By Deborah Borfitz 

March 3, 2020 | Study sponsors looking to improve recruitment and retention in clinical studies—particularly for rare disease research where patients are in scarce supply—might want to apply some of the proven tactics recently shared by Signant Health and UBC at the 11th Annual Summit for Clinical Ops Executives (SCOPE) in Orlando.

Technology providers share the responsibility to expedite patients’ journey, which is beset with obstacles, according to Signant Health CEO Mike Nolte. Nearly one-third of patients drop out of phase III trials and the delays are costing sponsor companies $8 million daily in lost revenues. Only half of participants adhere to a study’s drug regimen and 53% never receive any study results. 

Historically, technology has been deployed in a linear way around events such as randomization and data collection and a lot of the cleanup work was pushed to the end of a trial, says Nolte. Beyond drug safety, patients haven’t been the focus of attention. 

Study participants are predominantly white and live in North America or Europe, and 42% of them are male, he points out. Less than half are recruited through a clinician, and “most participate out of a genuine desire to help others, but this often goes unrecognized.”  

What’s most valuable to patients engaged in research, Nolte says, is “time outside of the trial”—e.g., trips with their spouse, visits with siblings and grandchildren, family weddings and staying active. 

Their first source of information about a trial, he continues, is “other” (20%) followed by their primary care physician (20%), a research clinician (19%) and an advertisement (16%). Their primary rationale for not signing informed consent documents is, first and foremost, the burden of participation (46%). Risks (33%) and insufficient information (12%) are other concerns. The reasons for dropping out of a trial are largely safety-related—adverse effects (40%) and general risks to health (33%). 

Alarmingly, 76% of the total investment in clinical research is for drugs that never make it to market, Nolte says. Geography, infrastructure and study visit complexity are “significant barriers” and support a shift toward remote clinical trials. 

“Don’t underestimate the importance of celebrating patient participation in research,” he adds. Only 39% of patients are getting follow-up communications after a study’s completion, although 91% of surveyed participants say it’s important to get news about a study’s outcome. 

Nolte’s advice boils down to this: 

Patient-centricity can be particularly important for rare disease studies, as was discussed by Shazia Ahmad, director of patient and physician services at UBC. Out of more than 7,000 rare diseases that have been identified, only a few hundred have a treatment approved by the Food and Drug Administration, she says. Patients and families are “extremely motivated” and patient advocacy groups play a significant role. 

Protocols also tend to be quite complex, driven by the scarcity of patients and special medical administration and adherence procedures, she adds. Investigators and staff at research-naïve sites may need training. 

Ahmad recommends several strategies for improving recruitment and retention in this arena, starting with a “deep understanding” of patients’ diagnostic odyssey, care journey and the impacts on caregivers. Sites will need a disease- and patient-specific recruitment plan, and the ability to flexibly respond to the needs of individuals and their family, Ahmad says.  

“Enlist patient advocacy [groups] early in recruitment planning,” she continues, incorporating whatever possible to ease the burden of participation. Helpful partners in this endeavor include the National Organization for Rare Diseases (NORD), the Genetic Alliance and Global Genes as well as local advocacy groups. 

One potentially useful tactic is to integrate value-added services, such as genetic testing to identity other carriers of disease within the family, she says. Mobile apps and wearables could also be deployed for the collection of patient-reported outcomes, and physical and physiological assessments, as well as to send out reminders.   

Patients may have to be identified via registries and ICD-10 codes in claims data, Ahmad says. Electronic health records can also be a valuable resource for geo-targeting patients for protocol-ready studies while improving physician awareness. “There are privacy concerns, but patients want to be found,” she emphasizes.  

Involving key opinion leaders early on can incite the participation of other physicians in the recruitment process, says Ahmad. Concierge services for people with rare diseases is critical, in terms of alleviating the inconvenience of getting to study sites at the appointed time and accommodating family members on overnight stays. Home nursing can also be a less burdensome alternative to in-person site visits.  

Online Tactics  

Digital recruitment strategies need to include Twitter, a favored social media channel among patient advocates, and engagement with online communities, she continues. “Take advantage of trending hashtags.” The call to action may simply be to improve understanding of the disease. 

“Engagement needs to be a two-way street,” Ahmad says, and should provide opportunities to learn how the disease impacts patients and ways they look for studies. “If you only talk about studies and recruitment, the message will become white noise.” 

Ahmad shares that her own daughter, at age 3 (she is now 17), was diagnosed with Kawasaki disease, which if not immediately treated attacks every organ in the body. Part of the credit goes to the Kawasaki Disease Foundation. Like many advocacy groups, its website provides a list of enrolling clinical studies. 

UBC was engaged by one sponsor for a 100-site global pediatric multiple sclerosis study with a targeted enrollment of 190 patients and a 60% screen failure rate. Parents were hesitant to sign up their child for the double-blind trial, which required weekly injections, says Ahmad. But using digital outreach, geo-targeted ads, pre-screening via a call center, knowledge of country regulations on outreach, advocacy groups and concierge services, 211 patients were ultimately enrolled. 

Next Steps 

For purposes of retaining patients in studies, Ahmad suggests using patient surveys on smartphones and handheld devices along with a system for quickly connecting patients with someone on the study team. Concierge services are also important, she adds, as is the patient stipend option and, especially in rare pediatric oncology, home nursing. 

Once a trial ends, best follow-up practices include providing access to study information, building relationships for future trials and empowering individuals, Ahmad says. UBC has created a weLink patient community platform as an avenue for sponsors and study participants to maintain contact and access study data and lay summaries, which are already required in Europe.