A blog post by Anne B. Cropp, Pharm.D., BCAP, Clinical Research Pathways Board of Directors
Many clinical trials fail in part because they can’t recruit or retain enough patients to accurately assess an investigational drug’s safety and effectiveness. That’s especially true for trials that involve patients with ultrarare diseases, which FDA defines as affecting 1 or fewer in 50,000 people.
In any clinical trial, challenges with enrollment often result in costly protocol amendments and program delays. To mitigate this risk, drug developers increasingly are taking the proactive step of seeking the input of the experts—patients and caregivers—early in study planning and design. Early engagement is making a difference, resulting in improved patient enrollment, reduced obstacles to clinical trial participation, shorter timelines, and greater trust in research.
What patients can tell us
This proactive approach recognizes that patients have information that’s essential to successful study design. Such information could include the types of specialists that patients see, the facilities where they seek treatment, the frequency and duration of visits, and the feasibility of some study requirements.
For example, patients may have a neurological condition wherein they seek the assistance of multiple specialists. The diagnosis might not always be by members of the medical specialty that a drug development team expected. Without speaking to patients beforehand, those responsible for study enrollment might target neurologists to help with recruitment when, in fact, most patient-provider interactions occur with another therapeutic specialist. Without these insights, drug development teams will lose valuable opportunities for effective clinical trial recruitment.
Patients also have essential insights on potential roadblocks to clinical trial participation and ways to overcome them. For example, does the patient require a caregiver and, if so, how does that affect trial design? How important is patient access to a clinical center of excellence? Are there physical limitations that would prevent most patients from traveling or meeting study requirements, such as spending an entire day at a trial site or enduring hours of fasting?
Benefits beyond the clinical trial
Addressing patients’ needs can prevent participants from dropping out of trials before they’re completed and can avoid time-consuming, costly protocol amendments. In addition, meeting with patients provides opportunities to collect meaningful insights on day-to-day life with their condition. These data can help inform the study team not just about the investigational drug but also about the disease itself.
The exchange of information works both ways. Patients gain a better understanding of the risks and potential benefits of research and how protocols are designed and finalized. Often, this understanding results in greater trust in research and increased willingness to participate in clinical trials. If they are part of a patient advocacy group or other disease-related network, patients also can influence the opinions and actions of others with their disease.
There also is an indirect but significant positive impact to the general public, which benefits from the new knowledge that’s gained with every completed clinical trial.
Anne B. Cropp, Pharm.D., BCAP, is the founder and chief scientific officer of Early Access Care, a healthcare firm that helps accelerate access to investigational drugs for patients in need. Dr. Cropp has more than 25 years of experience in pharmaceutical and biopharmaceutical drug development. She is a member of the Board of Directors of Clinical Research Pathways.