PharmaVOICE Blog Post

Clinical Outcome Assessment Selection for Rare Disease Trial Programs

Posted By: Dan Limbach
May 20, 2019

Evidera has published an informative article to provide some baseline knowledge about the “why, who, what, and how” when it comes to clinical outcome assessments for rare disease trials.

Read the article:

 Key points in the paper:

  • Patient inclusion throughout the drug development process is becoming a necessity. In rare disease product development, the patient perspective is particularly important because, often, not much is known about the disease experience
  • The patient’s perspective about their own experience should be reported directly by the patient. This may not be possible in rare disease as about 80% of the diseases hold a genetic component and nearly 75% of those affect children
  • When considering what concepts to measure in a trial program, begin by considering what is important, or meaningful, to the patient
  • The selection, or development, of a COA for a trial program should consider several factors.
    • Who is reporting the data?
    • How often are data being reported?
    • What challenges with mobility or ability to report does the population have?
    • What operational considerations exist (need for translations, mode of administration, and time to trial kick-off)?
  • It is possible that an existing COA measure has been developed and can be adapted to the rare disease
  • While there can be challenges in the selection and inclusion of COAs in a rare disease trial program, the patient’s unique perspective is a critical aspect of evaluating efficacy

Read the article:

About the Blog Poster: Dan Limbach

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