![]() More commonly, RWE has been used to augment evidence from RCTs, rather than accepting RWE on its own merit. 2 Suparna Wedam et al., “FDA approval summary: Palbociclib for male patients with metastatic breast cancer,” Clinical Cancer Research, March 2020, Volume 26, Number 6, pp. The US Food and Drug Administration (FDA), for example, approved the expansion of indications for Pfizer’s Ibrance to include male breast cancer on the basis of data from electronic health records and insurance claims. In a few instances, RWE has served as a primary source of evidence for the approval of therapies. In 2020, China’s National Medical Productions Administration published guidelines for RWE use to support drug development and review. ![]() 1 For example, in 2019, Japan’s Pharmaceuticals and Medical Devices Agency set up new consultation services for use of registry data in new drug applications and reexamination applications. ![]() As a result, regulators around the world are beginning to incorporate RWE into their approval processes. They recognize that real-world evidence (RWE) can help to accelerate drug development or indication expansion, minimize exposure of patients to placebo control arms, and help to offset rising drug-development costs. Regulators are beginning to consider evidence beyond randomized controlled trials (RCTs). The role that evidence generation plays among key stakeholders is evolving and becoming increasingly important. The end result is a significantly more efficient and effective use of resources in pursuit of better patient outcomes. From the outset, IEPs take into account the evidence needs of different functions and geographies across the life cycle of an asset, and then collaboratively determine how to meet them using a broad range of methods and data (Exhibit 1). Yet they tend to be bolted together, not integrated, and so are a far cry from the integrated evidence-generation plans (IEPs) being developed by a handful of companies. These may well be included in a master, asset-level plan. The status quo in most companies is one in which each function draws up its own evidence-generation plan. Increasing volumes and types of available data raise the potential to generate this evidence, but if it is to be realized, biopharmaceutical companies may need to change their approach to evidence generation, working far more strategically and collaboratively than they do at present. And patients need evidence to understand how a therapy might meet their needs. Payers need evidence to support patient access. Clinicians need evidence to support optimal treatment decision making. Evidence that a therapy is both effective and safe is the primary requirement. Understanding and improving patient outcomes through the generation of evidence is one of the most critical activities of an innovative biopharmaceutical company.
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