Building Bridges across the Valley of Death

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.