Keeping up with its obligations under the FDA Safety and Innovation Act (FDASIA), the FDA issued its strategic plan for accelerating the development of drugs and medical devices to treat rare diseases in children.
The plan, released Wednesday, outlines four agency objectives and promises a number of guidances on the subject.
To achieve its first objective – enhancing the foundational and translational science for pediatric rare diseases (PRDs) – the FDA will foster the conduct of natural history studies for the diseases and identify unmet pediatric needs in medical device development.
The second objective is to strengthen communication, collaboration and partnering for PRD therapies within and outside the agency. The challenges in developing products for rare diseases "are difficult to overcome by most single entities," the FDA said, so it plans to encourage public-public and public-private interactions and partnerships.
Thirdly, the agency intends to advance the use of regulatory science to aid clinical trial design and performance for the diseases. This will involve facilitating increased knowledge of biomarkers and appropriate clinical outcome assessments, early engagement of sponsors with the agency's study endpoints team and the use of web-based resources for education and navigation of regulatory processes.
The FDA is developing a number of guidances to help meet this goal. One draft guidance in the works will discuss selected clinical, scientific and ethical issues with the intention of helping sponsors obtain the data necessary to support the use of drugs in pediatric populations.
A companion draft guidance will focus on regulatory considerations in complying with the Pediatric Research Equity Act and Best Pharmaceuticals for Children Act, which were made permanent by FDASIA. The agency also plans to draft a guidance on developing cancer drugs for children and to finalize, within the next two years, a 2013 draft guidance on designing early phase trials for cellular and gene therapy products. (See BioWorld Today, May 25, 2012.)
In addition to the guidances, the FDA is developing training programs for pediatric clinical investigators and is exploring the use of modeling and simulation approaches in designing clinical trials for potential PRD therapies.
The final strategy in the plan is to enhance the review process for drugs and devices intended to treat PRDs. This includes gaining a better perspective of patients' and caregivers' preferences, implementing a benefit-risk framework in the review process and continuing the agency's rare pediatric disease priority review voucher program.
Under the voucher program, a sponsor that develops a qualifying therapy for a PRD is granted a transferable priority review voucher, which provides for a six-month review of another new drug or biologic instead of the standard 10-month review. A draft guidance on the voucher program is in the mix, the FDA said. (See BioWorld Today, Oct. 3, 2011.)