FDA draft guidance on drug development for rare diseases

FDA draft guidance on drug development for rare diseases

On 1st of Feb. 2019 the FDA announced the availability of draft guidance “Rare Diseases: Common Issues in Drug Development“. This guidance revises and replaces the previous draft guidance of the same name from 2015.

Comments and suggestions regarding this draft document should be submitted to FDA within 60 days of publication in the Federal Register of the notice announcing the availability of the document.

The purpose of this guidance is to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs.

Although the statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs, these issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, natural history data, and drug development experience.

This guidance addresses the importance of the following elements in development programs for rare diseases:

  • Adequate description and understanding of the disease’s natural history
  • Adequate understanding of the pathophysiology of the disease and the drug’s mechanism of action
  • Nonclinical-pharmacotoxicology and human toxicology considerations to support the proposed clinical investigation or investigations
  • Selection or development of outcome assessments and endpoints
  • Evidence to establish safety and effectiveness
  • Drug manufacturing considerations during drug development (e.g. pharmaceutical quality system considerations)
  • Participation of patients, caretakers, and advocates in development programs
  • Interactions with the FDA

Early consideration of these issues gives sponsors the opportunity to efficiently and effectively address the issues and to have productive meetings with FDA. 

Link to the draft guidance:


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