How do the Institute for Clinical and Economic Review's Assessments of Comparative Effectiveness Compare With the German Federal Joint Committee's Assessments of Added Benefit? A Qualitative Study.

OBJECTIVES: We compared the Institute for Clinical and Economic Review's (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee's (G-BA) added benefit ratings, and explored what factors may explain the disagreement between the 2 organizations. METHODS: We included drugs if they were assessed by ICER under its 2020 to 2023 Value Assessment Framework and had a corresponding assessment by G-BA as of January 2024 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER's proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA's certainty ratings. We also determined whether each pair was based on similar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement. RESULTS: Out of 15 drug assessment pairs matched on indication, patient subgroup, and comparator, none showed agreement in their assessments when based on similar evidence. Disagreement was attributed to differences in evidence evaluation, including evaluations of safety, generalizability, and study design, as well as G-BA's rejection of the available evidence in 4 cases as unsuitable. CONCLUSIONS: The findings demonstrate that even under conditions where populations and comparators are identical and the evidence base is consistent, different assessors may arrive at divergent conclusions about comparative effectiveness, thus underscoring the presence of value judgments within assessments of clinical effectiveness. To support initiatives that seek to facilitate the exchange of value assessments between countries, these value judgments should always be transparently presented and justified in assessment summaries.

An empirical ethics study of the coherence of NICE technology appraisal policy and its implications for moral justification.

BACKGROUND: As the UK's main healthcare priority-setter, the National Institute for Health and Care Excellence (NICE) has good reason to want to demonstrate that its decisions are morally justified. In doing so, it has tended to rely on the moral plausibility of its principle of cost-effectiveness and the assertion that it has adopted a fair procedure. But neither approach provides wholly satisfactory grounds for morally defending NICE's decisions. In this study we adopt a complementary approach, based on the proposition that a priority-setter's claim to moral justification can be assessed, in part, based on the coherence of its approach and that the reliability of any such claim is undermined by the presence of dissonance within its moral system. This study is the first to empirically assess the coherence of NICE's formal approach and in doing so to generate evidence-based conclusions about the extent to which this approach is morally justified. METHODS: The study is grounded in the theory, methods and standards of empirical bioethics. Twenty NICE policy documents were coded to identify and classify the normative commitments contained within NICE technology appraisal policy as of 31 December 2021. Coherence was systematically assessed by attempting to bring these commitments into narrow reflective equilibrium (NRE) and by identifying sources of dissonance. FINDINGS: Much of NICE policy rests on coherent values that provide a strong foundation for morally justified decision-making. However, NICE's formal approach also contains several instances of dissonance which undermine coherence and prevent NRE from being fully established. Dissonance arises primarily from four sources: i) NICE's specification of the principle of cost-effectiveness; ii) its approach to prioritising the needs of particular groups; iii) its conception of reasonableness in the context of uncertainty, and iv) its concern for innovation as an independent value. CONCLUSION: At the time of analysis, the level of coherence across NICE policy provides reason to question the extent to which its formal approach to technology appraisal is morally justified. Some thoughts are offered on why, given these findings, NICE has been able to maintain its legitimacy as a healthcare priority-setter and on what could be done to enhance coherence.

The ethical canary: narrow reflective equilibrium as a source of moral justification in healthcare priority-setting.

Healthcare priority-setting institutions have good reason to want to demonstrate that their decisions are morally justified-and those who contribute to and use the health service have good reason to hope for the same. However, finding a moral basis on which to evaluate healthcare priority-setting is difficult. Substantive approaches are vulnerable to reasonable disagreement about the appropriate grounds for allocating resources, while procedural approaches may be indeterminate and insufficient to ensure a just distribution. In this paper, we set out a complementary, coherence-based approach to the evaluation of healthcare priority-setting. Drawing on Rawls, we argue that an institutional priority-setter's claim to moral justification can be assessed, in part, based on the extent to which its many normative commitments are mutually supportive and free from dissonance; that is, on the ability to establish narrow reflective equilibrium across the normative content of a priority-setter's policy and practice. While we do not suggest that the establishment of such equilibrium is sufficient for moral justification, we argue that failure to do so might-like the proverbial canary in the coalmine-act as a generalised warning that something is awry. We offer a theoretical argument in support of this view and briefly outline a practical method for systematically examining coherence across priority-setting policy and practice.

Assessing clinical benefit in the Medicare Drug Price Negotiation Program: A 2-step approach for improving transparency, consistency, and meaningful patient engagement.

