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OBJECTIVES: To demonstrate how health technology assessment methods can be used to support Medicare's price negotiations for apixaban and rivaroxaban. METHODS: Following the statutory outline of evidence that will be considered by Medicare, we conducted a systematic literature review, network meta-analyses, and decision analyses to evaluate the health outcomes and costs associated with apixaban and rivaroxaban compared with warfarin and dabigatran for patients with nonvalvular atrial fibrillation. Our methods inform discussions about the therapeutic impact of apixaban and rivaroxaban and suggest price premiums above their therapeutic alternatives over a range of cost-effectiveness thresholds. RESULTS: Network meta-analyses found apixaban resulted in a lower risk of major bleeding compared with warfarin and dabigatran and a lower risk of stroke/systemic embolism compared with warfarin but not compared with dabigatran. Rivaroxaban resulted in a lower risk of stroke/systemic embolism versus warfarin but not dabigatran, and there was no difference in major bleeding. Decision-analytic modeling of apixaban suggested annual price premiums up to $4350 above the price of warfarin and up to $530 above the price for dabigatran at cost-effectiveness thresholds up to $200 000 per equal value of life-years gained. Analyses of rivaroxaban showed an annual price premium of up to $3920 above warfarin and no premium above that paid for dabigatran. CONCLUSIONS: Although health technology assessment is typically performed near the time of regulatory approval, with modifications, we produced comparative clinical and relative cost-effectiveness findings to help guide negotiations on a "fair" price for drugs on the market for over a decade.

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INTRODUCTION: The use of expensive oral targeted agents for advanced prostate can be influenced by those who stand to gain from their use. The 340B drug pricing program allows eligible hospitals to purchase medications at steep discounts, generating millions of dollars in savings. The extent to which hospitals engage in higher-risk prescribing due to program incentives is unclear. METHODS: Medicare claims were used to perform a retrospective study of men with advanced prostate cancer. The primary outcome was targeted therapy use in men with high noncancer mortality risk. Secondary outcomes included androgen biosynthesis inhibitor use in men with cardiovascular history, androgen receptor inhibitor use in men with neurocognitive history, and therapy within 14 days of death. Proportional hazards models were used to assess time-to-event outcomes, while logistic regression was used for binary outcomes. RESULTS: In men with high noncancer mortality risk, targeted therapy use did not differ at 340B participating compared to nonparticipating hospitals (hazard ratio [HR] 1.1, 95% CI 0.67-1.5). There was no difference in androgen biosynthesis inhibitor use in men with a prior cardiac event (HR 0.96, 95% CI 0.70-1.3) or androgen receptor inhibitor use in men with a prior neurocognitive event (HR 1.5, 95% CI 0.65-3.4) in those treated at 340B participating compared to nonparticipating hospitals. Therapy use in the last 14 days of life did not vary by 340B participation (odds ratio 1.3, 95% CI 0.86-1.9). CONCLUSIONS: In men with advanced prostate cancer, high-risk prescribing and futility measures did not vary by participation in the 340B drug pricing program.

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Health authorities use value-based pricing models to determine the value of innovative drugs and to establish a price. Pharmaceutical companies prefer value-based pricing over cost-based pricing. It is ambiguous whether value-based pricing has the same meaning to these stakeholders. We aimed to identify the elements that attribute to value-based pricing of innovative drugs from a pharmaceutical industry's perspective and as possible starting point for (value-based) contracting of drugs. We performed a scoping review of publications available in scientific databases with terms such as 'value-based pricing', 'pharmacoeconomics', 'drug cost', 'innovative drug' and 'drug therapy'. We included 31 publications, covering value elements of innovative drugs from a pharmaceutical industry's perspective. Overall, all found elements of value-based pricing were congruent with the elements of value-based pricing from a health authority's perspective. However, the emphasis placed on the elements differed. The most frequently mentioned elements in our review were economic considerations and cost aspects. Least mentioned were elements regarding cost-effectiveness, disease characteristics and patient characteristics. Although all elements in the drug value framework were present which indicate congruity, there seems controversy on the importance of cost-effectiveness as an element of value. Consequently, establishing a coherent and to all stakeholders' acceptable framework to value and price innovative drugs seems complicated. Mutual understanding can be found in the value elements societal considerations and healthcare process benefits. Our results supported the importance of economic and cost aspects regarding determination of prices of innovative drugs. Further research is required to quantify the weights of all relevant elements in the drug value framework, observe their possible interlinkages, and to weigh them over time.

