As healthcare costs in the United States have continued to grow, there has been an increasing desire to move towards “value-based” care in which patients and payers would be paying for the “value” of the treatment patients receive. This idea has been gaining particular momentum as policymakers look for strategies to curtail spending on pharmaceuticals. While there is merit in paying for services and treatments that work and eliminating wasteful spending, it is important to step back and consider to whom “value” is being provided. As the ultimate beneficiary, we would advocate that the measure of “value” in a healthcare setting should focus on value to the patient, but currently, we are concerned that the trend is to look towards “value” to the payer by prioritizing reduced cost over care outcomes for patients in the form of cost-effectiveness analyses. 

​Cell and gene therapies (C&GTs) are revolutionary advancements that offer potentially life-altering therapies – and in some cases even cures – for patients with rare and severe diseases. Around the globe, various health technology assessment (HTA) and value assessment organizations are attempting to estimate the health and economic value of new C&GTs to better inform healthcare decision making. These organizations most commonly employ cost-effectiveness analysis (CEA), which seeks to determine whether the costs of a given therapy are justified by its benefits; that is, whether it is a “good buy.”

PIPC has partnered with Xcenda to quantify the extent to which ICER incorporates stakeholder input in its final assessments, particularly patients.  Upon review of submitted comments, and comparing those comments to ICER’s final value assessments, Xcenda was able to quantify that patient perspectives were half as likely to be incorporated than other stakeholders. The report published today by PIPC bolsters their argument that ICER needs to take steps to improve not only its process for engagement, but also its consideration of input received from patients. 

Value assessment in general, and the use of cost-effectiveness in particular, is receiving renewed interest as a tool for controlling health care spending. Currently, the most common method for determining incremental cost-effectiveness of healthcare interventions is based on a calculation of quality-adjusted-life-years (QALYs). While the model has a basic appeal for making population-level decisions (by reducing patient populations to single, aggregate numeric values), it also poses several significant concerns from the vantage point of patient-centeredness and efforts to preserve access to needed care for individual patients and people with disabilities.  Of particular concern to me are the implications of use of QALYs for discrimination against people with disabilities, and its conflicting goals from the goals of personalized medicine.

The health care system is moving to models that seek to pay for value. In January 2015, the U.S. Department of Health and Human Services (HHS) accelerated the movement towards value-based payments by setting a goal to shift 50 percent of Medicare fee-for-service payments to alternative payment models (APMs) by 2018.1 The Center for Medicare and Medicaid Innovation (CMMI) within the Centers for Medicare and Medicaid Services (CMS) is playing a significant role in advancing value-based or alternative payment models. In addition, many State health agencies and private payers are exploring similar changes. However, CMMI and other payers lack sound procedures and standards for patient-centeredness. Patient-centered approaches to value-based payment are needed to ensure that new payment models support value that matters to patients. To get it right, patient engagement is essential.

​Value frameworks and similar value assessment tools are playing an increasingly prominent role in health care as insurance companies, Medicare, state agencies, and other stakeholders all are working rapidly toward "value-based" payment models. But will they pay for care that patients value? The Partnership to Improve Patient Care (PIPC) was founded on principles of patient-centeredness that cannot be achieved without engaging patients and people with disabilities. For the transition to a value-based health system to achieve outcomes that matter to patients, patients must have a seat at the table in defining value through the development of the value frameworks and tools.

Today, the Partnership to Improve Patient Care (PIPC) released a new White Paper on patient-centeredness in alternative payment models (APMs).  This White Paper represents an important step in PIPC’s discussion of the application of comparative effectiveness research, and should serve as an important tool for policymakers in considering the future of value-based payment models

The issue of how to set priorities for comparative research and health technology assessment has long been recognized as an important one. The statute creating the Patient-Centered Outcomes Research Institute (PCORI) also recognized this, describing specific factors and procedural considerations for defining national priorities for research. The question of how best to define research priorities, and whether to do so by proactively identifying specific topics or issuing broader solicitations for investigator-initiated prioritization, has been an area of ongoing debate.

In light of PCORI’s mandate to conduct comparative clinical effectiveness research, CER communication tools and materials should present patients with sound, clinical evidence to help inform their individual decision-making process. Based on the focus on patient care management found in PCORI’s research agenda, is it clear that the communication of the results of that research will have a direct impact on individual patient decision-making. Therefore, as PCORI begins to play a more active role in CER communication, PIPC believes these proposed best practices represent elements for all CER organizations to consider when communicating CER results to physicians and patients. While this document reflects extensive research and engagement with the patient and provider communities, we also consider it to be a living document that will continue to evolve based on feedback we receive.