be writing again about the clinical implications that tougher choices pose for patients and the providers who treat them.

Earlier Warnings of Cancer Drug Shortages? Alarm Bells Should Have Sounded 50 Years Ago

noreply@blogger.com (Debbie Kossman) — Wed, 2 Nov 2011 15:51:00 -0400

Right now in the US, over 40 million people are 65 years of age or older. And today -- right now -- we cannot meet the cancer drug demands of this age group. We cannot meet the demand for the wave of very expensive, new branded medications because the cost of these agents prohibits use for many patients. We cannot meet the demand for much cheaper generic cancer medications because -- among other reasons, some of which I addressed in a previous post about generic oncology drug shortages -- manufacturers do not get the financial returns to remain committed to continued supply.

To try to begin to remedy generic drug shortages that have already delayed care for many patients -- indeed, may already have led to preventable deaths -- President Obama signed an executive order earlier this week, directing the Food and Drug Administration to take action to reduce prescription drug shortages. The order instructs the FDA to do three things: broaden reporting of potential shortages of certain prescription drugs; speed reviews of applications to begin or alter production of these drugs; and provide more information to the Justice Department about possible instances of collusion or price gouging.

But none of those actions will increase incentives for manufacturers to make adequate supplies of generic cancer therapies. And none of those things will alter the fact that by 2030, the number of people ages 65 and older in the U.S. will have increased to over 70 million people. By simple math we have every reason to believe that in the next 20 years, demand for generic cancer medication such as doxorubicin and leucovorin -- mainstays of therapy for breast and colorectal cancer respectively, and drugs particularly hard-hit by current shortages -- will increase by at least the same proportion as the over-65 population. Cancer is largely a disease of aging, and the aging population is growing in this country. Neither of these statistics is news.

President Obama has called drug shortages “one of those slow-rolling problems that could end up resulting in disaster for patients and health care facilities all over the country.” One might say that the aging of the U.S. population is also a slow-rolling problem. Yet we have known for decades that demand for products that treat diseases of aging would explode. And still -- with all these years of advance warning -- we are unprepared. Why would we think that an executive order that requires drug manufacturers to report impending shortages will have a meaningful impact when changing demographics -- one of the most reliable indicators of future demand that can be found -- has not yet motivated us to prepare?

As we've discussed previously, this problem of generic drug shortages requires us to think harder about how to align the commercial vitality of our healthcare industry with the health needs of our society. That problem is a thorny one -- not just because it is intrinsically challenging to solve, but also because we seem unmotivated to solve it. In this matter as in others, we’ve been inclined to treat future as mere possibility -- a bridge to be crossed if and when we come to it. It is easier to deny and temporize than to tackle tough issues if the consequences are not literally upon us. Well, clearly they are upon us now.

Just this Sunday, The New York Times issued the chilling assessment that we are every bit as unprepared for an anthrax attack as the day it first crossed our radar screen, even though prudence would dictate that we take readiness more seriously. In matters of public health, as in every other aspect of public policy, we need to cultivate a keener future orientation.

Soda and Teen Violence: Hazards of Correlational Analysis Can Put Truth in Harm’s Way

noreply@blogger.com (Anonymous) — Tue, 1 Nov 2011 16:17:00 -0400

Statistical Ammo for the Twinkie Offense: Guns Don’t Kill People, Soda Kills People?

No one with an interest in public health and a zest for social engineering can go wrong by criticizing soda. That particular cup runneth over with empty calories and plausible links to obesity, as well as tooth decay. The carbonated beverage industry is fighting a pitched battle in municipalities across the US to avoid punitive taxation meant to price soda beyond the reach of citizens who seem most at risk of making poor nutritional choices -- choices that ultimately all of us must pay for. No matter what you think of coercive taxation, it’s really hard to defend soda.

Now, though, thanks to research published by two Harvard-trained researchers, Sara Solnick of University of Vermont and David Hemenway of the Harvard School of Public Health, soda has come under another sort of fire.

An analysis performed on the Boston Youth Survey database shows a significant correlation between soda consumption and violence in teens, including a propensity to carry guns and exhibit violent behavior with partners. The paper, which appeared in the Journal of Injury Prevention, got a two-day flurry of press attention because it has a provocative thesis that keyed off the famous “Twinkie Defense.” It gives everyone even more reason to worry about what dangers may lurk in our refrigerators.

Or does it?

Causality and Correlations

Notably, this kind of ad hoc, ad hominem statistical analysis is becoming increasingly commonplace as researchers mine survey and diary data for links between diet and just about everything, hoping to demonstrate in many ways that we are what we eat. But when hunt-and-peck analysis is behind so much nutritional science, it’s important to ask: Are all these correlations worthy of media coverage -- sensational or sober? And although they’re always good press, are they good science?

Scientists and social scientists are trained to recognize that association is not the same as causality. Still, researchers who scavenge databases for statistical links sometimes issue that caveat about causality in an almost pro forma way, then go on to offer up imaginative speculation about what causal relationships there may be.

In this case, Solnick and Hemenway suggest the possibility that all that sugar might be crowding out other nutrients (a concept whose underlying science is not actually clear), as well as the possibility that the stimulating effects of caffeine may be the root cause of the problem. As it happens, the researchers are handicapped in speculating about which of these two is more plausible because the Boston Youth Survey database does not include information on the type of soda consumed (e.g., diet vs. regular; caffeine-free vs. caffeinated). Not a minor detail.

