FDA proposes Standards Recognition Program for regenerative medicine therapies

Center for Biologics Evaluation and Research issues draft guidance on use of voluntary consensus standards

US Government Agencies are directed by the US Federal Government to use voluntary consensus standards rather than developing their own, unique standards, unless voluntary consensus standards are inconsistent with law or otherwise impractical. Furthermore, the FDA is directed to “recognize all or part of an appropriate standard established by a nationally or internationally recognized standard development organization for which a person may submit a declaration of conformity in order to meet a premarket submission requirement or other requirement”. The FDA centre of Devices and Radiological Health (CDRH) has an established programme for recognising standards. A list of consensus standards that CDRH has recognized or decided to recognize is available on the FDA Recognized Consensus Standards Database.

Standards can be used to meet premarket submission requirements and support the premarket review process for medical devices. However, in most cases the standards would only satisfy part of the submission requirements. Standards can be used to support different types of premarket submissions to the FDA, such as:

• Any 510(k)-submission including Abbreviated Premarket Notifications 510(k)s
• De Novo Requests
• Investigational Device Exemption (IDE) Applications
• Premarket Approval (PMA) Applications
• Product Development Protocols (PDP)
• Humanitarian Device Exemptions (HDE) Application

The FDA Center for Biologics Evaluation and Research (CBER) has recently issued a draft guidance on use of voluntary consensus standards for regenerative medicine therapies. This programme is modelled on the formal standards and Conformity Assessment Program for medical devices in CDRH. The guidance extends the existing guidance on standards development and the use of standards in regulatory submissions reviewed in CBER from March 2019 to regenerative medicine therapies. Regenerative medicine therapies include cell therapies (allogeneic and autologous), therapeutic tissue engineering products, human cell and tissue products, and combination products using these therapies or products. Additionally, the FDA interprets the definition of regenerative medicine therapies as including human gene therapies, such as genetically modified cells that lead to a sustained effect on cells or tissues, and xenogeneic cell products.

The CBER guidance outlines the requirements to be met in the process for development of voluntary consensus standards for a standard to be recognized. It also lays out the process for requesting recognition of a standard and the way that standards will be reviewed for recognition. The guidance indicates that using voluntary consensus standards can eliminate costs associated with the development of government-unique standards and promote international harmonization of standards that are acceptable to the FDA. The FDA intends to maintain a list of voluntary consensus standards that have been recognized in whole or in part under the programme on the FDA Regenerative Medicine website. The draft guidance indicates that the CBER intends to update information on the website twice a year.

Manufacturers of products for regenerative medicine therapies should consider this draft guidance and watch out for its finalization. When the recognition programme is operating, manufacturers will need to monitor the listing of recognized standards and consider participating in the development of potentially recognisable standards.