Mid-last year, the US Congress called on the FDA to sponsor a...

Mid-last year, the US Congress called on the FDA to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union.

The National Academies (for Sciences, Engineering, and Medicine) are private, non-profit institutions that provide independent, objective analysis and advice to solve complex problems and inform relevant public policy decisions.

The resulting 300 page report, which was released this week, contains recommendations to enhance and promote rare disease drug development.

Recommendations include:

• that the FDA take steps to make information publicly available and accessible on authorization submissions, review milestones, negative reviews and approvals, and the use of regulatory flexibilities for rare disease drugs. It was suggested that if researchers and sponsors working on rare disease drug development had a better understanding of the reasons for successes and failures of drug authorization applications, they could design new therapeutics that had a higher likelihood of success.
• that the FDA strengthen its mechanisms for using input from patients and caregivers throughout the drug development process, including by establishing an approach for advisory committee meetings.
• that the FDA enable the collection of natural history data and enhance the quality and accessibility of natural history data. It was stated that “gold standard” random controlled trials can be challenging, if not infeasible for drugs for very small rare disease populations. It was suggested that natural history data, along with other types of data such as information from patient registries, claims, and electronic health records, could help inform regulatory decision-making.
• that the FDA and EMA work together more closely to facilitate efficient global development of therapies for rare diseases.
• that the US Congress take action to encourage and incentivize more research to develop therapies for rare diseases in paediatric populations . It was recommended that Congress assess additional incentives to spur the development of drugs to treat rare diseases or conditions.

To what extent and how quickly the report will be acted on is not clear, but adoption of these recommendations could certainly be beneficial for drug sponsors with a focus on rare disease drug development.

https://www.agencyiq.com/blog/fda-q...nform-future-rare-disease-regulatory-reforms/

https://www.nationalacademies.org/n...nce-information-sharing-improve-collaboration

https://www.fiercebiotech.com/biote...open-collaborative-accelerate-rare-disease-rd