FDA Lays Out Work Plan for 21st Century Cures Act

Discovery, Development and Delivery

FDA Lays Out Work Plan for 21st Century Cures Act

In December 2016 Congress passed the 21st Century Cures Act as a bipartisan push to speed the creation and use of new medicines and therapeutic devices. Title III of the Cures Act provides funding to speed up the approval process. It adds $500 million over a decade to the Food & Drug Administration’s Innovation Account for this purpose.

This July FDA released its Work Plan for the new Title III findings. As reported by Daniel Kracov and Pari R. Mody in Advisory, the agency will focus on further review and approval, largely by hiring new staff whose skills match the priorities of Title III. These priorities in turn are based on three issues: need for research; quickest impact on providing critical services; and lack of other funding for a project.

The Work Plan’s Subtitle A looks at patient experience and will spend $25.8 million to collect data on this and incorporate patient experience into the approval process.

Subtitle B will spend $95.3 million in direct efforts to produce new medicines. It will allow for new hiring and contracting of staff to produce more “drug development tools” for research and testing. It will support the production of “genetically targeted” and “variant protein targeted” drugs aimed at rare diseases by easing the approval process for manufacturers. It will also award grants for monitoring the ongoing production of existing drugs.

Subtitle C deals with clinical trials. It allocates $57.8 million to develop new designs for clinical trials and to hire experienced researchers to implement them. “Real world evidence,” defined as facts about the use and risks of a drug being developed, that came from outside formal clinical trials, will be considered in the approval process and will mean hiring statisticians and data analysts. Health and Human Services (HHS) regulations for the safety of human clinical trial subjects will be synthesized with those of the FDA.

Subtitle D involves patient access to treatment. It provides #185.2 million to improve patient access both to new and existing drugs, especially cancer treatments, and much of the funding would go to the FDA’s Oncology Center of Excellence. There will be funding to fast-track approval of Regenerative Advanced Therapies.

Subtitle F deals with therapeutic devices and equipment, applying $109 million to define “breakthrough technologies” and speed up the review and approval process for them. There will be significantly more contact between FDA staff and manufacturers, up to 20 percent more interaction per year. The “Humanitarian Device Extension,” which applies to devices that treat rare diseases, will be doubled. A new process for the creation of standards for devices, which coordinates with existing standards, maintained by professional groups, will be developed.

Subtitle G allocates $27 million to implement a plan that requires the FDA to establish one or more Intercenter Institutes for a major disease area or areas. One such institute — the Oncology Center of Excellence (OCE) – has already been established. It establishes a “unified policy approach and clinical review of all drugs, biologics and devices used in medical oncology.” Funds would be used to support FDA medical and professional staff's coordinated review of oncology product applications throughout all the medical product centers, as well as policy development and collaboration with external stakeholders.