Conference Coverage

FDA Encourages Investment in Pediatric Rare Diseases



ALEXANDRIA, VIRGINIA—The FDA is establishing new policies to encourage industry to invest in and develop new products for pediatric rare diseases, said Margaret Hamburg, MD, Commissioner of Food and Drugs. “While about half of all rare diseases affect children, for many reasons, the development of drugs, biological products, and devices for pediatric rare diseases has been more challenging than for rare diseases more generally,” she added in a speech at the National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit.

“Our goal is to reduce medical product development times to ensure that patients receive promising products as quickly as possible, while adhering, of course, to our high standards for safety and efficacy,” said Dr. Hamburg. The agency sees great benefit in providing orphan drug incentives to industry to develop or improve products for rare diseases. It is equally important, said Dr. Hamburg, for the agency to offer enhanced guidance for research and development, early input on clinical study needs and design, expedited regulatory pathways, and targeted assistance for needed products.

Margaret Hamburg, MD

Incentives for Drug Development In 2014, the agency issued a strategic plan outlining how it will encourage the development of therapies for pediatric rare diseases. The plan relies on several laws intended to address the needs of the pediatric population, including the Best Pharmaceuticals for Children Act, the Pediatric Research Equity Act, and the Pediatric Medical Device Safety and Improvement Act.

These laws provide various incentives for drug development, the newest of which is the Rare Pediatric Disease Priority Review Voucher program. Under this program, the sponsor of a drug for a rare pediatric disease may be eligible to receive a voucher upon the drug’s approval. The sponsor can redeem the voucher for priority review of another drug application.

Earlier in 2014, the FDA approved elosulfase alfa, which received the first voucher under this program. Elosulfase alfa is the first FDA-approved treatment for Morquio A syndrome, a rare, autosomal recessive lysosomal storage disease. After receiving the voucher for priority review, the drug’s sponsor sold it, and the proceeds from the sale will allow the company to invest in research and development.

New Review Processes for Drugs and Devices
In addition to providing incentives for research and development, the FDA has adapted its regulatory review processes to enable drugs for rare diseases to be approved and marketed more quickly. To date, the agency has developed four expedited drug review programs: fast track, priority review, accelerated approval, and the breakthrough therapy designation.

“We’ve already seen positive results from the breakthrough therapy designation,” said Dr. Hamburg. As of the beginning of October 2014, the FDA had received 211 requests for breakthrough designation and granted 63 of them. Since Congress established the program in 2012, the FDA has approved nine new drugs for breakthrough designation and four new indications. Of these new approvals, 11 have been for rare diseases.

These developments are part of what Dr. Hamburg called the agency’s “broader success” in approving therapies for rare and pediatric diseases, as well as common disease therapies that gain indications for rare diseases. In 2013, the FDA approved 33 orphan drugs. Of the 27 new molecular entities approved, nine were for orphan drugs. As of October 2014, the agency had approved 11 of 29 new molecular entities for rare diseases—more than it had approved during 2013. “This is clearly an area of considerable and growing activity,” said Dr. Hamburg.

In parallel to its changes in drug review processes, the FDA’s Center for Devices has been streamlining and modernizing review processes. The center also has proposed a program for expedited device review. A draft guidance issued in 2014 suggested the establishment of a voluntary program that would speed to market products that address unmet needs for life-threatening or irreversibly debilitating diseases or conditions.

Openness to Flexible Study Designs
In the interest of providing new therapies for children with rare diseases, the agency has begun to accept study designs that it might not permit for other types of drugs. For example, the FDA accepts data from single-arm studies when the natural history of the disease is well characterized but the patient populations are exceptionally small. When appropriate, regulators also encourage the use of adaptive trial designs, which can be modified as information about drug response accumulates. This approach “can lead to more efficient studies or enrichment strategies to enroll patients more likely to respond to drugs under study, thereby reducing trial size and helping to direct drugs to patients who will benefit the most from them,” said Dr. Hamburg.

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