Even more important, they have little in the data to discipline their speculation because the survey database was merely mined, not designed, by these two researchers to test their own hypotheses, and thus, it cannot speak to many other relevant considerations that might potentially explain the statistical link. All these researchers had available to them were socio-economic variables and information about whether the teens ate meals with their parents, a handy but limited surrogate for parental oversight and family functionality or social integration.

Demon Coke: Sensationalism Over Plausibility

In the hard sciences, investigations are designed and evaluated on the basis of pre hoc hypotheses, so as to discourage “shopping” for statistical effects. In the social sciences, analysis of large survey databases allows researchers to browse for findings of interest while often leaving them ill-equipped to test more rigorously for cause and effect. The ability to do that places added onus on researchers to develop more stringent, science-based criteria before elevating correlations to the status of meaningful findings.

Much as I personally dislike soda and wish that so many Americans did not drink themselves fat, it is only one of many sources of excess carbohydrates in our diet and should not be demonized quite so recklessly. Under the heading of cause and effect, let’s agree that it’s much safer to have science -- real science -- drive politics than the reverse.

Statistics and the Search for Truth

Speaking more broadly, however, researchers who discover correlational links between foods or environmental phenomena and health effects should be more cautious and more disciplined in their rush for publicity. Researchers are under the obligation to look harder for logical explanations that square with known science before waving correlations at us in the literature and the press. They actually need to represent ad hoc correlations as something closer to hypothesis than fact.

The same argument can be made on the other side of the ledger, when researchers identify strong associations between healthy lifestyle behaviors and desirable outcomes. For instance, a recent University of Aberdeen study identified a strong negative correlation between self-reports of apple consumption during pregnancy and asthma in children. It’s actually difficult to trace a logical connection between the two -- something the authors freely acknowledge -- but they argue that it is, at very least, benign to endorse apple consumption in pregnancy, even if the finding is coincidental.

Here is a more cynical take. When a study uncovers only two (negative) correlations (the other was fish consumption and infant eczema), you can bet those findings will be published.

Admittedly, our ability to detect events sometimes gets ahead of our ability to explain them. Quantum physics is a perfect example -- we don’t discard findings because they violate current cosmologies. But nutritional science is not astrophysics. It is played out in the public arena on a daily basis, and people are prone to run with half-baked ideas in the world they wish desperately to control and understand: their own health.

With all of these quasi-findings given legitimacy in the scientific press, it’s no wonder that the public is often so distrustful of scientific data -- and so quick to propagate their own principles of magical causation. We’ve taught them how.

Challenging Bioethics of Organ Transplantation: ‘The Book of Jobs’ Raises New Moral Lessons

noreply@blogger.com (Anonymous) — Fri, 14 Oct 2011 16:52:00 -0400

Gods of Technology Burden Us with Tough Moral Questions

In an uncharacteristic shift from the remote third-person voice of our typical posts, I want to begin by saying that my husband strongly advised me not to write this particular blog. Observing that Steve Jobs was being lionized, almost deified, by the press and public, he warned that no good could come of advancing the view that Steve Jobs’ transplant two and a half years ago, presumably after a Whipple resection, might potentially be a waste of a perfectly good liver.

Which is why, as I type this, I am fingering my adored iPhone (which I actually consider to be a vital organ of my own) like a protective talisman. At least I’m not writing on an iPad.

Even in a society that reveres digital technology, it is rather surprising how delicately, almost timidly, the entire topic has been handled in the press, and how little editorial cross-talk there has been among bio-ethicists, medical professionals, and cultural commentators. There is clearly fear of inviting discussion about the medical or social merits of the case for transplant when the patient is one of society’s most important members as well as one of its most wealthy. In a Medscape commentary from the Cleveland Clinic, John Marshall used the topic as an opportunity to laud our growing ability to differentiate among different kinds of tumors, consigning the transplantation debate to a small parenthesis.

Transplantation, though life-saving in many circumstances, is clearly fraught with practical and ethical issues – most especially, the appropriate role of market forces in rationalizing distribution of scarce resources, and the complex social value calculus associated with selecting deserving recipients. Transplantation is a cumbersome, limited solution to grave medical problems. Thus, finding alternatives to conventional whole organ transplantation, such as promising new methods of pancreatic cancer treatment, remains a crucial and challenging frontier in medicine. Unfortunately, it is not one medical frontier but many: multiple diseases for which other curative approaches would be preferable to organ transplant, and multiple approaches to improved organ viability and transplantation success. And on the ethical side, we are still struggling to figure out who goes to the head of the line, among other equally tough questions.

A Parable of Two Innovators

Circumstances surrounding Jobs’ liver transplant and what he had to do to get one are still veiled by deft PR and legitimate patient privacy considerations. The spotlight has remained firmly fixed on Jobs’ commendable efforts to help rebalance supply and demand by calling vivid attention to the need for organs and lobbying successfully to add a donation question to the CA driver’s license application, as some other states have done. What is not happening – or, at least, not happening broadly enough and loudly enough – is the open, awkward scrutiny of transplant ethics and economics that this situation invites. Presumably, no one wants to have to state explicitly that it was more important to give Jobs a few more years of life so that he could steward the development of iPhone four and a half than to extend – potentially even save – the life of someone else. We all revere our sacred digital devices.

