Guiding Practices: Looking at Value Assessment Through a Broader, Patient-Focused Lens

There are many aspects to the U.S. health care system, as well as many different stakeholder viewpoints on how to address current challenges as our system shifts from one driven by the volume of health care services to one focused on the value of health care that is provided. While it is relatively easy to measure volume, it is quite different altogether to try and measure value. As part of this shift, there is an increased interest in understanding how to measure value. Yet most of this interest is focused on only one aspect of health care costs—biopharmaceuticals—causing us to lose sight not only of how all aspects of the system works together, but of how we can ensure that patients’ views and needs are recognized.

There are many aspects to the U.S. health care system, as well as many different stakeholder viewpoints on how to address current challenges as our system shifts from one driven by the volume of health care services to one focused on the value of health care that is provided. While it is relatively easy to measure volume, it is quite different altogether to try and measure value. As part of this shift, there is an increased interest in understanding how to measure value. Yet most of this interest is focused on only one aspect of health care costs—biopharmaceuticals—causing us to lose sight not only of how all aspects of the system works together, but of how we can ensure that patients’ views and needs are recognized.

To keep patient concerns front and center, it’s time to assess the issue of value through a broader, patient-focused, lens. Given the National Pharmaceutical Council’s (NPC) role as a policy research organization with a commitment to ensuring the use of sound methodology, we developed “Guiding Practices for Patient-Centered Value Assessment” to help stakeholders comprehensively consider value with the patient in mind.

Value assessments are a new and evolving area, and they have the potential to have a tremendous impact on patient treatment decisions, as well as on coverage and reimbursement decisions. In recent years, a number of organizations, such as the Institute for Clinical and Economic Review (ICER), Sloan Kettering Memorial Cancer Center (Drug Abacus) and the American Society for Clinical Oncologists (ASCO) have developed frameworks to assess new treatments.

While we recognize the importance of evaluating treatment costs, many of these frameworks fall short in their efforts to balance this evaluation with a comprehensive consideration of benefits to the patient. By not incorporating the full value and benefits of medical innovation, frameworks will fall short of their goal to meaningfully assess value. We believe our guiding practices will provide an important perspective about measuring value and will complement other efforts currently underway within the patient community.

Our guiding practices offer a comprehensive way to take all of these concepts into account. These practices include 28 specific elements, which are broken out into six key aspects of value assessments:

  • Assessment process: Stakeholder input and feedback should be incorporated throughout the assessment process, from the announcement of topics to be examined through the entire review cycle. In addition, assessments should be regularly reviewed to keep pace with continued medical innovation.
  • Methodology: Assessments should focus on all aspects of the health care system, use established methods and transparent models and assumptions, and include guidance to help users understand key drivers behind the results.
  • Benefits: Assessments should include a broad array of factors that matter to patients and society, consider individual treatment effects and view the value of a treatment over a long-term horizon.
  • Costs: All health care costs and offsets over time should be considered, and these costs should be accurate and relevant to the user of the framework.
  • Evidence: All of the sound, high-quality evidence that is currently available should be utilized for the assessment. It should be gathered and synthesized in a transparent and robust manner, using accepted methods.
  • Dissemination and Utilization: Assessments should be clearly labeled for their intended use, easy for users to interpret, and disseminated only after they are finalized.

Our practices also take a step further by outlining how to consider budget impact assessments. Budget impact assessments are a measure of resource use, not a measure of value. These types of assessments can inform users about what they are paying, but not about what they are paying for—value. Our recommendations are based on existing principles developed by a task force of the International Society of Pharmacoeconomics and Outcomes Research. Recommendations include considering longer time frames, using realistic estimates regarding the uptake rate of a treatment, including all aspects of the health care system, not simply medications, in the review, and separating budget impact from value assessment.

These guiding practices are a complementary approach to principles outlined by PhRMA, both of which were detailed by our organizations in a commentary published in The Hill today. The principles—led by PhRMA--and our guiding practices—led by NPC—reflect our organizations’ unique roles in the biopharmaceutical industry. PhRMA’s principles are broader and more conceptual; NPC’s guiding practices are aimed at researchers and framework developers and decision-makers, outlining more specific points on how framework principles could be achieved.

Even with guiding practices and principles, one value assessment will not fit all purposes for all users. But if “best practices” are followed, perhaps we can achieve a more holistic view of what value looks like to the patient, and ensure that health decisions are guided by this holistic, patient-centered view.