SummaryA significant push towards patient-centered value has occurred in recent years, yet a validated instrument to collect patient-centered data has not been established. The Patient Perspective Value Framework (PPVF) Initiative offers an approach for addressing this missing piece.
What is Patient-Centered Value?
Since being included as one of the six aims for improvement in the original Institute of Medicine report “Crossing the Quality Chasm” in 2001, patient-centricity has been considered a core component of high-value health systems. Defined in the report as “providing care that is respectful of and responsive to individual patient preferences, needs, and values and ensuring that patient values guide all clinical decisions,” patient centricity has recently garnered greater attention from the quality community, payers, and regulators due to recognition that advancing health equity requires changing how care is delivered to account for the unique needs of different patient populations. For pharmaceutical manufacturers, the focus on patient-centricity has manifested most clearly in the Food and Drug Administration’s (FDA) push for patient-focused drug development. For health technology assessment (HTA) bodies and value assessors, patient-centered value has emerged as a branch of patient-centricity. While there is no standard definition, patient-centered value generally incorporates the patient perspective into assessments of treatment value. Traditional HTA methods and value frameworks focus on clinical benefit from a health system perspective, using metrics such as quality-adjusted life years that have historically been criticized. Despite calls to make these evaluations patient centric, current value assessment frameworks do not systematically account for the patient perspective.
Patient-Centered Value Benefits More Than Just Patients
Stakeholders across the healthcare spectrum have different perspectives on patient-centered value, as well as different opportunities to benefit from moving to patient-centered value assessment. For providers, payers, and employers who aim to improve health outcomes and manage costs in their respective populations, understanding patients’ preferences for treatment and the ways that different therapies address those preferences allows for care plans that most effectively and efficiently meet patients’ needs. Patient-centered value can be a component of payer coverage and access decisions, such as incorporating patient-centered outcomes metrics into value-based contracts or considering patient preferences in utilization management practices. Ultimately, providers, payers, and employers want to provide high-quality, cost-effective care to their patients, members, and employees. These entities may consider incorporating the patient perspective as both a core component of quality and a tactic to improve outcomes.
Manufacturers may be considering patient-centered value in the context of the Inflation Reduction Act (IRA), particularly as it gives Centers for Medicaid and Medicare (CMS) the authority to negotiate lower drug prices for Medicare. Though guidance for patient input is pending, CMS has indicated it will consider patient input submitted during the negotiation process but that it prioritizes research with a strong study design and established, validated methodologies. To balance CMS’s preference for rigorous evidence with its emphasis on the patient voice, developing standardized instruments that capture the patient perspective on treatment value could be an important strategy for manufacturers directly and indirectly impacted by IRA Medicare negotiation.
Manufacturers preparing for or responding to value assessments by third parties such as the Institute for Clinical and Economic Review (ICER) can leverage patient-centered value to highlight important considerations missing from cost-effectiveness analysis techniques. Higher-cost treatments (e.g., treatments for rare diseases, curative therapies) may receive negative valuations because standard cost-effectiveness analysis techniques are not designed to account for patient-centered value elements. Investing in methodologies to incorporate patient-centered outcomes into evidence generation plans may support manufacturers in navigating value assessments like the ICER review process and may ultimately improve the accuracy and patient-centeredness of HTAs in the future. Measuring value from the patient’s perspective can be a key product differentiator. This was a primary objective of the Patient Perspective Value Framework, which was designed to assess the value of drugs, devices, diagnostics, and services with the patient perspective in mind and to be applied to different conditions and value frameworks.
Overview of the Patient Perspective Value Framework
In 2017, stakeholders recognized the need for a conceptual model for assessing patient-centered value that could be used to build specific value assessment tools such as shared decision-making tools, clinical decision support tools, and HTAs. In partnership with FasterCures, Avalere led an initiative to create such a conceptual model, the Patient Perspective Value Framework (PPVF). The PPVF comprises five domains: patient preferences, patient-centered outcomes, patient and family costs, usability and transparency, and quality and application of evidence. The framework provides a high-level methodology to assess the value of multiple healthcare options from the patient’s perspective (Figure 1).