Meanwhile, in a parallel news story one could hardly have scripted, Ralph Steinman died during the same week of a different type of pancreatic malignancy, just days before winning the Nobel Prize (posthumously) for ground-breaking work in cancer immunology. His achievement – the discovery of dendritic cells – led to development of the first commercially available cancer vaccine, Provenge, which came to market just last year. Steinman used his intellectual resources in pursuit of personal life extension and medical advance by treating himself with his own experimental technology. Of course, his particular malignancy (a more pedestrian and more lethal form of pancreatic cancer than the type Steve Jobs had), would not have qualified him for any form of transplant – even if we admired the heroes of biomedical research half as much as we idolize wizards of technology. Jobs had a much slower-growing neuroendocrine tumor. Still, there’s no escaping the irony. And the poignancy.

Honoring Steve Jobs by Shouldering the Burdens of Innovation

We need to see the "Story of Jobs," a man who accepted no limits, as an invitation to think and talk openly about transplantation: what’s wrong with the medical technology, what’s wrong or right with the system that distributes it, and how we need to change the world to solve these problems. That requires thoughtful discussion of what we value – on many levels.

Those who believe that ability-to-pay is an appropriate basis for distributing organs need to be prepared for the implications: Rupert Murdoch could have gamed the same system by buying up just as many “lottery tickets.” And those who prefer, instead, to use a “meritocratic” social value scale need to keep in mind that the contest is rarely between Steve Jobs and some unrepentant scoundrel. It is often between ordinary people with value only to their families and friends.

Ask most Americans to name who invented penicillin and they will not be able to tell you, but by almost any reckoning, penicillin saved more lives in a year than Apple technology will save in a decade. Even on the day the Nobel Prize was announced, most people didn’t even register the names of the people who won it or what their contributions have meant for the rest of us. Citizens of the Western World see immortality as their due and they are angry at the pharmaceutical industry in no small part because drug companies can’t ultimately fix everything that ails us, even at premium prices.

We have a right to ask whether one of the things missing from this equation is a respect for the heroism of medical research. As we face hard choices in the next decade over how to spend our money and how to harness our technological resources and ingenuity, we had better teach people whom else to lionize.

Pharma on Facebook: The handwriting on the wall is about more than adverse-event reporting

noreply@blogger.com (Anonymous) — Mon, 12 Sep 2011 10:25:00 -0400

A bad case of “consumer package goods envy”

Pharmaceutical companies have long had a bad case of “CPG envy” because regulatory handcuffs constrain their customer outreach and limit their ability to develop brand relationships with any of their customers. Even in this golden age of “patient democracy,” pharma has no small challenge engaging with patients for a complex set of reasons – among them the rather frustrating truth that patients don’t generally care to love and know their drug brand in quite the same way they love their brand of beer. (More on that in another entry…) At the same time, pharmaceutical companies feel a pressing need to cultivate relationships with patients as a result of many factors that are redirecting their marketing efforts toward end-users – including tighter access to physicians, the coming shift in pay burden to patients, and the industry’s focus on revenue lost through non-adherence.

For that reason, the opportunity for pharma companies to talk to patients and potentially hear them talk back through social media was hard to resist – especially given FDA’s continuing refusal to offer guidance – until Facebook announced new rules of the game requiring that everything be said out loud. Companies began rather quickly to reexamine their strategy and, in some cases, shut down the conversation.

There’s always a chaperone in the room

Did it make sense for pharmaceutical companies to retrench like that? Probably. Did it ever make sense for them to be using a social media strategy for patient outreach in the first place? Probably not – and for reasons that extend far beyond the Pandora’s Box of adverse-event reporting that open forums can easily become. The problem has to do with how pharma can, and cannot, talk back to vocal customers when there’s a chaperone in the room. I recently had the opportunity to speak with Mitch Wagner, Editor in Chief of The CMO Site, for an article he wrote about this same topic. In that conversation, I noted to him that critics who scold the pharmaceutical industry for turning tail and running from real dialogue miss the point. The dialogue is definitely not open. Certainly not open the way it is for CPG brands.

CPG have problems of their own in managing customer dialogue, even without regulatory oversight, but they can’t resist or dodge the opportunity; two-way conversation is considered mandatory for consumer brands. And disgruntled customers have a way of making themselves heard whether or not brands give them airtime on Facebook.

For the pharmaceutical industry, the problem is different, and AE reporting is certainly a big part of it. Industry veterans recognize that the current AE reporting system is an unreliable vehicle for post-marketing surveillance. They’re also right to worry that social media will make that system more unwieldy by pumping many more unfiltered attributions onto the web – a flow that’s cumbersome for the regulatory process to manage and virtually impossible for consumers to interpret. Pharmaceutical companies worried about AE reports or other commentary posted on open walls are right to be concerned. They can expect lots of clamor, lots of misconception, lots of unwelcome attention – and almost no ability to manage or muzzle the noise.

Scripted dialogue is safer than improv

This is the ultimate irony. Social media has redressed the balance for CPG companies in a way that enables both brands and consumers to hold the floor, but for pharmaceutical companies, the dialogue will be stilted once patients get to speak their minds in ways that companies and brands cannot. Facebook walls give patients a freedom of speech that the pharmaceutical companies themselves don’t have. When patients get to talk in social media forums and companies must largely listen, neither the marketing outcomes, nor the public health outcomes, are likely to be good ones.

