This was not a gabfest for geeks. The inaugural “TransMed Partnership Forum” (www.transmedpartnership.com) held in September brought experts together to find solutions for accelerating delivery of promising medical research to patients. These are particularly tough financial times for early research, with government funding down and venture capitalists focusing on less risky, later-stage products. This was not all about money, however. The forum aimed to foster partnerships between divergent stakeholders to solve translational problems that clearly require integrated, multidisciplinary solutions.
Pivotal medical meetings tend to occur on the coasts, but this forum drew experts from across the U.S. and Canada to St. Louis, Missouri. The forum was founded and sponsored by the Ewing Marion Kauffman Foundation and the School of Medicine at Washington University (WU).
The forum had the atmosphere of a founders’ meeting with palpable energy. The organizers realized that integration of silos of knowledge is an important translational issue, so the format was interactive and designed to encourage communication. There were no slides, no data: just open discussions. Most of the agenda was comprised of 90-minute open discussions where little was held back about significant translational hurdles. These sessions quickly brought attendees in contact with like-minded stakeholders. These roundtables focused on translational challenges for the top-ten therapeutic areas and new research technologies, policies, and models for collaboration.
The plenary speakers tackled broad issues of personalized medicine, new funding models, and partnering with industry. The funding session was dedicated to the expanding role of philanthropic foundations in translational research.
Foundations are stepping up to fill the current funding gap and to provide translational education and guidance. Speakers provided a balanced perspective on funding strategies. Lesa Mitchell, Ph.D., vp, advancing innovation, at Kauffman, who provided vision and guidance for the forum, commented, “The critical role that scientists play in our economy is compromised because faculty and graduate students need a better understanding of commercialization to empower them to recognize and advance their own innovations.”
The keynote session, moderated by William Peck, Ph.D., director of the Center for the Study of Health Policy at WU, addressed how the modern health care economy will adapt to accelerating technological change. Dr. Peck presented the harsh reality of a complex U.S. health care system under siege. “Health care costs are high and rising, amounting to $2.2 trillion this year with an anticipated doubling by 2016 to $4.4 trillion,” he noted.
“The U.S. is spending more on health care than any other developed country in the world, but still there are 46 million uninsured and countless underinsured that do not have timely access to the best care possible.” He noted a crucial need to improve quality and challenged the panel to address how the health care system can improve itself and its economy.
Ralph Snyderman, M.D., James B. Duke professor of medicine and chancellor emeritus for Health Affairs of Duke University, looked to the consumer to play a bigger role and carry more of the financial burden. The greatest problem, he said, is that innovations are not translated into clinical practice and are used randomly and sporadically. His solution was to develop robust bioinformatic disease-delivery models that include personalization and prevention. He suggested that the Center for Medicare and Medicaid fund the development of several learning models for personalized prevention across the U.S.
Harlan Krumholz, M.D., Hines professor of medicine, director, Robert Wood Johnson (RWJ) clinical scholars program at Yale and director of the Yale-New Haven Hospital Center for Outcomes Research and Evaluation, suggested building health care reform on a foundation of new science, aiming to enroll large numbers of patients in trials. He also highlighted the importance of educating patients about discretionary care and the importance of shared decision-making.
“We should create a show-me culture that requires evidence of how best to practice,” Dr. Krumholz said. He envisioned collaboration between industry, academics, advocacy groups, and payers that would improve the way medicine is practiced. In later discussion, it was noted that involving more patients in trials would address the problem of force-fitting increasing amounts of foreign patient data to the U.S. population.
Peter Corr, Ph.D., an academic veteran and retired svp of science and technology at Pfizer (www.pfizer.com), thought that big companies should disaggregate to form smaller, more innovative groups. He pointed to the increasing number of precompetitive partnerships that bring worldwide data together and should make a huge impact on medical practice. “In the U.S., we do not have a health care system, we have a sick care system,” Dr. Corr remarked. “The biggest threat to innovation is the hype and regulation surrounding conflict-of-interest issues.”
Is the solution becoming a single-payer system like many other countries? Garry Neil, M.D., president of Johnson & Johnson (J&J) Pharmaceuticals Development (www.jnj.com), did not think so. Dr. Neil placed his bet on the laboratories of democracy in several states that are working on mandatory insurance schemes that would cover everyone. This solution would involve a combination of increased emphasis on health care and personal accountability, better end-of-life intervention systems, and utilization of information technology, more personalized medicine, and new disruptive therapies.
Collaboration was a recurring theme throughout. Stakeholders simply have to work together. In the plenary, Partnering Effectively with Industry moderator David Rosen, developmental and commercial head of strategic alliances at Pfizer, asked panelists to give real-world examples of successful partnerships between academia and industry.
The new president of the Epilepsy Therapy Development Project and long-term Yale School of Medicine researcher, Joyce Cramer, said, “The table is not balanced when academic scientists and investors initiate discussions about intellectual property (IP) with university tech-transfer people. Both sides have pockets of knowledge and vacuums of inexperience that create difficult negotiations.” She related an example of a deal that was on the rocks simply because the tech-transfer group was ignorant of what others have done and of a pricing and deal structure that would be competitive.
David Chen, Coulter program director, department of biomedical engineering at the University of Virginia, talked about the potential dichotomy between research and commercialization objectives. His example demonstrated that it is possible to accomplish both. For example, allocate translational research funds to do modest animal testing to advance commercialization but in parallel use other institutional funds to advance research by writing a follow-on RO1 to the NIH Research Project Grant program.
Joni Catalano-Sherman, Ph.D., corporate director of the corporate office of science and technology at J&J, talked about the company’s long-standing partnership with early researchers in academia. About $60 million of no-strings-attached gifts from J&J have helped over 300 researchers with proof-of-principle experiments.
Ward Peterson, Ph.D., vp of research and preclinical development at Inspire Pharmaceuticals (www.inspirepharm.com), used his company as a success story for in-licensing university technology. He stressed that the relationship begins, not ends, when the deal is signed. Inspire has had many win-win experiences by simply providing masked compounds for university labs to test. As IP is bolstered by the company’s work, investors recognize the value-added. Dr. Peterson said this was a sustainable business model because when IP is diluted into the academic institution it can hamper development. “All of the IP comes from the university, Inspire’s responsibility is to turn the technology into new molecular entities,” he summarized.
Another successful translation from university research to company formation was provided by Stuart Peltz, Ph.D., president and CEO at PTC Therapeutics (www.ptcbio.com), who negotiated his company’s IP out of his own lab at the RWJ Medical School. Through a combination of funding vehicles the company accumulated over $200 million. “Companies can take a staged approach to building relationships with principal investigators. Let the PIs experience the risks and rewards to get them both excited and humbled about the road from concept to full commercialization,” he suggested.
The conversations outside the sessions were equally valuable. There were about 230 attendees, with over 40% of the attendees being M.D.s, Ph.D.s, or both. It was a gathering of some of the most intelligent people on the planet. Conversations were eager and substantial and often started by the single phrase: “Tell me about your work.” These conversations helped advance the forum’s goal of fostering collaborations among this vanguard with the hope of advancing research from bench to bedside.
During the forum, the silver-haired articulated their successes, failures, and insight. They put their hopes for improved patient outcomes squarely in the hands of the next generation who were receptive, responsive, and offered provocative solutions.
“This year our theme was Defining Success in Translational Medicine. Clearly we identified several key translational issues and began to think about solutions. The forum will be a true success if this ardent, seminal group returns next year and brings colleagues to work on solutions to bring about needed changes,” concluded Morrie Ruffin, CEO of LifeTech Innovations, who co-organized the event.