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Over 80% of people living with HIV in low-and-middle-income countries (LMICs) take first-line TDF/XTC/DTG (TLD). Due to hard-fought activism, in >100 LMICs TLD now costs under $45pppy under Voluntary License. With final DTG patents expiring by 2029, generic TLD will soon be available globally. We identify seven critical benchmarks underpinning TLDs success which novel ART should now meet, and an eighth for which novel ART should aim. These are superior efficacy; a high genetic barrier to resistance; safety in hepatitis B coinfection; favourable drug-drug interaction profiles including with antimycobacterials; efficacy in HIV-2; safety in pregnancy, long-acting formulation availability and affordable pricing from the outset. We illustrate when generic TLD will become available worldwide and compare this with trial programmes and approval timelines for two case-study novel ART combinations: islatravir/doravirine and cabotegravir/rilpivirine. We demonstrate that currently these regimens and trial programmes will not meet key benchmarks required to compete with TLD.

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Eligible pediatric hospitals can purchase clinician-administered drugs at discounted rates through the 340B Drug Pricing Program and charge payers prices exceeding drug acquisition costs, but the magnitude of these markups is not known. In a study of newly approved oncology drugs at pediatric 340B hospitals, median negotiated prices ranged from 102% (interquartile range [IQR]: 91%-156%) of average sales price (ASP) at Phoenix Children's Hospital to 630% (IQR: 526%-630%) at Driscoll Children's Hospital. Pediatric hospitals participating in the federal 340B Drug Pricing Program can extract steep payments on new drugs from commercial insurers, though with wide variation between and within hospitals.

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AIM: To investigate the most matchable price of tirzepatide (TIRZ) compared with semaglutide (SEMA) in the treatment of type 2 diabetes in China. METHODS: The patient cohort and clinical efficacy data were derived from the SURPASS-2 trial. Cost-utility analysis and a binary search were performed to identify the most matchable price of TIRZ from a Chinese healthcare provider's perspective. RESULTS: After lifetime simulation, the quality-adjusted life years of TIRZ 5, 10, 15 mg and SEMA 1 mg were 11.17, 11.21, 11.27 and 11.12 years, respectively. Despite an initial assumption that the annual cost of TIRZ equals that of SEMA, our analysis revealed that TIRZ is probably more cost-effective than SEMA. A thorough evaluation of pricing showed that the cost ranges for TIRZ at doses of 5, 10 and 15 mg were $1628.61-$1846.23, $1738.40-$2140.95 and $1800.30-$2430.81, respectively. After adjustment in the univariate sensitivity analysis, the cost ranges for TIRZ 5, 10 and 15 mg were $1542.68-$1757.57, $1573.00-$1967.16 and $1576.54-$2133.96, respectively. These cost intervals were validated through robust probabilistic sensitivity analysis and scenario analysis, except for the cost range for TIRZ 5 mg. CONCLUSIONS: This study shows that, using SEMA as a reference, the annual costs for TIRZ 10 and 15 mg are $1573.00-$1967.16 and $1576.54-$2133.96, respectively, for patients with type 2 diabetes in China.

