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(The RAND Blog)

April 25, 2016

What Do Stakeholders Say About Comparative-Effectiveness Research?

Photo by sturti/iStock

by Thomas W. Concannon, Virginia Kotzias, Dmitry Khodyakov, Gavin Fahey, Jennifer Graff

For decades, medical research has focused on making sure that new drugs and medical devices are safe and effective. This research has helped people to live longer and healthier lives, even in the face of chronic or life-threatening conditions. Successful research over many years means that patients, clinicians, and other stakeholders can now choose among several treatment options for most medical conditions. The Patient-Centered Outcomes Research Institute (PCORI) is a research agency created by the Affordable Care Act to provide information that can help patients and clinicians make better health care decisions. To meet this goal, PCORI sponsors patient-centered comparative-effectiveness research—or research that compares different treatments—as a resource.

The success of comparative-effectiveness research depends in part on the active involvement of people and organizations the research is meant to inform. Insurers, employers, and industry are some of the stakeholder groups that are critical to the success of comparative-effectiveness research. Last summer, researchers from the RAND Corporation and the National Pharmaceutical Council set out to learn what these stakeholders say about such research and PCORI's work to date. Results show that some stakeholders are interested in participating in comparative effectiveness research but that current research models may not provide stakeholders with the information they need to support their involvement. Their specific recommendations about how to make comparative-effectiveness research better include these points:

Design and Conduct Research That Answers Stakeholder Questions

Employers, insurers, and industry stakeholders would like to know that their decisions are informed by high-quality evidence. To make the connection between evidence and decisions, researchers should ask stakeholders about the questions they need answered. If researchers fully understand and can articulate these questions, they are more likely to develop research that will be useful. By tailoring research to the stakeholder's information needs, researchers are more likely to see stakeholders participate in and use research results.

Address Misunderstandings About Use of Cost Information in the Research

Many stakeholders we spoke with said that cost information is critical to the decisions they make about health and health care. They were also concerned that PCORI does not include cost information in its studies. PCORI's actual stance is that cost information can be included but cost-effectiveness analyses cannot. To involve insurers, employers, and industry stakeholders successfully in its work, PCORI must clear up this misunderstanding.

Use Existing Information Streams to Reach the Target Audience

Employers, insurers, and industry representatives make health-related decisions every day using information provided by insurance brokers and professional or trade organizations. Researchers could effectively share information with employers, insurers, and industry by partnering with their trusted information sources.

Describe How Research Can Help Stakeholders Make Better Decisions

Successfully engaging with stakeholders also means emphasizing both the research usefulness and the specific types of products that will be available to them. Stakeholders stressed that research results need to be actionable, have “near-term applicability,” and demonstrate an appreciable impact on employee health, client services, patient care, or service delivery efficiency. In other words, they want results that translate into real-world actions.


Thomas W. Concannon is a policy researcher, Virginia Kotzias is a project associate, Dmitry Khodyakov is a sociologist, and Gavin Fahey is a research assistant at the nonprofit, nonpartisan RAND Corporation. Jennifer Graff is vice president of comparative-effectiveness research at the National Pharmaceutical Council. The study described above was sponsored by the Patient-Centered Outcomes Research Institute.

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