ecruitment and retention in clinical trials pose significant challenges, often due to the burdensome nature of participation. Despite widespread recognition that patient input can alleviate these burdens and improve recruitment and retention rates, the industry has been slow to standardize this approach. This article outlines the transformative journey of a leading global pharmaceutical company towards embedding systematic patient engagement in clinical research.

Within the last three years, the company has revolutionized its approach to clinical trials to significantly enhance the patient experience. During this time, the percentage of our trial designs addressing patient needs increased from less than 1% before 2021 to 88% in 2024.

For example: In one early trial in a new indication for the organization, patient feedback led to changes in the endpoint strategy, randomization ratio, and co-medications, and to the co-creation (with patients) of patient newsletters and visit visuals. This trial completed recruitment in half the allocated time. In another trial, a trial simulation was conducted to understand patient and caregiver needs. Home assessments, caregiver reimbursement, and concierge services were implemented in the trial based on these insights. This trial recruited four months faster than planned and maintained a very high retention rate and high levels of patient satisfaction with their trial experience.

In addition, 95% of trials implemented specific plans for patient-centric trial conduct. These efforts have led to an average overall trial participant experience of 3.8 on a scale of 1 to 5. (Because these surveys were implemented at the end of 2023, prior year scores are not available for comparison.)

8 key components that were the foundation for this success:

1. Clear Ambition

Fundamental to success was to have a clear ambition so that all internal and external partners understand where we want to go. In our example, the ambition is simple and concise: to become the preferred trial sponsor.

2. Strong Senior Management Support

A key factor for moving the needle from sporadic to systematic patient engagement was strong support from senior management, who made it clear that they take patient engagement in clinical research seriously by talking about the value of patient engagement for clinical trials, emphasizing its high priority, and setting up dedicated resources and budget. Return on investment was reviewed with senior management using the metrics described in section 8 below.

3. Guided by Patient Needs

The journey’s foundational principle was to be guided by patient needs and to not implement solutions based on assumptions. Because the trial experience for patients and for trial site staff are closely connected, we implemented a holistic approach that focused on reducing the burden on both the patient and trial site staff. For example: In a previous trial, the clinical team hoped to reduce the patient burden by offering home nursing. They spent substantial resources on implementing home nursing into the trial, but none of the trial participants used this service. The lesson from this example is clear: Always start with understanding and documenting patient needs on the basis of empirical evidence. This allows the clinical trial team to strategically focus resources on solutions that have the highest impact on creating a good trial experience for patients and avoids unnecessary burdens on trial sites, such as providing caregiver reimbursement to compensate caregivers for loss of income when patients need caregivers to accompany them to site visits.

In 2021, the organization established a standing patient and site advisory panel, consisting of external patient advocates and trial site staff from different countries and therapy areas, for focused collaboration. This panel prioritized three things: Implement trial experience surveys in all trials, optimize informed consent in all trials, and address patient needs in all trial designs (for example, by adapting the complexity, frequency, and duration of trial visits to each patient’s schedule and needs). The global informed consent template was optimized by shortening it and incorporating graphics and lay language in the main text body; this template is now implemented in all new trials. Since 2021, the work of this panel has contributed to clinical trial simulations to identify the challenges facing patient recruitment and retention when conducting clinical trials in patients with pulmonary fibrosis published by the European Respiratory Society, and to improve trial design and outcomes to overcome participation barriers for patients with palmoplantar pustulosis.

4. Co-Creation with Patients

The organization co-created clinical trial experience surveys with this patient and site advisory panel. These surveys have been implemented in more than 80% of its trials and have received more than 3000 responses (and counting). These surveys include questions about ease of technology use, quality of information, and satisfaction with travel and other logistic support. The responses allow the clinical trial staff to identify concrete actions that further improve the participant’s trial experience.

Patient needs are addressed systematically and very early in the trial design stage, based on past experience: In 2019, the company convened a group of patients to discuss a clinical trial endpoint and learned that this endpoint was not measuring the patients’ pain in a way that was meaningful for them. Unfortunately, this was discovered too close to the start of the trial to make changes to the endpoint without delaying the start. This example taught the indispensable value as well as the practical feasibility of understanding and addressing patient needs well before trial designs are “locked”, which is often 8 to 12 months before trial start. This early engagement of patients ensures that the incorporation of patient input is not delaying the trial start.

5. Making it Everyone’s Business

An African proverb says: “If you want to go fast, go alone. If you want to go far, go together.” The organization made patient engagement in clinical research everyone’s business by assigning patient engagement responsibilities across more than 700 company staff involved in clinical trial design and conduct.

6. Creating Awareness and Ownership

One key to driving change across a large organization is to create awareness, which the organization accomplished by leveraging internal meetings and their internal social media network, and by creating a group of passionate ambassadors who were responsible for creating awareness in their organizational units. Including patient engagement in clinical research as one of four themes of a department meeting attended by more than 1,000 staff made the biggest impact, based on subjective observations instead of a quantitative assessment.

After establishing awareness of a need for change, it is important to create a sense of ownership for that change. Ownership was increased through promoting direct interactions with patient advocates and sharing patient testimonials with staff to illustrate the value of their work and recognizing company staff members with annual awards selected by the standing patient and site advisory panel.

7. Building Capabilities

The organization also documented and established the processes, tools, and knowledge required to implement patient engagement in clinical trials into its everyday work: A playbook serves as central resource, patient engagement has been integrated into existing processes, patient engagement training keeps staff skills current, and additional processes and tools were established as needed. Patient engagement capabilities were organized around recurring patient-centric “hot” topics: Feedback mechanisms (including patient surveys, interviews, and sharing trial status/results), decentralized trial planning, and recruitment and retention. To reinforce and make lessons learned more tangible, case studies and lessons learned are exchanged between company staff members in a dedicated quarterly presentation forum and in a monthly video series.

There is no single methodology for understanding patient need. The approach will vary depending on available time, budget, and relationships with pertinent patient advocacy groups. Different approaches included conducting example trial simulations or market research with patient groups, or scheduling patient advisory board meetings:

• In one early trial in a new indication for the organization, patient feedback led to changes in the endpoint strategy, randomization ratio, and co-medications. In addition, patient newsletters and visit visuals were co-created. This trial completed recruitment in half the allocated time.
• In another trial, a trial simulation was conducted to understand patient and caregiver needs. Based on these insights, home assessments, caregiver reimbursement, and concierge services were implemented in the trial. The trial recruited four months faster than planned and maintained a very high retention rate and high levels of patient satisfaction with their trial experience.

8. Measuring What Matters

Lastly, metrics were developed and leveraged to ensure that patient engagement remains one of the organization’s most visible, top priorities. Key metrics that were developed by a dedicated team and endorsed by senior management were percentage of trial designs addressing patient needs, percentage of trials implementing specific plans for patient-centric trial conduct, and overall trial experience rated by trial participants. These metrics were reviewed by senior management and shared with the organization twice per year with a comparison over time, between therapeutic areas, clinical trials, and countries.