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Cancer treatment has become increasingly expensive, partially due to the use of specialty drugs. The costs of these drugs are often passed down to patients, who may face the consequences of paying for more than they can afford, leading to financial toxicity. The 340B drug pricing program is a health care policy that may provide an opportunity to mitigate the financial consequences of cancer care. The 340B program requires manufacturers to sell outpatient drugs at a discount to hospitals caring for a significant number of socioeconomically disadvantaged individuals. The program intended for hospitals to use savings from discounted purchases to expand their safety net to vulnerable patients. Some studies have shown that participating hospitals do this by offering more charity and discounted care, whereas others have demonstrated that hospitals fail to sufficiently expand their safety net. A potential flaw of the program is the lack of guidance from governing bodies on how hospitals should use savings from discounted purchases. There has been growing discussion among stakeholders to reform the 340B program given the mixed findings of its effectiveness. With the rising costs of specialty drugs and associated prevalence of financial toxicity in patients with cancer, there is an opportunity to address these issues through reform that improves the program. Directing hospitals to offer specific safety net opportunities, such as passing along discounted drug prices to vulnerable populations, could help the growing number of patients who are financially burdened by medications at the core of the 340B program.

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Discovery research is the starting point for the development of more effective anti-cancer treatments. It requires an interdisciplinary research environment with first-class infrastructural support in which curiosity-driven research can lead to new concepts for treating cancer. Translating such research findings to clinical practice requires complementary skills and infrastructures, including high-quality clinical facilities, access to patient cohorts and participation of pharma. This complex ecosystem has yielded many new but also "me too" treatment regimens, especially in immuno-oncology resulting in an extremely high pricing of anti-cancer agents. The costs of antibodies, vaccines, and cell therapies charged by pharma stand out although the concepts and methodologies have been largely developed in academia, financed from public funds. Comprehensive Cancer Centres (CCCs) covering a coherent stretch of the cancer research continuum are well-positioned to make these personalized treatments more affordable, but this will require restructuring of the way the translational cancer research continuum is funded.

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OBJECTIVES: Decision makers considering using cost-effectiveness analysis (CEA) to inform health-technology assessment must contend with documented and controversial shortfalls of CEA, including its assumption of disease severity independence and static pricing. ISPOR has recently introduced novel value elements besides direct healthcare cost and effectiveness for the patient, and these should be captured in CEA. Although novel value elements advance our understanding of "what" should be measured (value of hope, severity of disease, health equity, etc), there is limited direction on "how" to measure them in conventional CEA. Furthermore, with Medicare empowered to set drug prices under the Inflation Reduction Act, it is not clear what role CEA might have on where prices are set, given objections to the quality-adjusted life year in conventional approaches. METHODS: We critically reviewed the evidence for expanding conventional CEA methods to a more generalized approach of generalized CEA (GCEA). RESULTS: GCEA accounts for methods that address objections to the quality-adjusted life year and incorporate novel value elements. Although GCEA offers advantages, it also requires further research to develop "off-the-shelf" resources to help inform, for example, maximum fair price in the context of Medicare drug price negotiation. CONCLUSIONS: Should a shift toward GCEA reveal that the societal value of novel medicines exceeds their market-based costs, which will raise the key question of what market failure Medicare negotiation is meant to solve, if any, and therefore what the appropriate role of such negotiation might be to maximize the value society might garner from the development of novel medicines.

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Priority-setting policy-makers often face moral and political pressure to balance the conflicting motivations of efficiency and rescue/non-abandonment. Using the conflict between these motivations as a case study can enrich the understanding of institutional design in developed democracies. This essay presents a cognitive-psychological account of the conflict between efficiency and rescue/non-abandonment in health care priority-setting. It then describes three sets of institutional arrangements-in Australia, England/Wales, and Germany, respectively-that contend with this conflict in interestingly different ways. The analysis yields at least three implications for institutional design in developed democracies: (1) indeterminacy at the level of moral psychology can increase the probability of indeterminacy at the level of institutional design; (2) situational constraints in effect require priority-setting policy-makers to adopt normative-moral pluralism; and (3) the U.S. health care system may be in an anti-priority-setting equilibrium.

