Life science organizations increasingly rely on real-world data (RWD) and real-world evidence (RWE) to support clinical trial activities like study design, observational studies and even new treatment paradigms. Applying innovative methodologies like co-creation and patient registry insights to data-driven clinical trial models contribute, to more effective and efficient clinical research and, as an improved patient experience.1
Newly refined FDA definitions, expanded use guidelines
In October 2021, the Food and Drug Administration (FDA) issued guidance on expanding the expanded use of real-world data and real-world evidence, acknowledging their increasing role in health care decisions beyond post-marketing safety and efficacy studies.
According to the FDA, real-world data are the data relating to patient health status and/or the delivery of health care that are routinely collected from a broad variety of sources, including2:
Electronic health records (EHRs)
Claims and billing activities
Product and disease-specific registries
Patient-generated data, including in home-use settings
Data gathered from other sources that can inform on health status, such as mobile devices
“These data hold [sic] potential to allow us to better design and conduct clinical trials and studies in the health care setting to answer questions previously thought infeasible. In addition, …with the development of sophisticated, new analytical capabilities, we are better able to analyze these data and apply the results of our analyses to medical product development and approval,” the FDA said in a statement.
Clinical registry data is longitudinal, fit-for-purpose and designed to help answer research questions that require large volumes of structured data among specific patient populations. This level of reliable diagnostic detail, for instance, to validate long-term safety events related to drug exposure, are typically collected across a broad network of sites. These registry studies enroll qualified patients who then share structured data, usually via questionnaires (including validated patient-reported outcomes (PROs)), at designated intervals for a specified period. Physician-reported outcomes often are integral to the data generated by these registry studies.
Because of the granularity of this type of real-world data, clinical registries are typically used to satisfy post-authorization safety studies, typically required by the FDA. Clinical registry data can also be used to support expanding indications for product use.
Bespoke patient registry model
Co-creation is a philosophy that reflects a desire to partner with patients (and their caregivers and support persons) living with a given disease or condition to determine the ways health care is obtained, accessed and delivered. This includes pharmaceutical and clinical research processes. One proven co-creation technique for collecting real-world data during the peri-approval phase is via the bespoke, disease-based patient registry.
Single-sponsor patient registry studies can provide relevant, valuable patient and clinical data for any number of research questions, including safety and efficacy. They also can collect voice- of- the- patient insights on topics like unmet needs, therapeutic burden, patient preference, how treatment decisions are made and prescribing patterns. Additionally, patient registries provide participants with an active and rewarding way to contribute data that will transform health care delivery in the years to come.
Incorporating single-sponsor patient registry data and feedback into the peri-approval process aids product decisions surrounding the sponsor’s approach to marketing and commercialization.
More broadly, the single-sponsor patient registry model can capture both granular and longitudinal data on a sub-group of patients and combine that dataset with other types of data (e.g., patient experience insights, EMR data, etc.). These comprehensive data expand the lens through which the sponsor can view patients living with a given disease or condition, informing changes needed in medical care access, treatment practices, guidelines and health policies
In order to truly understand the lived experience of a patient with a given condition or disease, several types of real-world data sources should be analyzed. While granular clinical data may communicate one aspect of patient health, adding in qualitative data and experiential descriptions show the real story - one that may be more complicated. The potential to partner with patients in this real-world sense provides an innovative opportunity to transform the power of the patient voice in its application to clinical development, expanding the breadth of real-world evidence.
1) Measuring the Impact of Patient Engagement and Patient Centricity in Clinical Research and Development. Stella Stergiopoulos, Debra L Michaels, Barbara Lopez Kunz, Kenneth A Getz. Ther Innov Regul Sci 2020 Jan;54(1):103-116. Epub 2020 Jan 6.
2) Real-World Evidence. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Accessed 1/22/22.