Faster Ways to Get Actionable Evidence to Stakeholders

Beyond Primary Research

Our Evidence Synthesis Initiative falls within our broader Research Synthesis program, which takes advantage of a wide variety of tools to pull together and analyze results for use by the public. The program includes PCORI activities already under way, such as our portfolio analysis and the topic nomination pathway that informs PCORI funding announcements. It will also branch out in new directions that will speed useful clinical information into stakeholders’ hands. Two of the approaches we plan to use are updating systematic reviews of the results of already-completed CER studies and reanalysis of data from such studies. These activities allow us to release products relatively rapidly within a set of clearly specified, rigorous, reproducible, and transparent methods.

Systematic reviews. The most common type of evidence synthesis is the systematic review. It provides answers to specific clinical questions by analyzing published and unpublished results from all relevant studies on a given topic. Systematic reviews identify, select, and summarize findings of available research to make clear what is known about a topic—and what is still not known. PCORI already has methodology standards for such reviews.

Systematic reviews often use statistical methods to combine published or unpublished study findings to produce estimates of an intervention’s effect and to illustrate how consistent and strong the effect is across the research. The most common approach is to use aggregate data meta-analyses, which typically summarizes the average treatment effect for each study population across all studies in the systematic review. One PCORI-funded study uses a variant of this method to compare treatments for kidney disease in patients with lupus (see box).

Based on input we have received from stakeholders, our initial efforts will include updated systematic reviews comparing treatment options for

• Atrial fibrillation
• Post-traumatic stress disorder
• Rheumatoid arthritis
• Urinary incontinence

We’ll implement this initial effort in coordination with the Agency for Healthcare Research and Quality.

Individual patient–level data (IPD) meta-analysis. Today, most research studies, including systematic reviews, provide the average result for the average individual—someone who may not actually exist. But by combining the granular specifics about each individual participant across several studies on the same topic, we have an opportunity to see how treatments may have different effects (i.e., greater benefit or greater harm) for individuals with different characteristics.

An IPD meta-analysis obtains and examines all of the relevant measured characteristics of each participant from all of the included studies in a systematic review. It can indicate whether treatments should be targeted for use in specific groups of individuals. It is also an important method to reconcile differences when studies examining the same treatments have conflicting results.

Other research data reuse opportunities. Besides IPD meta-analyses, a statistical approach called predictive analytics can be applied to previously conducted trials or other research data to provide more personalized results that can optimize the use of specific treatments. This approach can be helpful in studies with a diverse group of people and a wide range of individual responses to an intervention. An example of this approach is PCORI’s recently funded study on who benefits from diabetes prevention efforts (see box).

Next Steps

As our primary research results become available, our Dissemination and Implementation program will be working diligently to place individual study results into a context to best inform users. At the same time, our evidence syntheses will be important products to get into the public domain. Thus, the next question we face is: How would stakeholders prefer to receive this information?

To get a better focus on what would work best for a variety of healthcare decision makers, our Dissemination and Implementation program recently convened a stakeholder workshop. Participants included patients, clinicians, insurers, and employers. The participants expressed a strong need for brief and clear presentations of what evidence can be brought to bear on specific healthcare decisions, considering differing contexts and needs. They emphasized the importance of actionable information and tailoring findings to audiences. Finally, PCORI products should help patients and clinicians share in making health decisions.

All of us at PCORI are excited about evidence synthesis as an additional avenue for rapidly bringing useful evidence to patients, their families, clinicians, and other healthcare stakeholders. We look forward to sharing more details as our plans mature.

Editor's note: At the time that this blog post was published, Evelyn P. Whitlock, MD, MPH was PCORI's Chief Science Officer. She served in that role from September 2015 to June 2018.