The FasterCures Blog

Who decided that “top 10” lists should have 10 things on them?

noreply@blogger.com (FasterCures) — Wed, 25 Jan 2012 20:01:00 +0000

By Kristin Schneeman, Program Director, FasterCures

For five years now FasterCures has been producing its “Top 10” list of medical research trends to watch in the coming year. And every year we have to go through the painful process of deciding what to leave on the cutting room floor.

We relish the opportunity to tell people about some of the interesting trends, ideas, initiatives, events, and people that we think are worthy of attention in the 12 months ahead. But there is more that we’re thinking about, of course. The Top 10 list tends to be focused on things that we think are likely to be the subject of action or debate over the coming year, but there remain big, critical issues that span across years for all of us to focus on and work on together.

We’re concerned about the financial sustainability of the whole research enterprise. We’re hearing that venture capital is fleeing from life sciences, and those who are sticking around are investing in less risky opportunities. We’ll be fortunate if government funding remains flat – we’re not predicting any big increases in the current fiscal environment. So if more money isn’t likely to be pouring into the system anytime soon, what new sources of funding are out there, and what new financial models might be applied? Could constrained resources be an impetus for greater focus and efficiency in the research process?

The “business model” of university innovation is changing, and we’re working to understand the landscape of new approaches to commercializing university inventions: Who are the new partners? What are the new sources of capital? Do we have the human capital needed? What are the new intellectual property policies? When is the right time to license?

We’re interested in what appears to be an increasing openness on the part of big pharma to the need for new business models of their own, not just doing things better the same old way. They’re groping their way toward it – embracing more truly “open innovation,” more pre-competitive collaboration, new ways of interacting with academia, working more closely with patient groups – but the pressure is on to “evolve or die.”

Supporting and improving the FDA will continue to be a major focus for us. We’d like to see FDA be able to better leverage NIH’s research capacity to improve its ability to evaluate the new science coming down the pipeline. We’d like to see the Reagan-Udall Foundation established on firm footing so it can become the kind of resource that the Foundation for the NIH has become for the NIH. We’d like to see more patient-driven research foundations engaging in a conversation with FDA about patients’ needs and priorities. And we’ll be doing our best to ensure a productive public dialogue about the role and record of FDA; a “blame game” doesn’t serve any of us well.

As if our mission weren’t ambitious enough, we’re also considering how the states on either end of the medical R&D continuum – prevention/wellness and the delivery of care – fit in with our work to improve the system that discovers and develops treatments for disease. And we are considering what the impact of an increasingly global research enterprise, as well as global sources of capital, could have on the U.S. biomedical research system.

With so much work to be done, there is no time to waste!

On Huffington Post: Top 10 Medical Research Trends to Watch in 2012

noreply@blogger.com (FasterCures) — Fri, 06 Jan 2012 11:31:00 +0000

Margaret Anderson, Executive Director, FasterCures

1. NCATS: Who moved my cheese?
This year, the National Institutes of Health will establish its new National Center for Advancing Translational Sciences (NCATS), created to speed the translation of basic discoveries into therapies that will improve public health. In light of our current fiscal reality (and limitations), we breathed a sigh of relief to see it up and prioritized in the appropriations process. Solutions can be tough to develop, and tougher to implement. How will NIH translate its translational focus to its cadre of basic research-focused scientists? Will NCATS strengthen and streamline existing efforts at the 26 other institutes and centers? Will it help to move discoveries out of the lab and to patients more quickly? NCATS is one way to get moving (and, as we know, "movement in a new direction helps you find new cheese"). The stakes are high with more than 100 million patients waiting for meaningful treatments options. But with Dr. Francis Collins as the visionary behind this effort, the same man who defied expectations by completing the Human Genome Project ahead of schedule and under budget, we like the odds.

2. FDA: The science of failure.
Companies usually turn to the FDA to help them succeed (and with 35 approvals in FY2011, it helped many succeed), but perhaps it can also help them fail, faster. Commissioner Hamburg seems committed to having FDA play a role in improving R&D efficiency for all. FDA is sitting on mountains of data, and she has made it a priority -- through the agency's Strategic Plan for Advancing Regulatory Science and specific efforts like the PACES Initiative -- to figure out how to share more of it, particularly from compounds that were not approved, to help researchers and companies learn from failures. Will the industry see its self-interest in this and allow data to be shared -- and recognize that there is a business case for opening up the file cabinet?

3. Intellectual property: Keep it simple, stupid.
Everyone in medical research seems to agree that intellectual property is a significant issue slowing progress, from disagreements over the value of discoveries to the time it takes for agreements to be negotiated, and the aversion to standardized contracting. We say, why does this need to be so complicated? Can't we come up with a simple set of guiding principles or values that we all agree to abide by -- something along the lines of Google's "Don't be evil"? Ideas, anyone?

4. Cures on the stump.
Last time we checked, 2012 was a presidential election year. Will life sciences make an appearance on the agenda? The Obama Administration is working on a National Bioeconomy Blueprint to "harness biological research innovations to address national challenges in health, food, energy, and the environment" -- oh, and of course to "grow the jobs of the future." What commitments will the candidates make to support this critical economic driver -- whether NIH funding, tax policies for industry, workforce training, etc.? Can we make innovation in the life sciences a topic du jour in the election season?Venture capital investment in life sciences is on the wane, but venture philanthropy investment in drug development is on the rise. We're hearing discussion in several quarters about the idea of blending venture capital and venture philanthropy in some sort of hybrid investment fund. The wider world of social entrepreneurship has been experimenting with business models and forms of capital all along the spectrum from not-for-profit to for-profit for some time; the field of medical research is slow to catch up. We'll be watching for the launch of one or more of these efforts in 2012. Look for more discussion of financial innovations for translational research from FasterCures in 2012 as well.

5. 2012: Year of the Patient?
TIME magazine notably called 2011 the "year of the protester." Could 2012 be the "year of the patient"? We offered up a roadmap to effective activism in 2011, built on the example of the HIV/AIDS advocacy movement. We identified its ingredients for success as attention, knowledge and solutions, community, accountability and leadership. Many of us point to the successes this movement created in HIV/AIDS drug development. Thirty years later, will more groups seize the mantle and make 2012 as much about changing the research and regulatory paradigm as about raising more money and awareness?

6. Reimbursement is the new approval.
Even if we are successful at getting new treatments through the R&D and approval process, the question remains whether payers (government and private) will be willing to pick up the tab. Insurance coverage decisions -- and insurers themselves -- are playing a role farther upstream in the R&D and investment process, as companies begin to craft their research priorities and programs to maximize the possibility of reimbursement. Can payers position themselves as part of the solution to getting truly innovative therapies to patients? CMS is currently seeking comments on a revamp of its "coverage with evidence development" program. Could this become a model for other insurers to get patients access to new treatments while continuing to generate data about its effectiveness? Will smaller companies start reading the tea leaves and work with payers to avoid "failure to launch"?

7. HIT: If we build it, will research come?
The federal government has succeeded in driving a doubling of providers' adoption of health IT over the last two years through its stimulus funding. But their interest in ensuring all this data is available for research to drive new, more effective treatments for patients is still not evident. "Meaningful use" of electronic health records (EHR) to qualify for federal funding seems to have been an effective carrot/stick combination for driving adoption of EHR systems for patient care. Let's use it in the service of research by requiring that using EHR systems for clinical trial screening and matching be included as a measure for meaningful use in the next round of definitions. Unlike the "Field of Dreams," just because we built the infrastructure, research use will not magically appear.

8. CTSAs: Who you gonna call?
The first five years of NIH's Clinical and Translational Sciences Awards (CTSA) program are over, and by many accounts the focus at most of the 60 institutions has been on standing up the program on their own campuses, not on maximizing the power of the network. That may start to change in 2012 with the creation of a new Coordinating Center at Vanderbilt, which is already ramping up development of tools and resources, such as a shared IRBs, contract language, IP database and research data capture. Its director, Gordon Bernard, wants to make it a "virtual institute of drug development," a one-stop shop for companies, investors or nonprofit funders looking for academic partners. At Partnering for Cures 2011, he invited participants to contact him with their needs, saying that until the right partners are in place "we'll own that problem." So give him a call.

9. Putting old wine in new bottles.
Enthusiasm continues to grow in academia and among patient groups for the idea of repurposing existing compounds. Companies, if they're interested at all, are looking for some sort of exclusivity mechanism to allow them to make money. NIH sees this as a central opportunity for NCATS (see #1), and NIH's TRND program is piloting a repurposing effort with the Leukemia & Lymphoma Society and the University of Kansas as partners, with the aim of recruiting an industry partner. But pilots won't be enough; there needs to be funding sources identified, IP policies sorted out and regulatory pathways clarified. Could 2012 be the year we create a business model to support repurposing? Tick tock goes the clock. Patients are waiting.

10. Convergence of venture capital and venture philanthropy.
Venture capital investment in life sciences is on the wane, but venture philanthropy investment in drug development is on the rise. We're hearing discussion in several quarters about the idea of blending venture capital and venture philanthropy in some sort of hybrid investment fund. The wider world of social entrepreneurship has been experimenting with business models and forms of capital all along the spectrum from not-for-profit to for-profit for some time; the field of medical research is slow to catch up. We'll be watching for the launch of one or more of these efforts in 2012. Look for more discussion of financial innovations for translational research from FasterCures in 2012 as well.

