ow do we move from “checking boxes” to truly meaningful patient involvement in clinical trials? From emerging global trends through redesigning the consent process to creative solutions to real-world barriers, meaningful collaboration across the ecosystem is key—and the reason why we’re only at the beginning of what’s possible. Several recent initiatives are aiming to bring these possibilities into practice in the EU.

The question on everyone’s mind: If patients aren’t part of the process, then who are we doing this for?

To attract patients to join clinical trials, researchers must ensure that these trials address the needs and concerns of patients; consequently, early involvement of patients in trial design is key to running successful trials and efficient drug development programs. Industry sponsors generally recognize the importance of engaging with patients and involve patient organizations while developing a product; however, as mentioned in the EMA patient experience reflection paper, guidance in this area can seem fractured or outdated. As a result of this confusion, such engagement is often not reflected in the trial documentation that sponsors submit for assessment to regulators and ethics committees.

It is also essential that patients fully understand the commitment they make when agreeing to join a trial. Improving the informed consent template by making it shorter and its language easier to understand, and then further refining the form to accommodate any specific information needs of (potential) participants (e.g., second languages), will also help encourage trial understanding and participation. (See below for explanation of the Clinical Trials Coordination Group (CTCG) informed consent initiative in The Netherlands.)

The debate is no longer if patient involvement matters but how to make it more meaningful.

Understanding the Landscape

The CTCG has initiated a project on patient involvement in clinical trials as part of the Heads of Medicines Agencies (HMA) Clinical Trial Coordination Group (CTCG) workplan for 2025 (CTCG Workplan) aimed at fostering patient involvement in clinical trial design. Current regulations (e.g., EU Clinical Trial Regulation and Declaration of Helsinki) encourage documenting patient involvement in trial protocols and emphasize the need for meaningful engagement throughout the research process.

A survey about initiatives and review of patient involvement in clinical trials was conducted by the CTCG and MedEthicsEU in 2024, and responses were received from national competent authorities (NCAs) of 20 member states and from Medical Research Ethics Committees (MRECs) of 18 member states in the EU/EAA. Its key takeaways include:

• Consultation Rates: 25% of NCAs and 75% of MRECs reported consulting patients during their assessments.
• Involvement Gaps: Despite consultation, patient involvement in the actual trial approval process remains limited.
• Reviewers do not systematically assess patient involvement unless it is explicitly described in the protocol.
• Benefits of patient involvement included better endpoint selection, improved informed consent, and more inclusive and acceptable (to patient) studies.
• Challenges included difficulty in identifying relevant patient groups, particularly for niche medical conditions, and language barriers that hinder effective communication and understanding.

To address these challenges, the CTCG organized several follow-up sessions on meaningful patient participation in which some stakeholders advocated for making patient involvement mandatory, while others suggested that it may not be feasible or meaningful for every trial. Other feedback identified the need for clearer guidance to prevent tokenism in clinical trials and ensure that engagement with patients adds real value to the research process, and does not become a “checkbox” exercise that merely increases the administrative burden.

Industry Perspectives

Key insights from a survey on patient involvement in clinical trial design conducted amongst industry members of the European Federation of Pharmaceutical Industries and Associations (EFPIA) at the end 2024 and evaluated in the beginning of 2025 include:

• Importance of Patient Involvement: Industry involves patients in clinical trial design, as their input is seen as valuable and essential to improve the trial setup, reduce the burden on patients, and increase patient enrollment and retention. However, companies do not always reflect patient involvement in individual clinical trial protocols. To more systematically ensure patient involvement, many companies have developed internal frameworks to define patient involvement in trial design.
• Engagement Timing: Patient engagement is most prevalent during phases 2 and 3 of clinical development but is deemed essential throughout the entire process.
• Limited Feedback from Authorities: Industry sponsors do not often receive feedback regarding patient involvement from national competent authorities and ethics committees during the assessment of clinical trial applications. Many patient engagement initiatives are defined and implemented at the research program level, which can create confusion when submitting an application for a specific protocol within that program.
• Guidance Needs: Industry stakeholders expressed a desire for clearer guidance on what to include in clinical trial applications relating to patient input and how this information impacts the regulatory assessment of those applications.

Redesigning Informed Consent

In The Netherlands, a project initiated by the Central Committee on Research Involving Human Subjects (CCMO) on patient involvement in clinical trials, as part of the CTCG Workplan, has worked to make informed consent forms (ICFs) more accessible and comprehensible to patients. The initiative grew out of feedback from clinicians, research professionals, potential participants, regulators, and investigators, indicating that traditional ICFs are often too lengthy and complex. Their proposed new ICF template features:

• Reduced Length: The revised ICF contains 55% fewer words.
• Simplified Language: Text is kept at a B1 language proficiency level, so it is easier for patients to understand.
• Highlighted Key Information: A short introductory page provides essential trial information, enhancing clarity for potential participants; a layered (step-by-step or section-by-section) reading approach gives patients the opportunity to mark sections and request more information about that topic.

Next steps for this project include digitalization, user testing, and a launch planned by the end of 2025.

Ways to Emphasize Ongoing Patient Engagement

Recent regulatory updates from other regions, such as the revision of ICH E6 (R3) and FDA guidelines on patient-focused drug development, emphasize the need for including diverse patient populations in clinical trials and the importance of continuous patient engagement beyond product approval. Key strategies from these documents designed to accomplish these goals include:

• Patient Engagement: Involving patients and caregivers from the beginning helps identify unmet needs and challenges with new and existing treatments and ensures that product development aligns with patient priorities.
• Regulatory Focus on Diversity: Recent FDA and EMA guidelines and initiatives emphasize the importance of diverse and representative study populations in clinical trials to improve generalizability of the trial results in terms of both safety and efficacy outcomes.
• Iterative Design and Prototyping: Utilizing human-centered design principles, including iterative prototyping and real-world feedback, is essential for creating intuitive and accessible biomedical products that meet patient needs.
• Real-World Evidence: Incorporating real-world evidence and digital health tools into the development process enhances decision-making and allows for continuous tracking of patient data during trials.
• Patient-Centric Product Development: Ensure a holistic approach that integrates patient feedback into every phase, from initial ideation through commercialization.
• Continuous Improvement and Surveillance: Post-market surveillance and ongoing patient feedback are crucial for refining products and ensuring patient satisfaction, which can lead to better health outcomes.
• Affordability and Accessibility: Strategies must address barriers to access and ensure that products are affordable and available to diverse patient populations, particularly marginalized and underserved groups.

Conclusion

Meaningful collaboration with patients, not just during recruitment but from trial design through implementation and beyond, can significantly enhance the relevance, inclusivity, and ultimate success of clinical research. Addressing barriers to participation and fostering genuine partnerships are not just best practices; they are ethical imperatives.

In a rapidly evolving healthcare landscape, embedding the patient’s voice at every stage of innovation is no longer optional—it is, as demonstrated by the wide range of contributors to the below Key Takeaways, a shared responsibility. It is essential to ensure that treatments are not only effective but also aligned with the real needs and lived experiences of those they are meant to help.