September 6, 2022
Real-world data (RWD) from clinical drug registries can and should be used throughout the drug development lifecycle. At all stages, registries ensure that assumptions about the current standard of care are accurate and evidence-based.
The following highlight example use cases for clinical registry data as a source for real-world evidence (RWE):
- Ongoing benefit-risk assessments
- Patient journey
- Pharmacovigilance monitoring activities
- Regulatory decision-making
For use cases where it’s critical to have access to consistent, reliable diagnostic detail, biopharmaceutical companies can leverage clinical registry data. These data have several advantages over other real-world data sources:
- Data is collected in a structured, prospective manner
- Questions are carefully designed to capture accurate, objective responses
- Records are complete, including all relevant data points
- Such complete registry data supports extensive comparison (e.g., an individual’s treatment over time, effectiveness across diverse patient populations, etc.)
Clinical registry data is especially fit-for-purpose when answering research questions that require large volumes of detailed information collected across a broad network of sites. Highly structured clinical registry data lends itself well to both simple and sophisticated statistical analyses.
“We engage a large number of physicians working in specialty areas that align to the diseases we cover in our registries,” says Christine Barr, Vice President, Drug Safety and Pharmacovigilance at CorEvitas. “In our registry studies, physicians and patients complete questionnaires on a set schedule. This allows us to collect accurate information about drug use and dosages, adverse events that may be experienced, disease activity and disease characteristics over time. We also collect targeted source documentation that supports adjudication by expert physician panels. Our registries include broader patient populations that may not be well represented in clinical trials. This helps address uncertainties about long-term use of therapeutics in real-world populations.”
Across Phase II and Phase III clinical trials, clinical registry data can be leveraged in several different manners. With a single-arm comparator study model, RWE is collected in lieu of a control group. This dramatically reduces patient enrollment targets. Using registry data can also simplify recruitment where the targeted disease is severe, and fewer treatments are available.
Once a drug has received regulatory approval, clinical registry data are also utilized. By design, clinical trials are relatively short in duration and examine only a limited number of patients. Many subgroups or vulnerable populations are rarely, if ever, represented in clinical trials, including pregnant women, people with background comorbid conditions (for example, cancer or heart disease) and patients who have already been exposed to or failed treatment in the same class of drugs.
“Once a drug has passed through clinical trials, it has demonstrated efficacy and relative safety in a cohort of patients over a limited observation period,” Barr explains. “But many questions remain that can and should be answered. Clinical registries are frequently used in the post-approval space to help answer these questions. These data provide missing information to contextualize what was seen in the trial, filling in the blanks that inevitably appear because clinical trials can never have enough time, enough patients or enough patient diversity to accurately mirror real-world conditions.”
Clinical registry data are especially valuable for use in formal post-authorization safety studies that pharmaceutical companies can employ to demonstrate to regulators that they are applying an appropriate level of rigor to answer questions about effectiveness and safety. Regulators may choose to require these sorts of studies, but pharmaceutical companies may also perform them voluntarily. These voluntary studies may be part of a risk management plan and strategy, to show regulators they are making a good-faith effort to assure the safe use of the product in the real world, complementing the insights derived from their clinical trials.
Read the original article, here.