BIO 2016: Why Patient Input Matters


Here at the BIO International Convention, we've heard calls from advocates, industry, and the government to further incorporate patient feedback and perspectives into both the regulatory process, in which medicines are reviewed, and the medicine development process. An integrated approach has the potential to benefit everyone. For scientists and developers, further insight into the lives of patients can guide research and help finetune new treatments. For regulators, hearing firsthand patient experiences helps them assess whether the benefits of a potential medicine outweigh the risks for those suffering from disease. Most importantly, for patients, sharing their stories and perspectives can lead to more effective treatments.

A seat at the table
These days, patients play an increasingly proactive role in the development of new medicines. "Patients have gone from being passengers to copilots in drug development," noted Annie Kennedy of rare disease advocacy group Parent Project Muscular Dystrophy, at a panel yesterday. Paul Hastings, CEO of cancer-focused pharmaceutical company OncoMed, agreed: "Working with patients is not only a nice thing to do," he said, "It's an imperative."

And, at a panel earlier this week, Food and Drug Administration Commissioner Dr. Robert Califf noted that his agency actively listens to patient input. "As patients, we shouldn't just be recipients of the end process of what someone else determines is important, we should be involved from the beginning," said Califf. "What are the problems that need to be addressed?"

At the FDA, that means holding frequent meetings with patient advocates and families to get a first-hand understanding of patient priorities and perspectives, which then informs the FDA's efforts and resource decisions. This Patient-Focused Drug Development program, first passed as part of the fifth PDUFA reauthorization, has proven to be a resounding success for the agency and patients alike.

The ultimate goal is developing and delivering safe and effective medicines to patients. Obtaining their input and insight throughout the development and review process is a natural fit, and one we hope to see continue to evolve as Congress begins to discuss new PDUFA legislation this year.