The FDA's Center for Drug Evaluation and Research (CDER) has outlined its agenda for new and revised draft guidance documents in 2023, offering insights into the agency's priorities for clinical trial design and drug development. This includes long-awaited guidance on decentralized clinical trials (DCTs), new considerations for psychedelic research, expanded guidance on master protocols, and international harmonization efforts for adaptive trials.
Decentralized Clinical Trials (DCTs)
The draft guidance on DCTs is expected to be released by the end of 2023, as mandated by the Fiscal Year 2023 omnibus spending bill. Craig Lipset, co-founder and co-chairman of the Decentralized Trials & Research Alliance (DTRA), suggests the FDA will likely draw inspiration from existing documents, such as the Clinical Trials Transformation Initiative (CTTI) recommendations and the EMA’s recommendations paper. This guidance aims to provide clarity, reduce perceived risks, and sustain the adoption of DCTs, which accelerated during the COVID-19 pandemic.
Psychedelic Research
A notable addition to the agenda is draft guidance on "Psychedelic Drugs: Considerations for Scientific Investigations." This is particularly significant given the rapid expansion of psychedelic research. Albert Garcia-Romeu, PhD, of Johns Hopkins University, notes the FDA likely aims to standardize research methodologies, covering cardiovascular monitoring, mental health screening, drug interactions, and clinical licensure requirements. The guidance is expected to improve transparency and safety in psychedelic drug development, though further refinement for different psychedelic subclasses will likely be needed in the future.
Master Protocols
Building on its previous guidance for oncology drug development using master protocols, the FDA plans to release draft guidance on "Master Protocols for Drug Development and Biological Product Development," extending the framework to non-oncology clinical trials. Dr. Ben Saville of Berry Consultants anticipates this will address data sharing issues, shared placebos, and the use of previous controls. The goal is to promote the benefits of platform trials and accelerate clinical development across various disease areas.
International Harmonization on Adaptive Trials
The FDA is also working to harmonize its guidance with international regulators through the "E20 Adaptive Clinical Trials" initiative with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). Jay Park, PhD, of Core Clinical Sciences, emphasizes the importance of international harmonization to avoid competing or inconsistent advice across different regulatory jurisdictions. The ICH guidance is expected to complement existing FDA guidance on adaptive design clinical trials, covering Bayesian adaptive clinical trials and the use of real-world data.
Disease-Specific Guidance
The agency is also developing disease-specific draft guidance documents, including those for early Lyme disease, neovascular age-related macular degeneration, efficacy endpoints for clinical trials investigating antidiabetic drugs and biological products and preventive migraine treatment. A planned draft guidance is focusing on chronic pain drug development.
Other Notable Plans
Additional planned guidance includes institutional review board (IRB) review of individual patient expanded access requests and key information on the informed consent process for FDA-regulated clinical investigations. The agency also aims to provide guidance on using clinical practice data in randomized controlled trials for regulatory decision-making for drug and biological products.
