ISPOR’s Initiative on US Value Assessment Frameworks: A Missed Opportunity for ISPOR and Patients
2018; Elsevier BV; Volume: 21; Issue: 2 Linguagem: Inglês
10.1016/j.jval.2017.12.002
ISSN1524-4733
Autores Tópico(s)Healthcare cost, quality, practices
ResumoRecently, various stakeholders have sought to advance how health care value is defined and evaluated, primarily through the lens of value frameworks. The National Health Council has sought full representation of the patient voice in this value discourse. It has taken the patient community little time to come up to speed on the proliferation of US value frameworks, becoming educated on and ensuring active participation in value assessment reports [1Perfetto EM, Oehrlein EM, Boutin M, et al. Value to whom? The patient voice in the value discussion. Available from: 〈http://dx.doi.org/10.1016/j.jval.2016.11.014〉. [Accessed October 24, 2017].Google Scholar, 2National Psoriasis Foundation. ICER Q&A with Leah Howard. October 2016. Available from: https://www.psoriasis.org/advance/icer. [Accessed October 24, 2017].Google Scholar]. We appreciate the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Special Task Force’s (STF’s) acknowledgment that patient centricity is key to value assessment. We also welcome a dialogue on sound, transparent approaches to value assessment that engage the patient community and allow for innovations in methods to advance the field forward—as strongly emphasized in the report. The STF is thorough in its critical assessment of current methods and offers insights on new methods development. However, we view the final recommendations as a missed opportunity for this field and, as a result, a missed opportunity for the patient community. Primarily, our concerns center on STF’s emphasis on cost-effective analysis (CEA), with a quality-adjusted-life-year (QALY) metric, as “the core,” overshadowing other key components comprising a patient-centered value framework. It is important to note that the STF acknowledges CEA’s and QALY’s significant limitations. But, it does not acknowledge the significant, short-term implications of a CEA focus for patients, and proposes no action plan for decision making while methods evolve or for how methods development will take place to achieve patient-centered value assessment. The lack of a link to current decision making makes the recommendations especially concerning. The STF describes this evolving field and suggests promising new methods under development. Yet, its recommendations fall back on a staid and unilateral approach, one that has clearly been rejected by a number of stakeholders both in and outside the United States [3Patient Protection and Affordable Care Act, 42 U.S.C. § 18001 (2010).Google Scholar, 4EUnetHTA. HTA Core Model®. Retrieved from http://www.eunethta.eu/hta-core-model. [Accessed November 16, 2017].Google Scholar, 5Pezalla, Edmund. Presentation at Health Policy Development Using Outcomes Research Issues Session on ISPOR’s Special Task Force Report on Us Value Assessment Frameworks: What Does It Say and Is It Helpful? (Invited Issue Panel). ISPOR 22nd Annual International Meeting (May 22, 2017).Google Scholar, 6Kaplan R. Value judgment in the Oregon Medicaid experiment.Med Care. 1994; (Available from: http://journals.lww.com/lww-medicalcare/abstract/1994/10000/value_judgment_in_the_oregon_medicaid_experiment.1.aspx. [Accessed October 24, 2017])Crossref Scopus (56) Google Scholar]. The STF appears to assume that value frameworks are interchangeable with a QALY-based CEA metric. On this basis, the report goes as far as explicitly recommending that health plan coverage and reimbursement decisions be based on CEA [[7]Garrison L.P. Pauly M.V. Willke R.J. Neumann P.J. An overview of value, perspective, and decision context—a health economics approach: an ISPOR Special Task Force report [2].Value Health. 2018; 21: 124-130Abstract Full Text Full Text PDF PubMed Scopus (110) Google Scholar]. The myriad limitations noted with respect to CEA and QALY should give pause to anyone considering CEA as the predominant driver of a decision—and is especially concerning to patients. Patients do not think in terms of units of utility. They are concerned with things such as function, quality of life, family impact, and social burden. CEA as the core or primary component of a value framework is fundamentally not a patient-centered approach to value, and reflects very narrow thinking about what a value framework is and is supposed to do [[8]Goetghebeur M.M. Wagner M. Khoury H. et al.Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal.Med Decis Making. 2012; 32: 376-388Crossref PubMed Scopus (122) Google Scholar]. Any approach to defining value cannot be overly rigid or allow a single metric to dominate what should be a multifaceted approach [[9]Thokala P. Devlin N. Marsh K. et al.Multiple criteria decision analysis for health care decision making—an introduction: report 1 of the ISPOR MCDA Emerging Good Practices Task Force.Value Health. 2016; 19: 1-13Abstract Full Text Full Text PDF PubMed Scopus (377) Google Scholar]. Although the report describes other domains to be considered, they are not reflected in final recommendations. It seems that other domains are to be considered, but in the context of building better CEA and a better QALY, rather than in the context of building better value frameworks. For example, the “Value, Perspective, and Decision Context: An Overview” Section in the article by Garrison et al. [[7]Garrison L.P. Pauly M.V. Willke R.J. Neumann P.J. An overview of value, perspective, and decision context—a health economics approach: an ISPOR Special Task Force report [2].Value Health. 