FDA: Another tool helping developers navigate the difficult road to approval of drugs for rare diseases

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Today, the FDA released a blog post about a new tool for drug developers.  The FDA writes, “With the vast majority of rare diseases still without FDA-approved treatments, we have recently released a new resource for drug developers — draft guidance document — designed to help them navigate the difficult and unique challenges of developing and bringing to market new FDA-approved drugs to treat rare diseases.”

The FDA intends for its draft guidance to help drug developers create more accurate and timely drug development programs by encouraging

  • a focus on understanding a disease’s “natural history,”
  • creation of study designs with clinically meaningful endpoints,
  • development of evidence needed to establish safety and effectiveness,
  • and the establishment of drug manufacturing specifications to ensure quality.

To read the draft guidance, visit: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf.