Patients’ views are not often heard – at least, not in formal publications like scientific papers or official reports. So, it is refreshing to read the newly-released U.S. Food and Drug Administration (FDA) document called the “The Voice of the Patient”, which summarizes the input from patients and patient representatives at the ‘Patient-Focused Drug Development Initiative’ meeting on April 25 this year. The discussion focused on two key topics: disease symptoms and daily impacts that matter most to patients; and the patient perspective on treatments.
Overall, the FDA’s conclusion was that ME/CFS is a “debilitating disease that can severely affect a patient’s day-to-day functioning and have a devastating impact on a patient’s life”. And key themes from the patient consultation exercise included:
• Many patients can pinpoint a specific time in their life when they contracted the disease.
• Patients struggle daily with their symptoms, and the most frequently mentioned included severe fatigue or exhaustion, impairments in cognitive functioning, chronic pain, sleep difficulties, blood pressure drops and dizziness, sensitivity to light, sound and temperature and susceptibility to infection.
• Post-exertional malaise is a severe exacerbation of those cognitive and physical symptoms. A crash can result from even minimal mental or physical exertion, can happen without warning, and can last for days, weeks, months, or even years.
• Patients use or have tried a complex regimen of drug and non-drug therapies to treat their disease and manage their symptoms…These treatments have been met with varying degrees of effectiveness, and for some, none is effective.
• The illness takes a devastating toll on the lives of many patients and their families, including loss of careers, decreased quality of family life, social isolation, and feelings of hopelessness. Patients are desperate for research and development of treatments.
The FDA reports that it is grateful to patients, carers, and others who so thoughtfully and courageously shared their experiences and perspectives, and says that it will carefully consider this input from patients as it fulfils its role in the drug development process.