Read FasterCuresTop 10 lists from previous years:

Top 10 Medical Research Trends to Watch in 2011

Top 10 Medical Research Trends To Watch In 2010

For more by Margaret Anderson, click here.

Business Is from Mars, Academia Is from Venus

noreply@blogger.com (FasterCures) — Wed, 04 Jan 2012 18:32:00 +0000

Universities are showing increasing interest in stretching their work into the "Valley of Death" to move their discoveries towards treatments. Companies are rethinking their alliances with universities as sources of innovation to fill their pipelines, beyond traditional sponsored research agreements. At the Partnering for Cures meeting in November, experts discussed where such rethinking is taking place, the replicability and scalability of these models, and the challenges of this new type of relationship.

Moderator Ross DeVol, Milken Institute, noted that innovation is increasingly shifting to universities and, with that, new thinking and practices are developing to ensure that these efforts advance outcomes without distorting academia's core mission.

Annette Kleiser, University of Southern California Stevens Institute for Innovation explained that "making innovations, or I should say inventions, available to the public has become a critical part of what we do."

Pfizer's Tony Coyle pointed out that the wealth of academic understanding and expertise cannot be kept siloed in the usual few organizations. The divide between basic science and clinical practice is where we should be focusing, he pointed out, with the patient's needs always at the center. Coyle suggested industry look to its colleagues in academia to help reflect on what the true unmet medical needs are, and what opportunities exist to design therapeutics that are fundamentally different from the existing standard of care. "We don't need to be so insular," he said. "Success in medicine or chemistry alone will not allow us to develop new therapeutics that are truly meaningful and will have an impact on patients."

Stephen Friend, Sage Bionetworks, who professed himself a "galactic traveler" - having spent a third of his career in Venus/academia, another in Mars/industry and the last "on Earth with the patient" - talked about the need for new models to support all of the data and information that is starting to flow. The linear drug development pipeline is no longer, he said, and industry is "quite awake" to the fact that the old models aren't going to work.

Kleiser described why academia needs private industry: "We need the insight from the industry on how to turn these discoveries into a product. We need the exchange of data, and we need this exchange of expertise." She went on to give an example of a partnership USC has entered into with Johnson & Johnson and how the flexible nature of their IP agreement has opened up new pathways for innovation.

Fernando Kreutz, FK-Biotec, discussed his perspective on Brazil as a blank slate for medical research. "Innovation requires industry," he said. "In Brazil most of the science is actually done inside of universities. How can we channel all of this knowledge into innovation? That's the biggest challenge in Brazil right now."

Todd Sherer, The Michael J. Fox Foundation for Parkinson's Research, talked about what patients could do to help improve outcomes and ensure that development partnerships remain focused on meeting the needs of people with disease. "We do see the model changing in terms of understanding that one entity cannot solve all these diseases." While there is an openness for these collaborations, there are also competing goals and reward structures between industry and academia. Patient organizations can provide a neutral "playing field," allowing partners to come together in a safe space without the complications of either side having a home-field advantage.

Friend then talked about the power of data, and, in particular, the power of patient ownership of data when it comes to sharing and generating information for research. He highlighted several of Sage's projects including The Clinical Trial Comparator Arm Project, a precompetitive disease biology initiative, which is working to push comparator arm data of clinical trials in a variety of therapeutic areas into the public domain .

Recently, Pfizer and the University of California San Francisco began a revolutionary partnership which Coyle discussed. Aimed at more rapidly moving new therapies into human clinical trials, teams from the University and Pfizer work together on experimental therapies developed by the UCSF scientists with a goal of testing them in people with five hard-to-treat, often deadly conditions, including lung and prostate cancer."This is not collaboration from afar, but an example of working together with people that are striving for the same thing. Physically being located in the same lab starts trust, and builds this new ecosystem relationship. Academia cannot do it on its own, and the same goes for pharmaceutical companies."

In closing the panelists discussed the one thing they would change in the current environment. Kruetz said, "The one thing I would change is the lack of urgency in academia. It is shocking." Friend made the point that the scientist, the academic institution and industry work pretty well together. It is the patients that are missing. "Until earth joins Mars and Venus, it's not going to work."

What is NIH's Role in Bridging the Valley of Death?

noreply@blogger.com (FasterCures) — Tue, 20 Dec 2011 16:00:00 +0000

The spending bill referenced in our post yesterday will, if signed into law, establish the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health. Good news for our medical research and development enterprise, and for the millions of patients who need treatments and cures, the inclusion of funding for NCATS in the omnibus will help to speed the translation of basic discoveries into real world applications that will improve public health.

Last month at our Partnering for Cures conference, a distinguished group of panelists from academic, advocacy, and government institutions examined the challenges and opportunities to bridging the "valley of death" between preclinical development and clinical research, and specifically explored the impact and appropriateness of NCATS’ role in this effort. Moderator

Anna Barker of Arizona State University (formerly of the National Cancer Institute), challenged the panel to consider the rapid advancement of accessible information - who is the appropriate convener to push forward translational research?

Kathy Hudson of NIH explained the nuts and bolts of NCATS: instead of focusing on individual projects, NCATS will identify bottlenecks in the pipeline that slow the research process for all players. Hudson acknowledged that it is an "awkward time" given the difficult economic climate, but emphasized that the goal of this new center is to collaborate to address shared problems - not to duplicate what is already happening in the pharma/biotech industry.
University of Pennsylvania professor Garret FitzGerald agreed that NCATS will be helpful to "shine a light on problems," and pointed to the erosion of human capital in translational research. NCATS could play a role in rebirthing the discipline through the training of heterogeneous skills, he noted, which could be especially important in the era of comparative effectiveness research. FitzGerald argued that this type of focus would be politically uncontroversial and relatively inexpensive.

Academic scientist and entrepreneur Peter Lansbury said that government had a huge role to play, particularly in neurodegenerative disease. He noted that there a special need for tools that could help facilitate clinical trials for these types of diseases, where progression is erratic and often slow.
Louis DeGennaro of the Leukemia & Lymphoma Society - which earlier this year signed an cooperative R&D agreement with the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the University of Kansas Medical Center to accelerate the development of potential clinical therapies for rare blood cancers - mentioned that an often overlooked topic during discussions of solution development is the role advocacy and disease advocacy groups play. The "fiduciary responsibility" he and his colleagues feel for patients served by LLS has driven them to develop creative partnerships to engage in translational research.

Chris Varma, Entrepreneur-in-Residence at Third Rock Ventures, noted that although the amount of life science venture capital funding continues to increase, a smaller quantity of individual investments are being made overall. Given this environment, NCATS "makes a lot of sense" in driving efficacy and reducing risk, especially when it comes to identifying failures early on in the development process, he pointed out.

The panelists suggested a few priority areas for NIH's engagement in this area including

Get the funding and support for NCATS to successfully get up and running
Develop human capital - increasing the expertise, capabilities, and attractiveness of the research discipline
Keep focus on patients when bridging the gap from academia to clinical trials
Take an analytical approach to the data available for new research

Value of Establishing the NIH Center on Advancing Translational Sciences to Patients

noreply@blogger.com (FasterCures) — Tue, 20 Dec 2011 15:37:00 +0000

Statement of Margaret Anderson, Executive Director, FasterCures on Value of Establishing the National Center on Advancing Translational Sciences at the NIH to Patients

December 20, 2011 (Washington) – The spending bill President Barack Obama is expected to sign into law later this week will establish the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health. The inclusion of funding for this initiative bodes well for our nation's medical research and development enterprise and for millions of patients who need new treatments and cures now. FasterCures has been a vocal proponent for the creation of this effort to speed the translation of basic discoveries to real world applications that will improve public health.

The bill includes an increase for the NIH ($299 million over last year) and approximately $576.5 million for NCATS. Of this amount, 2.3 percent is new funding while the remainder brings together resources from other NIH Centers for programs such as the Therapeutics for Rare and Neglected Diseases (TRND) and the Clinical and Translational Science Awards (CTSA). The bill allows NIH to spend up to $10 million to support the Cures Acceleration Network (CAN) which creates new funding mechanisms at the NIH through which contracts will be awarded to expedite the development of “high need cures.” In all, the bill provides NIH with $30.7 billion in funding.

The goal of NCATS is to save time in the way we pursue and conduct medical research and development across disease areas. To patients with limited or no meaningful treatment options, saving time means saving lives. The creation of NCATS demonstrates our strong national commitment to medical research by ensuring advances achieved in basic science are translated into new therapies, better prevention strategies, and cures more efficiently.

As envisioned, NCATS would create tools to speed the development of new therapies, and make these tools widely available, thereby fostering efficiencies. This vision provides an opportunity for the patient and disease communities to work together on addressing a problem that cuts across diseases.

