What are the challenges?
Central to the credibility of observational studies is the transparency of research methods used to conduct these analyses.
- Despite the call for real-world evidence (RWE) to be “transparent, reproducible, disclosed, accurate and valid,” there is little agreement on how to achieve these elements.
- NPC recommends that the Food and Drug Administration (FDA), along with other stakeholders, work to more clearly articulate the requirements for transparent, reproducible evidentiary or hypothesis-testing studies.
The development of guidance on the standards for real-world and observational studies should build upon the foundation of existing good practices and be developed in consultation with experts, including RWE scientists in the biopharmaceutical industry and professional research societies
Are there tools that can help?
Efforts to identify best practices and standards for collecting and analyzing real-world clinical experience evidence have been made by several organizations, including the NPC. In totality, these guidelines can help identify areas where consensus exists, when further alignment and external stakeholder engagement are needed and whether important gaps need to be addressed.
Among these resources are:
- Good ReseArch for Comparative Effectiveness (GRACE) Checklist
- IMI GetReal RWE Navigator (RWE Navigator)
- Center for Medical Technology Policy (CMTP) RWE Decoder
- Academy of Managed Care Pharmacy (AMCP)-International Society for Pharmacoeconomics and Outcomes Research (ISPOR)-NPC CER Collaborative Tools
- Real-World Evidence Assessments and Needs Guidance (REAdi) Tool
Which tool should stakeholders use?
To help stakeholders navigate tools, NPC researchers developed a RWE Evaluation Toolkit that compares the characteristics and features of five RWE tools based on how each one can be used to evaluate the quality and significance of RWE. Because use of RWE is low, research that explores awareness, usefulness, and barriers to use of these tools may result in their improvement, uptake in their use, and ultimately increased use of RWE for decision making.