The RPM Report :: Actionable Insight on Regulation, Policy, the FDA and CMS

Is Merck Less Bullish on Biosimilars?

krawson3@yahoo.com — Fri, 25 May 2012 00:00:00 -0400

Of all the Big Pharma manufacturers, none seemed more optimistic about the commercial potential of biosimilars than Merck. But over the last several months, Merck shut down its BioVentures Group and skipped an FDA public meeting on the abbreviated approval pathway.

Musical Tables in Generic Drug Litigation

michael.mccaughan@previsionpolicy.com — Wed, 09 May 2012 00:00:00 -0400

Generic drug litigation remains a prominent part of food and drug law practices. But with the growing complexity of the case law—and the blurring lines within industry—it is very hard to predict which client and which attorney will be on what side.

The Limitations of Antibiotic Incentives

michael.mccaughan@previsionpolicy.com — Wed, 09 May 2012 00:00:00 -0400

Congress is on the verge of enacting important new incentives for antibiotic development. At the same time, a federal court ruling on Vancocin generic approvals underscores the importance of doing things the right way.

The Impact of REMS on Generic Drug Approvals and Drug Competition

Thu, 03 May 2012 13:32:00 -0400

From the conception of the REMS in the FDAAA Act of 2007, Congress was concerned that giving FDA authority to impose new post-marketing controls could raise competitive issues about market access for generics. Congress was right to be concerned.

Weighty Issues: GW Obesity Drug Outcomes Project Tests a Model for Patient Input in Drug Development

michael.mccaughan@previsionpolicy.com — Tue, 01 May 2012 00:00:00 -0400

A stakeholder project on obesity treatment outcomes will complete its work this summer, setting the stage for FDA to rethink efficacy endpoints for weight loss drug development. The closed-door project is happening in parallel with public advisory committee discussions on pending applications and safety standards.

The Inevitable Outcome: Diabetes Safety Model Expands to Weight Loss…and Beyond?

michael.mccaughan@previsionpolicy.com — Tue, 01 May 2012 00:00:00 -0400

The cardiovascular outcomes standard used for diabetes drugs since 2008 is moving to weight loss. That is important in itself, but also for what it says about the lessons FDA has taken from the diabetes experience—and what it might mean about regulatory expectations for other chronic drug classes in the future.

Reimbursement of Biosimilar and Biosimilar-similar ESAs

ramsey.baghdadi@previsionpolicy.com — Mon, 30 Apr 2012 00:00:00 -0400

Affymax received FDA approval for the first new ESA in years. Now the company is trying to secure favorable reimbursement for its product, Omontys.

Prometheus Rebound: Where Weak Patents Can Benefit Pharma

cole.werble@previsionpolicy.com — Mon, 23 Apr 2012 00:00:00 -0400

Biotech and pharma companies were disappointed when the Supreme Court limited patent rights surrounding personalized medicine. But the ruling may end up simplifying the path to adoption of targeted therapies by reaffirming a much more critical point: the therapeutic—not the diagnostic—is the dominant partner.

Patient Advocacy 3.0: Outreach Stretches to First Stages of Regulatory Development

cole.werble@previsionpolicy.com — Mon, 16 Apr 2012 00:00:00 -0400

FDA is ready to bring patient voices and perspectives into the drug approval framework. Practically, that means that drug sponsors will have to develop new skills and structures for working with a familiar stakeholder group at an unfamiliar point in the drug development process.

The Supreme Court and Biosimilars: What if it All Goes Away?

michael.mccaughan@previsionpolicy.com — Wed, 11 Apr 2012 00:00:00 -0400

After the bruising health care reform debate in Congress, biopharmaceutical companies were comfortably on the periphery of the legal arguments playing out in the Supreme Court. But being peripheral has its disadvantages: if the Court sides with the challengers, it is possible that one of the “peripheral” provisions tossed aside will be the new biosimilars pathway.

Chelsea’s Northera: The Perils of Drawing Conclusions in Real Time

ramsey.baghdadi@previsionpolicy.com — Mon, 09 Apr 2012 15:17:38 -0400

Despite a “yes” vote from an advisory committee, FDA sent Chelsea’s Northera back to the development phase with a complete response letter asking for data from another trial. There may have been nothing Chelsea could have done to get a better result—but the company could have held off on a post-panel victory lap.

CMS and Opioids: The Role for Non-Drug Reimbursements

Laura.Helbling@PrevisionPolicy.com — Mon, 09 Apr 2012 13:08:58 -0400

The Centers for Medicare and Medicaid Services proposed Part D rules to give plans some responsibility for monitoring opioid abuse. But providers at a Senate Finance Hearing think CMS reimbursements beyond drugs can have a role in screening abuse – and in treating pain to begin with.