NCATS would also serve to strengthen the translational efforts underway at many NIH Institutes. Many NIH Institutes support both basic and translational research portfolios in an effort to move discoveries through the pipeline more quickly. Mid-sized and smaller Institutes that may not possess significant expertise in drug development would benefit greatly from having access to a central hub at NIH where such expertise exists.

At FasterCures, we fully support NIH’s efforts to create NCATS. Thanks to our vitally important national investment in scientific discovery, we know more about disease and biology than ever before. We must now maximize the return on this investment and have systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients’ health and quality of life.

We believe this new Center will strengthen our nation’s medical innovation infrastructure, helping spur economic growth and create jobs in the life sciences industry, and most importantly, it will accelerate the process of turning scientific discoveries into much needed medical solutions.

Top Innovators in Medical Research Come Together To Find Cures

noreply@blogger.com (FasterCures) — Tue, 08 Nov 2011 17:48:00 +0000

3rd Annual Partnering for Cures Meeting Focuses on Collaborative Approaches to Speeding R&D

New York – November 8, 2010. More than 800 leaders from across all sectors in medical research – philanthropists and investors; medical research foundations and institutes, biotechnology and pharmaceutical companies, academic investigators, payers, and policymakers – gathered today to find partners and forge the alliances necessary to accelerate medical progress.

Hailed as a “game-changing meeting” and medical research’s most outcomes-oriented, and patient-focused event, Partnering for Cures is convened by FasterCures – the Washington, D.C.-based center of the Milken Institute – for the sole purpose of speeding the time it takes to turn discoveries in the lab into therapies for patients.

“Serendipity is not going to cut it anymore. Turning scientific discoveries into better health requires all stakeholders in the medical research system to work together,” said Margaret Anderson, executive director of FasterCures. “This effort allows stakeholders to easily find each other, share ideas openly, develop concrete solutions, and create lasting partnerships.”

This year, 30 cross-sector partnerships took center-stage and presented novel approaches to commercialization, academic translational efforts, data sharing initiatives, drug repurposing programs, clinical trials innovations, and intellectual property management. These innovative programs pave a path forward that could yield results that will improve and save the lives of millions of patients.

“Translating the science that comes out of basic laboratories is the greatest challenge and opportunity we are facing,” said biotechnology serial entrepreneur John Mendlein of Fate Therapeutics/aTyr Pharma during the opening plenary. “The good news is that we are witnessing a transformative time--there has never been such an intense focus on collaboration as we are witnessing now.”

Margaret Hamburg, Commissioner of the U.S. Food and Drug Administration Commissioner, noted in this plenary discussion that “the agency needs to find ways to come together with strong science, industry leadership, and patients to identify opportunity and seek regulatory flexibility.”

Susan Desmond-Hellmann, Chancellor of the University of California, San Francisco, said that to speed the drug development process, we need to “pay more attention to surrogate endpoints and intermediate indicators of success, and not waiting for the final or perfect solution.” She urged that “scientists need to be exposed to the clinic so they better appreciate the urgency felt by patients, families, and their doctors.”

Now in its third year, Partnering for Cures connects innovators and inventors across sectors, diseases, and disciplines and provided a platform to share collaborative solutions. With 16 panels and 90 speakers, the meeting focused on solutions – those that must be developed and those that must be amplified. The meeting also featured a customized partnering system.

It has facilitated industry partnerships that led to commercialization agreements; empowered philanthropists to make more informed investment decisions; enabled medical research foundations to find the right partners and investors; and engaged academia to incorporate new business models that accelerate the research and development process.

About FasterCures:
FasterCures/The Center for Accelerating Medical Solutions is a nonprofit think tank and catalyst for action that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. A center of the Milken Institute, it is nonpartisan and independent of interest groups. For more information, visit http://www.fastercures.org/.

For more information, contact:
Cecilia Arradaza
Communications Director, FasterCures
carradaza@fastercures.org

Think Medical Research, But Think Smart

noreply@blogger.com (FasterCures) — Thu, 03 Nov 2011 11:33:00 +0000

by Margaret Anderson, Executive Director, FasterCures/The Center for Accelerating Medical Solutions

As published in the Philanthropy News Digest:

We all know that philanthropic capital is uniquely positioned to play a powerful role in medical research. But fewer of us are aware of the importance of appropriately leveraged capital in creating impact and advancing promising research.

In 2010, philanthropies in the United States invested over $1.1 billion dollars in medical research. While an impressive number, it represents just 2.3 percent of the $140 billion invested by government, industry, and other groups in health and medical research that year. While funding for medical research has been growing incrementally on a year-to-year basis, public and private budgets are under increasing pressure, leaving a lot of promising research underfunded. In these tough times, therefore, it's more important than ever that we focus on doing more with our philanthropic dollars by investingsmarter.

Leading the flow of "passion capital" into medical research are a relatively new type of philanthropic entity known as "venture philanthropies" — organizations of a philanthropic nature that operate in a venture-capital mode. In addition to bringing a sense of urgency to the medical research community, venture philanthropies and the medical research organizations they partner with are beginning to change the financial infrastructure of medical research by providing risk capital for innovative research at critical (and traditionally underfunded) stages of drug development. These investments are also succeeding at drawing in more traditional capital, including public investment, which further leverages the original funds by expanding the pool of available capital at critical stages. Venture philanthropies also play another important role, as their close relationships with patient communities enhance their understanding of and expertise in specific diseases, resulting in forward-thinking research programs focused on reducing the overall impact of disease and solving problems most commonly faced by patients.

The fact that venture philanthropies are proving to be major players in the medical research arena does not mean that all foundations and philanthropic entities need to adopt the venture funding model in order to make meaningful investments that advance the medical research field. Indeed, at FasterCures we believe it is better to focus one's philanthropic resources on building a medical research investment portfolio that is strategic, intelligent, and...smart. And we want to help. To that end, we've created a pair of giving guides: Getting Started: The Medical Research and Development Primer and Giving Smarter: Building a High-Impact Medical Philanthropy Portfolio. Both are designed to help foundations and individual philanthropists better understand the medical research process, identify critical funding gaps, and explain the unique role of medical philanthropy in accelerating cures. They will also assist you in defining your organization's philanthropic priorities, evaluating the potential impact of your investments based on established criteria, and articulating the key questions you need to ask any research team, company, or nonprofit institution you are thinking about supporting or investing in.

In addition to our publications, we are continuing to facilitate the flow of philanthropic capital into medical research by convening major stakeholders at our annual meeting, Partnering for Cures, which this year will be held November 6-8, in New York City. At this year's meeting, more than eight hundred venture capitalists, philanthropists, policy makers, biotechnology and pharmaceutical company executives, patient advocates, and medical researchers will gather for a series of engaging panels and one-on-one meetings, with the ultimate goal of advancing the field of biomedical research and finding cures to a range of diseases. Partnering for Cures is uniquely positioned to introduce new and emerging foundations and philanthropists to the fundamentals of medical philanthropy and to provide more seasoned givers with complete due diligence opportunities.

We hope you can be a part of Partnering for Cures — it is a unique effort that promises to jumpstart your due diligence process. We also encourage you to download the toolkit, which we have designed with your needs in mind. And we look forward to your working with us to develop the financial infrastructure and create the outcomes-focused approaches needed if we are to speed up the process of getting to much needed therapies more quickly.

Margaret Anderson is executive director of FasterCures/The Center for Accelerating Medical Solutions, an "action tank" working to improve the medical research system and speed up the time it takes to get important new medicines from discovery to patients. Prior to her appointment as executive director, Anderson was FasterCures' COO for five years, served as deputy director of the Academy for Educational Development, and led programs and studies at the Society for Women's Health Research, the American Public Health Association and the Congressional Office of Technology Assessment.

Paying for Tomorrow's Medical Breakthroughs

noreply@blogger.com (FasterCures) — Tue, 25 Oct 2011 15:42:00 +0000

By Margaret Anderson, Executive Director, FasterCures

How are we going to pay for innovation and who will pay? Two big questions.

Let me share a few things we are doing in these areas. It used to be that in discussions about filling the R&D pipeline so that treatments and cures would magically come out the other side, the conversation would linger on the FDA as the final frontier. Now in addition to issues around regulatory hurdles, the discussions have evolved and include:

1.) whether financing will exist to even “get” things into the pipeline AND then
2.) if approved, whether patients can get reimbursed.

These are critical considerations that need attention and solutions. FasterCures and the Milken Institute held a Financial Innovations Lab in July to explore financial tools and other ground-breaking approaches that are being used to advance initiatives in medicine and other industries. The Lab spotlighted models for partnering in research and development, and identified finance instruments and incentives. A few are described below.

• Fast Forward, a wholly-owned subsidiary of the National Multiple Sclerosis Society, is a venture philanthropy model that provides leveraged, philanthropic funding to translate academic research and further develop biotech research into new treatments. The technologies are identified and evaluated by scientific and business advisors. Fast Forward makes $250,000 to $1 million investments.

• Israeli Life Sciences Fund uses a standard venture capital-structured fund with the government and private sector as limited partners. The Israeli government, which takes the first loss before the other limited partners, has committed $80 million to boost returns for private investors.

