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Content and Format of the RDEA Proposal | Rare Disease Endpoint Advancement Pilot Program


The RDEA Pilot Program proposal should include a one to two page(s) executive summary and be no longer than 12 pages long. The information below should be included in the RDEA Pilot Program proposal. The sponsor should indicate in their RDEA Pilot Program proposal if any items listed below do not pertain to their proposal. RDEA Pilot Program proposals that do not adhere to these format and content requirements will not be considered further. A sponsor can provide information not listed below as they deem relevant to their RDEA Pilot Program proposal.

  1. Product name, if applicable
  2. IND or pre-IND Application Number
  3. Proposed indication or disease being studied in a natural history study, as applicable
  4. If proposal is for a rare disease natural history study intended to study the proposed endpoint, include:
    1. Prior knowledge of disease epidemiology and natural history as described in the medical literature, registries, or other data sources
    2. What additional information the proposed natural history study will provide
    3. How the design of the natural history study will support endpoint selection for future studies
    4. Projected timeline to design and conduct the natural history study (if not already initiated)
  5. If proposal is for a common disease development program that includes novel endpoint(s) that could be applicable to a rare disease: Justification to support that the novel endpoint element(s) (including the specific endpoint and/or the endpoint methodology being developed) could apply to a rare disease.
  6. Justification that the proposed endpoint is a novel efficacy endpoint intended to establish substantial evidence of effectiveness for a rare disease treatment. An endpoint is considered novel if it has never been used to support drug approval or if it has been substantially modified from previously used to support drug approval.
  7. Scientific justification for why the endpoint is being explored to measure meaningful clinical benefit in the disease/condition and detailed description of endpoint attributes and characteristics, to include:
    1. Basis of endpoint: clinical outcome assessment, biomarker, digital-health technology, multicomponent, etc.
    2. Disease characteristics measured by the endpoint
    3. How the endpoint (or assessment tool used to collect data to support the endpoint) will be developed, verified, and validated
    4. How patient and caregiver input will be considered when developing the endpoint
    5. High level description of how the endpoint measures a clinically meaningful change in the studied population
  8. As applicable: Brief history of the development program, status of product development, and relevant summaries of pertinent information related to the endpoint from prior studies.
  9. Brief overview of study design, objectives, conduct, and analysis methods.
  10. Elements of the proposed novel endpoint development and/or study design the sponsor considers non-disclosable, along with a rationale for exclusion.
  11. A list of questions for discussion with the Agency about the specific proposed novel endpoint(s).
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