In early 2024, the Centers for Medicare & Medicaid Services (CMS) will send initial price offers to the manufacturers of the first 10 drugs selected for the Medicare Drug Price Negotiation Program, established under the Inflation Reduction Act. However, CMS has not specified exactly how it will adjust the starting point for an initial price offer based on assessment of a drug's clinical benefit. This article addresses unanswered questions relating to CMS' methods for assessing clinical benefit. Specifically, we address how CMS can weigh various measures of evidence, ensure transparency and consistency, meaningfully incorporate patient and other stakeholder perspectives, and support addressing evidence gaps. We propose a 2-step approach for assessing the overall clinical benefit of a selected drug compared with its therapeutic alternatives that builds on the framework outlined by CMS. In step 1, CMS would evaluate conventional clinical benefit, defined in terms of outcomes commonly used in clinical studies for the selected drug and indications. In step 2, CMS would evaluate other outcomes broadly related to patient experience that are not adequately represented in the clinical literature. Overall, our approach incorporates the advantages of both qualitative and quantitative approaches to value assessment and decision-making. We describe a set of loose decision rules to improve transparency and consistency, recommend incorporating ranks and weights to signal to researchers and manufacturers which elements of clinical benefit and sources of data are the most important, and center meaningful deliberation with clinical experts, patients, and caregivers.

Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program.

INTRODUCTION: The United States has begun assessing the value of pharmaceuticals to inform negotiated prices in the Medicare program. Given strong political objections in the United States to the use of QALYs, Medicare will need to adopt an alternative approach to measuring value. AREAS COVERED: In this narrative review, we identified six alternative approaches to measuring value (equal value life-years, health years in total, generalized risk-adjusted cost-effectiveness, severity weighting based on absolute or proportional shortfall, comparative effectiveness based on conventional clinical endpoints, and comparative effectiveness based on both conventional endpoints and patient-centric value elements) and five criteria for assessing these approaches (responsiveness to concerns about discrimination, feasibility, transparency, flexibility, and the ability to incorporate factors beyond traditional value elements). EXPERT OPINION: Four of the alternatives are broadly aligned with the cost-effectiveness framework, but none fully addresses all aspects of the stated concerns that QALYs may be used to unintentionally implement discrimination. We note, however, that the extent to which these concerns lead to discrimination in practice is unknown. Finally, we recommend an approach for measuring value in terms of comparative effectiveness that combines quantitative ranking and weighting of distinct criteria (including patient-centric value elements) with deliberation.

Using Real-World Data to Inform Value-Based Contracts for Cell and Gene Therapies in Medicaid.

OBJECTIVE: High upfront costs and long-term benefit uncertainties of gene therapies challenge Medicaid budgets, making value-based contracts a potential solution. However, value-based contract design is hindered by cost-offset uncertainty. The aim of this study is to determine actual cost-offsets for valoctocogene roxaparvovec (hemophilia A) and etranacogene dezaparvovec (hemophilia B) from Colorado Medicaid's perspective, defining payback periods and its uncertainty from the perspective of Colorado Medicaid. METHODS: This cost analysis used 2018-2022 data from the Colorado Department of Health Care Policy & Financing to determine standard-of-care costs and employed cost simulation models to estimate the cost of Medicaid if patients switched to gene therapy versus if they did not. Data encompassed medical and pharmacy expenses of Colorado Medicaid enrollees. Identified cohorts were patients aged 18+ with ICD-10-CM codes D66 (hemophilia A) and D67 (hemophilia B). Severe hemophilia A required ≥ 6 claims per year for factor therapies or emicizumab, while moderate/severe hemophilia B necessitated ≥ 4 claims per year for factor therapies. Patients were included in the cohort in the year they first met the criteria and were subsequently retained in the cohort for the duration of the observation period. Standard-of-care included factor VIII replacement therapy/emicizumab for hemophilia A and factor IX replacement therapies for hemophilia B. Simulated patients received valoctocogene roxaparvovec or etranacogene dezaparvovec. Main measures were annual standard-of-care costs, cost offset, and breakeven time when using gene therapies. RESULTS: Colorado Medicaid's standard-of-care costs for hemophilia A and B were $426,000 [standard deviation (SD) $353,000] and $546,000 (SD $542,000) annually, respectively. Substituting standard-of-care with gene therapy for eligible patients yielded 8-year and 6-year average breakeven times, using real-world costs, compared with 5 years with published economic evaluation costs. Substantial variability in real-world standard-of-care costs resulted in a 48% and 59% probability of breakeven within 10 years for hemophilia A and B, respectively. Altering eligibility criteria significantly influenced breakeven time. CONCLUSIONS: Real-world data indicates substantial uncertainty and extended payback periods for gene therapy costs. Utilizing real-world data, Medicaid can negotiate value-based contracts to manage budget fluctuations, share risk with manufacturers, and enhance patient access to innovative treatments.

