Clinical Research: Phase 1 - Phase 4

Realizing the Full Value of Patient Centricity in Rare Disease: 5 Ways to Maximize Your Engagement Efforts

As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders, sponsors need to be proactive in demonstrating their commitment to addressing unmet therapeutic needs.   

Beyond the buzzword: Patient centricity rarely utilizied to its potential 

Patient engagement is a key component to successful clinical trial execution in every indication, but especially in rare diseases. Utilizing the patient voice to drive the study should be incorporated throughout the entire drug development process; otherwise, you risk losing the full value of patient centricity. Gathering input from patients and family members on the specifics of the disease, standard of care, the type and frequency of assessments, visit schedules, support needs, and even recruitment or retention strategies is essential for ensuring that the voice of the patient is incorporated into every aspect of clinical development. Engaging patients and seeking their feedback on a clinical trial enables sponsors to develop the study in a patient-centric manner and design protocol parameters that lead to successful study enrollment and patient retention.

Why it’s never too late to engage patients

Ideally, patient engagement efforts should begin before the protocol is finalized so any feedback can be incorporated into the study design. However, as long as these efforts are underway before study start-up, input can still be implemented. In actuality, it is never too late to seek patient input, as insights gained can be shared or incorporated into future studies.

5 ways to keep the patient at the center of your development plans

Several approaches can be leveraged to increase patient engagement in rare disease studies:

  1. Involve patient advocacy groups and patient communities. Partnering with local, regional, and national patient advocacy groups or patient communities will help sponsors identify potential participants and increase awareness about the trial. These groups are a valuable resource for unpublished information about the natural history of the disease and unbiased insight into gaps in care and may even be willing to circulate study-related surveys to their members. Working with established, reputable groups also helps build trust among patients who may be unfamiliar with or wary of clinical research.

  2. Use social media and other digital platforms. Utilizing social media and other online platforms extends reach to a larger number of potential participants and provides a forum for discussion and support. Listening to and participating in these digital conversations allows sponsors to better understand the patient journey and the real-world impact of the disease on patients and caregivers. Sponsors should be aware that the policies and regulations for promoting clinical trials online vary by platform and country. It is also important to keep in mind that social media may not always be representative of the general population of patients with a particular rare disease, so digital engagement strategies should only be used to supplement, not replace, other more traditional methods for reaching potential participants.

  3. Make study participation as convenient as possible. While patients with rare diseases may be more likely to adhere to clinical trial protocols due to lack of alternative treatment options, willingness to participate in a study may still be affected by its impact on their daily lives. Given that many rare diseases are pediatric in onset, the trial logistics often involve parents and caregivers who must balance study participation with other personal or professional responsibilities. Ensuring that participation is not just feasible, but also positive helps ensure that patients and families will participate in the study for its duration.

    Integrate offers of transportation or other logistical support to make it easier for potential participants to participate in the trial. Decentralized trials afford another opportunity to expand access, enhancing diversity and equity. Incorporating telehealth visits, eConsent, ePRO platforms, and in-home nursing visits into the study design may make trial participation more attractive, especially if patients are ill or have limitations in mobility.

  4. Provide education and support. It is helpful to provide education to potential participants to help them understand the study process and potential risks and benefits. Creating educational materials that are easy to understand is essential for establishing clear communication and overcoming potential barriers to participation, such as misconceptions about clinical research. Being transparent about the goals of the study and the data that will be collected inspires trust. Enrolling in a clinical trial is a big decision—particularly if enrollment makes the patient ineligible to participate in other studies—so it may be useful to provide a dedicated patient navigator whose primary responsibility is to support patients and caregivers.

  5. Offer patients access to their data. In recent years, there has been increased interest in giving patients access to their own study data. When legally, ethically, and technically feasible, allowing study participants to view and download certain data and share them with others can be a powerful way to keep them engaged in rare disease trials. This data access creates a sense of ownership and reminds participants of the contribution they are making to the advancement of knowledge, not just for themselves but for others. If providing data access is not possible, sending periodic updates on study progress or relevant, new scientific discoveries is another way for sponsors to demonstrate their ongoing appreciation for the patient’s commitment to the clinical trial.

While the average dropout rate in clinical trials across all indications is 19.1 percent, the dropout rate in rare diseases studies is only 6.5 percent, demonstrating the willingness of these patients to remain involved in research once they have enrolled.1 For sponsors, the challenge is to engage the hearts and minds of potential participants, turning them into study ambassadors who can help spread the word.

[1] CenterWatch. Recruitment Rates Rising, but Retention Rates Fall, According to New Study, February 2, 2020. Available at