Recently Dr. Janet Maynard, who currently is the director of the US Food and Drug Administration’s (FDA) Office of Orphan Drugs Development, wrote an article in which she discusses the agency’s recent accomplishments in the field of rare diseases and also discusses ways in which the FDA plans to help facilitate and improve the development process for rare disease drugs and other products in the future.
2018 was a big year for rare disease treatments in which more innovative therapies designed to treat rare disease were approved by the FDA than ever before. This included 35 products that were approved under Orphan Drug designation. This is the largest number for any year since the passage of the Orphan Drug Act in 1983. Overall, the pace of development for rare disease therapies is as rapid as it has ever been.
However, the FDA is still working to improve its process for rare disease therapies and products. The agency is currently working to gather greater input from stakeholders from a variety of backgrounds, including politicians, patients, and scientific experts.
In addition, the agency is hosting a public meeting on April 29th that is intended to center the voices of rare disease caregivers and patients. The goal will be to improve the FDA’s understanding of the daily impacts of rare diseases on these populations. In addition, the agency hopes to gather new information about commonalities across different indications that can improve the regulatory development process for rare disease products. Dr. Maynard also announced changes to the FDA’s grant review process that will center feedback from patients gathered in recorded listening sessions in which patients discuss the impact of their disease.
Other actions that the agency plans to implement in 2019 include a patient portal. This portal is intended as a more streamlined method for patients and caregivers to communicate with the FDA and set up in-person meetings with agency representatives. The FDA will also establish an all-new FDA Orphan Products Grants Internal Review Committee which will include regulatory and rare disease experts. This will allow for a more informed grants assessment process that will include feedback from rare disease researchers.