False or Misleading Claims in Online Direct-to-Consumer Ketamine Advertising in Maryland.

This cross-sectional study evaluates the prevalence of false or misleading information in online direct-to-consumer advertising for off-label and unapproved ketamine in Maryland.

Estimated Savings From Using Added Therapeutic Benefit and Therapeutic Reference Pricing in United States Medicare Drug Price Negotiations.

OBJECTIVES: US Medicare will begin negotiating prices for top-selling drugs in 2023. This study describes and estimates potential savings from a therapeutic reference pricing approach, linking comparative effectiveness with the costs of existing therapeutic alternatives, that Medicare could use to adjust the starting point for price negotiations. METHODS: First, we identified target drugs likely to be selected for Medicare negotiation. Second, we identified comparative effectiveness ratings for target drugs based on French Haute Autorité de Santé reports. For target drugs with minor or no added benefit, we identified therapeutic alternatives based on the French reports and US clinical guidelines. For each target drug with minor or no added benefit, we computed the difference between spending based on the drug's estimated statutory ceiling price and spending based on the weighted average cost of therapeutic alternatives or the lowest cost therapeutic alternative. Finally, we calculated potential annual savings from using a starting point in negotiations based on costs of therapeutic alternatives. RESULTS: Potential drug-level savings to Medicare from using a starting point in negotiations based on average spending across therapeutic alternatives, compared with using the statutory ceiling price alone, ranged from $186 541 340 to $2 173 441 197. Potential savings from using a starting point based on the lowest cost alternative ranged from $199 872 163 to $3 605 904 765. CONCLUSIONS: Although we do not expect Medicare to rely on French comparative effectiveness assessments, this study demonstrates the potential for additional savings when using comparative effectiveness and costs of therapeutic alternatives to inform the starting price for negotiations.

We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment.

It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny.

Moral and Social Values in Evidence-Informed Deliberative Processes for Health Benefit Package Design Comment on "Evidence-Informed Deliberative Processes for Health Benefit Package Design - Part II: A Practical Guide".

An evidence-informed deliberative process (EDP) is defined as "a practical and stepwise approach for health technology assessment (HTA) bodies to enhance legitimate health benefit package design based on deliberation between stakeholders to identify, reflect and learn about the meaning and importance of values, informed by evidence on these values." In this commentary, I discuss some considerations for EDPs that arise from acknowledging the difference between social and moral values. First, the best practices for implementing EDPs may differ depending on whether the approach is grounded in moral versus social values. Second, the goals of deliberation may differ when focused on moral versus social values. I conclude by offering some considerations for future research to support the use of EDPs in practice, including the need to assess how different approaches to appraisal (eg, more quantitative versus qualitative) impact perceptions of the value of deliberation itself.

Incorporating Added Therapeutic Benefit and Domestic Reference Pricing Into Medicare Payment for Expensive Part B Drugs.

OBJECTIVES: Identify expensive Part B drugs and evidence for each drug's added benefit and model a reimbursement policy for Medicare that integrates added benefit assessment and domestic reference pricing. METHODS: A retrospective analysis using a 20% nationally representative sample of 2015 to 2019 traditional Medicare Part B claims. Expensive drugs were defined as having average annual spending per beneficiary exceeding the average annual social security benefit ($17 532 in 2019). For expensive drugs identified in 2019, added benefit assessments conducted by the French Haute Autorité de Santé were collected. For expensive drugs with a low added benefit rating, comparator drugs were identified in French Haute Autorité de Santé reports. For each comparator, average annual spending per beneficiary in Part B was computed. Potential savings from 2 reference pricing scenarios were calculated: reimbursing expensive Part B drugs with low added benefit at the level of each drug's (1) lowest cost comparator and (2) beneficiary-weighted-average cost of all comparators. RESULTS: The number of expensive Part B drugs grew from 56 in 2015 to 92 in 2019. Of the 92 expensive drugs in 2019, 34 offer low added benefit. Implementing reference pricing for these expensive drugs with low added benefit could have saved an estimated $2.1 billion if prices were set based on spending for their lowest cost comparator, or $1 billion if prices were set based on the weighted average of spending for comparators. CONCLUSION: Reference pricing based on added benefit assessment could be used to address the launch prices for expensive Part B drugs with low added benefit.

