Rare Disease Recruitment: 5 Strategies for Differentiating Trials in a Competitive Research Environment

Recruitment for rare disease research has always been difficult due to myriad factors inherent to the low incidence and prevalence of these conditions. In recent years, however, recruiting for rare disease trials has become even more challenging due to increasing competition in the clinical research environment. In addition, as technologies advance and information becomes more accessible, some rare diseases have become easier to diagnose, and new rare conditions are being identified. This progress has spurred increasing interest among sponsors, healthcare providers, patients, and caregivers to find solutions for addressing unmet therapeutic needs.

This blog, discusses key rare disease recruitment challenges and explores strategies for differentiating trials in today’s competitive landscape.

Challenges in rare disease clinical research

Longstanding rare disease clinical trial recruitment obstacles include small, geographically dispersed patient populations, disease heterogeneity, delays in diagnosis, and low—or even lack of—disease awareness. Further, approximately half of rare diseases affect children, adding layers of ethical, legal, and logistical complexity to study planning and execution.

As more sponsors turn their attention to rare and ultra-rare diseases, recruitment has become even more difficult. Study designs sometimes require naïve patients, limiting an already small pool of potential participants to those who have never enrolled in an interventional clinical trial. Moreover, many trials include long-term extension studies that keep participants on treatment, making those patients either uninterested or ineligible to enroll in another clinical trial. Consequently, the funnel of qualified study participants has gotten narrower.

Differentiating rare disease studies

To build awareness and create enthusiasm for their studies, sponsors need to adopt an approach to study planning that begins and ends with patients—and their families—and prioritizes them throughout every step of clinical development.

Below are five strategies that may help sponsors differentiate their rare disease trials.

1. Understanding the patient journey.

For many patients with rare diseases and their families, the journey to a correct diagnosis takes at least five years, with countless physician visits and tests along the way.^[i] Lack of awareness and limited data on disease pathology and progression leads to uncertainty and delays. Even when a diagnosis is made, therapeutic options are limited. Understanding the diagnostic odyssey—and where the opportunities lie to identify ready, willing, and eligible patients to participate in clinical trials—can help sponsors develop an effective recruitment strategy.

2. Helping stakeholders feel invested in the study.

Allowing stakeholders to inform and influence study design can be a powerful motivator for clinical trial participation. Engaging key opinion leaders, patient advocacy groups, patients, and families early and often during the study planning process helps ensure that the design and endpoints are measurable and meaningful. If stakeholders believe that a clinical study will make a difference in participants’ daily lives, they may be more likely to encourage enrollment.

To help sponsors engage with patient advocates locally, Premier offers patient advocacy liaisons (PALS) who perform regional research to identify and collaborate with local advocacy groups.

3. Designing studies with patients and their families in mind.

Whether limiting the number of study visits or selecting a clinical outcome assessment (COA) that accurately captures a patient’s experience, sponsors should challenge themselves to design meaningful and acceptable trials for participants and their families. Given that most people with rare diseases have limited treatment options, it is also important to ensure that, where feasible, the study design offers an opportunity for every patient to receive treatment.

4. Making study participation as easy as possible.
Participating in a clinical trial is a significant commitment for patients and their families. Sponsors can ease the burden of participation by:

5. Expanding recruitment to emerging regions.

To increase the pool of eligible patients, sponsors may consider including investigators and sites in countries or regions where clinical research has been less competitive. For example emerging markets such as Asia Pacific, Latin America, and the Middle East have increased their participation in clinical research. Globalizing trials benefit from enhancing access and diversity, and increasing complexity due to logistics and nuances in standard of care and culture.

To increase the pool of eligible patients, sponsors may consider including investigators and sites in countries or regions where clinical research has been less competitive. For example emerging markets such as Asia Pacific, Latin America, and the Middle East have increased their participation in clinical research. Globalizing trials benefit from enhancing access and diversity, and increasing complexity due to logistics and nuances in standard of care and culture. At Premier, we have experience studying some of the rarest conditions. Our work spans all aspects of drug and clinical trial development, encompassing more than 242 rare disease studies across multiple indications. Click here to learn more about how Premier can help move rare disease research forward.

^[i] National Organization for Rare Disorders. New Patient Journey Infographic Gives a Glimpse into the Diagnostic Odyssey, April 29, 2021. Available at https://rarediseases.org/new-patient-journey-infographic-gives-a-glimpse-into-the-diagnostic-odyssey/.