• Flow-through shares, which originated in the Canadian resource industry, encourage higher-risk investments through tax incentives. Oil and mineral exploration companies issue these shares as a way to pass government tax deductions for exploration on to investors, effectively halving the risk of investment.

There was broad agreement that the medical research system has much to learn from financial models that have worked in other industries such as filmmaking, telecommunications, and oil and gas. But participants cautioned against simplistic analogies, noting the unique circumstances that define the medical research process. Science is unpredictable, and failure to fully vet a product has consequences that are measured in terms of life or death. More discussion will take place on a paper from this lab and the models at a breakfast session our upcoming Partnering for Cures meeting so stay tuned.

On the topic of “Who Will Pay for Tomorrow’s Breakthroughs?” we’ve organized a plenary panel at Partnering for Cures with a terrific lineup of panelists. Brook Byers of Kleiner Perkins Caufield & Byers; Ron Cohen of Accorda Therapeutics; Sir Michael Rawlins of the UK’s NICE; Ellen Sigal of Friends of Cancer Research and PCORI; and Reed Tuckson of United Health Group will all look at the role that insurance coverage decisions and insurers themselves may be playing roles that are farther upsteam in the R&D and investment process. They’ll also tackle whether patient groups can bring harmony to the marriage of comparative effectiveness and personalized medicine.

We need more discussion and most of all solutions on both the front end of financing biomedical solutions AND the back end in terms of who will pay – JOIN US!

A Virtual Meeting with FDA Commissioner Hamburg

noreply@blogger.com (FasterCures) — Thu, 20 Oct 2011 12:24:00 +0000

“A lot of regulatory uncertainty is grounded in scientific uncertainty,” said FDA Commissioner Margaret Hamburg at a FasterCures web meeting held earlier this week that brought together hundreds of leaders from all sectors in medical research.

Hamburg underscored the essential role of the agency in advancing biomedical innovation and assuring our nation remains at the forefront. She noted that the FDA needs to be at the cutting-edge of science across all areas and disciplines and to do so would require having both the in-house expertise and strong collaborations with external partners. Additionally, she said FDA needs new tools to increase understanding of disease and more importantly identify opportunities that could make a difference in effectively diagnosing, treating or preventing disease. These knowledge, tools, standards, and approaches are at the center of the FDA’s regulatory science agenda.

“The FDA has approved more medical products this year than it has approved in decades, and that it has done so with considerable flexibility and timeliness,” Hamburg said. These include groundbreaking therapies that treat lupus, hepatitis, and metastatic melanoma.

She said that though this trend is encouraging, it is important to recognize that the number of new drugs in the pipeline is not as robust. She noted the need to work collaboratively across sectors and bring stakeholders together to create a regulatory pathway that’s transparent, consistent, and predicable and one that builds on the best available science.

Reflecting on more than two years as head of an agency that regulates products that account for 25% of consumer spending, Hamburg said the FDA is evolving and doing more to communicate with external stakeholders in a transparent and efficient manner, having earlier conversations with sponsor companies to determine how products benefit patients, and developing new ways to ensure patient voices are brought into the decision-making process.

When asked about how the agency reconciles the need for science and balancing risk and knowing people's lives are at stake, Hamburg responded by saying that FDA is trying to achieve an integrated approach that emphasizes the safety of the public and health of patients. The agency carefully weighs all decisions based on the known risks and benefits in a systematic and dynamic process. She noted that FDA approves “a lot of drugs that have a lot of risk” when the benefit to the patient is great.

In a recent survey released by the National Venture Capital Association, the FDA and regulatory barriers were pointed to as reasons for capital to be invested outside the U.S. Asked to comment on this data, Hamburg acknowledged that regulatory barriers are a considerable concern to people investing in medical product development but noted that a big percentage of products fail before it even gets to the FDA. She said that venture capital investors will have to be willing to take a considerable amount of risk.

Noting that regulatory reform is necessary to increase investment, she underscored the agency’s efforts to make the regulatory pathway clearer, more consistent, and more predictable. She emphasized the need to apply business approaches to streamline and improve the regulatory process. For example, one of the things FDA is now doing is adopting totally quality management or lean techniques from other industries and applying them to targeted areas to improve process.

A number of webinar participants posed questions about synergy between the FDA and the National Institutes of Health. Hamburg said that it is important to think more creatively about how clinical research resources are used.

“We have to make sure that we have the kind of clinical trial networks and investment in training next generation of researchers that will support a robust research agenda,” she said. “We need to rethink how we structure clinical trials institutionally and organizationally and also how to innovate and develop new clinical trial designs to enable us to get scientific answers in a more timely, less costly way.”

Hamburg said this is the time to develop a national strategy to look at the continuum of factors to advance medical product innovations and development. “We have an obligation to work across sectors to ensure science and technology are leveraged in ways that matter to patients,” she added.

View an archive of this webinar.

Related Event:
Hear more from FDA leaders at Partnering for Cures, November 6-8, 2011, NYC. FDA officials Margaret Hamburg, Jeffrey Shuren and Stephen P. Spielberg are among the confirmed speakers to address mission-critical challenges in medical research. View the Partnering for Cures Program.

Q&A opportunity with FDA Commissioner Hamburg on Regulatory Science

noreply@blogger.com (FasterCures) — Thu, 13 Oct 2011 18:43:00 +0000

Have you heard about Regulatory Science at the FDA but you are not sure what it means for your work? We’re pleased to have FDA Commissioner Hamburg join us for a Q&A webinar on Regulatory Science Initiative Implementation. Come and learn more about how this effort could stimulate personalized medicine programs, develop medical countermeasures to protect threats to global security, and ensure agency capability to evaluate new, innovative technologies.
In the recently released report from FDA titled Driving Biomedical Innovation: Initiatives toImprove Products for Patients, Commissioner Hamburg said, “ It is clear that our nation is at an important crossroads, where the science before us presents unprecedented opportunities to create new and better medical products and promote better health for the public. But we must act now to capitalize on this groundbreaking science in order to bring safter and more effective treatments to American families and keep our position as the global leader in scientific innovation. “
Be part of this important and unique opportunity to learn more about the FDA’s vital work, and participate in the discussion. This webinar is open to anyone, so please share this with your networks.

WHEN:
Monday, October 17, 2011, 2pm (Eastern)

WHERE:
Register Now for the FasterCures Webinar: (required).

This Webinar is part of FasterCures’ Webinar Series on research and innovation in medical research.

Why Come to Partnering for Cures?

noreply@blogger.com (FasterCures) — Wed, 12 Oct 2011 18:49:00 +0000

For three years in a row, hundreds of leaders in medical research participate in Partnering for Cures to find partners and explore collaboration opportunities that are crucial to speeding up the time it takes to get important new medicines from discovery to patients. This year, it will be held November 6-8, 2011 in New York City.

We, the medical research community, recognize that we have to build a system that can meet and anticipate the needs of patients. To be able to do this, we know we must engage patients in the research process. But, what does this entail? What are we asking of patients? What do we need from patients? And, most importantly, what are we doing to bring them into the system?

Similarly, we know we’ll all be patients at some point. As patient advocates, what are we doing to make sure our voices are heard and our contributions impactful? How do we become a force that drives medical progress?

And, at a time of limited fiscal resources – both private and public – we must ask: who will pay for tomorrow’s breakthroughs?

We have thoughtfully structured Partnering for Cures to help us answer these critical questions.

Problem-solve with medical research leaders. Every panel discussion will be focused on addressing some of medical research's top concerns in concrete ways. The goal of each panel is not just to inform (or entertain) – but to leave you with real transformative ideas you can run with, best practices you can emulate, and lessons learned you can build from.
See collaboration in action. Hear case study presentations from 30 cross-sector programs with innovative paths to advance their R&D goals. A unique opportunity to get a pulse on trends and best practices in real time.
Get expert advice to help you make informed decisions. From structuring collaborations or deals to crafting appropriate intellectual property policies, experts are on-site for one-on-one, free consultation sessions to help address your challenges.
Find the partner who could make a difference. Whether you're looking for an investor or a collaborator, a customized partnering system allows you to easily find potential allies from all sectors of medical research.

Getting to faster cures requires academia, government, industry, investors, nonprofits, and patients to work together...No one can do this alone. But we need an easier way to find each other, explore opportunities to collaborate, and develop sustainable partnerships.

That’s why we’re doing this. And that’s why we hope you will come and join us.

Revving the Engine that Propels Us

noreply@blogger.com (FasterCures) — Thu, 29 Sep 2011 12:56:00 +0000

by Gillian Parrish, Manager, Alliance Development and Communications, FasterCures

Over the past few decades, the U.S. has created and refined an extremely productive framework for developing new biomedical innovations and bringing them to the marketplace. In fact, the biomedical sector directly and indirectly accounts for some 5 million U.S. jobs, including 1.2 million high-wage private sector jobs in pharma, biotech, medical devices, research and testing.

However, for the first time since the 1980’s, U.S. preeminence in biomedicine is in jeopardy, vulnerable to falling behind growing powers in Europe and Asia thanks to regulatory, policy, and funding efforts in those nations that offer incentives for innovation and support entrepreneurship in a way the U.S. does not.

