The use of real-world data in drug development

Real-world data (RWD) is information related to patient health that is routinely collected from a range of sources like electronic health records, medical claims, disease registries and digital health technologies. Real-world evidence (RWE) is the clinical evidence about a medical product and its potential benefits or risks that is derived from analyzing RWD. RWD can play a crucial role in enhancing drug discovery, clinical development, post-approval studies and supporting use of medicines and vaccines. It can also inform regulatory decision-making, especially when randomized controlled trials (RCTs) are not feasible or ethical, offering a potential cost-effective and time-efficient alternative.

When it comes to developing medicines, stakeholders ranging from academics, industry, regulators and health care providers have long recognized the opportunity of using RWD to evaluate the benefits and risks of therapeutic interventions.

Here’s just a few benefits:

RWD can help inform the benefit-risk profile of medicines for both new drug applications and post-approval uses.

• This involves evaluating the effectiveness and/or safety of a medicine in real-world settings. By leveraging RWE, assessments can be made about a medicine’s current use and potential use in the future, both during the drug development process and after it’s been approved.

RWD can help make effective and safe treatments available to patients with rare diseases and/or limited treatment options.

• RWD can provide valuable insights into the real-world performance of medications, particularly in populations where RCTs may not be feasible, ethical or may not fully represent the diversity of patients who will ultimately use the treatment. RWD can be used to provide additional insights into use of a medication across diverse patient populations, ultimately leading to more information about the use of medicines by patients who need them the most.

RWD can help satisfy FDA requirements post initial approval.

• After approval of a medication, FDA can require sponsors to conduct studies to gather additional information about a product’s safety, efficacy or optimal use. RWD can be leveraged to help satisfy these requirements. Additionally, RWD can be used to support confirmatory evidence for the effectiveness of a medicine approved through accelerated approval.

With the emergence of novel technologies and the commercialization of a wide range of data sources, researchers now have access to larger and richer datasets. The reauthorization of the Prescription Drug User Fee Act (PDUFA VII) marked a significant step forward in leveraging RWE for regulatory decision-making in drug development.

PDUFA VII introduced a new pilot program to advance the use of RWE for regulatory decision-making, including for new indications and to satisfy post-market study requirements. Through this initiative, the U.S. Food & Drug Administration will also conduct public workshops to share insights from the program and issue related guidance documents. Furthermore, FDA is holding public workshops, conducting demonstration projects and issuing guidance documents to advance the use RWE analytical methodologies and better support pregnancy post-marketing studies.

To understand how to better leverage RWD in drug development, PhRMA and BIO recently partnered on a workshop and subsequent workshop summary featuring industry leaders, regulators, academics, data partners and patient advocates. Read the summary here.