For many, terms like patient-centered outcomes research and patient-focused drug development are new. For others, they are simply buzzwords that are aimed at putting a new spin on existing practices. As a researcher who has dedicated the majority of my career to the scientific study of the patients’ point of view, I rejoice in renewed interest in the patients’ perspective.
A paradigm shift is occurring. Patients were once viewed as passive, deferential recipients of medical products and services developed for them. Today, there is growing recognition that patients are a vital force for transformative change to address serious unmet medical needs and improve public health.
Parent Project Muscular Dystrophy has long held the belief that new approaches are required to expedite the development of treatments for rare diseases like Duchenne. Patient advocates have worked tirelessly with Congress, over time through legislation, to increase the engagement of the patient voice within the regulatory review process for therapeutics. Passed in 2012, The Food and Drug Administration Safety and Innovation Act (FDASIA) was a culmination of those advocacy efforts by patients.
This is not rocket science! Running a molecule through all of the traps is really hard. Understanding what people feel and think and experience with regard to the drugs they take and those they need is simple. In most other areas of our lives, various industries know quite well what features we want on our smartphones, that we suddenly all want Greek yogurt, and that we don’t want to pay to use the bathroom on a plane. In medicine, we have not yet realized the same dynamic and robust methods, tools and studies for understanding the needs and values of the end user. The needs, wants, va