The history of our country’s rise to leadership in biomedical innovation, and a suggested roadmap for overcoming the factors that now challenge our dominance in the global market, is the focus of a new Milken Institute Study, The Global Biomedical Industry: PreservingU.S. Leadership. Released last week at a Washington D.C. briefing co-hosted by the Council for American MedicalInnovation, the report offers specific policy recommendations that will ensure the industry continues to grow and flourish here, including:

Increase R&D tax incentives and make them permanent.

Cut corporate tax rates to match the OECD average.

Extend support for emerging biomedical research fields.

Provide adequate resources for the FDA and NIH to expedite regulatory reviews and clinical trials.

Leverage existing strengths in medical devices.

Build human capital for biomedical innovation.

Promote and expand the role of universities by adopting best practices in technology transfer and commercialization.

It makes a compelling case for action, and reinforces FasterCures’ long-held position that no one sector can do this alone. It will take collaboration – universities and businesses, corporations working together, private equity entrepreneurs willing to invest in high-risk, high-impact R&D models, innovative intellectual property arrangements, etc. – to maintain U.S. leadership in biomedicine.

“As recently as 1980, European companies dominated the industry,” said Ross DeVol, Chief Researchers at the Milken Institute. “Unless we act soon and act smartly, we’ll wake up in five to ten years with no products and nothing in the pipeline. This is our industry to lose.”

“The alarm this report sets off is very real,” said Jim Greenwood, President and CEO of the Biotechnology Industry Organization (BIO), at last week's event. “The fear that the U.S. is losing its competitive edge is well-founded. If we want to have more cancer cures than iPads, we have to invest in them.”

Strengthening the biomedical sector should be central to our country’s economic platform. Government, particularly the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), should work with the private sector to get more and better therapies to patients.

“The 21st century is going to be the century of life sciences,” said Stephen Ubl, President and CEO of the Advanced Medical Technology Association (AdvaMed). “The question is: will it be made in America or somewhere else?”

Many countries are building the infrastructure for biomedical research, and courting the companies who drive it. Our current leadership is in question, and comes with no long-term guarantee. But if the advocacy and policy communities can come together to tap into our nation’s most unique attribute – our collaborative, innovative spirit – we can take the decisive steps necessary to not only maintain, but grow our leadership in biomedicine.

Five reasons why you should be at Partnering for Cures

noreply@blogger.com (FasterCures) — Tue, 13 Sep 2011 11:48:00 +0000

Partnering for Cures is an opportunity to find allies who can help you advance your medical research goals, and a platform for solutions needed to accelerate progress.

Five reasons why you should register now and be in New York City on November 6-8, 2011:

Find the partner who could make a difference. Whether you're looking for an investor or a collaborator, a customized partnering system allows you to easily find potential allies from all sectors of medical research.
See collaboration in action. Collaboration is the name of the game at this meeting. Hear case study presentations from 30 cross-sector programs with innovative paths to advance their R&D goals. A unique opportunity to get a pulse on trends and best practices in real time.
Problem-solve with medical research leaders. Every panel discussion will be focused on addressing some of medical research's top concerns in concrete ways. The goal of each panel is not just to inform (or entertain) – but to leave you with real transformative ideas you can run with, best practices you can emulate, and lessons learned you can build from.
Diverse and dynamic panels include: • Regulating Risk: Clearing New Pathways for Progress at FDA
• Streamlining Drug Repurposing and Approval
• Philanthropy As Risk Capital
• Business Is from Mars, Academia Is from Venus
• Intellectual Property: The Lawyers' Debate
• Staying Afloat in a Sea of Data
• Convergence: The Death of Disciplinary Science
• And many more
Get expert advice to help you make informed decisions. Whether it's structuring collaborations or deals, developing marketing strategies, or crafting appropriate intellectual property policies, experts will be available on-site for one-on-one, free consultation sessions to help address your challenges.
Experts on-site to provide you with pro bono analysis and feedback on:• Strategic planning
• Structuring deals and collaborations
• Attracting philanthropic capital
• Communications and marketing
• Interacting with the FDA
Share your ideas openly. Expand your echo chamber and significantly increase your friends and family list. Speeding medical progress requires unconventional approaches from nontraditional allies. Partnering for Cures provides you with a safe haven to explore the possibilities.

For more information, visit www.partneringforcures.org.

RELATED CONTENT: Podcast of BIOTech Now interview with FasterCures executive director Margaret Anderson on the importance of partnering across sectors.

FDA’s Work IS Essential

noreply@blogger.com (FasterCures) — Wed, 07 Sep 2011 20:11:00 +0000

By Margaret Anderson, Executive Director, FasterCures

“The U.S. Food and Drug Administration provides essential benefits to consumers and patients, and there is no backstop, no other agency, that performs this critical work.” –Alliance for a Stronger FDA* ad, September 2011

No other agency touches American lives daily – consider the products we consume the first 30 minutes of each day– the list of products regulated by this one agency is long. And yet, despite such big responsibilities, its budget has not always kept pace.

As the US Senate Appropriations Committee considers funding levels for the agency. Here’s hoping that the Committee consider the vast needs of this chronically underfunded, but critically important entity:

- Increased funding at the FDA will allow the agency to bolster its scientific infrastructure and expertise and be able to more effectively take the baton of medical innovation from the medical research community and bring it into the marketplace, where scores of patients impatiently await access to potentially life-saving therapies and life-enhancing scientific advances.

A few weeks ago, FDA provided details on exactly how it will go about doing this through its strategic plan for regulatory science. Priorities include stimulating personalized medicine programs, developing medical countermeasures to protect threats to global security, and ensuring agency capability to evaluate new, innovative technologies.

In an editorial in The Wall Street Journal, FDA Commissioner Margaret Hamburg said that the agency is “actively taking steps to improve the clarity, consistency and predictability of our regulatory systems. At the same time, we are working with the broader scientific community to address issues of scientific uncertainty that slow medical-product development and review.”

- Increased funding at the FDA will support industries that are essential to our nation’s growth and prosperity and create high-paying American jobs. Consider that the bioscience industry, which depends on FDA for regulation of its medical device and therapeutic products, directly employed 1.42 million people in the United States in 2008 and generated an additional eight million related jobs**.

An FDA that’s functioning at peak performance can spur economic growth and accelerate the process of bringing to market promising breakthroughs that can help improve the quality of life, and even save lives.

*FasterCures is a member of the Alliance for a Stronger FDA. Margaret Anderson, FasterCures’ executive director is the Alliance’s vice president.
** The U.S. FDA: A Cornerstone of America’s Economic Future

New Opportunities for Patients to Engage

noreply@blogger.com (FasterCures) — Mon, 29 Aug 2011 15:49:00 +0000

Patients are the most underutilized resource in medical research – and they have the most to gain. Two things this past month struck us as truly promising in the effort to build a research culture that encourages and inspires broader public participation.

1) The National Institutes of Health (NIH) invited patient advocacy and disease research organizations to partake in the strategic planning process for the NIH Common Fund. The NIH Common Fund was enacted into law by Congress through the 2006 NIH Reform Act to support cross-cutting, trans-NIH programs that require participation by at least two NIH institutes or centers and that can be achieved over a 5-10 year period. This program supported NIH initiatives in nanomedicine, molecular libraries and imaging, the Clinical and Translational Science Awards, and the newly formed Center for Regenerative Medicine.

With this request for input, NIH is seeking innovative and transformative ideas from the outside community to drive the formation of new programs to be funded in 2013. This provides an opportunity for patients to help NIH clinical researchers prioritize novel research ideas and collaborations likely to drive new solutions to medical challenges. NIH lists a host of concepts on which they are seeking input, from artificial organs to Big Data initiatives to mobile health technologies. With this announcement, NIH is providing the public with an opportunity to peruse its strategic planning site and share its thoughts on the Nation’s biomedical research plans for the future. It is NIH’s hope that public input will help drive innovative new ideas through the Common Fund.

2) The Department of Health and Human Services (HHS) recently slated an update of the requirements for protecting human subjects in research – referred to as the Common Rule. This update, the first major overhaul in the 20 years since the regulations were enacted, is designed to enhance protection for research subjects while and reducing administrative burdens, delays, and ambiguity for researchers. The Common Rule covers a number of areas specific to Institutional Review Boards (IRBs), which review the ethics of research, and the need to obtain informed consent from research subjects.

HHS is requesting input on a number of key provisions. The department is proposing to simplify the informed consent process and allow consent on biospecimen research to extend broadly to future new studies. Stored biospecimens are an important source of information and material for researchers, and the reuse of existing data and materials can be an efficient resource for a new research study. If appropriately modified, this rule would respect patients’ contribution to science, as well as their rights and welfare, while removing unnecessary timely and costly administrative burdens to researchers.

In addition, in terms of oversight, HHS is proposing to restructure the missions of IRBs by prioritizing their workload based on the risks posed to subjects. The department also is evaluating the use of a single IRB of record to oversee studies conducted at multiple sites. Such modifications could greatly improve efficiency in coordinating research studies.