Comparison of Out-of-Pocket Spending on Ultra-Expensive Drugs in Medicare Part D vs Commercial Insurance.

Importance: Little is known about how out-of-pocket burden differs between Medicare and commercial insurance for ultra-expensive drugs. Objective: To investigate out-of-pocket spending for ultra-expensive drugs in the Medicare Part D program vs commercial insurance. Design, Setting, and Participants: This was a retrospective, population-based cohort study of individuals using ultra-expensive drugs included in a 20% nationally random sample of prescription drug claims from Medicare Part D and individuals aged 45 to 64 years using ultra-expensive drugs included in a large national convenience sample of outpatient pharmaceutical claims from commercial insurance plans. Claims data from 2013 through 2019 were used, and data were analyzed in February 2023. Main Outcomes and Measures: Claims-weighted mean out-of-pocket spending per beneficiary per drug by insurance type, plan, and age. Results: In 2019, 37 324 and 24 159 individuals using ultra-expensive drugs were identified in the 20% Part D and commercial samples, respectively (mean [SD] age, 66.2 [11.7] years; 54.9% female). A statistically significant higher share of commercial enrollees vs Part D beneficiaries were female (61.0% vs 51.0%; P < .001), and a statistically significantly lower share were using 3 or more branded medications (28.7% vs 42.6%; P < .001). Mean out-of-pocket spending per beneficiary per drug in 2019 was $4478 in Part D (median [IQR], $4169 [$3369-$5947]) compared with $1821 for commercial (median [IQR], $1272 [$703-$1924]); these differences were statistically significant every year. Differences in out-of-pocket spending comparing commercial enrollees aged 60 to 64 years and Part D beneficiaries aged 65 to 69 years exhibited similar magnitudes and trends. By plan, mean out-of-pocket spending per beneficiary per drug in 2019 was $4301 (median [IQR], $4131 [$3000-$6048]) in Medicare Advantage prescription drug (MAPD) plans, $4575 (median [IQR], $4190 [$3305-$5799]) in stand-alone prescription drug plans (PDPs), $1208 (median [IQR], $752 [$317-$1240]) in health maintenance organization plans, $1569 (median [IQR], $838 [$481-$1472]) in preferred provider organization plans, and $4077 (median [IQR], $2882 [$1075-$4226]) in high-deductible health plans. There were no statistically significant differences between MAPD plans and stand-alone PDPs in any study year. Mean out-of-pocket spending was statistically significantly higher in MAPD plans compared with health maintenance organization plans and in stand-alone PDPs compared with preferred provider organization plans in each study year. Conclusions and Relevance: This cohort study demonstrated that the $2000 out-of-pocket cap included in the Inflation Reduction Act may substantially moderate the potential increase in spending faced by individuals who use ultra-expensive drugs when moving from commercial insurance to Part D coverage.

Integrating Health Technology Assessment and the Right to Health in South Africa: A Qualitative Content Analysis of Substantive Values in Landmark Judicial Decisions.

The World Health Assembly has encouraged WHO member-states to establish capacity in health technology assessment (HTA) as a support for achieving universal health coverage (UHC). Simultaneously, the WHO has stated that UHC is "a practical expression of the concern for health equity and the right to health." This has prompted questions about potential tensions between priority-setting efforts and the right to health on the road to UHC. South Africa (SA) is an ideal setting in which to explore how the priority-setting work of an HTA body may be integrated with an existing rights framework.

Association Between Drug Characteristics and Manufacturer Spending on Direct-to-Consumer Advertising.