Those who lament the loss of free-flowing cross-talk between patients and pharmaceutical companies are taking a romantic view of interaction. Pharma marketers need to stop wishing they were stewards of brands like Activia. And they need to listen hard to all their customers in more controlled research environments where they can learn from patients without necessarily having the privilege of talking right back to them. Sometimes, everyone talks way too much.

Generic Oncology Drug Shortage Means Unintended Healthcare Rationing

noreply@blogger.com (Debbie Kossman) — Thu, 25 Aug 2011 09:09:00 -0400

In cancer therapy, expensive drugs extend lives, while some cheap drugs save them.

Renowned oncologist and bio-ethicist Ezekiel J. Emanuel, MD, PhD inaugurated a regular New York Times op-ed gig earlier this month with commentary on generic cancer drug shortages.

Taking up a topic that has already been much discussed in both the professional media and the lay press, Emanuel observed that these drug shortages appear “to be the consequence of corporate decisions to cease production, or interruptions in production caused by money or quality problems, which manufacturers do not appear to be in a rush to fix.”

The gravity of this situation is widely acknowledged throughout the medical community. Dr. Hagop Kantarjian of the MD Anderson Cancer Center has gone so far as to observe that, in his opinion, the shortages of cytarabine have “affected life and death situations in the United States.”

It’s not difficult to understand why companies would be demotivated to manufacture generic cancer drugs which, like all generics, deliver low margins. Unfortunately, many of the drugs in short supply – cytarabine, etoposide, and vincristine, among others – happen to deliver cancer cures to children while readily available branded cancer drugs sometimes deliver benefits measured in just months of survival for patients of advanced years, and at costs that can exceed $100,000 per patient. This is, Emanuel noted, a form of de facto rationing that stems from legislated attempts to constrain market forces that might otherwise have worked to make generic drugs more profitable.

The villain of the piece is the Medicare Prescription Drug, Improvement and Modernization Act of 2003, which sets Medicare reimbursement for intravenous medications at six percent above their average selling price. Sales data must be compiled by drug manufacturers and supplied to the government to establish a new average selling price, a process that takes time. As a practical consequence, the legislation restricts drug prices from increasing more than six percent every six months since reimbursement would otherwise lag behind the cost to hospitals and physicians.

Controlling drug prices suppresses supply, resulting in inadvertent rationing

When healthcare costs are rising exponentially, one tool that people are tempted to use is price controls, and Medicare is certainly an instrument of cost containment policy. But you don’t have to be an unrepentant capitalist to observe that price controls are not entirely benign and often have unintended consequences that are difficult to manage. Here, we have just such a case in which market forces – had they been left unfettered – might have allowed for a more rational and humane distribution of healthcare resources.

Emanuel offers two ways in which generic cancer drugs could be more profitable: (1) amending the Medicare Prescription Drug Act so that after several years of availability, reimbursement for generics would rise to an average selling price plus 30 percent or (2) more dramatically removing Medicare from coverage of generic cancer drugs entirely, leaving private pharmacy plans to respond to market forces.

Tackling drug shortages and health rationing head on

While arguing that the market needs to be given more leeway to dictate effective outcomes, Emanuel also took a moment to scold those who have, as he put it, engaged in “scare-mongering about death panels and health care reform.”

The mere mention of healthcare rationing is political suicide. But as this generic drug shortage scenario illustrates, we make decisions all the time as a society that result in healthcare rationing – whether anyone wants to see it, acknowledge it, or call it what it is. We are not facing up to the fact that when we have finite resources, we have no choice but to make difficult trade-offs. And demonizing good-faith efforts to grapple with difficult choices about how to spend limited public healthcare resources sets the process back.

Generic drug shortages are a potent reminder that we are using economic policy to set social policy without tackling the tough issues head on. It would be far better to have an open, explicit debate in which all participate deliberately, than a conspiracy of silence, through which dollars are wasted and lives lost – inadvertently.

Helping Patients Assess the Benefits of their Drugs: Reading Inside the Box and Between the Lines on Drug Efficacy

noreply@blogger.com (Anonymous) — Mon, 1 Aug 2011 09:49:00 -0400

Nothing creates advertising memes faster than DTC promotion – not even Super Bowl commercials – because health professionals and “civilians” alike take prurient interest in the conversation between pharmaceutical companies and patients. It’s not surprising, then, that Rx drug ads are both the inspiration for cultural jokes (can anyone look at a claw-foot bath tub without thinking of erectile dysfunction?) and the launch point for serious public health commentary.

Case in point. In a recent op-ed article in the NY Times, “Think inside the box,” authors, Steven Woloshin and Lisa M. Schwartz, two Dartmouth Medical School professors, urge use of patient Fact Boxes that feature not just drug side effects but also data on efficacy – which, in many cases, turn out to be less impressive than what optimistic patients imagine them to be. The presumption, which seems hard to refute, is that patients can make more educated choices about the risk-benefits of drugs when they know more – not just about the risks, but also about the benefits, or lack thereof.

Clearly, in the modern age of pharmacovigilante-ism, this is a high priority for FDA, which is squirming awkwardly in the space between two stools: the public health benefits that redound when patients take the prescribed drugs they might need but currently avoid taking, and the public health risks averted when patients reject drugs they feel are unsafe. The authors point, as object lesson, to Abilify, a drug slotted as second-line (add-on) antidepressant therapy for patients who, by definition, are difficult to treat, if not refractory. (Full disclosure: the makers of Abilify have never sought my advice on that product and I hold no particular brief for it.)

