Kevin Marsh, executive director, commercial strategy and new product development, patient-centered research, Evidera, and Margaret Vernon, vice president, general manager, patient-centered research, Evidera, discuss trial design and strategies for success.
The last 10 years have seen the complexity of clinical trials increase while the number of eligible patients per site has decreased, leaving recruitment targets unmet and higher budget and time costs. These issues are only compounded in rare disease populations where recruitment can be difficult due to several factors, including small populations distributed over wide geographic areas, a large number of pediatric patients in many rare diseases, patients’ health making travel difficult and a scarcity of diagnosis centers and investigators.
Furthermore, there is often little, if any, experience in conducting studies with rare disease populations. This means there is a lack of knowledge about which trial designs work for these special populations. To conduct a successful trial program, it is imperative that trials are designed and implemented in such a way to enhance patients’ and caregivers’ experiences and reduce burden and complexity. In January 2019, the FDA published its Draft Guidance on Rare Diseases, including a section on the importance of patient and caregiver engagement in rare disease clinical trials. The guidance specifically states that engaging patients with rare diseases can greatly facilitate both scientific accuracy and operational efficiencies in terms of identifying outcomes meaningful to patients and finding patients willing to participate in experimental studies.
Patients can be instrumental in providing input on designs and operational implementation of trials that enhances enrollment and retention. Patient input on protocol design can identify potential barriers to participation and retention and support development of appropriate solutions in various ways, such as:
- modifying design elements of the trial to ensure that patients think there is value in the study objective;
- development of key messages and outreach materials to enhance enrollment;
- development of logistical support, such as transportation, in the case of obstacles such as format, location, scheduling, length and timing of assessments;
- engagement and retention strategies such as providing patient-friendly communications, gamification or incentives in the case of a trial design that has been identified as lengthy or potentially burdensome;
- tailoring solutions to ensure the feasibility of trial participation in specific populations, perhaps identified by geographic location.
Involving rare disease patients and their caregivers in drug development, and particularly protocol design and operational implementation planning, can provide myriad benefits to the trial sponsor and the target patient and caregiver community at large. Benefits for sponsors include faster enrollment and reduced drop out, as well as lower associated costs. Perhaps the greatest benefit is for the patients—reducing the hurdles to participating in trials, minimizing unnecessary burden and complexity for patients participating in trials and getting drugs to market faster for patients who need them the most.
For more information on patient engagement in clinical trial protocol design and recruitment strategies, read the entire white paper on this topic.