2018; 21: 124-130Abstract Full Text Full Text PDF PubMed Scopus (110) Google Scholar] describes what patients might consider in their decisions (e.g., out-of-pocket costs), individualizing value expectations. It also describes current barriers to achieving such a model, likely the methodological barriers framework developers [10American Society of Clinical Oncology. ASCO Value Framework. Available from: https://www.asco.org/advocacy-policy/asco-in-action/asco-value-framework-update. [Accessed October 24, 2017].Google Scholar, 11National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) with NCCN Evidence Blocks. Available from: http://www.nccn.org/evidenceblocks/. [Accessed October 24, 2017].Google Scholar] are struggling to overcome—presenting an opportunity for ISPOR. But the report does not address how such barriers could be overcome and does not elaborate upon these domains as part of a framework of value considerations. It discusses them, but then reverts to CEA, the backbone of STF’s recommendations. Patients view value broadly, as reflected in recently updated value frameworks that use this broader lens. Most are multifaceted; some include or exclude CEA [10American Society of Clinical Oncology. ASCO Value Framework. Available from: https://www.asco.org/advocacy-policy/asco-in-action/asco-value-framework-update. [Accessed October 24, 2017].Google Scholar, 11National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) with NCCN Evidence Blocks. Available from: http://www.nccn.org/evidenceblocks/. [Accessed October 24, 2017].Google Scholar, 12Institute for Clinical and Economic Review. Value assessment project. Available from: http://www.icer-review.org/impact-and outcomes/value-assessment-project/. [Accessed October 24, 2017].Google Scholar, 13Memorial Sloan Kettering Cancer Center. Welcome to Drug Abacus. Available from: http://www.drugabacus.org/drug-abacus-tool/. [Accessed October 24, 2017].Google Scholar, 14Innovation and Value Initiative. Open-source value project: transforming the process of value assessment. Available from: http://www.thevalueinitiative.org/open-source-value-project/. [Accessed November 15, 2017].Google Scholar]. For example, the FasterCures Patient Perspective Value Framework does not currently include CEA [[15]FasterCures – A Center of the Milken Institute. Patient perspective value framework. Available from: http://www.fastercures.org/programs/patients-count/patient-perspective-value-framework/. [Accessed October 24, 2017].Google Scholar]. If added in the future, CEA would be but one component. The Innovation and Value Initiative states that its model is not a value framework, but a model to be used for CEA or multicriteria decision analysis within any value framework [[1]Perfetto EM, Oehrlein EM, Boutin M, et al. Value to whom? The patient voice in the value discussion. Available from: 〈http://dx.doi.org/10.1016/j.jval.2016.11.014〉. [Accessed October 24, 2017].Google Scholar]. Similarly, EUnetHTA’s Health Technology Assessment Core Model includes costs and economic evaluation as one of nine elements [[4]EUnetHTA. HTA Core Model®. Retrieved from http://www.eunethta.eu/hta-core-model. [Accessed November 16, 2017].Google Scholar]. Last, the Institute for Clinical and Economic Review framework is often criticized by opponents for heavily weighting CEA as its core and lacking quantitative attention to contextual considerations [[16]National Pharmaceutical Council. NPC evaluate ICER’s revised value assessment framework. Available from: http://www.npcnow.org/blog/npc-evaluates-icers-revised-value-assessment-framework. [Accessed October 24, 2017].Google Scholar]. Still, CEA is one component to the Institute for Clinical and Economic Review framework. Admittedly, value frameworks are works in progress and their approaches have been grounds for debate. Most now try to consider real-world factors important to patients. One can create a perfect, rigidly designed clinical trial; however, the output has limited utility without real-world context. Similarly, one can create a perfect, rigidly designed CEA. Again, the output will have limited utility without consideration of real-world context. Just as thinking has advanced in use of the real-world evidence to complement traditional trial data, we must contemporize thinking about other domains complementing CEA, and how to include them in an overall framework. The STF has called for value to be anchored to an admittedly flawed metric with limited real-world pertinence, one that ignores the social and political environment in which health care decisions are made. In the United States, we have just witnessed a tug-of-war regarding repeal and replacement of the Affordable Care Act. During the tumultuous discourse, Senator Bill Cassidy (R-Louisiana) stated that any repeal and replacement bill must “pass the Jimmy Kimmel test,” meaning it must ensure a newborn with congenital heart disease gets needed care and future insurance coverage despite a preexisting condition. This reflects the real-world implications of domains beyond CEA. It is impossible to ignore social and political realities, because they reflect the values of our country. Like the element of equity, they cannot be overlooked in the short-term. How do we incorporate them in a workable value framework now, rather than wait for methods to incorporate them into better CEA in the future? Herein lies the missed opportunity for ISPOR, to lead in both harmonizing and contemporizing thinking about assessing value, comprehensively capturing patient views and needs, while encouraging innovation in methods to do so. The STF has missed a critical opportunity to lead the field forward by establishing standards for what value frameworks are, providing clear definitions and domains to be considered, and guiding incorporation of all domains, rather than setting them aside until methods are developed. This missed opportunity to advance thinking is important to patients because it is those domains outside of CEA that represent their decision-making considerations. In the last 10 years we have witnessed a seismic change, that of patient centeredness in health care research, policy, product development, and care delivery. Even the Food and Drug Administration, typically reserved when assessing new approaches and change to its processes, is embracing patient-focused drug development [17U.S. Food & Drug Administration. Public workshop on patient-focused drug development: guidance 1 – collecting comprehensive and representative input. Available from: https://www.fda.gov/drugs/newsevents/ucm574725.htm. [Accessed October 24, 2017].Google Scholar, 18U.S. Food & Drug Administration. Patient-focused drug development: disease area meetings planned for fiscal years 2013-2017. Available from: https://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm. [Accessed October 24, 2017].Google Scholar]. The patient centricity movement is here to stay. But, we have a long way to go to get it right. We are still learning, developing, and testing methods for best capturing and incorporating patients’ views, needs, goals, and real-world experiences to inform all we do in our health care ecosystem. Assessing health care value is no different and patient centricity must be front and center. We need strong leadership to provide direction and guidance on frameworks and methods that transcend a narrow, QALY-centric approach, taking us to a sound, transparent, patient-centric approach. The STF has chosen not to lead this movement. Unfortunately, the result is a missed opportunity for the field and for patients whose access depends on it.
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