he Patient Engagement Council for Parkinson’s Research (PECPR) serves as a powerful example of how integrating patient voices from the outset can help a pharmaceutical company create a more inclusive, effective, patient-focused drug development process. Created in collaboration with The Parkinson’s Foundation and Parkinson’s UK, the PECPR is a strategic steering group that integrates patient perspectives throughout the company’s Parkinson’s research pipeline. This council brings patients and caregivers into the core of decision-making, supporting the argument that patients are essential crew members in navigating the complex journey of patient-focused medicines development.

Key Initiatives: Turning Patient Engagement into Action

Building a truly patient-focused approach requires more than good intentions—it demands concrete actions that embed patient perspectives at every stage of research and development. One multinational pharmaceutical company, through the Patient Engagement Council for Parkinson’s Research (PECPR), has implemented a series of structured initiatives designed to ensure that patient voices directly shape decision-making.

The following initiatives highlight how the PECPR is actively driving change—from establishing a strong governance model to fostering co-creation, addressing barriers to clinical trial participation, enhancing communication on treatments, and measuring impact. These efforts demonstrate how a strategic, systematic approach to patient engagement can lead to more inclusive and effective therapeutic innovations.

1. Setting the Course: Embedding Patient Engagement in the Core

Much like a well-built ship, successful patient engagement begins with a strong foundation. The PECPR was designed as a strategic steering group to ensure that the lived experiences of patients and caregivers influence decision-making directly. The company’s approach to patient-centricity is rooted in structure, governance, and transparency. By establishing a clear governance model, the company ensures that patient insights are not just gathered but are systematically integrated into research and development. The PECPR patient charter provided clarity on the council’s purpose and structure, roles and responsibilities, decision-making by consensus, communication and publications strategy, confidentiality and sharing of information, deliverables, how success will be measured, compliance, and guiding principles.

2. Harnessing the Tailwinds: Co-Creation for Greater Impact

A key element of success in patient-focused medicines development is the ability to capture and utilize external energy—the insights and expertise of the patient community. The PECPR’s work in co-developing a Target Product Profile (TPP) framework exemplifies this by actively integrating patient expertise into both process design and decision-making.

The initiative began with a kickoff and alignment meeting, bringing together the company’s Patient Engagement (PE) team, patient experts in clinical research, and patient organization (PO) partners to establish a shared vision and ways of working. This was followed by a series of interactive workshops: first, mapping and identifying gaps in the current TPP process and then defining the desired future state to shape a more patient-focused framework.

Beyond these group discussions, one-on-one calls with patients and PO partners were crucial in refining the process. These conversations helped define critical success factors, co-develop a preliminary TPP framework, and align on key impact metrics to measure the effectiveness of patient involvement. A dedicated playbook was created to document the co-creation process, providing guidance and encouraging broader participation. A guidance for the public is available on request.

The final readout meeting translated these efforts into actionable recommendations, embedding patient engagement into the operational framework. The TPP template itself was updated with an additional column to systematically track how patient insights informed development decisions. With this structure in place, the company has ensured that patient perspectives are not just considered but actively shape therapeutic innovation from the earliest stages. For example, the product development team is shifting away from the traditional approach of reaching out to Key Opinion Leaders (KOLs) to understand the patient/caregiver perspective, which often differs significantly from the patients’ views; the team is now exploring various alternative methodologies to gather these insights such as patient panels, one-on-one interviews, and literature searches.

3. Navigating the Waters: Addressing Barriers to Inclusion

Despite strong momentum, ensuring diverse representation in clinical research remains a challenge. To address some of these challenges, PECPR convened two dedicated patient councils, bringing together African-American, South Asian, and Hispanic individuals from the UK and US—communities that are often underrepresented in Parkinson’s disease research and care.

Held online in June 2023, these three-hour sessions gathered participants at various stages of their Parkinson’s journey, from those recently diagnosed to those who had been living with the disease for years. To deepen the discussion, each participant engaged with five members of their community beforehand, gathering additional insights on barriers and enablers to clinical trial participation across different ages, genders, and backgrounds.

