August 4, 2017
Richard J. Willke
Chief Science Officer
International Society for Pharmacoeconomics and Outcomes Research
505 Lawrence Square Blvd. South
Lawrenceville, NJ 08648
RE: Comments on Special Task Force Initiative on US Value Assessment Frameworks Draft Report
Submitted electronically via VAF@ISPOR.org
Dear Dr. Willke:
The National Pharmaceutical Council (NPC) shares ISPOR’s interest in informing the shift toward a value-driven health care system and appreciates this opportunity to comment on the Special Task Force’s (STF) draft report, “A Health Economics Approach to US Value Assessment Frameworks.”
NPC is a health policy research organization dedicated to the advancement of good evidence and science, and to fostering an environment in the United States that supports medical innovation. NPC is supported by the major US research-based biopharmaceutical companies. We focus on research development, information dissemination, education and communication of the critical issues of evidence, innovation and the value of medicines for patients. Our research helps inform critical health care policy debates and supports the achievement of the best patient outcomes in the most efficient way possible.
NPC supports the key objectives of the Initiative on US Value Assessment Frameworks:
- identify and discuss key methodological and process issues in defining and applying value frameworks to health care resource allocation;
- convene a Special Task Force to collaborate on a white paper that reviews relevant perspectives, approaches, and methods to support the definition and use of high-quality value frameworks; and
- engage key stakeholders throughout the development of the white paper to help to frame the scope of work for this methodologically oriented white paper and to review and comment on the work progress and products of the Special Task Force (STF).
We also commend ISPOR for reaching out to its members for comment on the draft report. ISPOR’s membership is diverse and the knowledge this membership possesses is extensive. In its present form, however, the draft report is reflective of only the health economist perspective; incorporating a variety of perspectives representing ISPOR’s diverse and knowledgeable members into the report will enhance it greatly.
We note the phrase used to describe the draft report in the third bullet, “methodologically oriented white paper”. The draft report is a thorough methodological exploration of many of the facets of value assessment, and this methodological focus is squarely in ISPOR’s wheelhouse. There are sections where the report moves beyond this methodologic focus and makes policy recommendations. Delving into policy is beyond the stated intent of the paper, and is particularly ill-advised given the narrow health economist perspective of the STF, which does not represent the diversity of ISPOR’s membership.
We do believe the methodologic insights in the draft report are valuable. As stated in NPC’s Guiding Practices for Patient-Centered Value Assessment (Guiding Practices), value assessments can be an important tool for the complex decisions organizations and patients face when considering treatment options. We believe assessments that adhere to the Guiding Practices can support optimal value for patients. Several key areas of comparison between the Guiding Practices and the draft report’s recommendations are detailed below; suggested changes for your consideration are italicized.
- Value assessments should focus broadly on all aspects of the health care system, not just on medications (NPC Guiding Practice VII).
The Guiding Practices emphasize the importance of assessing value for all components of the health care system. We commend the STF for using language in the draft report that reinforces this broad focus, for example: “Notably, the purview in this report is broader than pharmaceuticals alone, and encompasses devices, diagnostics, procedures, and health programs.” (Section 1, lines 59-60).
- The measurement of value should include a broad array of benefits that are important to patients and society (NPC Guiding Practice XIII).
The importance of including a breadth of benefits that matter to patients and society is underscored in both the Guiding Practices, and in Section 3 of the draft report, “Defining Elements of Value in Health Care.” Section 3 presents a thorough exploration of the many dimensions of value.
We applaud the STF for highlighting these elements, which is an important step towards comprehensively characterizing value. We urge ISPOR to build on this step by seeking to develop ways to quantify and incorporate these elements into assessments of value. This is an area of great methodologic need, and an optimal fit with ISPOR’s strategic objectives and goals in the area of scientific and research excellence.
The recommendations in Section 7, “Summary: Key Points and Recommendations,” are not fully consistent with the inclusion of a broad array of dimensions of values, and we recommend rewording Recommendations 3.1 and 3.2 for consistency with this concept.
- ISPOR Recommendation 1.1: Begin with and build upon cost-effectiveness analysis
The framing for this recommendation makes clear that cost-effectiveness analysis (CEA) is only a starting point for assessment and will not fully capture all of the relevant benefits, such as impact on productivity or caregiver burden. Building upon a CEA is necessary to fully capture all relevant benefits and dimensions of value.
- ISPOR Recommendation 3.1: Apply cost-effectiveness analysis to inform public and private coverage and reimbursement decision making
This policy recommendation calls for CEA to be the core component informing coverage and reimbursement decisions. This is at odds with Recommendation 1.1’s identification of CEA as a starting point for assessment that should be built upon to incorporate the additional benefits that cannot be captured by a CEA, and seems inconsistent with the overall draft report. We recommend revising this particular recommendation to better reflect the spirit of Recommendation 1.1 and to eliminate the policy focus.
- ISPOR Recommendation 3.2: Support additional research that enhances the applicability, scope, and relevance of CEA for decision making
We strongly agree with the importance of supporting this additional research, and note that many of ISPOR’s members already have been conducting such research and developing useful value measures (e.g., patient reported outcomes) that can expand the types of benefits that a CEA could capture. This recommendation would benefit from clarification that the call for research is not intended to imply that these additional measures should be dismissed for the time being and simply relegated to future research. Where these measures already exist, every effort should be made to incorporate them and capture as broad an array of benefits as possible.
- Thresholds should be developed in a transparent manner, may vary by population and disease, and should undergo a multi-stakeholder evaluation process (NPC Guiding Practice XIX).