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INTRODUCTION: The Reference Drug Program (RDP) was established to steer patients toward equally safe and cost-effective medication under British Columbia's public drug coverage. Each RDP class covers at least one reference drug, and non-reference drugs are reimbursed up to the cost of the reference drug. In 2016, the RDP updated to include proton pump inhibitors (PPIs). This study evaluated the impact on drug expenditures, prescription patterns, and health services utilization. METHODS: We identified a cohort of individuals covered by Fair Pharmacare who used PPIs, and a control group of H2 Blockers users. We used interrupted time series analysis on administrative data from June 2014 to December 2019 on the following outcomes: new users, day supply, expenditures, drug costs, reference drug use, and physician visits and costs. RESULTS: The RDP had little impact on overall PPI use patterns. We did not observe any changes in reference drug uptake, new users, physician visits, cost-savings, or significant changes to days supplied post-policy. Cost expenditure results were likely biased due to co-occurring changes to drug prices. CONCLUSION: Inclusion of PPIs to the RDP saw no cost-savings for the provincial drug program and had little impact on prescribing patterns. Overall, our findings are consistent with existing evidence that the RDP is safe for similar therapeutic alternatives, but the impact on PPI costs remains unclear.

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OBJECTIVES: This study aimed to conduct a review of existing methods used to incorporate life cycle drug pricing (LCDP) in cost-effectiveness analyses (CEAs), identify common methodological challenges, and suggest modeling approaches for prospectively implementing LCDP in CEA. METHODS: Two complementary searches were conducted in PubMed, combined with hand searching and reference mining, to identify English language full-text articles that explored (1) how drug prices change over time and (2) methods used to apply dynamic pricing in cost-effectiveness models (CEMs). Relevant articles were reviewed, and authors discussed the common methodological practices used in the literature and their associated challenges on prospectively implementing LCDP in CEMs. For each key challenge identified, we provide modeling suggestions to address the issue. RESULTS: We screened 1200 studies based on title and abstract; 117 were reviewed for eligibility, and 47 individual studies were included across both searches. Variations in prices over a product's life cycle are complex and multifactorial, and models applying LCDP in CEA varied in their methodology. We identified 4 key challenges to modeling LCDP in CEA, including how to model price trends before and after loss of exclusivity, how to capture the effect of price changes on future patient cohorts, and how to report results. CONCLUSION: Accurately quantifying the impact of LCDP requires careful consideration of multiple aspects pertaining to both the evolution of drug prices and how to reflect these in CEA. Although uncertainties remain, our findings can aid implementation and evaluation of LCDP in economic evaluations.

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Policy Points The 340B Drug Pricing Program accounts for roughly 1 out of every 100 dollars spent in the $4.3 trillion US health care industry. Decisions affecting the program will have wide-ranging consequences throughout the US safety net. Our scoping review provides a roadmap of the questions being asked about the 340B program and an initial synthesis of the answers. The highest-quality evidence indicates that nonprofit, disproportionate share hospitals may be using the 340B program in margin-motivated ways, with inconsistent evidence for increased safety net engagement; however, this finding is not consistent across other hospital types and public health clinics, which face different incentive structures and reporting requirements. CONTEXT: Despite remarkable growth and relevance of the 340B Drug Pricing Program to current health care practice and policy debate, academic literature examining 340B has lagged. The objectives of this scoping review were to summarize i) common research questions published about 340B, ii) what is empirically known about 340B and its implications, and iii) remaining knowledge gaps, all organized in a way that is informative to practitioners, researchers, and decision makers. METHODS: We conducted a scoping review of the peer-reviewed, empirical 340B literature (database inception to March 2023). We categorized studies by suitability of their design for internal validity, type of covered entity studied, and motivation-by-scope category. FINDINGS: The final yield included 44 peer-reviewed, empirical studies published between 2003 and 2023. We identified 15 frequently asked research questions in the literature, across 6 categories of inquiry-motivation (margin or mission) and scope (external, covered entity, and care delivery interface). Literature with greatest internal validity leaned toward evidence of margin-motivated behavior at the external environment and covered entity levels, with inconsistent findings supporting mission-motivated behavior at these levels; this was particularly the case among participating disproportionate share hospitals (DSHs). However, included case studies were unanimous in demonstrating positive effects of the 340B program for carrying out a provider's safety net mission. CONCLUSIONS: In our scoping review of the 340B program, the highest-quality evidence indicates nonprofit, DSHs may be using the 340B program in margin-motivated ways, with inconsistent evidence for increased safety net engagement; however, this finding is not consistent across other hospital types and public health clinics, which face different incentive structures and reporting requirements. Future studies should examine heterogeneity by covered entity types (i.e., hospitals vs. public health clinics), characteristics, and time period of 340B enrollment. Our findings provide additional context to current health policy discussion regarding the 340B program.

