When Should Observational Studies Be Used for Decision-Making, or When Will the Emperor Have Clothes?

Researchers have been discussing the pros and cons of observational studies for decades, and it’s a conversation that will continue long after the Institute of Medicine meeting has concluded. In fact, if you are interested in hearing more about the topic, you should attend the International Society of Pharmacoeconomics and Outcomes Research International Meeting in New Orleans on May 18-22. During the meeting, NPC Chief Science Officer Dr.

Researchers have been discussing the pros and cons of observational studies for decades, and it’s a conversation that will continue long after the Institute of Medicine meeting has concluded. In fact, if you are interested in hearing more about the topic, you should attend the International Society of Pharmacoeconomics and Outcomes Research International Meeting in New Orleans on May 18-22. During the meeting, NPC Chief Science Officer Dr. Robert Dubois will be moderating a panel discussion, “When Should Observational Studies Be Used for Decision-Making, or When Will the Emperor Have Clothes?”

Dr. Dubois will be joined by Jean Slutsky, PA, MSPH, Director, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality (AHRQ); Paul Wallace, MD, Senior Vice President and Director, Center for Comparative Effectiveness, The Lewin Group; and Marcus Wilson, PharmD, President, HealthCore.

During the session, panelists will take a closer look at observational studies that examine real-world treatment comparisons with potentially large sample sizes and patients with varying ages, disease severity, and presence of comorbidities. Because these studies also have limitations that could lead to incorrect comparative assessments, it raises a larger question of whether this evidence can be used in clinical and policy decision-making, and is it already?

Panelists will explore these questions from a few vantage points:

  • AHRQ’s Effective Healthcare Program summarizes evidentiary questions that influence decisions by clinicians (e.g., academic detailing, clinician guides) and patients (patient guides).  How and when do they consider experimental and non-experimental data? Would a large well-designed observational study have more weight than a small and imperfectly designed RCT?
  • Professional societies synthesize evidence in developing practice guidelines that influence clinical decisions.  Based upon newly collected and not previously presented data, are they using observational studies in their decisions?  Should they use these data more or less than they do?
  • Payment decisions influence care.  When should payers use observational studies?  Are they doing so already? What barriers need to be addressed for non-experimental designs to potentially have a firmer role?

The panel session will take place on Tuesday, May 21 from 11 am-12 pm and is open to registered attendees of the ISPOR meeting.