Putting patients first in clinical trials

Clinical trials offer clinicians a unique opportunity to lead advances in medicine, granting access to cutting-edge therapies that address unmet patient needs. They are a cornerstone of clinical practice that enhance patient outcomes, particularly for those with complex or refractory cases who have exhausted standard treatment options. Clinicians understandably prioritize the well-being and interests of their patients, so they must be confident in a trial’s safety and efficacy. A well thought-out clinical trial protocol should balance scientific rigor with participant practicality, as overly burdensome protocols risk poor recruitment and retention, and may impact negatively on patient outcomes in the long term. 

The clinical trial landscape is changing rapidly in response to the industry-wide desire to enhance patient participation and experience. Trial designs in particular are becoming increasingly patient oriented, and regulatory bodies are already drafting guidance emphasizing the importance of matters such as patient involvement, diversity and inclusion, and accessible reporting. This article provides insights into the growing importance of patient centricity and the resultant ongoing and potential shifts in clinical trial protocol design. 

The Growing Role of Patient Associations

Organizations like patient associations and community networks, including those formed via social media, are increasingly being used to ensure trial design is centered around patients and their caregivers. Advocacy groups play vital roles in reviewing protocols and participant-facing materials, and are key contributors to making trials more accessible and relevant by providing valuable insights into patient journeys. They also help to ensure that protocols are practical, less burdensome to participants and comprehensible, and that patients’ needs are considered. Their input also helps to improve compliance to protocol requirements. As well as these functions, patient associations serve as critical bridges between patients and the clinical research industry, educating participants about the value of trials and addressing common misconceptions, with the aim of encouraging involvement and fostering trust between the public and medical professionals. Additionally, patient groups frequently provide crucial ethical input on sensitive issues such as pediatric trials, parental compensation and the inclusion of underserved populations, helping to shape ethical as well as practical approaches to trial design and execution.

Enhancing Transparency in Scientific Communications 

Stepping up patient involvement through associations is not the only way in which clinical trial communities are fostering patient-centered trials. There is also a push to ensure that participant-facing documents, including informed consent forms, protocol summaries, results and reports, are published and presented in a lay-friendly manner, in order to make them more comprehensive to a wider range of non-specialists. In fact, guidelines indicate that communications intended for trial participants should be presented in plain language that is understandable to a layperson, and should be available in a variety of formats for greater accessibility.^1-4 Clear content information presented in a user-friendly way, along with timely public access to trial data and results, all contribute to demystifying the trial process, and build trust and engagement within participant cohorts. 

Expanding Diversity for Greater Participant Inclusion

Clinical trials can also become more patient-centered by including more diverse demographics and by addressing long-standing challenges in recruiting underrepresented populations. There is a growing recognition that diverse participation is essential for generating equitable and broadly applicable research outcomes. However, socioeconomic barriers, mistrust of pharmaceutical companies and limited access to technology have historically hindered efforts to achieve representative study cohorts. Nevertheless, regulatory agencies including the FDA^5-7 and MHRA⁸ are now championing the inclusion of diverse demographics in clinical trials, and are publishing recommendations on enhancing diversity in clinical trials and including representative populations.

Overcoming these obstacles requires a multi-faceted approach. For example, collaborating with clinical trial sites to understand local communities and tailoring outreach strategies – such as engaging with community centers and using targeted advertisements – can significantly improve recruitment outcomes. Taking a holistic view of the patient journey, including the recognition of barriers to diagnosis and treatment accessibility, ensures that study protocols reflect the realities of diverse patient experiences. Decentralized trials – featuring remote monitoring and digital tools to capture easily quantifiable data – are also emerging as potential solutions to increase diversity and reduce barriers to participation. This approach may face limitations in fields such as dermatology and rheumatology, where, depending on the endpoints, in-person assessments remain essential to generate high quality data. Study designs should also incorporate flexible approaches by allowing both in-person and remote visits to reduce the burden on patients and sites.

Navigating Regulatory Shifts 

Other regulatory input includes programs advocating sponsors to incorporate patient feedback and assessments of participant burden into protocol development.9-12 This can be seen in recent draft guidance and initiatives, such as a recommendation for lupus research in minority groups, which was highlighted by the American College of Rheumatology.^13,14 Including these measures in official guidance aims to make studies both scientifically rigorous and accessible to a wider range of participants. Novel treatment options also mean that trial designs must continually adapt to increasingly complex requirements. Biologics, for example, introduce challenges related to batch quality variability, needing stringent global quality control measures to ensure compliance. Sponsors are likewise embracing real-time data access and interim analyses, which demand robust statistical support and innovative trial management strategies to ensure alignment with rapidly evolving regulations governing secure data acquisition and usage. 

