Parkinsons Disease in the United States

John F P Bridges, Ph.D. on PDUFA

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.

John F P Bridges, Ph.D.
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Kim McCleary on PDUFA

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.

Kim McCleary
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Ryan Fischer on PDUFA

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.

Sharon Terry on PDUFA

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

Sharon Terry
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Ryan Fischer
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Sascha on PDUFA

As important stakeholders in the drug development and approval process, patients provide a unique and valuable perspective when considering the benefits and risks of potential new and innovative medicines.  Patients can identify areas of unmet medical need, provide critical perspective on the impact of a disease and its manifestations, and uniquely inform the development of outcome measures that are meaningful to patients.  A scientific approach to gathering patient input is necessary to realize the shared goal of a patient-centered approach to drug development and regulatory review.  PhRMA

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