Life Science Compliance Update

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November 10, 2017

Insight into 21st Century Cures Implementation

CuresActCongress

According to a proposal of how 21st Century Cure funds will be used (subject to the congressional appropriations process), FDA released a document explaining the bulk would be dedicated to sections of the law on “Advancing New Drug Therapies” and “Patient Access to Therapies and Information.” Other funds will go to modernizing clinical trial designs, patient-focused drug development, medical device innovation and improving scientific expertise and outreach at FDA.

Cures Implementation

FDA proposes to allocate the authorized Innovation Account amounts to the activities that represent the best opportunity to achieve the goals of the Cures Act in conjunction with other funding sources subject to their availability. FDA’s proposed allocation does not spread the Innovation Account funds across all eligible sections. Some eligible provisions may assist FDA in carrying out its responsibilities but do not require any specific investment in new or expanded program activities. Others complement projects and commitments under current or anticipated reauthorized user fee programs and could be funded in part through some user fee resources subject to the availability of funds. Still others dovetail with on-going work supported by budget authority.

Charge to FDA Science Board

In the Cures Act, Congress requested “recommendations from the Science Board to the Food and Drug Administration, on the proposed allocation of funds … and on the contents of the proposed work plan.”

The Cures Act requires FDA to allocate Innovation Account funds for identified eligible activities. The resulting proposals were evaluated and prioritized according to the following criteria:

  • Provisions that present the greatest opportunity for FDA to foster innovation and integrate advances in biological sciences, engineering, information technology, and data science, to most directly improve the Agency’s product review tools and processes.
  • Provisions that address the greatest need for scientific modernization.
  • Provisions that have the most immediate impact on delivery of services to patients, the medical product industry, academia, and health professionals.
  • Provisions for which other funds may not be available.

Patient-Focused Drug Development

The Cures Act emphasizes the need for patient engagement and directs the Agency to include the patient’s voice in drug development and review. Section 3001 requires FDA to make public a brief statement regarding whether and how patient experience data and related information that was submitted, if any, was used in the review of a drug or biologic marketing application. Section 3002 requires FDA to issue one or more guidances for the purposes of drug development and regulatory decision making. Guidance should address such issues as acceptable methodological approaches for collecting, measuring, and analyzing patient experience data. Section 3004 directs FDA to issue reports at specified intervals assessing the use of patient experience data in regulatory decision making, especially focusing on the review of patient experience data and information on patient-focused drug development tools as part of approved applications.

In implementing these provisions FDA aims to facilitate a more systematic gathering and use of patients’ perspectives on their disease/condition and available therapies to treat their disease/condition. The work plan proposes to use Innovation Account funds to implement certain elements of these provisions.

Advancing New Drug Therapies

FDA must develop a new regulatory process under the Cures Act to qualify drug development tools (DDTs) in order to facilitate timely and consistent review of DDT qualification submissions and publicly disseminate broader information about DDTs under review and following a qualification determination. Once a DDT is qualified under this new process, any sponsor may use it for its qualified context of use to support an application for approval or licensure of a drug or to support the investigational use of a drug. Expert FDA staff and contractors will help develop evidentiary criteria needed to support qualification, develop regulatory informatics platforms, and integrate new review processes. This must occur quickly to ensure FDA can meet its obligations under this section within statutory timeframes, including developing and issuing guidance outlining procedures for the qualification process, holding public meetings, and posting public reports. Once fully implemented, this section has the potential to transform drug development and review.

Other Sections

FDA’s plan addresses several other important areas as mandated by the Cures legislation. First, under Modern Trial Design and Evidence Development, FDA notes Section 3021: Novel Clinical Trial Designs. This section directs FDA to assist sponsors in incorporating complex adaptive and other novel trial designs into proposed clinical protocols and applications for new drugs and biological products in order to facilitate more efficient product development. To accomplish this FDA must hold a public meeting months after the date of enactment of the Cures Act and issue guidance on, among other things, how to use such novel trial designs, how they can help to satisfy the substantial evidence standard, and recommended analysis methodologies.

Regarding Patient Access to Therapies and Information, Section 3036 is interesting: Standards for Regenerative Medicine and Regenerative Advance Therapies. This section requires the establishment of standards and consensus definitions to support the development and review of regenerative medicine therapies, including with respect to the manufacturing processes and controls of such products. In order to meet the requirements of the Cures Act, CBER will use innovation funds to facilitate a public process with the National Institute of Standards and Technology and other stakeholders to coordinate and prioritize the development of standards and consensus definitions of terms. Such standards and terms will help foster the development, evaluation, and review of regenerative medicine therapies, including with respect to the manufacturing processes and controls for such products. Innovation funds will support expert FDA staff engaged in this work, as well as help facilitate long-term engagement with stakeholders on regenerative medicine standards development and consensus definitions through the creation of a public-private partnership.

Regarding Medical Device Innovation plans, FDA writes about Section 3051: Breakthrough Devices. This section writes into law and expands FDA’s Expedited Access Pathway program, which allows for expedited development and review of devices that represent breakthrough technologies for life threatening or irreversibly debilitating diseases/conditions. The Breakthrough Devices program requires frequent and extensive interactions between device companies and FDA staff during the device development phase, as well as priority review for breakthrough medical devices. FDA estimates that this program will grow at a rate as high as 20 percent per year over the next 10 years. FDA expert staff will accommodate the increased workload and will need to acquire the IT systems needed to fully implement the program. The program will accelerate patient access to these lifesaving therapies.

FDA aims in another subsection of the document to outline its work to improve scientific expertise and outreach. Section 3073 relates to Intercenter Institutes. This section requires FDA to establish one or more intercenter institute(s) to help develop and implement processes for coordination of activities in major disease areas between the drug, biologics, and device centers. FDA has established the Oncology Center of Excellence (OCE) to create a unified policy approach and clinical review for all drugs, biologics, and devices used in medical oncology. It will leverage the combined talents and skills of all FDA regulatory scientists and reviewers who work in medical oncology product review.

OCE will also serve as a single point of contact for external stakeholders for our work in cancer, including professional societies and patient advocacy groups. FDA medical and professional staff will coordinate review of oncology product applications across the medical product centers, policy development, and collaboration with external stakeholders. This Center of Excellence will help expedite the development of oncology and hematology medical products and support an integrated approach in the clinical evaluation of drugs, biologics, and devices for the treatment of cancer.

Conclusion

The proposed FDA work plan describes activities that are intended to successfully achieve the Cures Act requirements in areas that are a high priority for any Innovation Account funding that is appropriated. Together with other funding sources, as available, FDA’s proposed Innovation Account funding allocations can help chart a path for advancing medical product development and reviews and help bring innovative new therapies and products to patients and health care providers in a more timely and efficient manner.

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