The value of patient-centric approaches has never been more evident in clinical trials. The FDA encourages patient engagement in study design, and there is much talk on the topic. A recent study conducted by Tufts CSDD on the Patient Engagement Capabilities Questionnaire (PECQ), or patient engagement scores, across various therapeutic areas sheds light on a critical aspect of clinical trials: the significance of patient engagement. But why is this metric so pivotal, and what can the latest findings teach us? Let’s delve into it.

The Importance of Patient Engagement in Clinical Trials:

First, let’s cover patient engagement, at its core, which refers to the active involvement of patients in every phase of a clinical trial, from design and execution to the dissemination of results. It’s not just about gathering data; it’s about building a relationship. Here’s why, according to FDA:

  • Enhanced Study Design: Incorporating patient perspectives for more relevant and representative studies.
  • Improved Study Execution: Facilitating better recruitment, adherence, and overall execution.
  • More Relevant Outcomes: Ensuring that the outcomes being studied matter to patients.
  • Addressing Barriers: Recognizing and working to overcome challenges associated with patient engagement.
  • Continuous Improvement: Leveraging patient feedback to refine and improve future studies.

The Data:

Some therapeutic areas are more prone to natural patient engagement risks compared to others. The Tufts study provides a unique insight into the PECQ (or patient engagement) scores across various therapeutic areas. Most notably, areas like kidney disease and genetic disease stood out with high scores, indicating a robust patient engagement. On the contrary, some therapeutic areas lagged, suggesting a potential gap in their patient-centric approaches.

PECQ Scores by Therapeutic Area
Genetic and Kidney Diseases Had the Highest Patient Engagement Scores


The intriguing finding from the study is the notably high PECQ scores associated with kidney and genetic diseases. While the paper does not explicitly detail the reasons behind these scores, we can postulate some potential factors based on recent research:

  • Nature of the Disease: Diseases, especially those that are chronic or have a significant impact on daily life, might naturally drive patients to be more proactive. For instance, a study titled “Precision medicine in kidney disease: the patient’s view” emphasized the importance of understanding the drivers of kidney disease from the perspective of those affected by kidney failure1. Patients’ daily challenges with severe conditions might lead them to seek research opportunities, leading to heightened engagement.
  • Potential of Treatment: The drive to find better treatments or potential cures can significantly motivate patient engagement. The promise of a research breakthrough can be a strong incentive, especially in areas where current treatments are limited or only manage symptoms. The aforementioned study on precision medicine in kidney disease highlights the potential of tailoring treatments to individual patients based on the underlying mechanisms of their disease1. Another example is oncology, where patients seek novel therapeutics after multiple failed treatments.
  • Awareness and Education: While the research paper does not delve into specifics, there may be more outreach and educational efforts in certain therapeutic areas, leading to increased patient awareness and, thus, engagement. For example, a study on Tuberous Sclerosis (TS), a rare genetic disorder, emphasized the importance of understanding the clinical and genetic characteristics of the disease, which can play a role in patient engagement2.

On the other hand, therapeutic areas with lower engagement might not have the same level of outreach, awareness, or perceived urgency from the patient community.

It’s crucial to note that these interpretations are based on general knowledge of patient engagement, supplemented by insights from recent research. Further studies would be required to pinpoint the exact reasons for the varying levels of engagement across therapeutic areas.

Recommendations for Sponsors:

Although the data highlights the influence of patient engagement scores across different therapeutic areas, it’s crucial for sponsors not to sideline patient engagement in their clinical studies. Here are some key suggestions:

  • Prioritize Early Patient Engagement: Engage with patient advisors during the initial phases of the clinical study design. This early involvement ensures that the study is patient-centric from the outset and can help identify potential challenges before they arise. Consider also involving patient advisors post-study to gather feedback for continuous improvement.
  • Establish Regular Communication with Regulatory Agencies: Initiate early interactions with the FDA or other regulatory agencies when considering incorporating patient advisors’ input into clinical study designs. Leverage the FDA’s guidance and feedback mechanisms to ensure that the study design and execution align with regulatory requirements and best practices. Regular communication can also provide clarity on any potential regulatory challenges.
  • Address Barriers and Challenges: Actively recognize and address potential barriers to patient engagement. This includes overcoming perceived barriers like misconceptions about FDA regulations, logistical challenges of in-person engagement, and finding knowledgeable patient advisors. This ensures a more effective and meaningful patient engagement process.

By following these recommendations, sponsors can enhance the design, execution, and overall success of their clinical studies while ensuring they are focused on outcomes that are most relevant to patients, regardless of which therapeutic area they’re in.


The role of patient engagement in clinical trials is undeniable, and there’s a spectrum of engagement across therapeutic areas. While patient engagement scores provides some insight into therapeutic areas, following the patient engagement process highlighted by FDA should not be ignored. For sponsors and researchers, the path forward is clear: prioritize patients, build trust, and ensure that clinical trials are as patient-centric as they are scientific.


  1. Brown, K., Campbell, C., & Roberts, G. V. (2020). Precision medicine in kidney disease: the patient’s view. Nature.
  2. De Sautu, E. C., Vales, J., Bermejo, C. L., Ramos, F., Padilla, M., Aleja, J. G., & Morales-Conejo, M. (2021). Clinical, Genetic and Quality of Life Study of a Cohort of Adult Patients With Tuberous Sclerosis. Research Square.
  3. Kim, J.Y., Acelas, M.P.B., Granville, C.A. et al. Benchmarking Patient Engagement Capabilities and Preparedness of Drug Development Sponsors. Ther Innov Regul Sci 57, 1040–1049 (2023).
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Moe Alsumidaie Chief Editor
Moe Alsumidaie is Chief Editor of The Clinical Trial Vanguard. Moe holds decades of experience in the clinical trials industry. Moe also serves as Head of Research at CliniBiz and Chief Data Scientist at Annex Clinical Corporation.