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In this latest update, we explore the Inflation Reduction Act (IRA) enacted by the US Congress in August 2022, with the Centers for Medicare and Medicaid Services (CMS) recently releasing the list of the first ten drugs it will negotiate prices on. We also cover the consequences of price controls and rigid value assessment in Germany which have led to the withdrawal of a number of medicines. It will be important to see how the IRA balances cost-saving with holistic value assessment, incentives for innovation and patient access to treatment.

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Purpose: The market price of adebrelimab, the first Chinese-developed anti-PD-L1 antibody used as a first-line treatment for extensive stage-small-cell lung cancer (ES-SCLC), has garnered significant public attention. This study sought to investigate the affordable price of adebrelimab for Chinese patients with untreated ES-SCLC through a cost-effectiveness analysis. Patients and Methods: We conducted a cost-effectiveness analysis using a Markov model, incorporating a what-if scenario of adding adebrelimab to first-line etoposide/platinum (EP) chemotherapy is cost-effective for ES-SCLC patients from the perspective of the Chinese healthcare system. The model included three mutually exclusive health states, with transition probabilities derived from the CAPSTONE-1 trial. Health state utilities and costs were acquired from a myriad of authoritative sources. We compared the incremental cost-effectiveness ratios (ICERs) for adebrelimab plus EP chemotherapy (AEP) versus EP with a willingness-to-pay threshold of $37,654 per quality-adjusted life-years (QALYs) to estimate the affordable price ceiling of the upcoming adebrelimab. Results: For the entire ES-SCLC population, the estimated price ceiling of adebrelimab/mg was $0.542 (95% CI, $0.542-$0.552). Subgroup analyses found that the highest price ceiling of adebrelimab/mg was observed in ES-SCLC patients with lactate dehydrogenase concentration ≤ upper normal limit [$0.824 (95% CI, $0.815-$0.830)]; and the lowest was found in ES-SCLC patients with liver metastasis [$0.252 (95% CI, $0.250- $0.256)]. Sensitivity analysis revealed a heightened probability of cost-effectiveness for the first-line AEP as the price of adebrelimab decreased, encompassing both the entire ES-SCLC population and its subgroups. Conclusion: The affordable price range for adebrelimab/mg Chinese patients with untreated ES-SCLC was estimated to be between $0.252 and $0.824, with variations observed across different subgroups. In the context of universal healthcare coverage, our study provides valuable evidence to inform the implementation of a value-based pricing strategy for cancer treatment.