Discussions explored personal experiences with diagnosis, support from healthcare providers and communities, and perceptions of clinical research. Participants provided direct feedback on the company’s trial design and shared recommendations on improving patient recruitment, retention, and engagement. A key focus was how study teams could better communicate with underserved communities, ensuring that information reaches those who need it most.

By fostering open dialogue and listening to lived experiences, PECPR identified critical gaps in research accessibility—such as a lack of community trust in research caused by insufficient engagement with these communities, a lack of knowledge in these communities about drug research and development, and a lack of training in community engagement and clinical research among local healthcare professionals—and refined its approach to building more inclusive, community-focused clinical trials in the future. These proposed refinements include developing lay audience information materials about the trial which patients can easily share with family and friends, and local site introduction or orientation meetings to offer the opportunity to answer questions from, and provide reassurance to, patients.

4. Mapping the Course: Enhancing Communication on Disease-Modifying Treatments

Clear and accessible communication is key to helping patients navigate complex treatment options. To improve how information about disease-modifying treatments (DMTs) is communicated, PECPR took a collaborative, multistep approach involving patient organizations, advisory boards, and direct community engagement.

The process began with in-depth insight gathering, including desk research on Parkinson’s disease and best practices from other therapy areas like multiple sclerosis and Alzheimer’s. These findings were discussed with a dedicated Patient Advisory Board, which operated in parallel with the PECPR and included patient organizations and individual patients, to shape an initial draft narrative on DMTs tailored for the patient community.

To ensure clarity and relevance, the narrative underwent multiple rounds of refinement. Message testing was conducted in the US, UK, Spain, and Germany to assess how well the information resonated across different languages and cultural contexts. A Strategic Patient Council—comprising six leading patient organizations—further adapted the messaging using a literacy model to ensure greater accessibility for a broad audience. In parallel, patient advocacy groups gathered feedback from their wider communities, which was incorporated in further refinements.

The final, patient-focused narrative was then disseminated through trusted patient organizations, with a focus on reaching underrepresented communities. By integrating patient insights at every stage, testing and refining with patient organizations, and using the Flesch-Kincaid Grade Level tool to evaluate its readability, PECPR ensured that information on DMTs was not only accurate but also meaningful and accessible to those who need it most.

5. Measuring Success: Tracking Patient-Focused Outcomes

Ensuring that patient engagement leads to real impact requires the right tools and a structured approach. The company, in collaboration with the Parkinson’s Foundation, Parkinson’s UK, and three patient experts, used the CTTI Prioritization Tool to guide decision-making. This tool helped each group assess potential topics for collaboration by weighing the required time and resources against the expected value and impact. Each stakeholder independently ranked topics, and when results were combined, those with the highest value, such as patient-informed endpoints and outcomes in the TPP framework, and patient contributions in the regulator and HTA processes—identified in the tool’s top-right quadrant—were prioritized. This approach ensured that efforts were focused where they could make the greatest difference.

In parallel, the company applied the PFMD Patient Engagement Quality Criteria to evaluate and refine its engagement efforts. These seven criteria—covering shared purpose, accessibility, representativeness, roles and responsibilities, capacity-building, transparency, and sustainability—served as a benchmark for PECPR’s activities. Continuous assessment against these standards reinforced high-quality patient engagement, ultimately earning the company the PFMD “Made with Patients” award in 2024 for its systematic and impactful approach.

By leveraging these tools, the company ensured that patient involvement was not just well-intentioned but strategically guided, measurable, and capable of driving meaningful change in therapeutic innovation. Patients’ perspectives are now systematically included from the start of the development of the Target Product Profiles, clinical study designs are more informed by patient input and preferences, and Informed Consent Forms have been adapted to make them more patient-friendly.

Steering into the Future: Sustaining Patient-Focused Transformation

Much like a well-navigated voyage, sustainable patient engagement requires continuous adaptation and commitment. UCB’s PECPR is now expanding its efforts to ensure that patient perspectives shape not only drug development but also policy decisions. For that, PECPR is raising awareness of how the patient community can meaningfully interact with regulatory and funding partners in patient-focused research initiatives, enhancing outreach to the broader research community, and identifying new areas for early-stage patient engagement. This commitment reflects the “North Star” approach of focusing on outcomes that truly benefit patients, aligning with the company’s mission to drive transformative impact within the Parkinson’s research community.