It’s ingenuous to argue that drug information boxes – terse and sterile, without context or conversation – can do a very good job of conveying efficacy to the lay patient. Drug efficacy has to be viewed amid data and clinical experience available for an entire category of drugs, as well as information regarding drug study design and FDA approval policies. As one who has countless times watched physicians struggle to interpret data put before them in market research, and who has observed striking levels of confusion about drugs unmasked in cocktail conversation with well-educated patients, I despair of relying on a few lines in a drug warning box – simply written or otherwise – to get the job done.

All therapies work idiosyncratically in some patients and not in others – and no market illustrates this better than antidepressant therapy, a market category in which sizeable proportions of patients do not respond to any given drug. In the future, we will have biomarkers to help screen promising patient candidates for certain types of therapies, but for now, we have principally trial and error to guide us. It’s the role of the physician to set and manage patient expectations – even for those patients who can be induced to read drug fact boxes. (Outside of market research settings, many of them can’t.) Indeed, patients with debilitating depression should not be left by themselves to decide whether the odds of success are high enough to warrant a 4-week trial.

Underscoring the point here, the authors represent Abilify’s odds of success as just 10% – the net of test minus placebo control – a calculus that fails to acknowledge the practical conundrum associated with the way we conduct and interpret clinical trials. Without meaning to cast aspersions on experimental design, I hypothesize that placebo-control may actually understate the true benefit of active drugs investigated for mood disorders. It’s medically unethical to give patients placebo in the real world, and no one really knows how to assess the durability of “placebo effects” – the chicanery of mind against body. We can look only at clinical experience with drugs and then make empiric judgments about prospects of success based on what we observe.

I am not about to argue that the dialogue between physicians and patients is always what it should be; nor can I inveigh against the “motherhood” proposition that patients should have access to straightforward information. But I am convinced that improved conversation between patients and physicians is the first and best hope for informed decision-making, and we should not count on telegraphic commentary from FDA to substantially improve it. For other points of view on this debate, please see:

The New York Review of Books, “The Epidemic of Mental Illness: Why?”
Huffington Post article by Irving Kirsch, Ph.D., “Antidepressants: The Emperor’s New Drugs?”

The New “Political” Science: Avastin Breast Cancer Debate Exposes Paradox and Contradictions

noreply@blogger.com (Debbie Kossman) — Fri, 8 Jul 2011 15:50:00 -0400

The FDA’s recommendation to rescind Avastin’s metastatic breast cancer indication provoked a mix of commentary, commendations, and outcry from across the spectrum of stakeholders with an interest in the decision – and in what the decision might mean for the future: the future of cancer therapy approvals, evidence-based medicine, coverage for off-label use, drug pricing, and healthcare rationing … among other things.

This debate lies at the center of a tangled web of science, psychology, politics, economics, and ethics. Science and psychology, in particular, collide here in a way that reminds us of the challenge of selling the public on evidence-based medicine.

We are built by evolution to see cause-and-effect. The subset of women with metastatic breast cancer who have done well on Avastin almost cannot help but believe that they have done well because of Avastin. And it is no easy thing to overcome this built-in visceral conviction with the dry logic of rationality or the weight of evidence from patients treated in groups rather than as individuals.

When we’re able to be objective, we know that cumulative failed trials of a drug – particularly one that comes with noteworthy risks of serious adverse events (as Avastin does) – nullify the original justification for drug approval.

But the very success of Genentech’s other breast cancer drug – Herceptin – is a nagging source of doubt, even for those of us inclined to accept what today’s best science tells us about Avastin’s breast cancer value. Herceptin confers clinical benefit only for the minority of breast cancer patients whose tumors overexpress the Her2-neu protein. We have laboratory markers for Her2-neu that allow us to pick out the subset of patients who will benefit from the drug and to forego the drug in the rest.

It is altogether possible – maybe even probable – that today’s best science just isn’t good enough yet to have identified a causal link between Avastin and the positive outcomes in the subset of patients who have done well on the drug. In fact, suggestive data already exist for the potential value of markers such as tumor and plasma VEGF, circulating E-selectin, ICAM-1, and VCAM-1, and Genentech tried to argue for confining Avastin use to selected patient genotypes. There are even provisional data suggesting that some genotypes (VEGF-634 CC and VEGF-1498 TT) are associated with less grade 3-4 hypertension – a finding that could change the risk-benefit calculation for the drug. But no marker as promising as Her2-neu has revealed itself for Avastin, and no prospective trials establishing the clinical utility of any biomarker have been completed.

Where does all this leave patients? For now, Medicare has said it will continue to pay for Avastin despite the FDA’s recommendation that the metastatic breast cancer approval be withdrawn. In fact, a CMS spokesman said that, “The FDA decision, when it comes, does not affect CMS.”

Those who defend the FDA’s unanimous vote to rescind Avastin’s breast cancer approval do so by saying that it reflects today’s best science. Perhaps CMS is saying that the best science just isn’t good enough, but we could just as legitimately conclude that CMS is saying that today’s best science does not affect CMS. And if that’s what they’re saying, it’s a near-victory for irrationality and optimism over science. Coverage for breast cancer is de facto endorsement. Shouldn’t we challenge that endorsement if, by standards governing every other drug, Avastin doesn't warrant confidence?

