Path to Hope Form
Rare Disease Represented
We are at a critical moment. Please take the time to....
Share challenges in the size of your rare disease community, fragile nature of the disease, whom it most often affects (children, teens, adults - what age?), impact on activities of daily living and quality of life, life-expectancy, difficulty with the current clinical trial process (required size of trials, criteria, endpoints, understanding of the patient experience), the burden placed on rare patients and families who have participated in natural history studies, clinical research, invested in research and development, etc.
We are working to help FDA understand the rare community better and determine how to translate patient input more effectively into this regulatory process.
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