History of the PPVF
The PPVF initiative began due to an acknowledgement that existing value frameworks excluded or did not adequately capture the patient perspective. From its inception, the objectives of the initiative were to:
- Design a framework that can be used for multiple applications and by multiple audiences, such as patients, clinicians, payers, and policymakers
- Make traditional value assessment processes more patient centered through collaboration and information sharing
- Serve as a call to action for researchers to generate more robust studies that consider and collect patient-centered metrics identified in the PPVF
- Directly impact decision making by patients, clinicians, payers, policymakers, and life sciences companies through purpose-built tools based on the PPVF
Led by a steering committee comprising healthcare industry leaders from the federal government, payers, and life sciences companies, the first version of the PPVF was released in May 2017.
The PPVF Initiative later published a scoring methodology to provide further introspection on patient preferences in each of the five identified domains and create a foundation for the technical advancement of PPVF components in external value assessments. To conclude its early efforts, the initiative developed consensus recommendations for the technical improvement of other value frameworks.
Broader Stakeholder Involvement in Patient-Centered Value
Since the PPVF was introduced, stakeholders have begun to incorporate the patient perspective in value assessment. In 2019, Congress updated the authorizing legislation for the Patient-Centered Outcomes Research Institute’s (PCORI) scope of work and research priorities, which led to revised guidance on how the studies PCORI funds should capture patient-centered data. This work is still in progress. PCORI recently released a draft report containing a patient-centered value “attribute inventory” for public comment.
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the professional society for health economic and outcomes research, is also active in patient-centered value. Previously, ISPOR developed the ISPOR value flower that highlighted additional elements of value that are not included in existing value assessment methods. In recent years, ISPOR regularly convenes a Patient-Centered Special Interest Group to facilitate inclusion of the patient voice in all stages of research and decision making. Additionally, it develops patient-centered research reports outlining best practices in health economics and outcomes research and publishes studies in its academic journals, including a recent roadmap for “navigating patient-preference studies for cross-stakeholder decision making” and a study describing a new cost-effectiveness framework, “Generalized Risk-Adjusted Cost-Effectiveness.”
ICER independently evaluates the cost effectiveness of treatments. In its latest value assessment framework released in 2019, the organization “seeks to encompass and reflect the experiences and values of patients.” However, its assessments vary in their incorporation of patient-centered value concepts. For example, in its amyotrophic lateral sclerosis assessment, ICER uses some patient-centered outcomes to define health states but uses the disease-agnostic EQ-5D tool to define health utilities. ICER may better incorporate patient-centered value in its upcoming value framework update (anticipated in 2023), but information is pending. Because patient-centered data is difficult to collect, ICER and other HTAs may not be fully able to incorporate the patient-perspective into value assessments today.
Despite efforts across the healthcare industry, the short- and long-term recommendations initially advanced by the PPVF Initiative have yet to be enacted. Value assessment stakeholders acknowledge that reflecting the patient perspective in value requires expanding the inventory of validated instruments for capturing and quantifying patient preferences, patient priorities, patient-centered burden of illness, and patient-centered outcomes so that researchers can generate robust patient-centric evidence.
The Future for Patient-Centered Value
The PPVF brought together a wide range of stakeholders to develop and validate a framework that adequately accounts for the patient perspective in value assessments. Since it was last convened, significant evolution has occurred in stakeholders’ understanding and prioritization of patient-centric value. However, through ongoing efforts, stakeholders have also identified gaps in the ability to quantify patient-centric value elements without creating undue burden to the system.
Since the PPVF Steering Committee was last convened, the industry recognition of the need for tools and resources to support patient-centered value has only grown. Given this current landscape and momentum, Avalere is committed to continuing the work of the PPVF initiative. A new steering committee and work groups will be forming soon. To learn more about how your organization can support and participate in this work, please connect with us.
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