In December 2018, FDA Commissioner Scott Gottlieb, MD, issued a statement about how real-world data (RWD) and real-world evidence (RWE) offer opportunities to understand clinical outcomes of pharmaceutical products. RWD comes from a variety of sources, including EHRs, medical claims, lab results, and wearable and home monitoring devices, such as glucometers and oximeters. “Because they include data covering the experience of physicians and patients with the actual use of new treatments in practice, and not just in research studies, the collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypotheses for further testing of new products to drive continued innovation, and inform us about the performance of medical products,” the statement reads.
A longitudinal database is a collection of de-identified RWD and RWE from many patients over many years, which can have several advantages for pharma companies, versus data collected from clinical trials alone. Some uses for a longitudinal database in the pharmaceutical sector include:
- More accurate representation of medication effects – Because the population of those enrolled in clinical trials is not necessarily representative of the overall U.S. population in terms of race, ethnicity, sex, or age, the effects of medications on patients in clinical trials may be substantially different from the effects on the overall population once the drug comes to market. There may also be bias because clinical trial participants can be incentivized to adhere to medication instructions (e.g., how often to take a medication, whether to take with food or water, not to take with certain vitamins, etc.) if they are being compensated for rial participation. In a real-world setting, patients aren’t compensated for taking medications, and circumstances such as education, income, literacy levels, and lifestyle may act as barriers to adherence.What this means for pharma companies is that the results they get in clinical trials may not be an accurate representation of the results they will see once a drug comes to market. However, a longitudinal database can provide pharma companies with many years of RWD and RWE from thousands of patients. Analyzing such information can show adverse effects and overall efficacy in ways that clinical trials of a few months or even a few years cannot.
- Reduced cost and narrowed focus of R&D – Pharma companies can use longitudinal data to gain valuable insights into the efficacy of their drugs. A longitudinal database can help them answer questions in real-world settings, including:
- Which cancer drugs result in longer survival rates for patients with leukemia?
- Which antibiotics result in lower rates of infection recurrence?
- Do patients taking a certain type of medication have more hospital readmissions than those taking another type of medication?
- Do asthma patients taking certain medications visit the ED with acute asthma attacks less frequently than those taking other medications?
Seeing these trends, over time, can inform the pharmaceutical industry about which molecules, mechanisms of action, and therapies result in better patient outcomes. This information can help pharma companies focus and narrow their R&D efforts, shaving off billions of dollars and several years in the process of getting new drugs to market. It can also help pharmaceutical sales reps target their marketing efforts, sharing data on certain medications with certain doctors based on the physicians’ target populations, or offering statistics about which medications historically show the best outcomes.
- Improved post-market monitoring –Many drugs undergo additional clinical trials and/or post-market surveillance once a drug has come to market. “FDA uses postmarketing study commitments to gather additional information about a product’s safety, efficacy, or optimal use,” according to the FDA. And an article in the journal Cardiology notes, “Postmarketing surveillance covers the range of observational studies undertaken after marketing including cohort studies, case-control studies, and spontaneous reports of suspected adverse drug reactions. These observational studies are less rigorous than clinical trials but have the potential to provide information from a representative sample of ‘real-life’ patients.”
In its December statement, the FDA recognized the value that RWD and RWE can have on such postmarketing studies. Longitudinal databases of such RWD over time can be even more valuable in this context. “Traditional post-market studies typically require years to design and complete and cost millions of dollars. By using RWD and RWE, we may be able to provide patients and providers with important answers sooner—identifying a broader range of safety signals and following up on them more efficiently,” the FDA statement says.
In addition, the FDA says it is also using RWD and RWE as part of the information it takes into consideration when evaluating new drug applications (NDAs) from pharma companies. “We currently have new drug applications under review where RWD and RWE are helping to inform our ongoing evaluation as one component of the total complement of information that we’re evaluating,” the organization says.