Standards for Real-World Evidence

Well-designed studies will help researchers and other stakeholders draw meaningful conclusions about which interventions are the most effective treatment options for specific conditions and patients.

By contrast, there is a cost associated with poorly designed research. In the comparative effectiveness arena, faulty studies could mean that consumers may make important treatment decisions based on incorrect data. To give patients the best chance to improve their health, it is crucial that they and their health care providers have sound evidence in hand to guide medical decisions.

Efforts to identify best practices and standards for collecting and analyzing real-world clinical experience evidence have been made by a number of organizations, including the National Pharmaceutical Council. Among these efforts:

Despite the increased availability of standards, there is little agreement among researchers and other stakeholders on the best methods and standards for conducting analyses using real-world clinical experience evidence.

  • A study conducted by NPC and others that compared and contrasted nine existing guidelines and standards for analyzing clinical experience evidence found that at a high level, there is general agreement on the basic elements required; however, there is variation in how various guidelines and standards recommend those elements should be conducted.
    • The research found that following one of these nine guidelines may be subsequently found deficient if measured against a different set of good practices or disparities in what is considered credible research and ultimately, what evidence is used to guide care.
    • To encourage the flow of high-quality research from researchers conducting real-world clinical experience evidence to health care decision-makers applying evidence, the paper suggests that a common set of agreed upon standards and guidelines is needed.

Frameworks for when studies from real-world clinical experience are useful exist, but they require adaptation for use in the regulatory environment.

NPC conducted several studies that examined, developed and tested frameworks for the consideration of research for use in health care decision-making:

As more resources like these are developed, it will help all stakeholders use meaningful criteria and standards in designing and evaluating research. Only well-designed studies will help researchers and other stakeholders draw meaningful conclusions about which interventions are the most effective treatment options for specific conditions and patients.