By Hilary Marston, MD, MPH, CMO, FDA
LinkedIn: Hilary Marston
LinkedIn: FDA
Kevin Bugin, PhD, Deputy Dir Operations,
Center for Drug Evaluation and Research
LinkedIn: Kevin Bugin
Breakthroughs in science and technology are advancing innovation in more areas of medicine, providing novel treatment options for patients. The scientific community has a deepening understanding of disease and human biology, and of the enormous potential afforded through new technologies.
The same spirit of innovation is enhancing the design and conduct of clinical research. As the U.S. Food and Drug Administration observes Clinical Trials Day, we reflect on the progress made and the widespread efforts underway to advance clinical trial innovation. This progress has been possible because of clinical trial participants and their families, health care professionals, medical product developers, federal partners and the entire clinical research community.
Advancing Innovative Trial Design
Clinical research plays a crucial role in supporting the development of new medical products to address unmet patient needs. The FDA has several ongoing initiatives to support innovative clinical trials and protect people participating in clinical research. This includes our work through collaboration with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the International Medical Device Regulators Forum.
Enhancing diversity in clinical trials is among the FDA’s top priorities to help improve the data the agency receives about the patients who may potentially use the medical products. The FDA has published a number of guidances to promote diversity and inclusion of underrepresented populations in clinical trials to help ensure the generalizability for the intended patient population, including guidances specifically addressing enrollment of children, pregnant people, older adults and underrepresented ethnic and racial groups, as well as guidances on innovative trial designs and technologies that should make clinical trials more accessible, such as decentralized clinical trials and digital health technologies.
The FDA works to ensure participant views are incorporated in all phases of clinical research. For example, Patient Focused Drug Development meetings and patient listening sessions enable us to hear real world experiences of living with a disease and help us understand when a patient may consider participating in a clinical trial.
We are also working to help ensure the informed consent process supports empowered decision-making for people considering participating in a clinical trial. Recently, the FDA published a draft guidance on key information in informed consent that includes strategies to help ensure consent information is easier to understand for prospective research participants.
The FDA’s Center for Drug Evaluation and Research (CDER) recently established the CDER Center for Clinical Trials Innovation (C3TI) to enable and amplify innovative approaches to clinical trials that are designed to improve the efficiency of drug development. C3TI will:
- Share knowledge and tools on clinical trial innovation topics through a centralized knowledge repository and amplify through enhanced internal and external communication.
- Enable targeted demonstration projects to test, implement and scale the integration of innovation into clinical trials.
- Provide external and internal parties with a single CDER contact to help coordinate across innovation-related inquiries.
Last October marked the 10-year anniversary of the FDA’s Center for Devices and Radiological Health’s (CDRH) Early Feasibility Studies (EFS) Program and the publication of the FDA’s Guidance on Investigational Device Exemptions (IDEs) for Early Feasibility Medical Device Clinical Studies, Including Certain First in Human (FIH) Studies (EFS Guidance). Since its inception, the program has supported device innovation and increased patient access to potentially beneficial new technologies. EFS allow investigators to gain early clinical experience with investigational devices, often to address unmet clinical needs, while incorporating enhanced risk mitigation strategies to protect patients. This early clinical experience provides initial insights into device proof-of-principle and safety, guides device and procedure improvements, and informs clinical study modifications and non-clinical testing strategies, which together enhance device development efficiency.
The FDA’s Center for Biologics Evaluation and Research (CBER) and CDER, recently hosted a public workshop, Advancing the Use of Complex Innovative Designs in Clinical Trials: From Pilot to Practice. This workshop facilitated discussion on innovative clinical trial topics including the use of external data sources, Bayesian statistical methods, and simulations in complex innovative trial designs as well as trial implementation. Three case studies of CBER- and CDER-regulated trials illustrating various aspects of complex innovative designs and implementation were presented, followed by panel discussions motivated by the case studies.
Additionally, the FDA’s Oncology Center of Excellence recently hosted a workshop with the American Association for Cancer Research on the topic of clinical trial designs for multiple phases of treatment regimens, an increasingly common issue in cancer treatment.
The Future of Clinical Trials
The future of clinical research will depend on generating evidence in simpler and more efficient ways, including incorporating innovation into routine clinical practice and expanding access to clinical trials.
C3TI and other initiatives to support clinical trial innovation will work to adopt transformative methodologies and technologies in clinical research to help accelerate the development of safe and effective medical products. Ongoing initiatives—including the Advancing Real-World Evidence program and discussion papers on artificial intelligence and machine learning—represent the agency adapting to new developments related to the field.
The FDA remains committed to supporting clinical research including engaging patients and caregivers to understand their unique perspectives to promote innovative clinical trial design. Collaboration among the clinical research community is key to help solve scientific challenges and ultimately help ensure patients receive the safe and effective treatments they deserve.
This article was originally published on FDA Voices and is reprinted here with permission.