Patient Driven Study Design

Case study looks at one lupus trial that used patient feedback upfront to support their participation needs

Recruiting is notoriously the most expensive and time consuming part of clinical research. It can also be very difficult to predict. A recent examination of recruitment strategies for cancer clinical trials showed that more than half of eligible participants simply declined to participate on initial contact. Another nine percent agreed orally to participate, but never completed the enrollment.

Unfortunately, this is an all too common occurrence in clinical research. According to a recent Tufts Center for the Study of Drug Development (CSDD) report, 11% of sites at any given trial fail to enroll a single patient, and more than a third fail to meet their recruitment targets in the desired timeframe. Recruitment and retention challenges significantly extend the trial timeline, delaying the progress of bringing innovative new treatments to market.

Patients have a lot of reasons for not participating in trials. Some are based on lack of awareness regarding clinical research, misconceptions about the safety of trial participation, or concerns about health insurance for their care; others have logistical or daycare challenges, or other quality of life issues that make participation seem overly burdensome. In many cases, these barriers could be addressed with minor adaptation – but only if sponsors and Clinical Research Organizations (CROs) talk to patients and incorporate their feedback into study design.

Incorporating the patients’ perspectives is becoming an increasingly important part of the entire clinical research process, and it should begin with understanding why patients participate in a trial – and what’s standing in their way. Historically, sponsors and CROs rarely incorporated patient feedback into trial design, which may have caused them to alienate the very patients they wanted to recruit.

Lupus patients steer study design

Deploying a patient-centered study design process to engage directly with patients and use their feedback creates a more patient-centric trial experience. Patient-driven study design helps sponsors understand patient interest in participating in their trials, and identify barriers to participation. This feedback helps sponsors adapt their study design to overcome patient-identified challenges and better gauge how difficult it will be to meet recruiting goals.

QuintilesIMS recently demonstrated the value of this service for a lupus clinical trial. Before recruiting began for the trial, we met with 10 representative patients, jointly identified through the Lupus Foundation of America North Carolina chapter, and general market access channels, to ensure we had a mix of patients with varying levels of engagement in both disease advocacy and personal health management. Each patient was individually interviewed via WebEx for 90 minutes using a detailed interview guide. Moderators were trained to ask non-leading questions to eliminate bias and create consistency in the quality of feedback gathered. The interviewees were also shown information about the trial process to ensure they all had a baseline knowledge of the proposed clinical trial, and a general idea of what the lupus study would involve. Their responses were recorded, but kept confidential.

What they want: Comfort, convenience, and comprehension

Overall, patients were receptive to participating in the study as it was designed, even though it required many in-office appointments. This was valuable, as it helped the sponsor gain a sense of the lupus patients’ feelings about trial participation. Notably, a high level of hesitation about taking part in the pharmacokinetics sub-study, which requires daily blood draws, surfaced. While many patients saw the draws as unduly burdensome, others said they would be more likely to consider it if the site experience and logistics were appealing enough. Patients also reported a desire to feel connected to the trial. They wanted to better understand why procedures would be performed; they had general concerns about the safety of trial participation, and the impact it would have on their day-to-day lives.

Based on this feedback, we defined a set of tools for the sponsor to create a more engaged and informed patient population and improve trial recruiting and retention. These include providing patients with educational materials and customized study messaging, a calendar of visits explaining what will happen at each site visit, an opportunity to engage with site staff to provide periodic feedback, and reminders of appreciation to show the patients they are a valued part of the process.

By planning up front to talk to patients, we not only identified barriers to participation, but we signaled to these specific patients, and the patient community in general, that this is a sponsor who cares about patient concerns, and is eager to incorporate the patient perspective into the trial design. In a highly competitive clinical research landscape, being known as the company that prioritizes patient needs could make a real difference in a patient population that may be considering multiple trials or treatment options.

As sites compete for limited patient populations and the cost of trials continues to rise, sponsors need to do everything they can to make trial participation as easy and engaging as possible. Working directly with patients through patient-driven study design initiatives can give sponsors the insight they need to achieve their recruiting goals.

Marie Lux, Senior Director, Site & Patient Networks, QuintilesIMS