Prioritizing the Patient Voice
The passing of the 21st Century Cures Act (Cures) carries with it a prominent focus on the involvement of patients in every stage of the pharmaceutical development process. In some respects, this legislation renews the commitment to patients that the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) spearheaded through the Patient Focused Drug Development program. Considering the amount of time and resources dedicated to the identification of new treatments, healthcare entities operating in a variety of sectors are hungry for insight into patients’ perceptions and experiences.
As the FDA previously declared, patients should play a vital role in treatment development. Being responsible for drug and medical device approval is a challenge that requires the agency to weigh potential side effects against outcomes. The first-hand insights patients offer into the day-to-day struggles and successes of living with a condition affords context to validate these decisions. To this aim, the FDASIA-initiated disease-focused meetings held from 2012 to 2017 were a leap forward in the prioritization of such patient experiences which Cures will carry on.
Formalizing the patient involvement process will take time, and many questions still exist. Since Cures requires the FDA to create a framework for considering real-world evidence (RWE) in the drug approval process, the next two years will likely yield a focus on considerations outside the clinical setting. This concept challenges the standing status quo which views medical evidence gathered through clinical trials as superior. The use of RWE includes information derived from more than one source beyond strict research settings. Such sources may include disease registries, personal device data, electronic health records and product registries. Considering information from these perspectives will provide rich information about relevant patient experiences.
While RWE is not a replacement for clinical evidence, its blend of patient insights will add pertinent and colorful information for regulatory decision making. One thing is for certain, the increased discretion that Cures affords the FDA regarding the studies it utilizes when considering drugs approvals will further propel the patient voice to the front and center.
Meagan Bates is a Senior Account Executive at JPA