Whipsawed by the Long Tail: How Patient Advocacy Groups May Distort Our View of Patient Customers

noreply@blogger.com (Debbie Kossman) — Fri, 24 Jun 2011 11:54:00 -0400

Advocacy groups are vocal and powerful, and you don’t want them working against you. But they don’t necessarily have the same perspectives or agendas as less connected patients.

The industry began looking tentatively at patients as customers two decades ago but it is only since the Web created virtual disease communities that we’ve been able to find and talk with patients who have obscure diseases. This is occurring just as the industry is, ironically, being forced to look for new commercial opportunities in diseases that were once regarded as not profitable enough to target in the old industry block-buster model.

The list of patient advocacy groups is long and growing now that the Web has enabled patients with exceedingly rare diseases -- for example, alpha-1 antitrypsin deficiency or rasopathies -- to assemble and find their collective voice. Current estimates place the number of “rare” diseases at about 6,000 (JAMA, June 8, 2011).

Web access has also transformed the model of traditional advocacy groups (e.g., American Heart Association, American Cancer Society) to a true community of patients who speak more directly through the organizations that represent them. The Internet makes it possible to find not only “the long tail” of small markets for commercial products (Chris Anderson, editor-in-chief of Wired), it allows isolated, geographically dispersed sufferers and treaters of better-known, more visible diseases to share stories, pool resources, and build clout.

Concentrated clusters of advocacy groups are tantalizing targets for pharmaceutical and biotech manufacturers because we gain relatively easy, inexpensive access to relevant patients as both marketing research informants and as partners for public relations and legislative lobbying. Indeed, drug manufacturers have invested heavily in collaborating with advocacy groups to the point that, in many cases, industry is the biggest supporter. An article by New York Times Reporter Gardiner Harris notes that the majority of donations made to the National Alliance for Mental Illness come from drug makers.

Industry partnerships with patient advocacy groups are necessary and valuable, but they should not be assumed to be representative of the broader population of patients.

A community of people who talk a lot and form a political perspective on the world can develop orientations that are not necessarily representative of others who don’t. People who join advocacy groups may be more prepared to be defined by their disease than others, and that characteristic may have different implications for approaches to therapies, etc.

As a consequence, disproportionate reliance on advocacy groups, without corresponding investigation of the perspectives and priorities of the larger numbers of patients navigating their disease on their own, can lead us to abandon outreach to patients who are less easily accessed. And, by definition, advocacy group members are challenging to use as proxies for newly diagnosed patients who have not been in daily or weekly virtual dialogue with other patients and healthcare advocates.

By way of example, research comparing advocacy group members to representative samples of respective patients has yielded opposing conclusions about the value of patient services provided by pharmaceutical and biotechnology product manufacturers.

The Web has certainly democratized our commercialization efforts by enfranchising patients whom we might never have heard from. But we need to be mindful that large groups of people, however they assemble, become political entities. Self-selection bias is still present whenever we tap into any community of “volunteers” and their perspectives can be shaped by community interaction.

When we attempt to forecast demand for new therapies or preferred methods of new drug delivery by vetting them through patient members of advocacy groups, we may find unrealistic expectations about those therapies, over- or understatement of interest in new options, and dismissive attitudes toward support services with real potential to advance therapy adoption and persistence.

Clinical Practice Guidelines: Not Just For Cancer Anymore

noreply@blogger.com (Debbie Kossman) — Mon, 2 May 2011 11:01:00 -0400

The lack of standardization of healthcare in the U.S. has led to shocking variations in treatment outcomes and equally shocking cost differentials, to the point that in some places, and for some conditions, more money is spent to deliver worse outcomes.

As a consequence, clinical practice guidelines will be developed and refined to achieve the goal of standardizing care across providers and geographies.

Oncologists are the Vanguard of the Guidelines-Centered Future

Only reluctantly do we give up our autonomy in this country, but oncologists -- for whom the stakes and challenges are especially high -- were early practitioners of evidence-based medicine and have already embraced clinical guidelines.

The National Comprehensive Cancer Network's (NCCN) Clinical Practice Guidelines in Oncology have become a staple resource to practicing oncologists. Insurers now look to the guidelines to help decide on coverage policies for cancer therapy. Patients motivated to search for online cancer information will find the NCCN Guidelines for Patients.

Clinical Practice Guidelines for Everything?

The move to optimize and standardize other areas of patient care gained momentum this past December when The Dartmouth Institute for Health Policy and Clinical Practice announced a collaborative initiative among seven premier U.S. healthcare organizations to share data on outcomes, quality, and costs.

The participants are: Dartmouth-Hitchcock, Cleveland Clinic, Denver Health, Geisinger Health System, Intermountain Healthcare, and the Mayo Clinic -- organizations which together manage 10 million patients. They'll be focusing first on a handful of conditions associated with rapidly increasing costs and wide variations in quality and outcomes across the country. Among them are asthma, diabetes, heart failure, and depression.

The collaborative aims to develop best-practice models that will, in their words, "impact the clinical care of patients across the country by disseminating these models quickly and working with providers and health systems to adapt them to local conditions."

