By Sarah Wells Kocsis, SWHR Vice President of Public Policy
Patients have the greatest stake in the success or failure of medical products, and their voices must be taken into consideration throughout the entire development process, regardless of whether the product is a drug, device, biologic, or other therapeutic.
The Food and Drug Administration (FDA) has achieved significant milestones in its efforts to more systematically integrate patient experience information into the development and regulatory review of new medical products. As FDA expands this work, the Society for Women’s Health Research (SWHR) seeks to ensure that this process includes meaningful input from women.
Women account for more than half the U.S. population, make more than three-quarters of health care decisions for their families, and provide the majority (66%) of caregiving. Thus, appropriate inclusion of women — such as female patients, caregivers, and health care providers — and their input is necessary for the successful development of medical products.
What is patient-focused drug development?
“Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.” — Food and Drug Administration
In 2012, Congress enacted legislation that required FDA to conduct disease-specific PFDD meetings to provide key stakeholders with an opportunity to hear from patients directly. So far, FDA has held 26 meetings on a variety of diseases and conditions, including many that disproportionally or exclusively affect women, such as breast cancer, female sexual dysfunction, chronic fatigue syndrome/myalgic encephalomyelitis, and fibromyalgia. FDA captures what is heard at these meetings in Voice of the Patient reports.
“There are still a lot of aha moments that our FDA review staff and others have at these meetings,” FDA PFDD Program Senior Advisor Pujita Vaidya told SWHR’s Policy Advisory Council in December. “A lot of FDA reviewers, we realized, just had not ever engaged with or met an individual with a particular disease … so it’s been really impactful.”
Vaidya spoke to SWHR’s policy committee about the importance of incorporating patient input across the entire lifecycle of medical product development from the translational phase to the post-market stage. “We need to incorporate the patient experience data from very early on in development,” she said. “It has to be part of the initial plan.”
To supplement the PFDD meetings, the 21st Century Cures Act directed FDA to develop guidance “regarding the collection of patient experience data, and the use of such data and related information in drug development.” FDA is working on four methodological and two non-methodological PFDD guidance.
How is SWHR working to ensure women are appropriately represented in PFDD?
Engaging stakeholders — including patients, patient advocacy organizations, researchers, medical product developers and others — in the PFDD Program and guidance development is critical, Vaidya said. “We truly want to hear from folks,” she stressed, adding that FDA analyzes and revises its work based on the feedback it receives at public meetings and in comments to the docket. She encouraged stakeholders like SWHR to contribute through activities such as supporting research, coordinating stakeholder work, convening meetings, and contributing to guidance and policy development.
SWHR strongly supports the PFDD initiative to integrate the patient voice into drug development and regulatory decision-making. SWHR has provided input to FDA and will continue to do so. In comments to the docket on February 16, 2018, and December 14, 2018, SWHR stressed the importance of addressing the unique considerations of women as patients, caregivers, and family decision-makers across the lifespan and emphasized the following:
- Sex and gender play critical roles in the risk, pathophysiology, presentation, diagnosis, treatment, and management of disease, and thus, are important variables for consideration in all phases of patient-focused research. Many conditions and diseases are either unique to women, occur disproportionately in women, or affect women differently than men. The study of sex and gender differences is leading to important discoveries of how women and men differ in fundamental ways and how these differences affect disease risk, symptoms, diagnostic sensitivity and specificity, and response to therapy.
- The terms “sex” and “gender” as defined by the Institute of Medicine (IOM) should be added to the PFDD glossary. Establishing standardized terminology is paramount to achieving shared understanding of principles and meaningful dialogue pertaining to PFDD. As defined by IOM:
- “Sex” refers to the classification of living things according to reproductive organs and functions assigned by chromosomal complement.
- “Gender” refers to the social, cultural, and environmental influences on the biological factors of women or men. Gender is rooted in biology and shaped by environment and experience.
Moving forward, SWHR will continue to engage in the PFDD initiative and is preparing a set of principles that are essential to ensuring women are carefully considered within medical product development.