The importance of involving patients throughout the trial process and ensuring their needs are at the forefront of drug development and clinical trial research cannot be stressed enough. In this chapter, we will explore the main challenges associated with patient engagement and provide strategies and best practices to actively engage and empower patients in your drug development. The goal is to help you enroll trials faster and promote a patient-centered approach that enhances the quality and success of your clinical trials.
Why engage patients
Rare disease patient communities are often great forces for progress. Informal groups may exist on social media platforms to share information. When patients and caregivers formally organize as nonprofits, these patient advocacy groups can become incredibly important and influential partners for gene therapy clinical trial developers. Depending on the size, mission, and scientific sophistication of the organization, patient advocacy programs can include websites, social media presence, webinars, support groups, patient educational conferences, medical and scientific conferences, patient registries, grantmaking, or even independent gene therapy program development.
Through these functions, patient advocacy groups or motivated individual advocates can contribute to the quality and success of your clinical trials in myriad ways. It can be valuable to engage members of the patient community to get feedback on enrollment materials prior to finalization. Patient advocacy groups can be a crucial partner in trial recruitment, due to their trusted position in the patient community and established communication channels. Engage patient leaders early to learn about how they partner with researchers. For example, do they disseminate information about upcoming, newly open or ongoing clinical trials?
Many patient advocacy groups maintain their own patient registries. These registries can be extremely useful in finding eligible participants for trials. IRB-approved registries can also contain valuable data about the disease state.
Patients are the undisputed authority on the outcomes that matter to patients. At all stages in the research process, researchers should make efforts to ensure that their efforts are aligned with the needs of the patient community. Patient priorities should be major factors in the clinical outcomes that are tested and the design of potential interventions. Patients with extremely rare diseases and their caregivers often have a great deal of expertise on their disease condition, which is a precious source of disease information for diseases with limited published clinical research.
The patient community can also be an unexpectedly powerful ally in overcoming roadblocks to research. While this role should not be expected of patient communities, research teams that are candid with patient communities about the causes of delayed progress will sometimes find that the patient community can be a powerful partner in identifying and enabling solutions. Sharing information about research progress is not only often ethical, but it is also sometimes helpful to the research team.
First steps in patient engagement should ideally begin well in advance of trial recruitment. Research teams can begin with simple engagements like sharing copies of new papers with patient communities along with a plain language summary, scheduling an introduction with patient advocacy group leadership, or asking to attend a patient educational conference in order to learn more about patient needs and priorities.
Patient communities are the single most affected stakeholder for research on their disease. Patient engagement empowers patients to partner, enables efficient recruitment, and yields better science. Every research team needs a patient engagement plan for ethical, logistical and scientific reasons.