Engaging Patient Perspectives Earlier in Clinical Trials for Better Outcomes

Authored by: Nicky Barclay-Prout

For many pharmaceutical companies, patient perspectives are traditionally considered during or after clinical trials. But there is growing recognition that the patient voice is just as valuable, if not more so, in the earliest stages of development. Patient input at this point can help ensure that clinical trials are designed to reflect what matters most to those living with the condition. Conducting pre-clinical patient voice research supports the development of patient-centric products and executing feasible clinical trials for better outcomes.

This shift has been reinforced by regulatory momentum; in the US, the FDA’s Patient-Focused Drug Development (PFDD) Guidance Series, first introduced in 2018¹, provides a framework for integrating patient perspectives throughout medical product development. In the UK, the MHRA has committed to embedding patient involvement at every stage of the regulatory lifecycle², while in the EU both the European Medicines Agency³ and the new EU Health Technology Assessment (HTA) Regulation⁴ call for early and meaningful patient input into trial design and endpoint selection.

In this article, we explore how patient perspectives help shape better products and clinical trial endpoints, as well as how early feedback on trial design improves recruitment, retention, and overall experience – including some examples from a recent chronic disease study where patients informed a more meaningful and patient-centered trial design.

Understanding What Matters: Informing TPPs and Trial Endpoints

When patient insight is introduced in the earliest stages of development, it provides a powerful lens through which to understand real unmet needs. What does meaningful improvement look like to a patient? Which symptoms most disrupt daily life? Which outcomes would be worth the burden of long-term treatment?

These questions are not always answered in clinical literature, and yet they are vital to building an effective Target Product Profile (TPP) and selecting endpoints that resonate.

In our recent research with people living with a chronic disease, patients spoke about how fatigue, dietary limitations, and emotional burnout shaped their experience more than the biometrics often used to measure disease status. Many were unaware of their precise disease staging but were attuned to fluctuations in energy levels and day-to-day functioning. This kind of insight helps ensure trial endpoints are meaningful, relevant, and aligned with what patients value most.

Designing Trials that Patients Will Join and Stay In

Equally important is understanding what it feels like to participate in a clinical trial.

Recruitment and retention challenges often arise not from unwillingness but from unclear expectations, logistical barriers, and emotional concerns.

In the same chronic disease study, participants initially felt overwhelmed by the trial schedule, but when the timeline was broken down into phases and explained in context, many became more open. Some expressed anxiety about joining a placebo arm or taking an investigational treatment that might destabilize their current care. Others flagged practical concerns, such as travel time and fasting requirements paired with long site visits. Morning appointments and on-site meals were among the simple changes that made participation feel more feasible.

These are the details that can be easily overlooked in protocol development, yet they make or break the trial experience.

Broadening the Lens: Who We Listen to, and Why 

To inform both clinical trial endpoints and participation strategies, it is essential to go beyond the “research-engaged” patient population. Listening to those who are less engaged often reveals different barriers, such as mistrust, competing priorities, financial strain, or care responsibilities, all of which can influence their perception of what outcomes matter and whether they are willing or able to join a trial.

In conditions where stigma, misinformation, or social isolation are common, input from caregivers, Patient Advocacy Groups, and community influencers can also add critical context. These perspectives help shape trials that are not only clinically relevant but also practically and emotionally accessible to a broader, more diverse population.

A Flexible, Human-Centered Research Approach 

Delivering this kind of insight requires a flexible research toolkit, tailored not only to the therapy area but to the population, culture, and local context. In-depth interviews, mobile and in-person ethnography, and enhanced social media insights all play a role in building a clear and nuanced picture.

In the chronic disease study, for instance, patients described the term “lifestyle counselling” as patronizing. Through qualitative exploration, we found that reframing the offer as “personalized wellness support” helped remove feelings of blame and made participants more open to behavior-change guidance. This insight helped inform how trial communications and support services could be better received and more widely adopted.

These kinds of details are only surfaced through human-centered research. They help avoid unintentional missteps and ensure that both trial outcomes and trial experiences are aligned with what patients actually value and need.

The Impact of Early Engagement

 When patient insight is gathered early, it has the power to shape the entire development pathway. It enables teams to define endpoints that reflect meaningful outcomes and to design trials that feel feasible, supportive, and worth committing to.

Ultimately, early engagement drives better science, stronger strategy, and more inclusive clinical research. It helps sponsors avoid barriers before they arise and ensures the patient voice is embedded not only in what we measure, but in how we deliver. It is not just the right thing to do; it is the smart thing to do. Contact us

References:

1. U.S. Food and Drug Administration. (2018). Patient-focused drug development: Collecting comprehensive and representative input. Guidance for industry. https://www.fda.gov/media/113653/download
2. Medicines and Healthcare products Regulatory Agency. (2021). Patient involvement strategy 2021–2025. https://www.gov.uk/government/publications/mhra-patient-involvement-strategy-2021-to-2025
3. European Medicines Agency. (2020). Regulatory science strategy to 2025. https://www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-2025
4. European Commission. (2022). Health technology assessment (HTA) regulation: Entered into force in January 2022, applicable from January 2025. https://health.ec.europa.eu/medicinal-products/health-technology-assessment_en