Drug development and clinical research methods evolve continuously through establishment of new regulations and through adaptations to changes in treatment accessibility, technology, evolving knowledge in biology, and appearance of new diseases.
Patient-centric trials, in which the perspective of the patient is considered in study design and performance, is one such new approach in clinical research that primarily focuses on reducing patient burden.
In 2012, FDA issued the Patient-Focused Drug Development Guidance for Industry, which was later integrated into the 21st Century Cures Act. The Act was designed to accelerate medical product development, bringing cures and treatments more efficiently and quickly to patients. In light of continued efforts to improve diversity, equity, and inclusion in all aspects of society, the FDA has also provided a Guidance for Industry (Enhancing the Diversity of Clinical Trial Populations) describing ways of increasing diversity in clinical trials.
What are the challenges to greater inclusion?
Patient recruitment is always a challenge. Every patient is unique, exhibiting different signs & symptoms, other medical conditions, and availability for study procedures and visits.
But what are some additional challenges to increasing diversity in clinical trials?
Mistrust of the clinical trial process and medical institutions due to historical events and systemic institutional racism.
Historical exclusion of women in trials, especially women of childbearing potential.
Exclusion of patients due to age, body mass index (BMI), lack of English language fluency, difficulty in accessing the clinical trial site (hours or location), and non-traditional gender identity.
How do we address challenges and provide a more patient-centric and inclusive experience?
Research by the Tufts Center for the Study of Drug Development has shown that involving patients and/or advocacy advisory boards in protocol design can be beneficial to patients and sponsors. To be included in a study, patients may need to meet fewer eligibility criteria and/or may experience fewer procedures per visit. Sponsors may benefit by needing fewer protocol amendments to adapt to patient and site experiences, shorter trial duration due to faster patient recruitment, and, as a result of these benefits, a lower budget than anticipated (Getz, Applied Clinical Trials Online, September 2021).
Another method to increasing patient engagement in trials is to bring the trial to the patient; i.e., a decentralized trial approach. The methods used in decentralized trials – mobile devices, visiting healthcare professionals - can make participation in a trial more convenient or even possible for patients and can engage a diversity of health care providers in clinical trials. These techniques help meet the goal of considering “diversity when selecting health care providers and study coordinators to assist with clinical trial recruitment” stated in the FDA guidance.
The benefits of patient-centric and inclusive study design
We’ve reviewed some of the challenges to inclusivity in clinical trials and some of the strategies to improve patient engagement.
Benefits of patient-centricity to the trial participants include:
More trials that address the greatest concerns of patients
Greater ease of participation due to clearer inclusion criteria, and fewer burdens of participation, such as fewer tests, use of mobile devices, and use of home healthcare services
Patient insight into their trial data
Benefits of patient-centricity to trial sponsors include:
Protocols targeted to patient needs and abilities
Greater patient/advocacy group engagement due to participation in design meetings, insight into trial results
Greater diversity of patient population due to involvement of advocacy groups, use of mobile devices, and use of home healthcare services
Faster enrollment and greater patient retention in trials
More robust data due to patient retention
Lower trial costs due to targeted assessments, faster enrollment, and greater retention
Consideration of patient experience, needs, preferences, and goals in the design and implementation of clinical trials has multiple benefits for patients and for sponsors. Patient engagement can lead to greater patient diversity in trials and result in higher patient retention. Sponsors will notice more robust data, reduced trial timelines and budgets, and more trials that successfully meet trial goals.