Real-world evidence (RWE) is only as meaningful as the perspectives it includes. While healthcare data (e.g., claims) can reveal patterns, it’s the patient voice that brings true depth, valuable context, and humanity to those patterns. Increasingly, regulatory agencies like the FDA are recognizing that patient experience isn’t just complementary, it’s foundational. For instance, the FDA has been mandated by the 21st Century Cures Act to incorporate patient experience data (PED) into drug and biologic application reviews, emphasizing transparency and accountability. According to a 2025 study, surveyed professionals overwhelmingly agree that expanding PED use is crucial for FDA decision-making.
The Patient Voice: A Strategic Imperative
The “patient voice” encompasses more than patient-reported outcomes or satisfaction scores. It includes the narratives, preferences, and values that patients bring to their healthcare journeys. Understanding patient needs, goals and desires as well as the challenges they face living with their unique set of healthcare needs daily helps medical affairs teams create HCP and patient educational materials, informs conversations with payors and policy makers and puts them in a position to not only advocate for the improved wellbeing and quality of life of the patients they serve but also to be a positive catalyst for change. Incorporating these insights ensures that RWE studies are not only scientifically robust but also resonate with the real-world experiences of patients.
FDA Initiatives Elevating Patient Voice
Over the past decade, the FDA has been increasing its emphasis on incorporating the patient into its assessments, examples have included:
- Project Patient Voice: Launched by the FDA’s Oncology Center of Excellence, Project Patient Voice is an online platform that provides patient-reported symptom data from cancer clinical trials. This initiative addresses the gap where such data is often absent from drug labels, offering patients and healthcare providers additional insights into treatment experiences.
- Patient-Focused Drug Development (PFDD) Program: The FDA’s PFDD initiative systematically gathers patient perspectives on specific diseases and treatments. Through public meetings and guidance documents, the program ensures that patient experiences inform drug development and regulatory decisions.
Integrating Patient Voice into RWE
Patient feedback can help improve internal processes and allows pharma and medical tech companies to design better clinical trials to reduce attrition, identify areas of challenge and gaps in care to provide more comprehensive and valuable educational materials, and uncover unmet needs that organizations can address to be considered the innovative thought experts in their disease state/therapeutic area. Integrating patient voice into healthcare research can take up numerous forms.
Establishing Patient Advisory Boards: In addition to traditional physician advisory boards, it is critical to collaborate with patients as well to gain insights into their experiences and preferences. Patient input can reveal factors that can impact quality of life that may be overlooked through other datasets.
Conducting Qualitative Interviews: Gather in-depth information about patient journeys to inform study design. This can guide the selection of relevant endpoints and improve study protocols as well as inform the implications of the research.
Partnering with Advocacy Groups: Leverage the expertise of patient organizations to ensure diverse and representative input. Organizations like Rare Patient Voice have taken this a step further by developing a community of patients whose voices can be heard directly in primary research efforts.
When patient perspectives are integrated into RWE, interventions can be tailored to better align with patient needs, leading to enhanced health outcomes.
Embracing the Future of RWE
Incorporating the patient voice into RWE is not just a methodological enhancement, it’s an imperative. By actively engaging patients throughout the research process, we can ensure that RWE studies are more relevant, inclusive, and impactful.
Author
Nicole Garberg
Head of Real World Evidence
Nicole has been consulting with clients across all areas of the healthcare industry for 20+ years. Her expertise in patient experience, QoL and journey mapping is leveraged to improve patient outcomes and business performance for her clients and their patients. Nicole holds a MS in Experimental Psychology and BA degrees in Psychology & Sociology.
