As new drugs and therapies make their way to the Food and Drug Administration (FDA) for approval, itís critical for government regulators to hear directly from patients. Their voice helps to shape the drug development process, educating the FDA about common symptoms, impacts on daily life and challenges in existing treatments.
The FDA recently published a report detailing the impact of Parkinsonís disease (PD) on patients and their loved ones. The report is part of the FDAís Patient Focused Drug Development Initiative, a program to gather patient perspectives on symptoms and available treatments. The initiative gives patients the opportunity to share their experiences and highlight areas of unmet need.
Information published in the report was gathered at a September 2015 FDA meeting featuring panels with Parkinsonís patients and caregivers. The FDA also solicited public comments from the PD community, and the Parkinsonís Action Network, 23andMe and PatientsLikeMe conducted surveys and submitted the responses to the FDA.
A major topic covered in the report is the impact of PD symptoms on daily life, as well as limitations patients experience as a result of their disease.
Patients identified motor symptoms, impaired balance and coordination, cognitive impairment and sleep disturbances at the symptoms with the greatest impact. They shared how Parkinsonís has affected their social lives, describing it as isolating, as well as the emotional burden of the disease, saying it can be frustrating and unpredictable.
The report also highlights patient experiences with Parkinsonís treatments, including prescription medications, alternative therapies and lifestyle changes. The importance of personalized treatment regimens was noted, and many participants expressed a desire for more effective therapeutic options because current prescription drugs have varying degrees of success.
ďThroughout the meeting, participants raised a number of other considerations that they believed were important relating to Parkinsonís disease treatment and drug development more broadly, including the progress of stem cell research, clinical trials for new surrogate markers, and the need for a greater focus of disease prevention in addition to treatment. Many participants commented on the need for increased awareness and education to properly diagnose and treat Parkinsonís disease. Participants also commented on the importance of early diagnosis for improvement of quality of life.Ē
In its report, the FDA thanked the Parkinsonís community for sharing its experiences, saying, ďThe insight provided . . . will aid in FDAís understanding of what patients truly value in a treatment and inform the agencyís evaluation of the benefits and risk of future treatments for Parkinsonís disease patients.Ē
The Michael J. Fox Foundation (MJFF) will continue to highlight opportunities for patients to provide feedback to the FDA on our blog and social media channels.
Individuals with and without Parkinsonís disease can participate in Fox Insight, MJFFís online study to gather long-term information about life with PD, to help advance Parkinsonís research and therapeutic development.