Importance: Some drugs are heavily marketed through direct-to-consumer advertising. Objective: To identify drug characteristics associated with a greater share of promotional spending on advertising directly to consumers. Design, Setting, and Participants: Exploratory cross-sectional analysis of drug characteristics and promotional spending for the 150 top-selling branded prescription drugs in the US in 2020 as identified from IQVIA National Sales Perspectives data. Promotional spending data were provided by IQVIA ChannelDynamics. Exposures: Drug characteristics (total 2020 sales; total 2020 promotional spending; clinical benefit ratings; number of indications, off-label use; molecule type; nature of condition treated; administration type; generic availability; US Food and Drug Administration [FDA] approval year, World Health Organization anatomical therapeutic chemical classification; Medicare annual mean spending per beneficiary; percent sales attributable to the drug; market size; market competitiveness) assessed from health technology assessment agencies (France's Haute Autorité de Santé and Canada's Patented Medicine Prices Review Board) and drug data sources (Drugs@FDA, the FDA Purple Book, Lexicomp, Merative Marketscan Research Databases, and Medicare Spending by Drug data). Main Outcomes and Measures: Proportion of total promotional spending allocated to direct-to-consumer-advertising for each drug. Results: The 2020 median proportion of promotional spending allocated to direct-to-consumer advertising was 13.5% (IQR, 1.96%-36.6%); median promotional spending, $20.9 million (IQR, $2.72-$131 million); and median total sales, $1.51 billion (IQR, $0.97-$2.26 billion). Of the 150 best-selling drugs, 16 were missing data and key covariates; therefore, the primary study sample comprised 134 drugs. After adjustment for multiple drug characteristics, the mean proportion of total promotional spending allocated to direct-to-consumer advertising for the remaining 134 drugs was an absolute 14.3% (95% CI, 1.43%-27.2%; P = .03) higher for those with low added clinical benefit than for those with high added clinical benefit and an absolute 1.5% (95% CI, 0.44%-2.56%; P = .005) higher for each 10% increase in total sales. Conclusions and Relevance: Among top-selling US drugs in 2020, a rating of lower added benefit and higher total drug sales were associated with a higher proportion of manufacturer total promotional spending allocated to direct-to-consumer advertising. Further research is needed to understand the implications of these findings.

The Value of Explicit, Deliberative, and Context-Specified Ethics Analysis for Health Technology Assessment: Evidence From a Novel Approach Piloted in South Africa.

OBJECTIVES: This article explores the perceived value, including associated strengths and challenges, of using a context-specified ethics framework to guide deliberative health technology appraisals. METHODS: The South African Values and Ethics for Universal Health Coverage (SAVE-UHC) approach, piloted in South Africa, consisted of 2 phases: (1) convening a national multistakeholder working group to develop a provisional ethics framework and (2) testing the provisional ethics framework through simulated health technology assessment appraisal committee meetings (SACs). Three SACs each reviewed 2 case studies of sample health interventions using the framework. Participants completed postappraisal questionnaires and engaged in focus group discussions. RESULTS: The SACs involved 27 participants across 3 provinces. Findings from the postappraisal questionnaires demonstrated general support for the SAVE-UHC approach and content of the framework, high levels of satisfaction with the recommendations produced, and general sentiment that participants were able to actively contribute to appraisals. Qualitative data showed participants perceived using a context-specified ethics framework in deliberative decision making: (1) supported wider consideration of and deliberation about morally relevant features of the health coverage decisions, thereby contributing to quality of appraisals; (2) could improve transparency; and (3) offered benefits to those directly involved in the priority-setting process. Participants also identified some challenges and concerns associated with the approach. CONCLUSIONS: The SAVE-UHC approach presents a novel way to develop and pilot a locally contextualized, explicit ethics framework for health priority setting. This work highlights how the combination of a context-specified ethics framework and structured deliberative appraisals can contribute to the quality of health technology appraisals and transparency of health priority setting.

Developing and piloting a context-specified ethics framework for health technology assessment: the South African Values and Ethics for Universal Health Coverage approach.

OBJECTIVES: While ethics has been identified as a core component of health technology assessment (HTA), there are few examples of practical, systematic inclusion of ethics analysis in HTA. Some attribute the scarcity of ethics analysis in HTA to debates about appropriate methodology and the need for ethics frameworks that are relevant to local social values. The "South African Values and Ethics for Universal Health Coverage" (SAVE-UHC) project models an approach that countries can use to develop HTA ethics frameworks that are specific to their national contexts. METHODS: The SAVE-UHC approach consisted of two phases. In Phase I, the research team convened and facilitated a national multistakeholder working group to develop a provisional ethics framework through a collaborative, engagement-driven process. In Phase II, the research team refined the model framework by piloting it through three simulated HTA appraisal committee meetings. Each simulated committee reviewed two case studies of sample health interventions: opioid substitution therapy and either a novel contraceptive implant or seasonal influenza immunization for children under five. RESULTS: The methodology was fit-for-purpose, resulting in a context-specified ethics framework and producing relevant findings to inform application of the framework for the given HTA context. CONCLUSIONS: The SAVE-UHC approach provides a model for developing, piloting, and refining an ethics framework for health priority-setting that is responsive to national social values. This approach also helps identify key facilitators and challenges for integrating ethics analysis into HTA processes.

