What’s In Your RWE Evaluation Toolbox?


Real-world evidence (RWE) – information collected from everyday health care experiences – is growing in availability, and many health care decision-makers are interested in understanding and using this information. In fact, one in every two FDA approvals last year included RWE studies in the regulatory submissions – a trend that’s expected to grow with forthcoming FDA guidance on RWE in 2021 and with data availability from routine patient care. Clinical guideline groups and professional societies use high-quality RWE studies to complement existing evidence from clinical trials or fill gaps in knowledge, and payers use it to inform their formulary and coverage decisions.

A sharper focus on RWE has led to an increased need for advanced analytical tools that make sense of the RWE. Over the past several years, professional scientific societies, organizations and researchers have designed such tools to aid the evaluation and use of RWE. However, the awareness and use of these tools may be lacking. A new Best Practices article published in the Journal of Managed Care and Specialty Pharmacy (JMCP) aims to help people learn more about what these tools do and how to use them.

“Although RWE has been growing, citations of these studies in formulary and coverage decisions remain infrequent,” said Jennifer Graff, PharmD, vice president of policy research at the National Pharmaceutical Council (NPC), and a co-author of the article along with authors from the University of Washington. “In a survey of health plan pharmacy and medical directors, most believe RWE can help make more informed decisions, but few feel adequately prepared to evaluate or use RWE. Over 50% of surveyed health plan pharmacy and medical directors noted a pivotal barrier to RWE use was their personal or their organization’s lack of experience interpreting RWE studies,” she said.

RWE Evaluation Tools

The article compares the characteristics and feature of the following five tools based on how each one can be used to evaluate the quality and significance of RWE:

1. Good ReseArch for Comparative Effectiveness (GRACE) checklist

2. IMI GetReal RWE Navigator (RWE Navigator)

3. Center for Medical Technology Policy (CMTP) RWE Decoder

4. Academy of Managed Care Pharmacy (AMCP)-International Society for Pharmacoeconomics and Outcomes Research (ISPOR)-NPC CER Collaborative tools

5. Real World Evidence Assessments and Needs Guidance (REAdi) tool

Dr. Graff notes that each tool has its own purpose, function, strength, complexity and limitations. “Some tools evaluate individual studies. Other tools are more encompassing, and they identify available studies, assess these studies and guide the decision process,” she said. “Some are checklists. Other tools have worksheets or online assessments. It’s important to find one that best fits your use and needs.”

The article reviewed the tools across 27 items and eight domains based on the tool’s objective, development and scope, platform and availability, features of design, assessment of internal and external validity, ability to summarize a body of evidence in addition to evaluating individual RWE studies, assistance towards decision-making, and the ability to share the tool results with others.

“Each of these five tools is publicly available and free for anyone to use,” said Dr. Graff. “Although most were designed for population health care decisions makers, these tools have been used by researchers designing studies, training programs educating students and clinical guideline bodies as they evaluate the evidence to inform care guidelines. Over time, users know what flaws to look for and become more efficient with practice.”

However, Dr. Graff also points out that using more RWE studies may not always equate to better decisions if quality is ignored. “In the COVID-19 pandemic, we’ve seen how important it is to consider all the available evidence as well as the quality of that evidence. Without considering both – all evidence and the quality of those studies – decision-making and patient health can suffer.”

Read the article’s full findings on the JMCP website.