The FDA and Patients: Partnering for Cures


Today's guest post comes from Robert Conley, Regulatory Leader for Biomedicines for Lilly and Distinguished Lilly Scholar in Neuroscience. Here's what he heard at the annual Partnering for Cures meeting hosted by FasterCures, an organization whose mission is to accelerate the transformation of scientific discoveries into innovative medicines that help patients.

Earlier this month, at the Partnering for Cures meeting, I had an excellent opportunity to interact with patient groups, the FDA, researchers and other stakeholders about the importance of incorporating the patient voice into drug development and review. I also spoke on a panel about benefit-risk assessment in drug review with representatives from the FDA.

The current regulatory benefit-risk paradigm is asymmetrical. Often, FDA regulators feel pressure to identify and avoid the risks of new medicines, rather than examine how best to balance those risks against the potential benefits to patients. The problem lies not in the regulators, but in the system itself, which creates incentives to err on the side of avoiding risk -- even if some patients might accept that risk in return for the potential benefits.

A new, possibly useful, initiative was created in PDUFA V that will help address this issue in a more transparent and systematic manner. This important initiative holds promise for speeding safe and effective medicines to patients in need. This year, the FDA also initiated several patient-focused drug development meetings to help the Agency better understand what patients value most in terms of their treatments, including their benefits and risks.

In order for the FDA initiative to succeed, several things need to happen.

  • First, The FDA should embrace standardized and repeatable processes throughout the review process. For innovation to thrive, we need a high degree of predictability so that innovators understand regulatory requirements. We're hopeful this will result in better understanding for patient-reported outcomes and endpoints for new medicines.
  • Second, input from patient groups must be used effectively by drug reviewers. The FDA will process a great deal of patient feedback at public meetings. Lilly is most interested in seeing how this information will be combined and used to inform the agency's benefit and risk assessment, which in turn will help Lilly and other pharmaceutical companies design our clinical programs.
  • Finally, I'm eager to see what this new initiative means for common and chronic conditions, such as cardiovascular disease, neurodegenerative disease, diabetes, stroke and lower respiratory disease. Chronic diseases affect the quality of life of 90 million Americans, and cause seven out of 10 deaths each year. This represents a huge public health need and opportunity.

I really think we've reached a new day in drug development. Both the FDA and pharmaceutical companies are more eager than ever to hear from patient groups and understand their thoughts on the tradeoffs between benefits and risks in new medicines. This systematic and transparent framework will strengthen the process for drug development, review, and approval, and I believe that such an approach will result in regulatory decisions that are more predictable, more balanced, and more timely.