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Clinical trials should meet two major goals – (1) being scientifically sound and – (2) being relevant and accessible for people living with a disease. Involving people living with a disease or their representatives (patient experts) in clinical trial design and conduct supports adequate consideration of their needs and realities.
The role, extent, and topics for the involvement of patient experts can range from full co-creation and co-design of the trial or even setting research priorities with multiple patient experts to involving one patient expert in the review of patient facing materials. Moreover, the methods and level of structuredness can range from group discussions, advisory board participation, surveys or document reviews.
How can we make this involvement a valuable and effective activity for both – the patient expert and the pharmaceutical company?
Together with Dirk Rohde, a patient expert, cancer survivor and founder of a regional patient group in head and neck cancer (Kopf-Hals-Mund e.V., Köln), who recently was awarded the Order of Merit of the Federal Republic of Germany for his longstanding and valuable engagement for cancer patients, we present the 10 most important considerations.
Organizational aspects
1. Find the right patient expert: Identify and connect with a patient expert who is recognized within the patient community as knowledgeable about the specific disease and in the field of medical research and clinical trial design. If there is no patient expert with this background, offering trainings on research basics might be an option to support the patient expert in their role.
2. Safeguard patient privacy and ethical integrity: Ensure the implementation and adherence to the pharmaceutical company’s guidance and local countries’ legislation on collaborating with patients and patient experts. Handle any personal patient expert data with confidentiality. Check for any conflicts of interest and inform the patient expert on potential conflicts of interest that may arise in future engagements (e.g. patient expert contribution to HTA process).
3. Define clear roles and expectations: Outline what the patient expert’s role will be and clarify how their insights will be integrated into the study design. At the same time, establish transparent decision-making boundaries and define whether the patient expert has an advisory, consultative, or decision-making role.
4. Contract & compensate fairly: Patient experts should be compensated for their time and expertise, to reflect the value of their contributions. Use fair market value guidance for determining appropriate compensations. The tasks and compensations should be agreed in a mutually agreed contract set up at the start of the collaboration.
Practical and collaboration related aspects
5. Set feasible deadlines and ensure accessible communication: Avoid overwhelming and unnecessary medical jargon and use clear, accessible language to ensure the patient expert can fully engage with complex medical and scientific information. Decide on a dedicated contact person, who can support the patient expert in case of questions. Plan regular check-ins to ensure the patient expert feels informed, valued, and able to contribute meaningfully throughout the collaboration. Understand that many patient experts aren’t working as full-time paid employees for a patient organization and might need more flexible schedules.
6. Ensure review cycles: After receiving initial feedback from the patient expert, revised documents or decisions should be reported back especially in case the patient expert's suggestions were dismissed. This will contribute to mutual understanding and acceptance of the final decisions. The patient expert should receive access to updated versions and be able to provide additional feedback.
Content related aspects
7. Understand and respect the patient perspective: patient experts can provide invaluable insight based on their lived experiences, which can help identify potential challenges, such as unrealistic treatment schedules or burdensome data collection. Patient experts should be consulted on the inclusion of patient-relevant outcomes (e.g., quality of life, important symptoms) in addition to traditional clinical endpoints. This ensures that trials are designed with outcomes that are relevant for patients and for regulatory and HTA authorities.
8. Check trial feasibility & understandability of patient facing materials: Patient experts can provide valuable reviews and feedback on potential barriers for participants, such as understandability of trial information and informed consent forms, travel requirements, explanations of complex procedures, the need for co-medication or the burden of time commitments.
9. Involve patient expert in trial recruitment: Patient experts usually have detailed insights into patients’ preferred modes of gaining and sharing information and can therefore contribute to the informational strategy related to the clinical trial. Patient experts might also be contacted in case trial participants share positive and negative experiences regarding their participation. This information can help to improve patient-centeredness or current and future trials.
Evaluating the collaboration
10. Seek constructive criticism and adapt the collaboration: Asking regularly for feedback from the patient expert regarding the collaboration and improving the collaboration based on this feedback helps to maintain a strong and trustful relationship. Patient experts are extremely important partners for making a clinical trial a success for the patient community and the pharmaceutical manufacturer.
Key Takeaway:
The inclusion of a patient experts in clinical trial design is a dynamic, collaborative process that can help produce a more patient-centered, practical, and ethical clinical trial. By respecting and incorporating their insights, you can develop a trial that not only meets regulatory and scientific requirements but also resonates with the lived realities of potential participants.
