Ongoing efforts to conduct research and develop tools to support evidence-based decision-making have taken on a renewed sense of urgency amidst the debate over rising health care costs, a debate that is often too narrowly focused on the up-front cost of treatment. Sound, well-structured tools for value assessment can help us move beyond a narrow, siloed focus to a more holistic perspective on value that is central to long-term solutions and patient needs.

Today, our organizations, the Pharmaceutical Research and Manufacturers of America and National Pharmaceutical Council jointly announced comprehensive positions in support of value assessment frameworks in health care. These principles and guiding practices are indicative of our sector’s commitment to advancing health care solutions that focus on the patient and support better value. This commitment is seen in our member companies’ focus on discovering and developing medicines that make a difference to patients and society, and support of patient-focused drug development.

The tools and solutions we develop to move health care toward a system driven by value must be grounded in sound science and centered on the needs and values of patients. Well-structured value assessment frameworks can help achieve this. Transparently developed, methodologically sound, patient-centered value assessment tools also can help patients and providers decide what treatment option is right for the patient, thereby achieving value for the health care system. Organizations such as the American Society for Clinical Oncology and the National Comprehensive Cancer Network have recognized the need for new value frameworks to support patient-physician decision-making.

However, the current debate over the development and use of value assessment tools illustrates that there is a lack of consensus around a single approach to determining the value of medicines. Methods and approaches to value assessment must continue to evolve. It is critically important to get this right, and much work remains to be done. Speaking to emerging value frameworks, one recent article in the New England Journal of Medicine stated that, “the frameworks will require refinement, however, before they’re ready to be broadly applied.” Ensuring that value frameworks are sound before using them in public policy or as the basis for decision-making is essential – value assessments that are not developed transparently, based on rigorous methods or are vulnerable to misinterpretation and misuse have the potential to hinder a patient-centric health care system and progress against unmet medical needs.

The importance of getting value assessment right was recently underscored by a proposed rule announcing a Part B Drug Payment Model released by the Centers for Medicare & Medicaid Services (CMS). The proposal would link Medicare payment policy to assessments of comparative clinical and cost effectiveness. It also references value assessment models that emphasize payer cost-control over patients’ needs and values and that have not been thoroughly vetted or validated. Rather than creating centralized standards and national one-size-fits-all value assessments, CMS should be advancing value assessment tools and regulatory reforms that help individual patients decide which treatment options are most valuable to them and accelerate market-based solutions for value-driven care.

Experiences in several European countries have shown the potential dangers of employing such centralized value assessments in public policy. Restrictions imposed by the United Kingdom’s National Institute for Health and Care Excellence (NICE) have created substantial barriers between patients and life-saving treatments—almost 80 percent of cancer medicines reviewed in the last seven years by NICE has been given some kind of access restriction. Organizations like NICE, which impose centralized value judgments, also create delays to accessing new medicines. Market authorization of new cancer drugs starts later in countries, like the United Kingdom, that use a cost-per-quality adjusted life year mechanism to restrict access and may never reach the levels seen in other countries without such mechanisms.

While there currently is great interest in novel value assessment frameworks, there also is great variability in the methods and approaches being used, as well as significant unresolved issues related to the process through which the frameworks are developed. Given this variability, no single framework can provide a universally applicable answer about a treatment’s value. As these emerging frameworks evolve, the principles and guiding practices we have developed can ensure that value assessment supports efficient, high-quality, patient-centered health care and continued innovation.

Our organizations have come together to provide complementary approaches to addressing these concerns. PhRMA’s principles provide a road map for a range of stakeholders to consider in developing or evaluating value assessment frameworks. NPC’s guiding practices provide additional technical detail for researchers and others developing or evaluating value frameworks. Both of our approaches reflect common positions and include these key concepts:
  • Describe a sound process that is open and transparent, with opportunity for input and a strong role for patients and physicians.
  • Support patient-centered care by considering patient preferences and heterogeneity, appropriately communicating results and avoiding misuse.
  • Deliver reliable, relevant information by using rigorous, transparent methods that rely on the full range of evidence and prioritize longer-term and broader outcomes.
  • Value continued scientific and medical progress by accounting for personalized medicine, the step-wise nature of progress and the inherent value of innovation.
  • Take a system-wide perspective on value by examining the full range of tests, treatments, care management approaches and health care services.

Our industry is supportive of the movement toward a patient-centered, value-driven health care system, and our participation in the conversation about value assessment will not end with the release of our principles and guiding practices. In fact, our work has just begun. As we move forward, we are firmly committed to meaningful engagement with other stakeholders, including patients, providers, payers and researchers, to support sound, evidence-based tools that aid in health care decision-making.

Steve Ubl is the President and CEO of Pharmaceutical Research and Manufacturers of America and Dan Leonard is the President of the National Pharmaceutical Council.