Guidelines vs. the Free Market

This trend means that the battle for product success will increasingly be fought before a shrinking audience of decision-makers who control outcome studies and write the clinical treatment guidelines -- guidelines that will be followed by front-line providers and used by payors to set reimbursement policies. The masses of community practitioners who implement those guidelines will more faithfully follow clinical pathways they've had little role in creating.

There will, of course, always be some room for interpretation by site-of-care decision-makers because clinical situations can be ambiguous, and patient "portraits" not always crisp. But leverage with end-users will diminish as the role of brands is established earlier and elsewhere.

Direct-to-Consumer Genetic Testing: What Happens After The Results Are Back?

noreply@blogger.com (Debbie Kossman) — Wed, 20 Apr 2011 09:18:00 -0400

The growing phenomenon of genetic testing will give consumers a blurry crystal ball that, like the fortune-telling of fairy tales or mythology, is certain to create as much conflict as it averts.

But manufacturers of direct-to-consumer (DTC) genetic tests -- as well as the companies that make the therapeutic interventions that might be used as a result of such tests -- have an opportunity as a result.

By satisfying customer cravings for guidance about when and how to use DTC genetic tests, manufacturers have the opportunity to establish a distinctive foothold in the marketplace.

And manufacturers need to do this before it is done for them in ways they might not want.

The Food and Drug Administration's (FDA) review of how to handle DTC test kits became more urgent in 2010 when Pathway Genomics announced an agreement with Walgreen Co to distribute the tests through its 6,000 neighborhood pharmacies.

Walgreen's suspended plans to sell Pathway's tests to consumers after an FDA warning that the test is not approved and could put consumers at risk, but the FDA is asking experts about:

The wisdom and practicality of sorting tests into categories, such as those that look for inherited disease vs. predict a risk of future disease vs. predict the chances of responding to particular drugs.
The need for professional genetic counseling to interpret test results correctly.

All customer constituencies are -- or should be -- hungry for valid, evidence-based guidance about (1) whether, when, and how to use DTC and physician-ordered genetic testing and
(2) when paying for it is a sensible healthcare investment.

The complexity of the relationship between DNA results, disease risk, treatment availability, and the value of knowing one's risks challenges even the most sophisticated experts. The task of arming consumers with even the most basic understanding of the utility -- or lack of utility -- of DTC genetic testing is daunting, and the area is just as fraught for regulators, physicians, and payors.

An Object Lesson for DTC Testing: Cholesterol Home Test Kits

Despite almost two decades of consumer education, and treatment and testing innovation, a 2003 Consumer Reports article maintained that cholesterol test kits didn't provide enough information to be helpful.

The array of genetic tests that will be directly available to consumers is going to increase very quickly, and those tests will tell us about things far more important -- and scary -- than bad cholesterol. While the opportunity to help patients and physicians more effectively manage health and illness is vast, so too are the risks of needless anxiety, misspent funds, and medicolegal liability.

Do the Math: Nurse Practitioner Scope of Practice Must Multiply

noreply@blogger.com (Debbie Kossman) — Tue, 12 Apr 2011 14:26:00 -0400

The expanding role of nurse practitioners will have to expand to fill the gap created by a physician shortage and to release some of the pressures on a system that's over-priced, over-burdened, and under-resourced.

The average primary care physician today carries an annual caseload of approximately 2,400 patients, requiring physicians to see patient after patient throughout the day at a breakneck pace. Even though there are many more diagnostic codes and therapies than ever before, the average patient spends far less time with his or her physician than at any time in the past.

In the next 20 years, both the number of people aged 65 and older, and the percentage of the U.S. population they represent, will increase substantially. Chronic illness affects over 80 percent of older adults and consumes disproportionate resources. Almost a quarter of older adults have five or more chronic conditions, regularly visit 13 different physicians, and fill over 50 prescriptions annually.

To meet the demand for primary care in 2030, each PCP would have to see 33 percent more patients than today. To avoid that, we will need 40,000 more physicians than we expect to have. Otherwise, 95 million patients -- over one in every four people -- will go without access to a physician simply because of the lack of providers to handle them. And the problem can only get worse once we bring another 32 million uninsured patients into an already stressed system.

Over the past 20 years, the number of NPs per capita in the U.S. has risen seven times faster than the number of MDs. In 2011, 16 states and the District of Columbia allow nurse practitioners to practice and prescribe independently of physicians, and 28 more states are now considering regulations that would allow NPs to practice a full range of responsibilities.

Within the Veterans' Administration, Kaiser Permanente, and Geisinger Health Systems, the expanded role of NPs has been linked to improved quality and patient satisfaction, as well as to cost reduction. Training programs across the country are moving toward curricula that will prepare NPs in gerontology as well as adult health, to ensure that graduates are ready to contribute to the care of older patients.

A debate and a political tug-of-war is now raging over whether nurse practitioners should be allowed to assume responsibilities that have historically been granted only to physicians. On one side of the rope is a coalition of four physician specialties, which includes the American Academies of Family Physicians (AAFP) and Pediatrics (AAP), the American College of Physicians (ACP), and the American Osteopathic Association (AOA), along with the Accreditation Association for Ambulatory Health Care. On the other is the National Committee for Quality Assurance (NCQA) and the American Academy of Nurse Practitioners.

But there is rarely a clear perch from which to make predictions about the future than demographics. And the demographics are clear: There will not be enough primary care physicians to serve U.S. demand for primary care.

