FDA Comment Deadline: Rare Diseases — Common Issues in Drug Development

Tuesday, April 2, 2019 - 5:00pm

The Food and Drug Administration (FDA) is announcing the availability of and seeking public comment on a draft guidance for industry entitled “Rare Diseases: Common Issues in Drug Development.” This draft guidance assists sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs through a discussion of selected issues commonly encountered in rare disease drug development. This draft guidance addresses the following important aspects of drug development:

  • Adequate description and understanding of the disease's natural history
  • Adequate understanding of the pathophysiology of the disease and the drug's proposed mechanism of action
  • Nonclinical pharmacotoxicology considerations to support the proposed clinical investigation or investigations
  • Reliable endpoints and outcome assessment
  • Standard of evidence to establish safety and effectiveness
  • Drug manufacturing considerations during drug development

This guidance revises and replaces the draft guidance for industry of the same name issued on August 17, 2015 (80 FR 49246). This revision includes the following:

  • Updates to the natural history section
  • Inclusion of issues for evaluation of biomarkers for consideration as surrogate endpoints
  • Description of nonclinical flexibility
  • Additional information on historical (external) controls and early randomization
  • Addition of safety section
  • Retitled Chemistry, Manufacturing, and Controls section to Pharmaceutical Quality Considerations
  • Additional information on changes to drug substance or manufacturing process with clarification on areas of flexibility
  • Addition of a considerations section addressing several topics including participation of patients, caretakers, and advocates; consideration of pediatric issues; and interactions with FDA

Comments may be submitted electronically through this portal at Regulations.gov, or in written form. For more information regarding this draft guidance and the submission process, please see this Federal Register notice.