Patients should have a role in deciding the value of medicines

Policymakers increasingly consult value assessment models to help price new medical interventions. Value models use prespecified approaches and selected health outcomes to match the price of an intervention to its expected benefits. Who chooses the approach and outcomes, however, is at the center of a debate about the value of pharmaceutical, biotech, and medical device-based interventions.

Patients, their family members, and the general public have historically been excluded from contributing to value assessment models. We believe that their voices and views should be essential elements in measuring value.

Their exclusion hasn’t been an oversight — it is by design. Setting aside the views of patients — who have the most to gain or lose from health care interventions — is standard operating procedure in pharmacoeconomic and policy research. Advocates of that approach say that summary, societal-level preferences are the only way to generate apples-to-apples comparisons of the benefits and costs of every health care option. We contend that obscuring patient perspectives hurts the long-term prospects of these methods and that limited resources will be allocated to interventions for a precisely but thinly conceived notion of “the public.”

Advocates of including a patient-centered approach say that is the only way to estimate what matters most to the individuals who would be directly affected by the new treatment. Rising civic movements of disability activists and others have adopted the phrase “nothing about us without us” that invokes widely held beliefs about fairness. Movements carrying this message have put research orthodoxy on notice, and some have begun participating in valuation and pricing debates.

Advocates of both patient-centered and societal-level approaches understand that a debate about these approaches needs to happen. But they are currently talking past one another and the polarization will only intensify if advocates on both sides become further entrenched. The core disagreement can be resolved, but it will require many actors — drug and device makers, insurers, researchers, policymakers, patients, and members of the public — to forge a common path forward.

Our experience with patient and stakeholder engagement tells us that methods for including patient voices in research are far less complicated or demanding than some researchers believe they will be. We recommend that advocates of the two approaches prepare for a new public conversation about health care valuation by:

• reinforcing that this conversation is needed to advance valuation methods
• recognizing that all stakeholders in this debate, including affected patient communities, can participate in a public conversation
• identifying what various stakeholders need to know about health care valuation in order to be productive contributors to the conversation
• setting clear aims for the conversation that describe both the desired effects on stakeholders and the desired effects on valuation methods

Once this groundwork has been laid, a national discussion should begin. At a minimum, valuation of a medicine should account for health outcomes that are important to patients. In addition, new deliberative procedures can be adopted to accommodate views of a wide range of decision-makers. For instance, multicriteria decision analysis offers an opportunity to combine differing views in challenging valuation work where value is defined by many factors.

Conversations about the value of medicine should be informed by an examination of the core outcomes used in value assessment. For instance, the COMET Initiative, a catalog of core outcome sets including some that are useful to valuation work, could be assessed for meaningful patient involvement in identification, development, implementation and evaluation of measures.

Once they are prepared for a national discussion, researchers, patients, family members, and others should engage in discussions that are based on well-established principles of stakeholder engagement.

The U.S. research enterprise has witnessed a dramatic growth in stakeholder engagement in recent years. With growing consensus that health care research can be conducted with support and engagement from multiple stakeholders, including patients and the public, we can forge a new and more productive path on even the most contentious topics in health care valuation.

Thomas Concannon is a senior policy researcher at the nonprofit, nonpartisan RAND Corp., assistant professor of medicine at Tufts University School of Medicine in Boston, and director of stakeholder and community engagement at the Tufts Clinical and Translational Science Institute. Lori Frank is a senior behavioral scientist with the RAND Corp. and president of the International Society for Quality of Life Research. She founded and served as program director of the evaluation and analysis program at the Patient-Centered Outcomes Research Institute, and currently serves on the Personalized Medicine Coalition board and the Alzheimer’s Foundation of America’s medical, scientific, and memory screening advisory board. RAND received a contract award from PhRMA Foundation to pursue work related to patient engagement in health care valuation. The opinions expressed here are those of the authors and do not represent the views of RAND or findings of research supported by the PhRMA Foundation.