Despite decades of progress in our fundamental understanding of human disease, our ability to translate exciting new discoveries into products that can help patients is severely lagging behind the pace of discovery. Panelists focused on the challenges that need to be addressed, what’s working and how these could be amplified, and the power of patients in efforts to transform the medical research system.

Margaret Anderson, Executive Director of FasterCures opened the discussion by noting how we all have a stake in medical research and a role to play to accelerate this process.  Whether you’re the parent of a child with Type I diabetes, a cancer survivor, a nonprofit foundation whose bottom line is getting treatments to the patients you care most about, working for a company committed to developing life-saving therapies, or an advisor to philanthropists wanting to make the greatest possible impact in the quest for new cures.

Kathy Giusti, Founder and CEO of the Multiple Myeloma Research Foundation (MMRF), said that "it's not that people don't want to collaborate - it's just that they don't have the time nor the processes to make it happen." MMRF serves as an end-to-end solution for drug development; business models have been developed to addressed barriers. And yet, they will “continue to struggle to determine the right drug for the right patient at the right time."

Robert J. Beall , President and Chief Executive Officer, Cystic Fibrosis Foundation, Kathy Giusti , Founder and Chief Executive Officer, Multiple Myeloma Research Consortium, Margaret Anderson, Executive Director, FasterCures, Billy Tauzin, President and Chief Executive Officer, PhRMA, and Ed Benz, Jr., President and Chief Executive Officer, Dana-Farber Cancer Institute

Billy Tauzin, President and CEO of PhRMA, noted that science is speeding along at a much faster rate than policies and decisions made in Washington. Tauzin noted that we need new, efficient clinical trial models supported by technologically-advanced regulatory policies.

Edward Benz, President and CEO of the Dana-Farber Cancer Institute, speaking with experience as both a health care provider and a medical researcher, noted that among the challenges that need to be addressed is the need for additional resources to take new scientific discoveries out of the laboratory and get them developed, and increasing participation in clinical trials.

Benz said that “in cancer, only 3-4 percent of patients join clinical trials.”

Robert Beall, President and CEO of the Cystic Fibrosis Foundation (CFF), said patient organizations can de-risk the environment and get companies involved. The CFF implemented a number of efforts to de-risk, including investing over $300 million in early stage development at biotechnology companies, creating a clinical trials network, developing a data registry, providing clinical and epidemographic data with patient genotypes, and allowing access to secondary assays.

Beall emphasized that foundations can “start by identifying patients. Patient power and patient organizations can create vital role for this transformation effort.”

Panelists also noted that philanthropic investment - relatively small compared with government and industry funding - plays a critical, strategic role in catalyzing progress, filling gaps, and leveraging public and private investment.