Successfully demonstrating product safety and efficacy in a randomized clinical trial is a monumental event, but it doesn’t always translate to market access and uptake one it’s launched. Since clinical trials are limited to a controlled sub-set of patients, observations recorded in the clinical trial setting can vary from what actually occurs in clinical practice. That’s where real-world evidence (RWE) can help pharmaceutical companies to inform development planning and also to demonstrate a product’s comparative effectiveness, safety and value from the viewpoints of various stakeholders, including regulators, payers, prescribers and patients.
This article discusses the role of RWE, the different information needs among stakeholders and potential solutions for meeting their evolving requirements.
Key sources of RWE
Information outside of traditional clinical trials
Large simple trials or pragmatic clinical trials
Prospective observational or registry studies
Retrospective database studies
Administrative and healthcare claims
Electronic health records
Registries (e.g., product, procedural or disease)
Data beyond the clinical trial
The controlled nature of a clinical trial allows researchers to carefully examine the effects of a treatment as compared to a placebo or the latest standard of care. But outside of a clinical trial, patient care can be more variable and complex. To understand current treatment patterns and unmet needs among a more heterogeneous population in the market, pharmaceutical companies are increasingly turning to RWE
RWE draws from sources outside of a traditional clinical trial, for example, from an existing database, or conduct of a prospective observational study or disease registry.
By better understanding patient care in routine clinical practice, pharmaceutical companies can inform internal decision points across the development lifecycle of their product, as well as support external decision making. For example, RWE can help:
Historically, RWE assets were employed near the end of development, but now many pharmaceutical companies are including RWE research earlier in their drug development process.
Satisfying stakeholders’ needs
Because RWE is intended to address the needs of different stakeholders and can be generated through myriad study designs, successfully planning RWE studies requires multidisciplinary expertise and input.
Stakeholders include external decision makers who each have their own objectives and information needs. For example, a regulator may be interested in real-world safety once the product is launched and more widely prescribed. The payer maybe interested in real-world clinical cost effectiveness to determine formulary placement.
Internal stakeholders in the company also approach RWE from distinct perspectives. Consider the differences and levels of familiarity with RWE between medical affairs, health economics and outcomes research, market access, statistics, regulatory, scientific and clinical/medical staff. These contrasting positions and roles in the RWE planning process stress the importance for internal stakeholders to understand each other's objectives and information needs so that they can coordinate better with one another.
Ongoing discussion and coordination between internal stakeholders offers many benefits and may be part of an ongoing business strategy, but the reality may contradict this ideal scenario. In complex corporate structures, teams may manage their own separate budgets and have separate reporting lines, different visions, priorities, objectives, as well as different vendor in data agreements with different access rights. In some cases, each team is generating its own RWE in an uncoordinated process.
Addressing evolving environments
Yet even with a defined process and cross-company communication, information needs change over time. Several key events in the product life cycle can trigger the need for additional evidence. For example, if a competitor launches a new product and the standard of care changes, the generation of RWE must account for this shift. Generating RWE is an ongoing, iterative process with multiple, concurrent research activities. This process often includes studies that may need to be revisited or repeated over time.
Whether gathering primary real-world data through prospective studies or registries or accessing secondary data from existing health records, or databases, RWE study planning is complex. And while RWE projects can involve smaller and more targeted research endeavors, evaluating all of the available options for a RWE study requires a multidisciplinary approach.
To help sponsors with this involved effort, several Covance and industry RWE researchers recently developed an online framework tool. Learn more about this unique effort and the RWE framework tool in the next blog in this series, or be sure to check out the webinar “Enhancing Study Design with a Real World Evidence Tool.” To speak with someone about your RWE needs, contact us or to learn more about Covance Market Access visit us at www.covance.com/marketaccess.