In this new era of transparency, patients and the broader public have an opportunity to redefine their role in research by sharing their ideas for innovative models of research and sharing perspectives on how to improve the informed consent process. These two opportunities are opening the door to more meaningful public input in the medical research enterprise.

At FasterCures, we are strong proponents of working to improve our medical research system to better work for patients. Similarly, we urge the public, and especially patients, to become engaged, proactive participants in the process.

A few months ago, we released the report Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, which highlights how people affected by HIV/AIDS rallied together and created an advocacy movement that demanded change and got results. We have much to learn from HIV/AIDS activists, how they redefined patient engagement in the medical research process, and changed the drug development paradigm. Thanks in large part to the groundwork they laid, the public is increasingly viewed as an essential ally and partner in advancing medical research.

RELATED EVENT:

REGISTER NOW for a free FasterCures webinar
Bits and Bytes: Making Patient Information Available and Meaningful to Researchers
September 16, 2011
1:00 - 2:30 p.m. (Eastern)

Three distinguished leaders will discuss opportunities for accelerating research through improvements in biobanking, the use of electronic medical records, and the release of personal health information for research. Don't miss this important discussion!

Speakers:

David Blumenthal, M.D., Director, Institute for Health Policy, Massachusetts General Hospital and Partners Health System; former National Coordinator for Health Information Technology

Carolyn Compton, M.D., Ph.D., Director, Office of Biorepositories and Biospecimen Research at the National Cancer Institute

Sharon F. Terry, President and CEO, Genetic Alliance

Moderator: Margaret Anderson, Executive Director, FasterCures

Fixes in Financing Needed to Bridge the Valley of Death

noreply@blogger.com (FasterCures) — Wed, 03 Aug 2011 20:37:00 +0000

Turning medical science into medical solutions requires innovators to leap all kinds of hurdles, but one of the biggest is the financial “Valley of Death,” where many underfunded, early-stage ventures meet their demise.

To help bridge this funding gap, dozens of investors and innovators convened for a Financial Innovations Lab to explore financial tools and other ground-breaking approaches that are being used to advance initiatives in medicine and other industries. Organized by FasterCures; and the Milken Institute, the Lab was designed to explore whether these financial tools could be applied to translational medical research.

The Lab spotlighted models for partnering in research and development, and identified finance instruments and incentives, some of which are described below.

Distributed Partnering Model utilizes product definition companies (PDCs) that identify and license promising early-stage assets from research institutes, manage product definition research, and sell de-risked assets to later-stage stakeholders. The PDCs make $3 million to $5 million investments aimed at attracting additional funding.

BioPontis Alliance is a hybrid investment fund and product development company that aggregates technologies from a consortium of universities, screens these assets, develops them through a translational development network, and then licenses them to strategic pharmaceutical partners. Their activities are supported by a $50 million fund. The model encourages sourcing of early-stage assets from academia by pooling and sharing the value of intellectual property, and helps ensure later-stage funding by establishing relationships with pharmaceutical companies. This proposition is made more attractive to universities via a master IP licensing agreement where the process is expedited and universities receive a pro-rata share of the total value created by the assets.

Fast Forward, a wholly-owned subsidiary of the National Multiple Sclerosis Society, is a venture philanthropy model that provides leveraged, philanthropic funding to translate academic research and further develop biotech research into new treatments. The technologies are identified and evaluated by scientific and business advisors. Fast Forward makes $250,000 to $1 million investments.

Israeli Life Sciences Fund uses a standard venture capital-structured fund with the government and private sector as limited partners. The Israeli government, which takes the first loss before the other limited partners, has committed $80 million to boost returns for private investors.

Flow-through shares, which originated in the Canadian resource industry, encourage higher-risk investments through tax incentives. Oil and mineral exploration companies issue these shares as a way to pass government tax deductions for exploration on to investors, effectively halving the risk of investment.

There was broad agreement that the medical research system has much to learn from financial models that have worked in other industries such as filmmaking, telecommunications, and oil and gas. But participants cautioned against simplistic analogies, noting the unique circumstances that define the medical research process. Science is unpredictable, and failure to fully vet a product has consequences that are measured in terms of life or death.

NCATS: Increasing the odds of getting to faster cures

noreply@blogger.com (FasterCures) — Mon, 18 Jul 2011 15:26:00 +0000

By Margaret Anderson, Executive Director, FasterCures

This is old news: the drug development paradigm takes too long, it costs too much, and it’s laden with failures.

But for the thousands of people diagnosed each day with a disease for which there are no cures, or limited treatment options, the reasons – real as they may be – pale in comparison to the great need for living a healthy and productive life.

Of course we need to continue spotlighting the “broken R&D system” and need for a “new business model”. Identifying the problem is half the battle. But what are solutions to these problems? That is what matters most.

Solutions can be tough to develop, and tougher to implement. In the pursuit of solutions, we may second guess: Have we picked the “right” one? How can we hedge against risks? What about failure? Who is on board? Is it the right time to implement?

The road to success is paved with good intentions. It is clear that actions to address these vexing challenges will require bold vision, steadfast leadership, and diverse support. Thanks to our vitally important national investment in scientific discovery, we now know more about disease and biology than ever before.

The opportunity we must seize now is contingent upon having systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients’ health and quality of life. If indeed we are in an era where the scientific knowledge and possibilities are abundant but they are getting stuck in the translational pathway, we have to get moving.

The proposed NIH National Center for Advancing Translational Sciences is one way to get moving. Knowing full well that there are no guarantees in the pursuit of treatments and cures, this proposed center was conceptualized to reengineer the process of developing diagnostics, devices, and therapeutics to increase our odds of success, and streamline a process to make it work better for all sectors and across all diseases.

In a paper published last week, NIH Director Collins laid out the possibilities that his proposed center could yield to reengineer the process of translational research . Among the specifics, he noted that the proposed NCATS will:

support broadly applicable rather than disease-specific target-validation approaches and the investigation of nontraditional therapeutic targets that are considered too risky for industry investment.

encourage innovations in chemistry for drug delivery, such as nanoparticles; imaging agents for use as biomarkers; and detection technologies for use in diagnostics.

aim to develop more reliable efficacy models that are based on access to biobanks of human tissues, use of human embryonic stem cell and induced pluripotent stem cell models of disease, and improved validation of assays.

serve as an honest broker for matchmaking between compounds that have been abandoned by industry before approval and new applications for which these compounds might show efficacy.

support innovative designs for testing combination therapies, as optimal treatment of many diseases is likely to require multiple therapeutic agents.

All of these are essential to all the sectors, and the NIH has the ability to share the insights and system improvements coming out of this work across every entity in the medical research system. But the NIH can’t go it alone. The entire medical research community must come together to ensure this proposed center does not become part of the road paved with good intentions. And we urge our Congressional leaders to support the establishment of this important effort.

Patients need these improvements in translational research, and that means you and me.

Relevant Links:
1) Science Translational Medicine Commentary: Reengineering Translational Science: The Time Is Right
2) Podcast (MP3 - Dr. Collins summarizes the goals and functions of the proposed National Center for Advancing Translational Sciences)

Building Bridges across the Valley of Death

noreply@blogger.com (FasterCures) — Tue, 05 Jul 2011 14:35:00 +0000

Gillian Parrish, Manager of Alliance Development and Communications, FasterCures

I spent the early part of last week at the BIO2011 Convention, where the message on medical research seemed to be that there is cause to be optimistic about the future of drug development, but that in order for patients to see the payout of groundbreaking science, truly collaborative, outcomes-oriented partnerships – and a willingness to take risks – are key. On Wednesday afternoon, a panel moderated by our own Margaret Anderson explored strategies for crossing the valley of death – the gap between a promising discovery in the lab and the point at which a company is willing to pick it up and moved its development forward – to fund innovative new medicines.

“What’s missing today is a sense of urgency,” said Dr. Ellen Feigal, Vice President for R&D at the California Institute for Regenerative Medicine. “Research cannot just be about fostering knowledge and understanding anymore. It has to be about translating that knowledge into products, and then working with industry to move those products into the clinic.”

Neil Warma, President and CEO of Opexa Therapeutics, emphasized the need to communicate around the long-term horizons of science – describing to funders and partners in clear terms the impact of research on patient’s lives five or more years down the road, and then demonstrating discernable progress along the way to keep enthusiasm and energy up. “The education process is long,” he said, “so it’s important to bring an entrepreneurial mindset to everything you do.”

“If you don’t have any failures, then you aren’t taking enough risk,” noted Dr. Bob Beall, president and CEO of the Cystic Fibrosis Foundation. He talked about how CFF’s successes – more than 30 drugs in the development pipeline, four of them already FDA-approved – have been a result of holding its partners’ feet to the fire, and keeping patients at the table every step of the way to create the urgency Feigal talked about.

But it goes beyond just having a seat at the table. While patient presence is critical to humanizing research and speeding progress, patient voices can’t just be loud, they also have to be smart on the science and knowledgeable about the regulatory environment. The recently released paper Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, co-authored by FasterCures and HCM Strategists, distills lessons learned from the HIV/AIDS movement that can be replicated to address today’s medical research advocacy challenges. HIV/AIDS activists were successful because they refused to accept that the system was unchangeable, and instead took the time to figure out what it should be and how it should work.

