The FDA should be more open and collaborative to unleash a surge in approvals of rare disease drugs, according to a report by the National Academies of Sciences, Engineering, and Medicine.
Congress asked the FDA to contract with the National Academies to conduct the study. The brief focused on the flexibilities and mechanisms available to regulators, the use of “supplemental data” in the review process and an assessment of collaboration between the FDA and its European counterpart. That brief has spawned a 300-page report that provides a road map for kick-starting orphan drug innovation.
Many of the recommendations relate to transparency and collaboration. The National Academies wants the FDA to strengthen its mechanisms for using input from patients and caregivers throughout the drug development process, including by establishing an approach for advisory committee meetings.
International collaboration is on the agenda, too. The National Academies is recommending the FDA and European Medicines Agency (EMA) implement a “navigation service” to advise on regulatory pathways and provide clarity on how to comply with requirements. The report also identified the underuse of the existing FDA and EMA parallel scientific advice program and recommends steps to increase uptake.
The focus on collaboration between the FDA and EMA reflects the National Academies’ conclusion that the two agencies have similar programs to expedite the review of rare disease drugs and often reach the same approval decisions. Despite the overlap between the agencies, “there is no required process for regulators to jointly discuss drug products under review,” the National Academies said.
To boost collaboration, the report suggests the FDA should invite the EMA to conduct a joint systematic review of drug applications for rare diseases and how alternative and confirmatory data contributed to regulatory decision-making. The National Academies envisages the review considering whether the data are adequate and useful for supporting regulatory decisions.
“EMA and FDA should establish a public database for these findings that is continuously updated to ensure that progress over time is captured, opportunities to clarify agency thinking over time are identified, and information on the use of alternative and confirmatory data to inform regulatory decision making is publicly shared to inform the rare disease drug development community,” the report states.
The report includes recommendations for lawmakers, with the National Academies advising Congress to “remove the Pediatric Research Equity Act orphan exemption and require an assessment of additional incentives needed to spur the development of drugs to treat rare diseases or condition.”