The impact of comparative effectiveness research: stakeholders’ views on what will improve healthcare decision-making

In a recent article [1], we described the results from 5 years of surveys that examine the effects that a new emphasis on comparative effectiveness research (CER) is having on healthcare decision-making in the USA. The surveys sought opinions from six stakeholder groups who are knowledgeable about CER: researchers, health plans, employers, business coalitions/human resources specialists and trade/advocacy groups.

The results from the survey's sixth year are now available [2], and it is even clearer that stakeholders are unwavering in the belief that CER is important, but its impact is still on the horizon. It is perhaps not surprising that in a span of 6 years, change in something as broadly significant as healthcare decision-making is gradual, showing only recognition of the need for change, the players likely to be influential and some of the elements that are evolving.

The most recent survey added questions to seek stakeholders’ perspectives on the role that patient groups will play in the CER enterprise. When the survey was initiated in 2011, the Patient-Centered Outcomes Research Institute (PCORI) was just being established, so patients were not yet actively engaged in PCORI or the CER process. Today, that has changed.

Stakeholders anticipate a key role for patient groups in establishing priorities for research. Half of the survey respondents believe that patient groups will play a significant role in establishing research priorities, along with PCORI, the Agency for Healthcare Research and Quality (AHRQ), and the NIH.

Stakeholders also were asked about key participants for four other activities related to research: establishing research standards, funding and monitoring research, conducting research and translating/disseminating research. As in previous years, PCORI and AHRQ are expected to play leadership roles in establishing standards; NIH, PCORI and the biopharmaceutical industry are anticipated to be the key players in funding and monitoring research; stakeholders expect academia and the biopharmaceutical industry to be leaders in the conduct of research; and AHRQ, academia and PCORI are anticipated to take the lead in the translation and dissemination of research.

Similar to previous surveys, the 2016 results show that most respondents foresee a moderate or substantial improvement in healthcare decision-making due to CER in 3 years (78%) and even more (92%) in 5 years. A third of respondents expect a moderate to substantial improvement in 1 year.

Because the effects of CER are not expected to be realized for a few years, we asked respondents about what changes they believe will increase the impact that CER will have on healthcare decision-making. As shown in Figure 1, respondents focused on translation and dissemination of CER results, relevance of CER questions and the infrastructure for using results.

The highest percentage of respondents, 59%, believe that better translation of CER results into actionable recommendations is needed to increase CER's impact. In total, 43% of respondents feel that improving the infrastructure and resources for decision-makers to use CER results will increase the impact of CER. While there are concerns about the quality of data as well as the methods and inputs to the research process, respondents believe translation, dissemination and application of CER are the areas most in need of improvement for enhancing the impact of CER on healthcare decision-making.

Several questions in the survey address specific issues about the healthcare decision-making environment that could be expected to change or be affected by the increased focus on CER. These issues include: the availability of accepted research standards, how adequately research priorities address treatment choices faced by patients and providers, use of objective and transparent processes for interpretation of evidence, consideration of quality, outcomes and value in treatment assessments, the completeness of the comparative effectiveness evidence base and use of real-world evidence. The first four issues have been tracked since the original survey in 2011; the latter two issues were added to the 2014 survey. Results for this year are as follows [2]:

The findings from the 2016 survey echo and reinforce the conclusions drawn from the first 5 years of the survey. Change is slow and gradual in any endeavor which is as large and significant as healthcare decision-making, and the as yet unrealized potential of CER to have a significant impact depends critically on an intensified focus on translation, dissemination and real-world application of research results.

The complete results and survey instrument are available at [2] and an infographic summarizing the results (Supplementary Figure 1).