“You have to keep going in the face of failure,” said Charles Queenan, Senior Vice President of New Ventures at the Juvenile Diabetes Research Foundation. “Failure is inevitable, but progress takes time.”

By taking a step-wise, incremental approach to risk, managing expectations from the get-go, and embracing the fact that research is not unidirectional, progress is not only possible, but fully attainable.

As John Mendlein of aTyr Pharma and Fate Therapeutics has proven time and again – you have to make a big bet by finding technologies that can shake up medicine, adding capable people and adequate venture financing, and creating a team-oriented culture to get promising science across the finish line and into the hands of patients.

US Faster than Europe When It Comes to Approving Cancer Drugs

noreply@blogger.com (FasterCures) — Tue, 28 Jun 2011 14:57:00 +0000

Adam Clark, Ph.D., Director of Federal and Scientific Affairs, FasterCures

In the world of drug development, there may be a tendency to believe the grass is always greener on the other side of the fence – or in the case of U.S. Food and Drug Administration, on the other side of the pond. Recent Congressional hearings have heard testimony on the slow pace of FDA approval of medical devices in the US compared to Europe.

A new study challenges these preconceptions and offers evidence that the FDA is in fact more efficient than its European counterpart in reviewing and approving new drug applications to bring new oncology drugs to market.

An analysis conducted by Friends of Cancer Research published in Health Affairs compared the review times of oncology drugs submitted to the FDA and the European Medicines Agency (EMA) between 2003 and 2010. The study showed that oncology drugs applications in the US were approved at approximately six months, while oncology drugs in Europe took nearly a year. In addition, this same study found that the FDA approved 32 oncology drugs over this time period compared to 26 by EMA.

Unfortunately for regulatory agencies like the FDA, it only makes headlines when something goes awry – safety alerts, food recalls, warning labels, and so forth. And headlines tend to shape public opinion, and in turn, could ultimately influence public policy. But the reality of the FDA’s task at-hand, regulating 25% of every consumer dollar is a tall order for any one agency. But there is no backstop, no other agency, that performs its critical work.

FasterCures is a member of the Alliance for a Stronger FDA, a broad-based coalition focused on ensuring Congress adequately funds FDA's essential missions. We need a strong and robust FDA to protect public health and safety and ensure the U.S. continues to pave the path to medical progress – and be able to apply lessons learned from cancer drug approvals across all other diseases.

Relevant Links:
• Roll Call Op-Ed: Sigal: FDA Requires Support, Funds to Continue Research
• NYT - U.S. Approves Cancer Drugs Faster Than Europe, Study Shows
• Exclusive: Cancer Drugs Reach U.S. Market Before Europe: Study

What Have We Learned from HIV/AIDS Activism? The Basics Make All the Difference

noreply@blogger.com (FasterCures) — Mon, 20 Jun 2011 20:27:00 +0000

Margaret Anderson, Executive Director, FasterCures

What a privilege it was to lead a discussion with esteemed leaders and officials in the HIV/AIDS advocacy space about our new paper Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change. The paper, authored by FasterCures and HCM Strategists, was intended to distill lessons learned from the HIV/AIDS model so we could examine if they can be replicated to address today’s medical research advocacy challenges. Five elements of the model rose to the top: attention; knowledge & solutions; community; accountability; and leadership. Thursday’s forum delved into each of these elements, and reminded us that while challenging the system is a Herculean task, even under the most difficult circumstances, it is absolutely possible to achieve change.

As my colleague from HCM Strategists and collaborator on this effort Michael Manganiello said in his opening, “If it weren’t for the leaders of this movement, I wouldn’t be standing here today.” I pointed out how personal this issue is for so many, and how many people who have died from HIV/AIDS paved the way. But, as Manganiello offered, “We don't question status quo anymore. We should not accept that the system is unchangeable.”

HIV/AIDS activists were successful in turning a cause into a movement because they were organized and knowledgeable about the issues, not just loud. It started with fear and anger, which led to theater, which led to getting smart on the science – and it was that combination of factors that was absolutely key. Getting to the table does not equal getting attention. We asked whether advocates today are lulled into a sense of complacency by sheer virtue of the fact that we are “invited in”. Are advocates part of a box that gets checked instead of a voice that gets heard?

We need to stop accepting that the system is what it is and focus on what it should be. Maureen Byrnes who was executive director of the National Commission on AIDS, had two important messages for all advocates today, “Be specific, and don’t wait to be invited.” Brenda Lein who was a member of ACT UP and worked with Martin Delaney at Project Inform said, “Getting angry isn't enough. That's where it starts, but then you need to understand process and be open to solutions.” She has since worked with other disease populations including breast and prostate cancer to help share the lessons learned.

Patients, not just patient groups or scientific representatives, must be at the center of advocacy efforts – and they need to invest time and energy into understanding the scientific, policy, and regulatory issues and how to address them. Byrnes asserted, “Success is not just asking for change, but understanding the system and thinking about what a solution looks like.” This report offers up an opportunity for all disease advocates to figure out how to apply this to their community.

So, what was the advice for today’s advocacy? Dr. Tony Fauci, director of the National Institute of Allergy and Infectious Diseases asserted “If you really want to shake cages you have to be persistent. This is very different than coming out for a meeting once a year. We knew [the HIV/AIDS activists] weren't going away.” Dr. Jim Curran who ran the HIV/AIDS Division at the Centers for Disease Control and Prevention and is now Dean at the Rollins School of Public Health said “As an activist, you have to have a certain amount of threat you bring to a meeting.”

I think what struck me most during this discussion was the shared insight that both Fauci and Curran offered: activism is NOT personal. Whether it was the 20,000 postcards with unfriendly images being delivered to your home, or full-page NYT ads saying you were an idiot, or relentless noisemaking and disruption at meetings, they both spoke over and over about how “they aren’t just numbers, they are people.” Leadership was one of the elements of the model we offered in the paper, and I think those who have worked with these officials, have benefited from their tireless commitment to getting things done, or have heard them speak can say they embody just what you want and need in a leader.

So, I urge everyone to read this report, and analyze how you can apply the elements that defined HIV/AIDS activism to advance your solutions. They’re basic but it’s a roadmap for how to achieve meaningful change. As Larry Kramer, founder of ACT UP said in our interview with him, “You always have to be in charge, and you have to be on top of it, and you have to not give up. It is never ending, and it is day after day after day after day, and it’s exhausting. And it really doesn’t work unless you realize that. It doesn’t’ work if you don’t do it every single day.”

Onward. Let’s get back to basics.

Relevant Links:

WSJ Health Blog: Lessons From AIDS/HIV Advocacy Efforts

Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

More Researchers Can Now Access Nation’s Research Crown Jewel

noreply@blogger.com (FasterCures) — Mon, 20 Jun 2011 11:10:00 +0000

By Kristin Schneeman, Program Director, FasterCures

We noted with interest a post from Sally Rockey, NIH’s Deputy Director for Extramural Research, highlighting progress being made in the NIH Clinical Center’s effort to open its doors to greater use by the extramural research community. We are fans of the Clinical Center, the nation’s largest and most sophisticated research hospital, and have long advocated that its resources should be available to and used by researchers outside the NIH’s Intramural Research Program (IRP) in addition to the Intramural Research Program (IRP) researchers it has historically served.

In 2008, FasterCures convened a task force chaired by Nobel Laureate Dr. David Baltimore, which called for the IRP to adopt a new mission that is outcomes-focused, is capable of responding quickly to new opportunities and challenges, and fully utilizes its world-class research hospital and other infrastructure. Among the task force’s key recommendations was that the Clinical Center be fully utilized (at the time it rarely reached 60% occupancy), and that NIH create streamlined mechanisms by which extramural researchers and industry can more fully use the Clinical Center for projects in collaboration with the IRP.

In 2010 FasterCures and 86 other patient organizations sent a letter urging the NIH’s Scientific Management Review Board to open up the Clinical Center facilities to external researchers. As the nation’s research crown jewel, it has the potential to excel in research efforts focused on rare and orphan diseases and on pre-clinical and methods research essential to building tools, platforms, and protocols for the entire clinical research enterprise.

We are very pleased to see that NIH continues to move forward with this important effort. We urge all stakeholders – patient groups, academic researchers, and industry alike – to take Dr. Rockey up on her offer “to provide input on your interest in accessing the NIH Clinical Center resources.” If they build it, will you come?

TIME Magazine and The Chronicle of Philanthropy Spotlight FasterCures’ Efforts to Step Up Medical Progress

noreply@blogger.com (FasterCures) — Thu, 09 Jun 2011 16:18:00 +0000

By Margaret Anderson, Executive Director, FasterCures

Speeding the pace of medical research by strengthening the connections between the people who can make it happen is what FasterCures all is about. We look for big ideas in small spaces and encourage financial and strategic risk-taking to turn discoveries in the lab into cures for disease. Among our greatest privileges has been our work with and development of The Research Acceleration and Innovation Network (TRAIN) program, a new class of medical foundations known as “venture philanthropies” – organizations of a philanthropic nature that operate in venture-capital mode – which are bringing a sense of urgency to the rest of the medical research community. By measuring the effectiveness and efficiency of a medical charity’s scientific outcomes, dollars can better be directed to projects and organizations making the greatest progress.

