Leveraging Patient and Public Involvement to Enhance Real-World Study Design

Real-world evidence (RWE) plays an increasingly significant role in healthcare research, providing insights into the effectiveness and safety of interventions in routine clinical practice. To optimise the design of real-world prospective observational studies, collaboration with patients is essential.

KKI has joined forced with the CL Foundation, under the leadership of CEO Susan Thornton, to advance research and improve patient care in cutaneous T-cell lymphoma (CTCL). Last month, Susan and I attended the Patients as Partners Europe Conference in London to highlight the collaborative efforts between our organisations and showcase the invaluable role of patient and public involvement in informing the design of real-world observational studies.

Leveraging patient and public involvement to enhance study design

During the conference, I presented a case study on the role that patient insights played in the design of a real-world prospective observational study, which aimed to capture patient experiences and symptoms during treatment. During the study design phase, patient interviews were conducted with five patients and three of their spouses, and key concepts were identified that were most important to the patient, such as disease impact on symptoms and health-related quality of life. The study design was further refined through consultation with the Lymphoma Coalition Community Advisory Board, enhancing the study's feasibility and appropriateness from the patient's perspective.

As well as playing a vital role contributing to study design and analysis, the CL Foundation’s collaboration with KKI includes sharing study results with CTCL communities and patient advocacy group (PAG) networks, ensuring that valuable insights reach those who need them most.

Continued collaboration to improve patient outcomes

During our presentation, Susan Thornton emphasised the importance of ongoing partnership with patients throughout the life cycle of clinical studies. This includes engaging patients in sharing study outcomes with the community, understanding the impact of studies on clinical practice, involving patient advocacy groups at every stage of the study process, and maintaining a continuous loop of information exchange. Such collaboration not only educates the patient community about the significance of their participation but also highlights how their involvement drives improvements in clinical practice and outcomes.

The collaboration between the CL Foundation and KKI serves as a powerful example of utilising patient and public involvement to inform the design of real-world observational studies. By incorporating patient insights, these studies become more patient-centered, relevant, and impactful. Patients and advocacy groups play a vital role in shaping research agendas, generating meaningful data, and driving positive change within the healthcare landscape.

Continued collaboration and engagement between patients, patient organisations, healthcare professionals, and pharmaceutical companies hold the potential to revolutionise the way real-world evidence is harnessed and translated into improved patient outcomes.

KKI/INT/KKI/0943 | June 2024