Recently, the United States Food and Drug Administration (FDA) issued a Draft Guidance, “Decentralized Clinical Trials for Drugs, Biological Products, and Devices.” The draft guidance provides recommendations for sponsors, investigators, and other stakeholders regarding the implementation of decentralized clinical trials (DCTs) for drugs, biological products, and devices.
The guidance defines a DCT as a clinical trial where either some or all of the trial-related activities occur at locations other than traditional clinical sites. In a fully-decentralized clinical trial, all activities will take place at locations other than traditional trial sites. Homes of trial participants and local health care facilities that are convenient for trial participants are considered alternate locations. In a hybrid DCT, some trial activities will require in-person visits by trial participants to traditional clinical trial sites, and other activities can be conducted at locations other than a traditional clinical trial site, such as their home. Some examples of decentralized elements include obtaining laboratory tests at a local facility not the traditional trial site, or using telemedicine for a follow-up visit instead of mandating the participant come to the traditional trial site.
The FDA’s regulatory requirements for investigations of medical products are the same for DCTs and traditional site-based clinical trials. The Food and Drug Omnibus Reform Act directed the FDA to “issue or revise draft guidance that includes recommendations to clarify and advance the use of decentralized clinical studies to support the development of drugs and devices” by December 29, 2023.
The agency hopes that the guidance will support DCTs and ultimately lead to diversified clinical trial participation and improved trial recruitment and retention. FDA Commissioner Robert Califf said, “As we seek to improve our evidence generation system, decentralized clinical trials may enhance convenience for trial participants, reduce the burden on caregivers, expand access to more diverse populations, improve trial efficiencies, and facilitate research on rare diseases and diseases affecting populations with limited mobility.”
Recommendations
The FDA notes that fully decentralized trials may be appropriate for investigational products (IPs) that are simple to administer or use, have well-characterized safety profiles, and do not require complex medical assessments. Hybrid models may be more appropriate for more complex medical assessments or in cases where the administration of an IP needs to be performed at a clinical trial site, with follow-up assessments being conducted in a remote manner, either through telemedicine or by local health care providers.
The FDA notes that for inspectional purposes, there should always be a physical location where all clinical trial-related records for participants under the investigator’s care are accessible and where trial personnel can be interviewed. The FDA also outlined potential concerns with DCT, such as remote assessments differing from on-site assessments, especially if trial participants are responsible for performing their own physiological tests, such as home spirometry.
Additionally, the FDA provided examples of what should be considered when planning a remote clinical trial visit or clinical trial-related activities, such as specifying when a telehealth visit is appropriate and when a participant should be seen in person. The FDA further noted considerations to make when digital health technologies are used in DCT, such as reviewing draft guidance for industry on the topic (Digital Health Technologies for Remote Data Acquisition in Clinical Investigations) and ensuring that digital health technologies are available and suitable for use by all trial participants.
The draft guidance also delineates roles and responsibilities of the sponsors as well as the investigators and delegation of trial-related activities. The draft guidance also includes recommendations on investigational products in a DCT (drugs and biological products as well as medical devices) and considerations for packaging and shipping investigational products in a DCT, when necessary.
Request for Comments
The FDA notes that the guidance document is solely issued for comment purposes and is not legally binding. The FDA has requested comments on the draft guidance be submitted no later than August 1, 2023. Even once finalized, it only represents the agency’s current thinking on the topic and will not establish any rights for any person, nor will it be binding on the FDA or the public.