While a simple value threshold for decision-making appears to be an elegant solution to a health economist, the quality-adjusted life year (QALY) has considerable shortcomings and may not be appropriate for decision-making in a diverse health care system like the US. Section 3 of the draft report effectively describes some of the issues with the QALY: “QALYs capture only a subset of benefits produced by a health care intervention. This simple framework neglects numerous alternative aspects of benefits that should also be considered” (Section 3, lines 126-128). The cost/QALY metric is not meaningful in all decision-making contexts, for example, during patient-provider interactions where both parties are unlikely to have sufficient understanding of this metric and how it was calculated.
- ISPOR Recommendation 4.1: Utilize value thresholds – expressed as in the cost-per-QALY metric potentially considering other elements – to help guide coverage and reimbursement decisions
Given the shortcomings and limitations of the QALY, we suggest that you reconsider this strong endorsement of the cost-per-QALY decision rule. For example, since the report does not contemplate an evaluation of the quality of individual cost/QALY approaches, such an endorsement leaves open the possibility that a low-quality cost/QALY approach would be preferred over a non-cost/QALY approach, even if the latter is of superior quality and/or is more appropriate to a given situation.
- Best available evidence should be used for the assessment (NPC Guiding Practice XXII). Accepted methods should be used to assess the quality of evidence, certainty of evidence and conflicting evidence (NPC Guiding Practice XXIII).
An important part of a value assessment is the selection and evaluation of relevant evidence for the evaluation. ISPOR and its members have a wealth of knowledge on this topic, yet it is not reflected in the draft report. We believe that the draft report would be strengthened by the inclusion of recommendations related to evidence selection and evaluation, including the use of real world evidence.
- Value assessment should clearly state the intended use and audience to avoid misuse (NPC Guiding Practice XXVII).
Different value assessments have different audiences, and it is critical to clarify the intended audience and purpose so as to avoid misuse of an assessment. In particular, assessments designed for payer decision-making are not appropriate for shared decision-making between patients and their doctors, and vice versa. We commend Recommendation 2.1 for underscoring this point, but are concerned about potential conflation between payer decision-making and patient decision-making (e.g., shared decision making between patients and providers) in several places in the draft report.
- ISPOR Recommendation 2.1: Clarify importance of perspective and decision context
Recommendation 2.1 underscores that the perspective, specific decision context, and specific meaning of value in that context must be made clear in the development and use of value assessment frameworks, which we fully support.
The narrative of the report emphasizes that “the primary focus of our recommendations is on payers” (Section 7, lines 6-7). However, the report states in many places that payers act as agents on behalf of their enrollees (e.g., Section 7, lines 44-45), which could be viewed as suggesting that the payer perspective also represents the patient perspective.
For example, Section 7, lines 8-9 state that payer use of CEA in decision-making “best serve(s) the interest of the plan members and patients who they represent.” Payer decision-making is generally based on a payer perspective, which is different than a patient perspective or a societal perspective. Decision-making that best serves the interest of patients is based on a patient’s perspective, not a payer’s. When a payer makes decisions based on a CEA that uses a payer perspective, they are making decisions that best serves the payer’s own interests. We strongly recommend clarification of all language that conflates the two perspectives.
The patient perspective is also mishandled in point 2 of Recommendation 6.1: “Well designed patient-level frameworks can help to guide individual treatment choices among clinically appropriate options that have been approved for reimbursement…” (emphasis added). This language suggests that patient framework decisions should be confined by the decisions made by a payer’s framework. Patient-focused frameworks should stand alone; we recommend this language be revised so as to not suggest the patient framework is only relevant within the context of a payer framework.
- Assessments of ways to address budget impact concerns should include all relevant stakeholders and consider all approaches (NPC BIA Guiding Practice VII).
NPC’s Guiding Practices emphasize that all interested stakeholders should be involved in conversations about ways to address affordability concerns. The recommendation in Section 7 bypasses these conversations and focuses on a limited set of approaches that do not take all stakeholders’ interests into account and may limit patient access to treatments.
- ISPOR Recommendation 4.2: Manage budget constraints and affordability
The narrative accompanying this recommendation provides specific direction on approaches for addressing affordability, which have not been vetted with relevant stakeholders. Many stakeholders would be concerned about option (b), which could delay access to new technologies. We recommend removing the specific policy recommendations and emphasizing the importance of considering all approaches and engaging all stakeholders.
We appreciate this opportunity to provide input on the Special Task Force’s draft report for the Initiative on US Value Assessment Frameworks Draft Report, and look forward to continuing to engage with you on this important topic.
Robert W. Dubois, MD, PhD
Chief Science Officer
Kimberly Westrich, MA
Vice President, Health Services Research
 US Value Assessment Frameworks Initiative Documents. https://www.ispor.org/ValueAssessmentFrameworks/Docs. Accessed July 31, 2017.
 Initiative on US Value Assessment Frameworks. https://www.ispor.org/ValueAssessmentFrameworks/Index. Accessed July 31, 2017.
 Guiding Practices for Patient-Centered Value Assessment. National Pharmaceutical Council. http://www.npcnow.org/sites/default/files/npc-guiding-practices-for-patient-centered-value-assessment.pdf. Accessed July 31, 2017.
 Section 7 Summary: Key Points and Recommendations. https://www.ispor.org/USValueAssessmentFrameworks/STF-White-Paper-Section7-DRAFT-for-Member-Review.pdf. Accessed July 31, 2017.