Tuesday, October 25, 2011

Paying for Tomorrow's Medical Breakthroughs

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!

Thursday, October 20, 2011

A Virtual Meeting with FDA Commissioner Hamburg

“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.

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.

Thursday, October 13, 2011

Q&A opportunity with FDA Commissioner Hamburg on Regulatory Science

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 ImplementationCome 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. 

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

Register Now for the FasterCures Webinar: (required).

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

Wednesday, October 12, 2011

Why Come to Partnering for Cures?

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.