Summary and Future Directions  

It’s clear that the growing emphasis on patient centricity is transforming clinical trial design and execution, marking a shift from viewing collaboration and commitment from patients as optional, to recognizing it as essential. Innovative trial designs, including adaptive and master protocols, are becoming more prevalent, enabling sponsors to address multiple research questions simultaneously.. The importance of patient feedback  is even more important to ensure these innovative trial designs remain accessible and inclusive, resulting in impactful research aligned with patients’ needs.

Additionally, increased investment in public and patient education promises to improve recruitment and retention, and elevate the overall quality of research outcomes. These public awareness campaigns and the adoption of patient-centric approaches may increase costs initially in some instances. However, the financial burden is far outweighed by the long-term benefits to the medical field, including streamlined participant recruitment, improved regulatory compliance and faster timelines to drug approvals. Together, these ongoing changes in the clinical trial sphere reflect the need to balance regulatory compliance, scientific progress, and patient-centered protocols. As clinical research continues to evolve into the future, the medical industry’s commitment to placing patients at the core of trial design and execution will pave the way for more inclusive, effective, and equitable healthcare solutions.

References

1. International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. GUIDELINE FOR GOOD CLINICAL PRACTICE EG(R3) . https://database.ich.org/sites/default/files/ICH_E6%28R3%29_Step4_FinalGuideline_2025_0106.pdf. Published online January 6, 2025.
2. Council of the European Union. REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL  of 16 April 2014  on Clinical Trials on Medicinal Products for Human Use, and Repealing Directive 2001/20/EC. https://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:32014R0536&from=EN#tit_1; 2014.
3. US FDA. Informed Consent Guidance for IRBs, Clinical Investigators, and Sponsors . https://www.fda.gov/media/88915/download. Published online August 2023.
4. US FDA. Key Information and Facilitating Understanding in Informed Consent. https://www.fda.gov/media/176663/download. Published online March 2024.
5. Fienkeng M. Overview of the Public Workshop to Enhance Clinical Study Diversity (November 2023). https://cersi.umd.edu/sites/cersi.umd.edu/files/0-1_MathildaFienkeng_v10_final.pdf. Published online September 6, 2024.
6. Mehta GU, Pazdur R, Bumpus NN, Califf RM. When Diversity Goals Meet Multiregional Trials. New England Journal of Medicine. 2025;392(1):4-7. doi:10.1056/NEJMp2409392
7. US FDA. Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies, Draft Guidance for Industry. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/diversity-action-plans-improve-enrollment-participants-underrepresented-populations-clinical-studies. Published online June 26, 2024.
8. NHS Health Research Authority. HRA and MHRA draft inclusion and diversity guidance. https://www.hra.nhs.uk/about-us/news-updates/hra-mhra-guidance-developing-and-submitting-inclusion-and-diversity-plan/. Published online September 30, 2024.
9. TransCelerate Biopharma inc. Patient Experience Initiative . https://www.transceleratebiopharmainc.com/initiatives/patient-experience/.
10. Medicine and Healthcare products Regulatory Agency. Patient Involvement Strategy: An Assessment of Progress.; 2025.
11. US FDA. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making. https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical. Published online February 14, 2024.
12. 

    Julie Massicotte

    US FDA. Patient Engagement in the Design and Conduct of Medical Device Clinical Studies. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-engagement-design-and-conduct-medical-device-clinical-studies. Published online January 22, 2022.

13. American College of Rheumatology. New American College of Rheumatology Initiatives Aim to Close the Gap on Racial Disparities in Lupus Clinical Trials. https://rheumatology.org/press-releases/new-american-college-of-rheumatology-initiatives-aim-to-close-the-gap-on-racial-disparities-in-lupus-clinical-trials. February 23, 2022.
14. Sheikh SZ, Englund T, Simkus A, et al. Training to Increase Minority Enrollment in Lupus Clinical Trials With Community Engagement: Enhancing Lupus Clinical Trial Recruitment Through Provider and Community Health Worker Engagement. Arthritis Care Res (Hoboken). 2025;77(2):201-208. doi:10.1002/acr.25419

Julie Massicotte is Senior Director of Regulatory Affairs, Indero.