On May 2, 2023, the U.S. Food and Drug Administration (FDA) released Draft Guidance regarding the implementation of Decentralized Clinical Trials (DCTs) for drugs, biological products, and devices.

What is a Decentralized Clinical Trial?

DCTs are clinical trials where some or all trial-related activities occur at locations other than traditional clinical trial sites. Examples of decentralized elements include obtaining laboratory tests at a local facility rather than an academic medical center or conducting a clinical trial follow-up visit in a participant’s home via telemedicine. A DCT can be fully decentralized (for example, all trial activities occur via telehealth) or hybrid (for example, administration of the investigational product occurs at a traditional site while follow-up visits are conducted at-home or via telehealth). As we discussed previously, reliance on local clinical laboratory facilities and access to telehealth providers and digital health technologies have greatly expanded the types of trial-related activities that can be conducted remotely and the scope of data that can be collected.

By utilizing technology, such as telemedicine, to deliver consent and education, remote monitoring, direct-to-patient distribution of investigational medicines and wearables, use of local labs and image centers, and providing the opportunity for a research participant to engage in research activities from the comfort of their home or other convenient location, DCTs offer a more patient-centric approach to clinical trials. In FDA’s press release, FDA Commissioner Robert M. Califf, M.D. remarks, “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.”

FDA’s New Draft Guidance

The Draft Guidance builds on FDA’s 2020 recommendations which were issued in response to the quarantines, site closures, and travel limitations resulting from COVID-19. While FDA maintains its normal requirements for traditional site-based trials, the Draft Guidance provides recommendations such as design considerations, conduct of remote clinical trial activities, use of digital health technologies to remotely acquire data, roles of the sponsor and investigators, informed consent and institutional review board oversight, determination of the appropriateness of investigational products, packaging and shipping of investigational products, and safety monitoring of trial participants.

FDA has expressed its commitment to working with sponsors to discuss how decentralized elements may fit into a clinical trial.

Considerations When Conducting a DCT

In the Draft Guidance, FDA outlines the importance of creating specific plans to facilitate the decentralization of the trial, such as how to coordinate trial activities, interact with local health care facilities, providers, and laboratories, visit participant homes, and distribute investigational products.

The DCT should be designed in such a way that all activities are under the oversight of the investigator regardless of the network of locations where trial-related services are provided. The plan should consider how to collect, store, access, and interpret data obtained from non-traditional sources. The plan should also consider the training of trial personnel, the appropriateness of remote visits, verifying participant identity, developing records, and addressing adverse events. When using digital health technologies, sponsors and investigators should refer to FDA’s draft guidance regarding Digital Health Technologies for Remote Data Acquisition in Clinical Investigations issued in December 2021.

Although DCTs are proving to be promising, close attention should be paid to state laws regarding corporate practice of medicine, licensure and scope of practice requirements for trial personnel, regulatory obligations associated with the use of telehealth or remote patient monitoring, direct-to-patient shipping considerations, payor reimbursement rules, federal and state privacy and confidentiality laws, and contracting issues related to interacting with various local providers and vendors.

FDA is accepting public comments on the Draft Guidance until August 1, 2023.

Health systems, clinical research organizations, and other emerging companies considering DCTs should take a serious look at this new FDA guidance and keep abreast of developments that can significantly reduce the barriers of participation in research and address certain public health needs.