Stakeholders agree that good research has the potential to inform better health care decisions. But how do we define "good research?" Although randomized controlled trials (RCTs) have long been considered the gold standard of scientific studies, many researchers agree observational studies play a central role in answering certain types of questions, particularly regarding real-world behavior. However, some stakeholders remain reluctant to rely on observational studies. Payers, for example, prefer to use RCTs instead of external observational data to inform drug coverage decisions, as NPC-funded research described in the April issue of the Journal of Managed Care Pharmacy.
Given the value of observational approaches in conducting comparative effectiveness research (CER), developing tools and guidelines to enhance the quality of observational studies can help ensure all stakeholders have confidence in the findings. The Patient-Centered Outcomes Research Institute (PCORI) has set out to do so by identifying 60 research standards in its draft Methodology Report released earlier this month.
NPC also is playing a key role in informing the conversation. A series of papers in the May Journal of Comparative Effectiveness Research sheds further light on the topic by exploring the need for better resources. To develop the series, NPC teamed up with the Center for Medical Technology Policy and Outcome, a Quintiles Company, to explore the need for a translation table researchers can use to determine the best approach to studying a given question. These three groups also developed Making Informed Decisions: Assessing the Strengths and Weaknesses of Study Designs and Analytic Methods for Comparative Effectiveness Research, a booklet that describes both experimental and nonexperimental study designs and methods that may be used to address CER study questions. In addition, NPC is collaborating with the Academy of Managed Care Pharmacy and the International Society of Pharmacoeconomics and Outcomes Research to develop a toolkit payers can use to evaluate observational research.
In addition, NPC is supporting an initiative to develop a Good ReseArch for Comparative Effectiveness (GRACE) checklist, a validated tool for the assessment of observational CER quality and usefulness for decision making. Watch NPC’s interview with Dr. Nancy Dreyer, Global Chief Scientist and SVP at Quintiles Outcome, as she explains the importance of observational studies and the GRACE initiative and checklist.
As more resources like these are developed, it will help all stakeholders use meaningful criteria and standards in designing and evaluating research.