By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease
Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around. This departure from traditional recruitment methods, where sites would typically initiate contact with patients, presents both opportunities and challenges, marking a pivotal moment in how we approach patient engagement and recruitment strategies to reduce site burden.
The Shift from Traditional Recruitment Methods
Historically, clinical trial recruitment relied on sites identifying and reaching out to potential participants, and most strategies for successful clinical trials were underpinned by finding the right sites with the right access to patients. However, this model does not always translate well into rare diseases for several reasons:
- Many rare diseases do not have treatments, so patients are not actively receiving care from a physician or site who could present the option.
- Patients are widely geographically dispersed and may be treated by community physicians unaware of clinical trial options.
- Many rare disease patients do not have a formal diagnosis for their cluster of symptoms.
Consequently, many patients and caregivers take matters into their own hands, turning to online resources, such as trial finders or advocacy groups, to search for relevant trials. Some trial finders offer pre-screening materials, where patients can put their disease specifics, stage of the disease, medications, age, location, and more to match with a trial.
Once matched, patients are provided with the information needed to reach out to a site to discuss enrollment, empowering patients and their caregivers to make informed decisions about their health. We see this increasingly happening within pediatric conditions or conditions like neurodegenerative disease, where caregivers are responsible for a large majority of healthcare decision-making.
Addressing the Burden on Clinical Trial Sites
One of the major challenges of this shift is the additional burden placed on rare disease clinical trial sites. When patients initiate contact, the risk of them failing to meet the eligibility criteria is likely – as advanced as online pre-screeners are, they often only select the most pertinent eligibility criteria – which can lead to wasted resources and time. This situation can be exacerbated when patients travel long distances only to be screened out immediately at the site, causing frustration and elevated stress for both patients and sites.
Enhancing the Recruitment Process and Patient Experience
To mitigate these challenges, it’s important to explore solutions that can streamline the patient recruitment process and improve site efficiency. Here are several key strategies to utilize:
- Develop Dedicated Study Websites
One effective approach is the implementation of dedicated pre-screening websites. These centralized platforms can filter patient inquiries based on eligibility criteria before they contact trial sites, reducing the workload for sites and improving the efficiency of the recruitment process. It is imperative, however, given the points mentioned above, that these pre-screeners are compiled thoughtfully and in a way that will screen out the likely reasons for eligibility while containing those items that the patient/caregiver will know themselves or have readily available to answer. Additionally, if these websites are created by the sponsor or CRO, partnering with advocacy to have them promote the study website further gains interest and increases the awareness and available pool of patients. - Educate and Partner with Sites
To address the reluctance of some trial sites to adopt new technologies, it’s crucial to demonstrate the benefits of pre-screening platforms through site education and case studies showing the advantages of using such a recruitment tactic. Fostering relationships with sites can additionally help cultivate a mutually beneficial partnership. Once site relationships are established, it can significantly increase communication between sponsor, CRO, and site, enabling greater flexibility from the sites in case obstacles arise during drug development. - Leverage Patient Advocacy Organizations
One critical way to reduce site burden is to partner with patient advocacy organizations (PAOs). These groups have a wealth of information on their rare disease and offer a way for patients to get information in a palatable manner, giving them a sense that they’re not alone in their condition. In addition, PAOs often go above and beyond to raise awareness of clinical trials, including family conferences, externally-led patient-focused drug development (PFDD) events, disease awareness days, and events. Partnership with these advocacy groups to engage and provide tailored information to patients at these events will increase knowledge and awareness of your trial and can increase interest in participation.
As previously mentioned, some advocacy groups (i.e., Michael J Fox) have very advanced clinical trial finders with pre-screening already built in. Others simply provide a list of all available trials with some pre-defined filters built in. While both are a wealth of information for patients and their caregivers, it’s necessary to review the publicly available options to make sure that it is a fit-for-purpose solution for your trial. - Address Cross-Border Enrollment Challenges
One factor that may negatively impact patient recruitment is cross-border enrollment challenges. Since few clinical trials are available for certain rare disease indications, the closest trial may be in another country, which can be especially common in Europe. It’s essential to implement systems that can manage cross-border data transfers and accommodate patients from different countries, ensuring that eligible patients are not excluded based on their location and instead are offered the option to participate in their closest country if they choose.
Get Rare Disease Expertise from Worldwide Clinical Trials
The shift towards patient-driven recruitment in rare disease clinical trials represents a significant change in how we approach trial participation. At Worldwide, our team of rare disease experts is dedicated to embracing innovative technology platforms, supporting more than 115 advocacy groups, and optimizing site processes. Together, we can better meet the needs of patients and caregivers while enhancing the efficiency of rare disease clinical trials. Learn more about our extensive rare disease services today.
