Telling Health Stories for Drug Development – Takeaways from the DIA 2020 Virtual Global Annual Meeting

Mary Stober Murray
National Minority Quality Forum
Patient Engagement Community Chair

hile discussions among representatives from industry and regulatory agencies often stress the importance of patient stories, only a few of these discussions give insights into how to elicit these perspectives, identify patients, and empower them to share their experiences. Further, industry requires a level of change management to convert these insights into meaningful changes in drug development structures and processes geared to improving patient experiences and outcomes. The live panel discussion “Telling Health Stories for Drug Development” at the DIA 2020 Virtual Global Annual Meeting  revealed the considerations and steps to take when integrating patient stories and experiences into drug development activities.

Through their own stories, panelists discussed their perspectives on how to elicit experiences from vulnerable populations, how to build a patient community where none exists, and how to aggregate patient preferences, perspectives, and other data into resources for researchers. Panelists noted where changes can occur beyond the protocol to fully integrate patient insights as fundamental elements of a drug development program. Finally, panelists gave their perspectives on the COVID-19 pandemic and Black Lives Matter movement, and the importance of making sure that diverse patient perspectives, particularly from communities of color that are disproportionately at risk for COVID-19 infection and serious disease, are integrated into the vaccine development programs now urgently underway.

Key Takeaways

  • Plan ahead to identify “representative” populations and individual patients.
  • Stigma may silence the very patient voices that are most necessary for relevant drug development. Methods from the arts, such as drama, and behavioral sciences can provide safe ways for people to share their experiences and insights with researchers.
  • Change management techniques are needed to get beyond an individual protocol and fully integrate patients into drug development programs. Consider changes to program-level eligibility criteria, endpoints, terminology, and even to internal organizational structures and processes.
  • The urgency of the COVID-19 pandemic and the Black Lives Matter movements shine a light on populations whose voices must be heard while potential vaccines and treatments are fast-tracked into development.

The panel discussion opened with a question: “Whose stories are important?” Panelists discussed different ways to think of patient populations and the variety of needs, including not only medical needs but socioeconomic needs and culture. Entire patient populations may be “invisible” for many reasons, such as poor access to disease screening and treatment, language barriers, and stigma. Panelists explored the critical role of overcoming stigma to empower patients in sharing their experiences openly and having data reflect their reality.

Alternative methods to help patients express themselves may be used to overcome stigma. Theater professionals and storytelling coaches can be engaged to help people participate in constructing narratives that accurately represent their experiences and perspectives, even if specific individuals remain anonymous. In a social science method called experience sampling, people are asked to write a daily diary, noting their thoughts, feelings, and environmental circumstances over time. These methods can help researchers understand not just demographic differences among patients, but differences in circumstances that inform the experience of living with a given disease, the feasibility of participating in a clinical trial, and ultimately of adhering to medication.

Similarly, a patient population may be “invisible” for scientific reasons. As an example, Lynch Syndrome is a genetic mutation associated with multiple cancers. Unless patients have been tested for Lynch, they may not know they are part of this patient population. As a result, their experiences are not sought or offered, and their data are not collected from this perspective. A research program and related protocols focused on Lynch Syndrome patients will look different than a research program dedicated to a specific tumor type. With this in mind, Alive and Kickn, a Lynch Syndrome patient advocacy organization, is building a disease registry where Lynch Syndrome patients enter their data and experiences via survey instruments to share with researchers.

Session Panelists

Mary Stober Murray (Moderator), VP, Collaborative Action Networks, National Minority Quality Forum

Adebola Adedimeiji, Associate Professor of Epidemiology and Population Health, Albert Einstein School of Medicine/Montefiore Medical Center

Dave Dubin, Founder, Alive and Kickn (Lynch Syndrome)

Amy Fesmire-Baus, Senior Scientific Writer, Bristol Myers Squibb

Even while declaring patients as partners, industry still faces major challenges in systematically integrating patient perspectives beyond individual protocols. The panelists discussed the importance of building awareness among clinical team members of diverse patient needs early in development, rather than waiting until a final protocol is being operationalized. Specifically, clinical teams can map protocol elements (such as eligibility criteria, endpoints, and terminology) to areas of a patient’s concern (such as worry) and then consider interventions to reduce the concern. In one protocol example, a patient expressed that one medication on her regimen was key to keeping her flares in check, and the study required a washout period. This patient was unwilling to go off that specific medication in her regimen, so she planned to decline participation if that requirement was mandatory. Some other experiences have been the move to liquid biopsies for screening genetic mutations of tumors to decrease the patient burden of traditional biopsies. Adjustments focused on reducing a known patient burden may have the additional benefit of facilitating the study’s operations.

Changes in organizational structure and processes can further integrate patients into drug development through their presence on Advisory Boards and drug monitoring Steering Committees as well as by including patients as authors on publications.

Addressing COVID-19, panelists suggested that researchers need to seek surviving patients and/or caregivers in communities of color which are disproportionately at risk for COVID-19 infection and serious disease. Collecting these experiences, researchers may consider inclusion/exclusion criteria, the schedule of activities, assessments, and baseline characteristics early on, in the context of these patients’ lives. For example, coughing, shortness of breath, and cyanosis are typical for many patients with heart disease. Researchers must determine if these symptoms are part of the underlying condition or of COVID-19 – from the perspective of inclusion/exclusion criteria as well as assessments to determine if the medication is working. Researchers need to understand the baseline levels in patients, such as oxygen saturation levels, in order not to exclude whole segments of the very ill heart disease population or to categorize them as non-responders.

Ultimately patient stories are “more than an anecdote” and must be pursued, collected, analyzed, and integrated in ways that capture the human complexities of living with disease and the consequences of treatment.

Speakers shared several resources at the end of the session that can help those involved in drug development systematically integrate patient perspectives and experiences. Notably, the newly-released Collecting Comprehensive and Representative Input Guidance from FDA’s Patient-Focused Drug Development drives home the expectation from regulators that patient needs are methodically articulated and incorporated into pharmaceutical development plans and submissions. Here are some of the other resources:

Please also visit DIA’s resources on Patient Engagement.