FDA officials update on orphan drugs, gene therapies at DIA

Regulatory NewsRegulatory News | 18 June 2020 |  By 

Officials from the US Food and Drug Administration (FDA) discussed the agency’s recent efforts to support the development of products to treat rare diseases during a session at DIA’s Global Annual Meeting on Wednesday.
Orphan and rare pediatric disease designations
While the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.
Janet Maynard, director of FDA’s Office of Orphan Products Development, gave an update on FDA’s orphan drug designation program. According to Maynard, orphan drug designation requests have stabilized at just over 500 requests per year since 2016. The number of designations granted have also remained stable, in the mid-300s since 2015, with a spike to 477 in 2017.
Maynard also discussed FDA’s rare pediatric disease designation and priority review voucher program, which is set to sunset later this year without legislative action.
“Between 2013 and 2016 we saw a steady increase in terms of the number of rare pediatric disease designations requests we have received, and that number has remained greater than 50 since 2016,” she said. FDA has granted more than 50 rare pediatric disease designations each year since 2017.
However, Maynard pointed out that, “After 30 September 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug and that designation was granted by 30 September 2020,” and that the agency will no longer issue vouchers regardless of designation after 30 September 2022.
Companies looking to get in under the wire will need to submit requests for rare pediatric disease designation soon, Maynard said. FDA guarantees a 60-day review for rare pediatric disease designation requests issued alongside a fast track or orphan designation request but does not commit to a specific timeline for requests submitted separately.
“The Offices of Orphan Product Development and Pediatric Therapeutics cannot commit to providing a response to rare pediatric disease designation requests by 30 September 2020 for any requests received after 31 July 2020,” Maynard said.
OND reorganization and ‘rare disease hub’
After undergoing a major reorganization earlier this year, FDA’s Office of New Drugs (OND) is better equipped to address rare disease drug development, said Hylton Joffe, acting director of the new Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine.
Joffe’s office oversees three clinical divisions, the Division of Pediatrics and Maternal Health, the Division of Rare Diseases and Medical Genetics and the Division of Urology, Obstetrics and Gynecology.
“You can imagine, given the significant burden of rare diseases in children, how having these three divisions together could lead to very strong collaborations in the rare disease space,” Joffe said.
Joffe pointed out that the newly created Division of Rare Diseases and Medical Genetics, which oversees the rare disease group and inborn errors review group, is the only division within OND that is solely focused on rare diseases. While other offices within OND also deal with rare diseases, “We see this new division and this office as the new OND rare disease hub,” Joffe said, adding that the goal of the hub will be to enhance communication and consistency for rare diseases.
Gene therapies
Following the approval of the first few gene therapies, Elizabeth Hart, branch chief at the Office of Tissues and Advanced Therapies within the Center of Biologics Evaluation and Research, said expectations are high for future gene therapies to treat rare diseases.
Hart said that the number of gene therapies in development has increased significantly in recent years, with investigational new drug applications (INDs) for gene therapies increasing more than 140% between 2016 and 2019. According to Hart, 70% of gene therapy INDs are targeting rare diseases.
While the approval standard for gene therapies and other rare diseases is the same as for other drugs, Hart said that the clinical development plans for those products could look very different from those for more common conditions.
“We do have the option to exert regulatory flexibility, and this is especially important for rare diseases. Clinical development programs for the different diseases may vary substantially, and so they really need to be individualized,” she said.
Hart also said that, “There are no specific minimum number of patients to be studied to establish effectiveness and safety of a gene therapy, the number of subjects needed is determined on a case by case basis taking into consideration the persuasiveness of the data, including comprehensiveness and quality; the nature of the benefit provided; the length of the treatment exposure, the patient population that would be treated after marketing approval; and concerns for potential harm from the treatment.”


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