Patient Engagement in Drug Development: Myth or Reality?
Is the patient voice adequately represented in the drug development process? The situation has improved but the patient community is not yet recognized as a co-equal in drug development or regulatory review according to Marc Boutin, executive vice president and chief operating officer at the National Health Council, a panelist at a BIO International Convention session on Wednesday. ’We scare you,’ declares Boutin who says there is an adversarial connection between patients, regulators and industry but the relationship has evolved over the last decade. Patient advocates have developed strategies to amplify their voice and demand access, providing the data and evidence to help transform healthcare delivery.
A section in the 2012 FDA Safety and Innovation Act fosters participation of patient representatives in appropriate FDA meetings with medical product sponsors and investigators, and a new transparent framework to communicate FDA benefit-risk approval decisions to the public. From the patient perspective, ’benefit-risk decisions are value judgments, they are not science,’ says Boutin. He acknowledges that FDASIA meetings are a critical step but sophisticated patient engagement is still lacking. In-depth interviews, focus groups, crowd sourcing and other strategies should be part of the equation, he adds. Getting the patient perspective in benefit-risk could change the discussion dramatically and improve healthcare delivery, Boutin says. Ensure that clinical trials are designed appropriately for people with chronic conditions, for example, to understand the benefit-risk for a subset of the population.
Kimberly McCleary, director of strategic initiatives, FasterCures, admits more work needs to be done to ’measure patient preference and expectation in an academic, rigorous way across different domains.’ To that end, FasterCures has formed a benefit-risk advisory council to work towards integration of the regulatory environment in a healthcare setting. The council will expand opportunities for patient perspectives to shape product development and influence regulatory decisions. The Patient Centered Outcomes Research Institute (PCORI) is also shifting the dynamic with patient involvement in a post-market setting. PCORI has identified critical points for patient engagement which is an opportunity for pharmaceutical companies to insert in their own business model, Boutin claims.