Public opinion regarding U.S. Food and Drug Administration approval of aducanumab and potential policy responses: A nationally representative survey.

BACKGROUND: Despite controversy among experts regarding aducanumab's approval by the U.S. Food and Drug Administration, little is known about public opinion on this matter. METHODS: We conducted a representative survey of U.S. adults ages 35 and older to (1) determine opinions regarding aducanumab's approval, (2) identify any evidence of reputational injury to the Food and Drug Administration, and (3) explore opinions regarding policy responses available to policymakers, such as those relating to a national coverage determination by the Centers for Medicare and Medicaid Services. The survey was administered online and in English and Spanish using a probability-based sample derived from the National Opinion Research Center's AmeriSpeak® panel. Selection probabilities in the panel and survey design account for differences in population distribution and expected response rates by demographic. The survey was analyzed using survey weights to adjust for selection probabilities and non-response. RESULTS: A total of 1025 respondents completed the survey. While approximately three-quarters of respondents were initially unfamiliar with aducanumab, respondents were less supportive of the drug's approval once given information about the drug's potential clinical and economic impact. Sixty-three percent of respondents support restricting aducanumab access to patients most likely to benefit. Seventy-one percent indicated a willingness to enroll a family member with mild Alzheimer's disease in a "waitlist"-style trial to further study aducanumab; sixty percent indicated a willingness to enroll a family member in a randomized placebo-controlled trial. Eighty-one percent agree aducanumab should be withdrawn from the market if confirmatory trials fail. The median respondent was willing to pay $1-5 in higher Part B premiums to cover aducanumab. CONCLUSION: These findings demonstrate support for a range of proposed policies in response to aducanumab's approval. The opinions of an informed public ought to be considered when developing policies in response to aducanumab's approval.

Integrating health technology assessment and the right to health: a qualitative content analysis of procedural values in South African judicial decisions.

South Africa's move towards implementing National Health Insurance includes a commitment to establish a health technology assessment (HTA) body to inform health priority-setting decisions. This study sought to analyse health rights cases in South Africa to inform the identification of country-specific procedural values related to health priority-setting and their implementation in a South African HTA body. The focus on health rights cases is motivated in part by the fact that case law can be an important source of insight into the values of a particular country. This focus is further motivated by a desire to mitigate the potential tension between a rights-based approach to healthcare access and national efforts to set health priorities. A qualitative content analysis of eight South African court cases related to the right to health was conducted. Cases were identified through a LexisNexis search and supplemented with expert judgement. Procedural values identified from the health priority-setting literature, including those comprising Accountability for Reasonableness (A4R), structured the thematic analysis. The importance of transparency and revision-two elements of A4R-is evident in our findings, suggesting that the courts can help to enforce elements of A4R. Yet our findings also indicate that A4R is likely to be insufficient for ensuring that HTA in South Africa meets the procedural demands of a constitutional rights-based approach to healthcare access. Accordingly, we also suggest that a South African HTA body ought to consider more demanding considerations related to transparency and revisions as well as explicit considerations related to inclusivity.

Assessing the Added Therapeutic Benefit of Ultra-Expensive Drugs.

OBJECTIVES: While the United States does not have a method for assessing the added therapeutic benefit of drugs, France, Canada, and Germany do. We examined the added therapeutic benefit of the most expensive drugs prescribed to Medicare Part D beneficiaries in the United States. METHODS: We identified ultra-expensive drugs with annual Medicare spending that exceeded $62 794 (United States GDP per capita in 2018) using Medicare Part D Prescription Drug Spending and Utilization Data. We used added therapeutic benefit ratings assessed by health technology assessment agencies in France, Canada, and Germany. RESULTS: We identified 122 ultra-expensive drugs in 2018. Sixty-five percent of these drugs (n = 79) were assessed by at least one of the countries. Based on these assessments, approximately 75% received a low added therapeutic benefit rating. CONCLUSIONS: Most ultra-expensive drugs prescribed in the United States and assessed by France, Canada, and Germany provide low added therapeutic benefit. Policy reforms in the United States could use added therapeutic benefit to inform coverage and pricing decisions for ultra-expensive drugs. Similar to Germany, one approach would be to allow the company to set a market price for a limited period of time before requiring a price reduction if the added therapeutic benefit is below a certain threshold. Another approach would be to identify when drug prices are substantially more expensive in the United States and conduct an added therapeutic benefit assessment and price review on these drugs.