How patient engagement improves outcomes in chronic illness

Patient engagement’ has become a buzzword in healthcare and drug development, and it’s easy to gloss over the phrase without pausing to appreciate its richness and value. What is patient engagement exactly, how does it relate to patient outcomes, and what are people doing about it?

As we speak to pharma colleagues and attend conferences, it is apparent that patient engagement has different meanings across different companies and departments. For some, it is a process of harvesting the opinions and experiences of patients and patient advocates to inform decisions about research and support strategies that meet their needs. For others, it is a reciprocal process – a collaborative partnership between sponsors and patients where patients are included in decision-making. Some see it as the behaviours patients exhibit when they actively participate in their own healthcare such as information seeking, self-management and adherence to treatment.

We think of patient engagement as encompassing all three views. It is both a process and a behaviour throughout the drug life cycle. We’d argue an important aspect is the role of patients as meaningful, active partners in health research and in management of their health.

Engaging patients, starting in discovery and preclinical phases, is becoming recognised as a crucial component in developing and rolling out effective, patient-centred therapies. In fact, the FDA advocates for patient perspectives through the regulatory process via initiatives like the Patient-Focused Drug Development (PFDD) series.

Patients are the end users of any treatment, and their insights and input into decision-making at each stage of drug development is critical to deliver a final product that they will be motivated to use as prescribed. Research shows that engaged patients are more likely to adhere to treatment plans, make informed decisions about their care and adopt healthier lifestyle behaviours.

It is not just patients who benefit. Multiple peer-reviewed publications (many funded by pharmaceutical sponsors) have shown significant value to sponsors, including an expected net present value from an investment in patient-centric initiatives that can exceed a 500-fold return for a pre-phase 3 project. This is equivalent to accelerating a pre-phase 2 product launch by two and a half years.

Across our work with biotech and pharma clients, we implement strategies to incorporate patient engagement for regulatory and commercial success. Three fundamental tenets include:

1. Develop drugs that are relevant to the needs of those experiencing the disease
Meaningful engagement with people who have a lived experience of the disease of interest in the discovery stage can highlight unmet needs, providing actionable direction for clinical research. We find patients value the opportunity to share their experiences, like the symptoms that most impact their quality of life. We also find that close liaison with patient advocacy groups (PAGs) ensures that these early studies align with patient priorities. A good ‘patient outcome’ in this context is a research direction that results in the development of a drug that has an impact on the most bothersome symptoms or alleviates the worst impacts of their disease.

2. Ensure diversity and inclusiveness as early as possible in clinical trial stages
A lack of representativeness in clinical trials samples leads to poorer outcomes, absence of external validity and exacerbation of health inequalities. Inclusive patient participation in clinical trials is vital for successful drug development, and patient engagement in trial design is important to enhance enrolment, adherence to trial protocol and retention. In our experience, best practices allow patients to:

• Evaluate eligibility criteria
• Inform the design of less burdensome protocols to enhance retention
• Support a ‘dry run’ of a protocol that can be more insightful to the realities of the trial than a protocol read-through
• Enhance recruitment strategies and materials by identifying barriers to participation and ways to overcome them
• Help identify outcomes that are truly relevant to patient needs and preferences, including giving insight into what a meaningful change looks like to them.