This model of cure entrepreneurship has begun to take root across diseases and sectors, and both TIME magazine and The Chronicle of Philanthropy have recently taken notice.

This week’s TIME magazine features the article “Check Your Charity!” about the lack of market discipline in the medical philanthropy space and the push to better measure and compete on scientific outcomes. The piece highlights organizations that are applying a results-oriented approach to their work, like the Multiple Myeloma Research Foundation (MMRF), Accelerate Brain Cancer Cure, and the Melanoma Research Alliance – all part of the FasterCures TRAIN program. “Critically, FasterCures has sought to link these evolving groups . . . . It's trying to spread best practices across a host of health care groups, a break from the past in which researchers and organizations shielded their work to protect their grant money and intellectual property.”

A recent Chronicle of Philanthropy article calls out other TRAIN members such as The Chordoma Foundation and the Cystic Fibrosis Foundation, for their innovative approaches to accelerating medical progress. The piece notes that “FasterCures amplifies the work of groups that have taken risks and been successful;” and that “by getting organizations and companies that are used to competing to instead work together, FasterCures hopes to break down walls that may slow the achievement of medical discoveries that could save lives.”

Check out the full articles here:

• “A Think Tank Seeks to Accelerate Medical Science’s Search for Cures”
• “Check Your Charity!”

For more on TRAIN organizations, initiatives, and best practices go to TRAIN Central Station, the online portal for and from venture philanthropy in medical research.

TRAIN Group at Center of New NIH Collaborative Effort

noreply@blogger.com (FasterCures) — Fri, 27 May 2011 13:42:00 +0000

Kristin Schneeman, Program Director, FasterCures

FasterCures aims to expedite cures by stimulating innovative collaborations across all sectors. We recognize that a collective approach is needed to successfully address the barriers to innovation. That is why we were fascinated to note this week the announcement that NIH has just signed a cooperative research and development agreement (CRADA) to move promising therapies for rare blood cancers into clinical proof-of-concept studies. This is an excellent example of collaboration that we’d like to see more of in the future.

While NIH has engaged in CRADAs for many years to make government facilities, intellectual property, and expertise available to further the development of marketable products, this one caught our attention because of the involvement of the Leukemia & Lymphoma Society, a major funder of blood cancer research. The press release announcing the agreement explicitly noted the role that philanthropic and patient organizations have in the drug discovery process. Also engaged in the collaboration is the University of Kansas Medical Center.

The agreement is notable to us also because NIH is viewing it as a “Learning Collaborative,” the goal of which is “to bridge the gap in time and resources that often exists between basic research and human testing of potential new treatments.” “We plan to learn more about and improve the drug development process for all diseases,” says Chris Austin, director of NIH’s Therapeutics for Rare and Neglected Diseases program, which is spearheading this effort.

FasterCures, through our TRAIN program, has long been a proponent of the important role that patient-driven philanthropic organizations can play in funding innovative approaches to medical research and in helping move promising discoveries across the translational “valley of death” toward new treatments. They bring not just risk capital but also the voice and priorities of patients into the research process.

This agreement is part of a heartening trend (no pun intended) at NIH to play a more active role in bridging the gap between the many promising discoveries it funds and the point at which biotechnology or pharmaceutical companies are willing to invest in expensive late-stage clinical research. We are pleased to see that Leukemia & Lymphoma Society has a seat at the table in this effort, and we hope to see more collaborations of this kind in the near future. We look forward to learning along with The Learning Collaborative about how government, academia, nonprofits, and ultimately industry can work more efficiently and effectively to get badly needed new treatments to patients.

The Data Deluge

noreply@blogger.com (FasterCures) — Tue, 17 May 2011 16:47:00 +0000

Adam M. Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures

Earlier this year FasterCures put together a Top Ten list of Medical Research Trends to keep an eye on in 2011. Coming in at number 5 on the countdown was Where is our GPS: Mapping the pre-competitive space. The trend outlined whether we could create a "marketplace" that will help facilitate the exchange of pre-competitive information and communal data sharing.

Recently, I saw first-hand a plan in action to work on this issue. I was among the participants at the second annual Sage Bionetworks Commons Congress. Sage Bionetworks is a new medical research organization with a focus on understanding biology as a network science and biological research as a community effort. Sage’s approach - shift to data intensive science built upon open access and broad collaborations to design network maps of disease built upon vast databases linking genotpyes, treatments, and medical outcomes.

Genomics is moving biology from an observational science to an informational science. Our way of thinking of disease is shifting from one gene, one disease to multiple genes with numerous phenotypes. In fact, diseases like cancer are now considered to be hundreds, if not thousands, of different diseases with a variety of underlying genotypes. This shift in understanding disease also means we need a shift in researching them as well. Finding cures for complex diseases in the 21st century will require us to fundamentally rethink how we conduct biological research.

Sage Congress participants noted that scientific disciplines such as physics and astronomy have benefited tremendously from data sharing and collaboration to build a fundamental new understanding or the universe. Similarly, the computer industry learned decades ago that open source access to software accelerated innovation, allowing tailoring of new applications to reach broader consumer markets. However, the field of biology has not yet undergone such a cultural shift. Despite technological revolutions in genomics and information technology, its approach to data sharing and knowledge generation still remains largely confined to closed networks and research silos with a focus on journal publications.

Sage is attempting to challenge this traditional paradigm by building a federated, open network commons for researchers to share large data sets, protocols, and research findings (positive or negative) to encourage collaboration and cross discipline interactions. This model supposes that the next biomedical revelations will be data driven and will require diverse talents and a community approach to research. Sage is currently underway building a data repository and the Congress featured a demo of Synapse, a portal that will ultimately include the repository, a cloud compute component, and tools for collaboration and workflows.

This is a decidedly different approach to biology, one that will require support from the research community as well as the patient organizations and the public. However, if this new world of data-driven medical science is to be a success, funding agencies and academia will also need to conform to the new reality. Incentives will need to shift to promote open access to data and collaborative science, both in terms of funding and career promotion.

It was noted at the conference that biomedical research is not so much limited by technology as it is by its culture. And cultural change can be extremely difficult. But if efforts like the Human Genome Project are any example, outcome-oriented research and collaborative scientific enterprises can transform our understanding of biology and with it, our ability to treat and to cure diseases. And at FasterCures, that is the bottom line.

Related Resource:
FasterCures webinar on “Data Sharing Policies and Opportunities for Nonprofit Funders of Research?” View an archive online.

Biomedical Innovation: Can the U.S. Keep Its Edge?

noreply@blogger.com (FasterCures) — Tue, 10 May 2011 12:28:00 +0000

by Adam Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures

Over the past decade, international investments in life sciences research and development (R&D) in the in the private-sector have shifted away from the U.S. An expert panel at the 2011 Milken Institute Global Conference examined this shift and explored policies that influence biomedical innovation in the U.S.

Ross DeVol of the Milken Institute framed the issue by noting that it was not until the late 1990s that the U.S. began to dominate global pharmaceutical R&D. U.S. leadership can be attributed to a number of factors, he said, including a more predictable regulatory framework and adoption of intellectual property policies such as the Bayh-Dole Act, which encouraged entrepreneurship. However, DeVol said that “our regulatory approval process has gotten more cumbersome,” which has resulted in a risk-averse environment that inhibits medical innovation.

Edward Holmes of Singapore’s Agency for Science, Technology, and Research (A*STAR) said that Singapore has made the biomedical industry a top priority and developed policies that have resulted in a four-fold growth in the biomedical industry in the past decade. Such policies created an environment with sound intellectual property strategies, efficient regulatory programs, effective ethical frameworks for clinical trials, and an educational infrastructure that strongly supports science and math education to secure a strong scientific workforce. Singapore continues to focus on biomedical manufacturing and a strong effort to build up efforts in R&D.

Michael Leavitt, former Secretary of the U.S. Department of Health and Human Services, emphasized that the next decade of U.S. biomedical innovation will be built upon the “Era of the Value Proposition” in which “the future innovation will have to involve a value proposition that will demonstrate to a payor that the innovation will result in a change in the cost curve.”

Bijon Dorri of General Electric, explained GE’s interest in the healthcare sector and highlighted GE’s Healthymaginations program as an area in which GE is increasing its global competitiveness in healthcare. For projects supported through the Healthymaginations, it must address at least one of three pillars of healthcare: reduce cost, improve quality, or increase access. These guiding principles, he said, are “driving a change in the value proposition of healthcare innovation.”

The panel discussed the growing costs of clinical trials as one of the primary drivers to move R&D out of the U.S. to countries where such costs are significantly lower.

Collaboration was another key theme the panel deliberated on. Partnerships between the private and public sectors are key to moving therapies forward. There needs to be open communication channels between industry and the National Institutes of Health, the FDA, and the Centers for Medicare and Medicaid to ensure a seamless continuum of R&D innovation from discovery to development to dissemination and access.

Lesa Mitchell of the Kauffman Foundation moderated this panel. Watch a video of this session.