Drugs

Recommendations on Rare Disease Collaboration

David McFarland

— 2 min read
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A new National Academies book, Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities, has recommendations based on the Academies’ study of processes for evaluating the safety and efficacy of drugs for rare diseases in the U.S. and the European Union. Congress called on FDA to contract with the National Academies to look into (1) flexibilities and mechanisms available to regulators; (2) the consideration and use of “supplemental data” submitted during the review process; and (3) an assessment of collaborative efforts between FDA and the European Medicines Agency (EMA). Recommendations in the book are:

  • congressional action is needed to encourage and incentivize more studies that provide information about the use of rare disease drug products in pediatric populations;
  • FDA should strengthen mechanisms to integrate input from people living with a rare disease or condition, their caregivers, and patient representatives, especially patient groups that are small and under-resourced, throughout the full continuum of the drug development process;
  • FDA and the National Institutes of Health, in collaboration with EMA, nongovernmental organizations, patient groups, and biopharmaceutical sponsors should implement a sponsor, investigator, and patient group navigation service to support the development of drugs to treat rare diseases and conditions;
  • FDA should assess the impact of new and ongoing programs and approaches that support drug development for rare diseases and conditions to improve the regulatory decision-making process, and publicly share the results of these assessments promptly, take steps to ensure that lessons learned across different programs are disseminated throughout FDA, scale up and expand successful programs, and modify or sunset programs that are not improving the regulatory decision-making process;
  • FDA should enable the collection and curation of regulatory-grade natural history data to enhance the quality and accessibility of data for all rare diseases;
  • FDA should invite EMA to jointly conduct systematic reviews of submitted and approved marketing authorization applications to treat rare diseases and conditions that document cases for which alternative and confirmatory data have contributed to regulatory decision-making;
  • FDA should collect and disseminate information on how state-of-the-art regulatory science; innovative study design and methods; tools, including biomarkers and surrogate endpoints, and effective applications of alternative and confirmatory data inform regulatory decision-making for rare disease drug products;
  • FDA should take steps to make relevant information on marketing authorization submissions, review milestones, approval and negative review decisions (refusal to file, clinical hold, and complete response letters), and the use of regulatory flexibilities for rare disease drug products publicly available and easily accessible to inform sponsors, patients, researchers, and reviewers on decision-making rationales and when and how available policies are applied;
  • to facilitate the efficient global development of orphan drugs, FDA and EMA should build upon existing clusters relevant for rare diseases in specified ways; and
  • FDA, along with EMA and other key stakeholders, should assess the impact of the Parallel Scientific Advice (PSA) program over the past decade on drug development for rare diseases and conditions, publicly share the results of this assessment, seek sponsor input on approaches to improve and enhance the use and utility of the program, and take action to increase access, use, and impact of the PSA program going forward.

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