The Popularity of “Special Pop”: Antibiotics Incentives and PDUFA V

Laura.Helbling@PrevisionPolicy.com — Mon, 09 Apr 2012 12:22:00 -0400

The Infectious Diseases Society of America’s “Special Population” approval mechanism has proponents within FDA and industry. That makes it look like a good balancing proposal to complement the market incentives for anti-infectives being pushed under the GAIN Act add-on to PDUFA V.

FDA’s Latest PR Push: Protecting the Reputation of Generic Drugs

krawson3@yahoo.com — Mon, 09 Apr 2012 11:40:00 -0400

The public’s acceptance of generic drugs is at an all-time high. But manufacturing issues, shortages of life-saving medications and questions about the overall quality of generics are testing the system like never before.

Last Chance to Partner with Major Biopharma at Pharmaceutical Strategic Outlook 2012!

Sun, 01 Apr 2012 00:00:00 -0400

Join us at the Grand Hyatt New York from April 11-13, 2012 for the 9th Annual Pharmaceutical Strategic Outlook Conference. Experience a dynamic three days of networking and discussion - with a personal and interactive setting, in-depth on-stage interviews, thought-provoking panels, one-on-one partnering meetings and innovative company presentations by carefully selected biotech companies.

The Supreme Court and the “Laws of Nature”

Wed, 28 Mar 2012 00:00:00 -0400

Shortly before taking up the health care reform law, the Supreme Court weighed in on the intellectual property framework for personalized medicine. Here is an excerpt from the court’s March 20 opinion in Mayo v.

The State of CER: Some Progress, Cautious Optimism

Tue, 27 Mar 2012 00:00:00 -0400

It isn’t a household name—yet—but the Patient-Centered Outcomes Research Institute has certainly increased its name recognition among health care stakeholders during the last year, especially as it works to meet the federally mandated requirements of the Affordable Care Act. This was just one of the findings by the National Pharmaceutical Council from the second year of surveying key stakeholders about comparative effectiveness research (CER) perceptions and its impact on health care decisions.

Maintaining a Sustainable Environment for Innovation: Ending the Evidence Arms Race for Regulatory and Reimbursement Decision-Making

Mon, 26 Mar 2012 00:00:00 -0400

Increasing attention on containing healthcare costs has spurred new business opportunities in data aggregation, real-time analytics, and rapid translation of data into evidence on the value of new innovation. This has become a race for more and faster evidence.

Shire’s Replagal Withdrawn: A “Non-Traditional” Review Ends Badly

michael.mccaughan@previsionpolicy.com — Mon, 26 Mar 2012 00:00:00 -0400

Shire’s decision to take its Fabry Disease therapy and go home sets up two possible narratives. One accentuates FDA’s apparent inflexibility and is sure to reinforce calls for legislative changes to the approval standard.

Too Much Success for Generics? GPhA’s Neas Defends Hatch-Waxman

krawson3@yahoo.com — Thu, 22 Mar 2012 00:00:00 -0400

Newly installed GPhA CEO Ralph Neas has a lot to juggle these days on Capitol Hill, between drug shortages and user free agreements for generics and biosimilars. But one thing Neas wasn’t expecting was a threat to Hatch-Waxman.

CMS Wades Into the Opioid Debate

michael.mccaughan@previsionpolicy.com — Thu, 15 Mar 2012 00:00:00 -0400

Reducing prescription drug abuse is a high priority item for several key legislators and for the Obama Administration. Proposed new rules for Medicare drug plan sponsors might help—but they also underscore the growing tension among the many different stakeholders in the pharmaceutical supply chain.

What’s Going On With Eliquis and Xarelto at FDA?

ramsey.baghdadi@previsionpolicy.com — Thu, 15 Mar 2012 00:00:00 -0400

A key date was removed from FDA’s tentative advisory committee calendar. But a panel is still expected to review BMS’s anti-clotting agent Eliquis and a new indication for J&J’s Xarelto in ACS.

Regulatory Innovation: A Dialogue (Part II)

cole.werble@previsionpolicy.com — Thu, 15 Mar 2012 00:00:00 -0400

Center for Drug Evaluation & Research Deputy Director Douglas Throckmorton and AstraZeneca R&D President Martin Mackay took part in a moderated discussion of new initiatives and ideas for drug regulation during The RPM Report’s FDA/CMS Summit in Washington DC in December 2011. The first part of the dialogue focusing on drug review activities was previously published; this second section addresses post-market activities and the impact of data from different types of clinical experience on FDA regulation.

GAIN Is Great: Now Who Gets the Benefits?

Laura.Helbling@PrevisionPolicy.com — Thu, 15 Mar 2012 00:00:00 -0400

Congress is set on enacting a package of antibiotic development incentives—but needs to sort out how best to define the targets.

COPD Post-Market Requirements: Cardiovascular Safety Could Evolve to Comparative Trials

cole.werble@previsionpolicy.com — Fri, 02 Mar 2012 00:00:00 -0500

Forest's aclidinium breezed through an advisory committee review with three strong votes in favor of approval. The FDA panel, however, also included an interesting call for more head-to-head trials in COPD from the outgoing panel chair.

Setting the Stage: Qnexa and the Power of REMS

michael.mccaughan@previsionpolicy.com — Thu, 01 Mar 2012 00:00:00 -0500

Vivus’ second trip to an advisory panel was a much happier one, with the company’s weight loss therapy garnering an overwhelming “yes” vote on approvability. The key change?

Crossing the Line Between Food and Drugs

Thu, 01 Mar 2012 00:00:00 -0500

In the era of “nutraceuticals,” the lines between a food, a dietary supplement, a drug can be hard to define—even though they make a huge difference in the regulatory standards that apply. In a March 5 Warning Letter to Breathable Foods Inc., however, the agency drew a very bright line when it comes to inhaled caffeine: the role played by the human epiglottis.

Regulatory Innovation: A Dialogue

cole.werble@previsionpolicy.com — Thu, 01 Mar 2012 00:00:00 -0500

Center for Drug Evaluation & Research Deputy Director Douglas Throckmorton and AstraZeneca R&D President Martin Mackay took part in a moderated discussion of new initiatives and ideas for drug regulation during The RPM Report’s FDA/CMS Summit in Washington DC in December 2011. Here is a portion of that conversation, transcribed by The RPM Report and lightly edited for clarity.

Conflict Over Conflicts: FDA Advisory Committee Rules May Be Ripe For Change

krawson3@yahoo.com — Sun, 26 Feb 2012 00:00:00 -0500

Recruitment of FDA advisory committee members has been a persistent challenge for the agency—especially since the conflict of interest rules were tightened five years ago. While FDA has improved the number of vacancies on its expert panels, it has yet to meet its goals—especially in the Center for Drugs.

Coverage With Evidence Development and Expedited Approvals: Policy Proposals For FDA and CMS

Tue, 21 Feb 2012 00:00:00 -0500

Based on stakeholder consultations, former FDA and CMS head Mark McClellan and former CMS official Sean Tunis present a potential upside for biopharma in broader use of Medicare coverage with evidence development. Pfizer executives participate in workshop on coordinating expedited drug approval, CED policies.

19th Annual Euro-Biotech Partnering Summit

Wed, 01 Feb 2012 00:00:00 -0500

Publisher’s Spotlight: Join us in Paris, France from May 30-June 1, 2012 for the 19th Annual Euro-Biotech Partnering Summit. This year’s theme is Changing the Business Model: Drawing on Global Examples of Innovation to Find New Products and Maximize Efficiency.

9th Annual Pharmaceutical Strategic Outlook (PSO) Conference, April 11-13, 2012

Wed, 01 Feb 2012 00:00:00 -0500

Join us at PSO 2012 where we will delve into the top issues affecting the pharma industry. Experience a dynamic three days of networking and discussion - with a personal and interactive setting, in-depth on-stage interviews, thought-provoking panels, one-on-one partnering meetings and innovative company presentations by carefully selected biotech companies.

What Will CMS Do With Avastin In Breast Cancer?

ramsey.baghdadi@previsionpolicy.com — Wed, 01 Feb 2012 00:00:00 -0500

With FDA’s decision to pull the indication for Avastin in breast cancer, CMS is now waiting and watching for residual use patterns.

Biosimilars: Inverting the Innovator Development Model

michael.mccaughan@previsionpolicy.com — Wed, 01 Feb 2012 00:00:00 -0500

When it comes to the new biosimilar pathway, it’s the IND—not the BLA—that matters.

Secondary Uses of Sentinel

michael.mccaughan@previsionpolicy.com — Wed, 01 Feb 2012 00:00:00 -0500

FDA’s new drug safety surveillance system is up and running, though the implications for safety oversight are only just beginning to be felt. But the conversation is already under way about what else Sentinel can do—and the answers may be very important to the future of drug regulation.

Primatene Mist – Adventures in Tangents at User Fee Hearings

Laura.Helbling@PrevisionPolicy.com — Wed, 01 Feb 2012 00:00:00 -0500

It’s hard to anticipate what will come up in the user fee hearing process.

Biotech Stocks and Advisory Committees: No News Is Good News?

michael.mccaughan@previsionpolicy.com — Wed, 01 Feb 2012 00:00:00 -0500

FDA has approved six new molecules already in 2012--and five were approved without going to an advisory committee. Meanwhile, FDA has held four advisory committees to review new drug applications, and all four ended in overwhelming "no" votes.

