The hard-hitting rhetoric of emerging electoral politics is quickly overwhelming the everyday business of government, making it easy to forget that many other important issues are continuing to unfold and develop. One of those issues is comparative effectiveness research (CER). It matters because most stakeholders on the left and right view CER as a key tool for improving health care quality. However, many of the details related to making CER a useful tool are still being worked out.
One year ago this month, 21 individuals from diverse health care backgrounds were appointed to serve on the board of governors of the Patient-Centered Outcomes Research Institute (PCORI), which was created by the Affordable Care Act. During this last year, PCORI has set its organizational infrastructure, hired an executive director, held public meetings, started stakeholder engagement, and initiated discussions to identify a set of national priorities for public comment. Throughout its process of defining patient-centered outcomes research, the Board has sought to involve a broad representation of health care stakeholders. In fact, more than 600 comments were recently received on the proposed definition. This is important because the definition will inform the types of research conducted and the priorities will set the tone for its work moving forward.
At the same time, the Board has done this with the landscape changing under its feet. Since its creation, health care stakeholders have begun to think about the type of information they will need to make decisions, and how they will use this information. And the answers to these questions are shifting from where many observers were on key questions even a year ago.
An employer stakeholder survey sponsored by the National Pharmaceutical Council (NPC) shows that they will be looking to PCORI as their main source of CER information. Employers also expect that PCORI will broaden its range of research to include "productivity at work and home" as outcomes. This last finding is of particular interest and importance and is a commonly overlooked metric in the discussion.
It’s becoming clear, too, that CER and PCORI won’t focus just on “blue pill vs. the red pill” research as some stakeholders had thought, but rather on evaluating a fuller picture of quality of life, quality of care and the potential for improved adherence to treatments. And, as NPC has supported in public comments, PCORI appears to be focusing on patient-centered outcomes research that would:
- Utilize a full range of evidence that considers both direct and indirect benefits to society, such as quality of life, patient functionality and economic productivity.
- Consider heterogeneity, or the needs of patients who may respond differently to medicines and treatments based upon age, genetic variation, co-morbidities, or patient preferences.
- Focus on clinical comparative effectiveness, not cost effectiveness.
Getting these issues right matters because PCORI must decide how this research will be translated and disseminated, particularly in a way usable by key decision makers. The Agency for Healthcare Research and Quality, which is tasked with assisting PCORI with dissemination, is already taking steps to consider how to tackle this issue. While the patient is at the heart of the decision, most of this information will be used by health care providers, and information dissemination is a highly regulated activity.
In addition, changes could also be in store for how research is developed as the PCORI methodology committee’s report is due next spring. This committee has been charged with developing standards for research to ensure that the research is of high quality. Another task is the development of a translation table, which would serve to assist the PCORI Board in determining which research methods are best suited for specific research questions. This is critical because research designed or conducted poorly leads to poor and useless results.
Another important consideration is whether -- and how -- the pharmaceutical industry will be able to respond to or assess these research results. As more public databases come online, such as patient registries or population-based information, the question arises -- will the industry be an active partner in designing research, conducting and reviewing newly accessible data? And will the industry be able to comment on any limitations or potential biases in the results? Typically, information exchange is limited by FDA regulations, so additional considerations for appropriate design and appropriate information exchange could be justified.
One year ago these issues seemed far in the future. But with real steps underway, the theory of CER is becoming the reality of CER. While perhaps not as exciting as political debates and electoral politics, we need to pay attention to the unfolding debate. The reason is simple: providers, patients and other decision makers need better information to help them make decisions to provide the best and most appropriate treatments or services that will deliver the best outcome, and in a way that provides the best value to all.