Healthcare cost savings from expanded access to preventive medicine could be dead on arrival

noreply@blogger.com (Debbie Kossman) — Mon, 21 Mar 2011 08:40:00 -0400

Enormous, and enormously unrealistic, hope is being placed on preventing illness and its complications as a means to improving health outcomes and containing healthcare costs.

A major criticism of the U.S. fee-for-service healthcare model is that it rewards the treatment of health problems over their prevention. Much of the effort at reform is aimed at revising incentives to motivate providers to deliver preventive medicine.

The logic is that if physicians and other providers are compensated for a focus on prevention, and patients have access to affordable primary care, then people will be motivated to use that care and do the things needed to stay healthy. The reality, however, may be quite different.

The impediments to preventive medicine and health maintenance may lie less with our healthcare system than with the psychological make-up of the patients it's meant to serve.

For all that patients may talk about wellness, it is actually very difficult to socialize or coerce them into greater proactivity or compliance. As a general rule, people want to see the doctor only when they're sick. That's why the people least likely to miss their medical appointments are those who are already very sick.

People who know the right choices to make for their health -- diet, exercise, annual physicals -- often fail to make them. Fear, denial, and other psychological forces keep us from facing our vulnerabilities and embracing steps that might extend our longevity but in ways we can't generally measure or know.

Many of us with prescriptions to prevent various life-threatening conditions are, for a variety of reasons, notoriously reluctant to take them, or take them regularly. The problem of insufficient adherence and persistence with drug therapies has unmeasured implications for the health status of our population but clear implications for the revenues of the pharmaceutical industry.

Complicating things further, any public debate about the merits of a particular therapy or test becomes rational grounds for people to abandon preventive or early intervention behaviors.

A case in point is the drop in rate of yearly mammograms among women over age 40 after the revised U.S. Preventive Services Task Force guidelines were announced in November 2009 saying that women in their 40s at average risk for cancer do not need annual mammograms.

Even before the Task Force issued its conclusions, and in spite of American Cancer Society endorsement of yearly screening, only 50 percent of women in their 40s were receiving annual mammography (and only 54 percent of women between 50 and 64, the group for which there is greatest evidence of, and agreement about, mammography's value).

Kocher & Sahni suggest in The New England Journal of Medicine that accountable care organizations (ACOs) should focus on the 10 percent of patients responsible for 64 percent of all U.S. healthcare costs by offering "better and more efficient management of chronic conditions," for example, by calling high-risk patients to return for timely follow-up care.

Unfortunately, even patients at high risk of future health problems are not necessarily easier to coax or coerce into dutiful prevention behaviors than anyone else.

Not long ago, I received a message from my physician's office telling me that my internist had renewed my prescriptions (a proton pump inhibitor for GERD, an ACE inhibitor/diuretic combination for hypertension, and five medications for asthma) and reminding me to schedule my annual physical. I haven't. I know I should. And I'm willing to bet that I'm one of the more compliant patients my physician has.

Emerging notions of 'Valued-Based Health Care' are raising the bar and extending forecasting horizons for new drug therapies

noreply@blogger.com (Debbie Kossman) — Fri, 18 Mar 2011 16:14:00 -0400

Not long ago, a New England Journal of Medicine article, "What is Value in Health Care?" by Harvard Business School professor Michael Porter, offered up an analysis of healthcare value and its measurement in health outcomes research. Porter, along with and his colleague and co-author Elizabeth Olmsted Teisberg, describe three types (tiers) of health outcomes:

Health status achieved or retained -- survival, degree of health/recovery
Process of recovery -- time to recovery/normal living, short-term liabilities of treatment
Sustainability of health -- time to/nature of recurrences, longer-term liabilities of therapy

This "Outcome Measures Hierarchy" is being used by a growing number of large multi-hospital providers, small group practices, health plans, and employers in the U.S. and abroad as they work toward implementing value-based principles to guide therapy choices and outcome assessments.

Notably, the third tier -- health sustainability -- introduces a criterion that has been notably absent from previous calculations of therapy benefits, often based on trials as short as three months or six, even for conditions we know to be chronic.

The injunction to healthcare forecasters and marketers of all types -- product manufacturers as well as the Wall Street analysts who look over their shoulders -- is clear: The timeframe for product performance is growing longer and new healthcare products will be judged by what they do for us over a period of years, not months, after they're launched.

Porter's injunction and its widespread adoption by providers have implications for how our industry prices products out of the gate, as well as the way marketers position them against existing therapies. The demand for yet more specific, detailed, wide ranging, and importantly, longer-term evaluation is increasing. These hurdles are destined to increase (1) the cost of clinical trials and post-marketing surveillance as well as (2) the risks of product failure.

The common wisdom today is that a new product's first-year performance is the sprint that sets and signals its lifecycle value. Not so tomorrow when the Food and Drug Administration approval and commercial availability are merely the starting gun of a marathon.

If full implementation of value-based healthcare means that the duration of clinical trials for FDA approval must increase substantially, then patent life might correspondingly increase. In fact, in this future world, patent life must increase for the rewards of new product R&D to be sufficient to compensate for the longer development phases and greater risks of product failure that the value-based world creates.

Porter is, more than anything else, a competitive strategist. In laying out his value-based framework, he is telling us not only what constitutes value in healthcare. He is telling us what he believes will be essential for competitive advantage in this industry.