The US Food and Drug Administration (FDA) on Friday released a plan submitted to Congress for how it will use $500 million in potential new funding from the 21st Century Cures Act over the next nine fiscal years.
The plan, similar to a proposal released in May, would see about $185 million of the total go to updates and modernizing FDA’s regulation of combination products, developing FDA standards and consensus definitions to support the development and review of regenerative medicines and new accelerated approvals for some regenerative advanced therapies.
In March, FDA issued its first Regenerative Medicine Advanced Therapy (RMAT) designation. CBER Director Peter Marks said recently at the DIA annual conference in Chicago that FDA has so far received 19 RMAT requests, of which it has acted on 18 and granted four.
FDA Commissioner Scott Gottlieb said Friday that the agency in September will announce "a comprehensive framework for the development and proper FDA oversight of regenerative medicine."
Advancing New Therapies
About $95 million of the total would also go to FDA’s establishment of a qualification process for drug development tools, the reauthorization of the priority review voucher program for rare pediatric diseases, grants for studying continuous manufacturing, and work targeting rare diseases that will allow some applications to rely on data and information previously submitted by the same sponsor (or agreed to by a different sponsor) in another application.
FDA is required to submit annual reports to Congress from FY 2018 to FY 2025 on the activities conducted using any of the funds outlined in the plan. Importantly, the funds may be appropriated by Congress, but are not required to be, on an annual basis.
Plan for Patient‐Focused Drug Development Guidance
In addition, FDA recently released a plan on its intentions to issue a series of four guidance documents focusing on approaches to bridge initial patient-focused drug development meetings to tools to collect meaningful patient and caregiver input for use in regulatory decision making.
By the end of the second quarter of calendar year 2018, FDA says it will publish a draft guidance describing approaches to collecting comprehensive and representative patient and caregiver input on burden of disease and current therapy.
"This draft guidance will address topics including: standardized nomenclature and terminologies, methods to collect meaningful patient input throughout the drug development process, and methodological considerations for data collection, reporting, management, and analysis," FDA said.
By the end of the second quarter of calendar year 2019, FDA says it will publish a draft guidance describing processes and methodological approaches to developing methods for sponsors, patient organizations, academic researchers and experts to develop and identify what are most important to patients in terms of burden of disease, burden of treatment and other issues.
By the end of the second quarter of calendar year 2020, FDA will publish a draft guidance describing approaches to identifying and developing measures for an identified set of impacts (e.g., burden of disease and treatment), which may facilitate collection of meaningful patient input in clinical trials.
And also by the end of the second quarter of calendar year 2020, FDA will publish a draft guidance on clinical outcome assessments, "which, when final, will, as appropriate, revise or supplement the 2009 Guidance to Industry on Patient-Reported Outcome Measures. The draft guidance will also address technologies that may be used for the collection, capture, storage, and analysis of patient perspective information. The draft guidance will also address methods to better incorporate clinical outcome assessments into endpoints that are considered significantly robust for regulatory decision-making," FDA said.
Meanwhile, FDA’s Center for Devices and Radiological Health (CDRH), which has already amended its current regulations to allow more devices to qualify for a humanitarian device exemption for small patient populations, will allow researchers to seek approval for device clinical trials through a central institutional review board rather than local review boards.
CDRH also published a list of reusable device types for which FDA will require validated instructions for use and validation data regarding cleaning, disinfection and sterilization in 510(k)s. These new requirements go into effect 8 August 2017.
Food & Drug Administration Work Plan and Proposed Funding Allocations of FDA Innovation Account
21st Century Cures Act Deliverables
21st Century Cures Act