Re-Earning the Public Trust

Wed, 01 Feb 2012 00:00:00 -0500

Pharma has made big changes in marketing and promotion practices and the systems to assure compliance, but the industry’s ethical image is lagging the efforts at reform. Pfizer Specialty Care/Oncology President Geno Germano says a shared commitment among companies to convince the public of the impact of the industry’s compliance reforms is the key to a healthy environment for the industry.

An Emerging Approval Trend At FDA: More First-Time Sponsors

krawson3@yahoo.com — Thu, 19 Jan 2012 00:00:00 -0500

There is a surprising trend in the list of FDA approvals in 2011: an increase in the number of small biopharma companies opting to forgo Big Pharma partnerships and submit new product applications on their own. Here’s how these “emerging sponsors” are changing the way FDA does business—for big and small companies alike.

The Real Obesity Drugs Hurdle: Paying for Them

ramsey.baghdadi@previsionpolicy.com — Thu, 19 Jan 2012 00:00:00 -0500

Obesity drugs are readying for round two at FDA. But if ever approved, given the airing of safety issues at advisory committee meetings, who would pay for them?

What Might Have Been? The Gingrich FDA Reform Platform

Thu, 19 Jan 2012 00:00:00 -0500

Former House Speaker Newt Gingrich hasn’t forgotten his interest in FDA reform: one theme of his Presidential campaign has been to revisit some of the arguments that underpinned the GOP reform legislation of the mid-1990s. At press time, the chances of Gingrich winning the nomination appeared remote—but for a brief time late in the fall it looked like FDA might take center stage in the upcoming elections.

All the Guidance They Need: Biosimilars Business Development Doesn't Wait For FDA

michael.mccaughan@previsionpolicy.com — Sun, 15 Jan 2012 00:00:00 -0500

Wall Street is eagerly awaiting formal guidance from FDA on the new biosimilar pathway in the US. Biotech companies large and small aren't waiting.

FDA “Flexibility” Presages Good Climate in 2012 for Approval Decisions, Shortage Situations

cole.werble@previsionpolicy.com — Sun, 15 Jan 2012 00:00:00 -0500

As FDA girds for the Capitol Hill phase of the user fee reauthorization push in 2012, the agency is projecting an image as a flexible problem-solver. FDA’s underlying message: the agency has sufficient authority and does not need tinkering with the legislative mandate.

FDA’s Banner Year for Drug Approvals: Time to Change the Focus

michael.mccaughan@previsionpolicy.com — Sun, 01 Jan 2012 00:00:00 -0500

FDA is entering a big legislative year with a good story to tell based on 30 new molecular entity approvals. But the level of submissions remains constant—suggesting it may be time to shift the focus from what comes out of FDA to what makes it in.

Specialty Tiers Ensure Part D Plans Cover High-Cost Biologics, Blum Says

c.kelly@elsevier.com — Sun, 01 Jan 2012 00:00:00 -0500

CMS Medicare director Jonathan Blum acknowledges there is a “robust debate” within CMS regarding specialty tiers in Part D but says he believes permitting plans to charge beneficiaries higher cost-sharing for expensive biologics preserves access to such drugs.

The End of an Era: Lipitor, Blockbuster Drugs and the Waxman/Hatch Model

michael.mccaughan@previsionpolicy.com — Thu, 15 Dec 2011 00:00:00 -0500

Pfizer’s Lipitor goes generic—albeit with some decidedly unusual wrinkles. The biggest generic launch in history is a good moment to take stock of the transition away from the blockbuster model.

An Involuntary User Fee: Two Ideas for Off-Label Promotion Fines

Laura.Helbling@PrevisionPolicy.com — Thu, 15 Dec 2011 00:00:00 -0500

What if off-label promotion fines were put to good use? A consumer representative and a Senator have thrown out ideas for how to use them.

Companion Drug/Diagnostic Pathway: A Question of Where and Who

cole.werble@previsionpolicy.com — Thu, 15 Dec 2011 00:00:00 -0500

FDA has put a lot of thought into preparing for the drug/diagnostic companion products that presage the era of personalized medicine; but the lure of fewer controls and less development costs is tugging some of the effort off-shore. FDA is also scaring off small companies from genomic test projects and their VC investors.

FDA's Patient-Centered Drug Center

michael.mccaughan@previsionpolicy.com — Thu, 15 Dec 2011 00:00:00 -0500

The new Prescription Drug User Fee Agreement includes a host of new projects, of varying potential impact. But it is clear that a plan to host a series of disease-specific meetings to identify key risk/benefit goals of patients is one of the most important from FDA’s perspective.

Who Makes a Drug Safe?

Thu, 15 Dec 2011 00:00:00 -0500

Former FDA Commissioner David Kessler submitted a brief in support of plaintiffs in a product liabilty case against Bayer Pharmaceuticals over the safety of the oral contraceptive brand Yasmin, made public on the eve of an Advisory Committee review of the topic. In the excerpt below, he discusses the importance of state tort liability to back up FDA regulation.