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Spotlight On Rare Disease Development – Q4 2023

FDA Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. 
Early in 4Q2023, the FDA announced a pilot program for Sponsors of drug or biological products currently in clinical trials under an active IND.  The benefit of participation in the pilot program is to engage the FDA in more frequent communication to discuss clinical development issues.  To be eligible, biological products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life.

Eligibility for drug products requires that the product must treat rare neurodegenerative conditions, including those of rare genetic metabolic type. The FDA will be accepting applications to the START program between Jan. 2, 2024, and March 1, 2024.  If you are interested in applying for the START pilot program, contact Facet today!

RARE DISEASE DAY – March 1, 2024
FDA will virtually host Rare Disease Day on March 1, 2024, in global observance of Rare Disease Week.  Panel topics include:

  • The legal framework for approving studies and medical products at FDA
  • What FDA does during review processes to approve medical products
  • Decentralized clinical trials and digital health technologies
  • Where to find important information and documents related to clinical trials
  • Information that can be obtained from medical product labels
  • Legal and ethical requirements for consent forms in clinical trials
  • FDA initiatives to advance medical product development for rare diseases
  • Ways for patients to engage with FDA

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