Statement from Margaret Anderson, Executive Director, FasterCures on the New NIH-FDA Partnership to Speed New Treatments to Patients

FasterCures applauds the U.S. Food and Drug Administration and the National Institutes of Health on launching an initiative that will fast-track the biomedical research and development process to deliver safe and effective treatments to patients sooner. This formalized effort to integrate and enhance two critical disciplines - translational science at the NIH and regulatory science at the FDA - that are fundamental to our medical research system, paves the way to an enterprise that will harness the promise of science and turn it into accessible medical solutions.

For all the human and financial capital flowing through our healthcare system, there remains a formidable list of diseases for which there are no cures or even meaningful treatment options. The coming together of the FDA and the NIH, complex entities with enormous priorities, to publicly commit to achieving a shared goal of “illuminating the pathway from microscope to marketplace,” is not only unprecedented, it is strategic, timely, and necessary to addressing patients' needs.

We believe that with Dr. Peggy Hamburg and Dr. Francis Collins at the helm of the newly created Joint Leadership Council, we will be able to actualize a more collaborative paradigm that will help connect the dots between discovery and development. We are pleased to see the initial resources to support this work, and hope that we will see additional funding carved out to strengthen and sustain this effort. And, we are delighted that the group is open to community input and look forward to participating in its first public meeting in the Spring.

This new initiative builds on some exemplary practices at FDA and NIH and is the first of many steps necessary to create an effective research enterprise focused on improving the lives of patients and their families. We congratulate Secretary Kathleen Sebelius, Drs. Collins and Hamburg for their leadership and vision – Americans for generations to come will benefit from this remarkable endeavor.

Translating the CTSA Forum

by Margaret Anderson, Executive Director, FasterCures
The CTSA Industry Forum meeting, convened by NIH's Clinical and Translational Science Awards (CTSA) consortium, was chock full of the type of commentary and discussion that has come to define any medical research meeting you go to these days. It was a two-day meeting held at NIH February 17-18 to look at collaboration among academia, government, and industry. Many of the topics raised could have been the focus of a week-long discussion. There were some presentations about CTSA initiatives. Following this meeting, I hope to see more and more coordinated information from the CTSA Consortium as fast as they have it. There is no time to waste since the issues raised represent some of the huge boulders standing in the way of drug discovery.

Let me start with the end of the meeting and work my way to the beginning because I think these points illustrate a key challenge.

• It's about the patients. We need to ensure that patients are placed at the center of these conversations. In a panel right before the closing remarks, Cystic Fibrosis Foundation CEO Bob Beall spoke about the need to look at patients and patient organizations as collaborators that cannot be overlooked. NIH Director Francis Collins did say in his opening presentation, “The person who is hurt by these silos in research is the patient,” but there was little patient focus in between these two bookends. If patients are not put at the center of research, the whole endeavor breaks down and we go from promoting efficient and effective collaborations to promoting the opposite. Bob Beall urged us to see how patient and disease research foundations can support and promote policy changes, build resource capacity, and generate public support and cooperation (among other things).
  
• Let's learn from examples. We have remarkable examples we can point to that elegantly illustrate Bob Beall’s point. AIDS activists transformed medical research in government, industry, and academia and changed how FDA conducted regulatory policy in the early days of the HIV/AIDS epidemic. Breast cancer advocates showed the power of organizing and, fundraising, and created training programs to educate champions so they could sit on scientific review boards. Now we see the power of venture philanthropy groups who are being looked at as the shining example of how a strategic mindset mixed with entrepreneurialism, tenacity and a relentless focus on outcomes can be applied to figure out the best way to do something can lead to systems change across all sectors.

Among the themes that percolated throughout the two days:

• So how do we cross the valley of death? We’ve all mastered the pictures for powerpoint, but now what? There seemed to be an interesting tension between the industry participants at the meeting and those representing CTSAs. Is innovation the purview of academia? I heard industry saying that a greater understanding of their targets and needs was required in order to close regulatory gaps and de-risk industry investments earlier. Misaligned and nebulous IP policies clearly continue to be a barrier, and the US is behind other countries/regions in creating and implementing successful pre-competitive partnering models. Defining what collaboration looks like amongst government, academia, industry and patients is still an ongoing endeavor, and one speaker noted that the motivations of the various parties both converge and diverge throughout research and drug development. Francis Collins referenced the Pharmaceutical Assets Portal project out of UC Davis as one example and potential antidote to getting more shots on goal. (Note: I don’t know my sports metaphors, at all. Just ask my husband and he will confirm. So when I start to understand one, we know we’ve reached the saturation point!).
  
• If venture capital is focusing on later stage investment, and is looking for an exit strategy, what are our options? Elizabeth Stoner of MPM Capital spoke about the lack of liquidity in 2009, and how there were fewer dollars invested and fewer deals made. Pay attention to NIH’s TRND and BRDG-SPAN programs, examples of NIH stretching into that valley. But if 25 percent of biotech companies are operating with less than six months worth of cash, it’s a problem.
  
• Conflict of interest is a burning issue. Whether real or perceived, it is a super-sized elephant in the room, and I appreciated the attention to the issue at this meeting. One speaker pleaded with participants to stop focusing on conflicts and instead focus on synergies of interest. Patients want treatments, so we need to collectively identify the solutions to any problems with conflict, and then move on to implementing them.
  
• The importance of regulatory science. FDA came up many times over. FDA needs more money to do its work, and it needs more tools in its toolkit to be prepared for innovation in the pipeline. FDA needs resources to attract the best and the brightest, but is using science from the last century to do its work. This is not sustainable. FDA needs to be able to reach out more effectively to partners to help it stay abreast of the latest regulatory science. We need everyone’s voices on these issues, so CTSA researchers’ involvement would be welcome. (Note: I sit on the Board of the Alliance for a Stronger FDA, and I encourage everyone to join our efforts.)
  
• Discovery needs to use information better. There was interest in open source platforms, and access to data was cited repeatedly. What low hanging fruit can the CTSA pluck off here?

Lots of fodder for all of us committed to improving our medical research system to chew on. Where do we meet next and what's on the agenda?

Advance Excerpts from Mike Milken's Closing Keynote Remarks to be Presented at the Russia Forum, February 4

Mike Milken on the Imperatives of Medical Science

Because human capital represents more than three-quarters of the world’s real assets, it follows that investments in education and health by governments, industry and individuals will provide the greatest returns. Over the past two years, the world has seen net financial losses in the range of 10 to 20 trillion dollars. But if we could eliminate death and suffering from heart disease and cancer – not curing them, but simply turning them into chronic, rather than fatal, diseases – the increase in global human-capital value would be more than US$200 trillion. That’s the estimated value of the resulting longer lives and higher productivity. In other words, small increases in the value of human capital more than offset any losses in recent financial markets.

This is one example of why medical research is an integral component of the global economy. In fact, health advances account for as much as half of all economic gains. After thousands of years of nearly imperceptible growth, the world’s wealth began doubling every few decades beginning in the last century and a half. The largest stimulus for this growth was increased human productivity made possible by improved health and extended life spans – the result of improved sanitation, disease prevention and cures produced by research laboratories. The advance of such technologies as genomics and proteomics give us hope that we will continue to make progress against deadly diseases everywhere around the world. That is why FasterCures, a center of the Milken Institute, is working to remove barriers to progress in medical research worldwide.

More on Mike Milken's remarks at the Russia Forum.