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Purpose: The aim of this study was to evaluate the cost-effectiveness of a recently approved first-line therapy (adebrelimab plus chemotherapy vs. chemotherapy alone) for patients with extensive-stage small-cell lung cancer (ES-SCLC) in the US and China, and to estimate the reasonable range of adebrelimab price from the decision-makers. Methods: Several partitioned survival models were built to compare the cost and effectiveness of adebrelimab plus chemotherapy vs. chemotherapy alone over a 10-year time horizon. Clinical efficacy and safety data were extracted from the CAPSTONE-1 trial. Costs and utilities were obtained from previously published studies. Sensitivity, scenario and subgroup analyses were performed to explore the uncertainty of the model outcomes. Price simulation was conducted at three thresholds of willingness-to-pay (WTP), including WTP of $100,000 in the US and of $37,422 in China, 0.5WTP of $50,000 in the US and of $18,711 in China, and 1.5WTP of 150,000 in the US and of $56,133 in China. Findings: Base-case analysis at $1382.82/600 mg of adebrelimab price indicated that adebrelimab plus chemotherapy would be cost-effective in the US at the WTP threshold of $100,000, but not in China at the WTP threshold of $37,422. If PAP was taken into account, the regimen would be cost-effective in China at the given WTP. The results of price simulation indicated that adebrelimab plus chemotherapy was completely favored in the US if adebrelimab price was less than $8894.98/600 mg (total quality-adjusted life years [QALYs] were calculated with progression-based utility [PB-utility]) or $8912.51/600 mg (total QALYs were calculated with time-to-death utility [TTD-utility]) at the WTP threshold of $100,000; if adebrelimab price was reduced by at least $202.03/600 mg (total QALYs were calculated with PB-utility) or $103.06/600 mg (total QALYs were calculated with TTD-utility), the regimen was also cost-effective in China without PAP at the WTP threshold of $37,422. The above results were stable in the sensitivity analyses. Subgroup analysis found that the subgroup with better survival benefits tended to have a higher probability of cost-effectiveness, which was also associated with adebrelimab price. Implications: First-line adebrelimab plus chemotherapy represented a dominant treatment strategy comparing with chemotherapy alone in the US and also did in China with PAP at $1382.82/600 mg of adebrelimab price. Decision-makers could benefit from pricing strategy provided by this study in making optimal decisions. More evidences were needed to verify and improve the results.

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CONTEXT: Positive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes. OBJECTIVE: We analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes. METHODS: Data were extracted from publicly available HTA reports on drugs that received marketing authorisation between 2009 and 2018 (N = 1415). The outcomes of interest were the probability of positive HTA outcomes and the time-to-HTA outcome; these were examined with respect to clinical, regulatory, product- and disease-related, evidence uncertainty and contextual variables. Econometric models utilising survival analysis and multinomial logistic regression were specified. FINDINGS: Positive HTA outcomes accounted for 87.3% of the sample (n = 1235), of which 71% (n = 1004) were restricted. Drugs with positive HTA outcomes were subject to clinical restrictions (n = 652, 46%), financial risk-sharing (n = 439, 31%) or had been rejected at least once (n = 282, 20%). Significant predictors of positive HTA outcomes were orphan drugs with cancer indications, high quality of evidence linked to clinical and economic evidence uncertainties which had been overcome, and contextual considerations, particularly innovativeness and unmet need. Comparative analyses revealed systematic differences between countries in their propensity to accept the same drugs, particularly oncology and orphan drugs. CONCLUSIONS: Our results are contextual and reinforce arguments in favour of explicitly accounting for social value judgements, establishing separate assessment frameworks for highly uncertain products, adopting risk mitigation strategies for novel therapies with early phase evidence, and sharing of HTA practices across settings. Lastly, HTA agencies have adopted an extra-welfarist approach to value assessment and resource allocation.

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Growth in the cost of prescription drugs in the US has generated significant interest in the use of external reference pricing (ERP) to tie prices paid for drugs to those in other countries. We used data from the Pricentric ONE™ database, an international drug pricing database, to examine product launch timing, launch price, and price changes from January 2010 - October 2021 in both ERP and non-ERP settings, with a focus on 100 high-priced drugs of interest to Medicare and Medicaid. We found that ERP policies were associated with a 73% reduction in the likelihood of drug launch within 9 months of regulatory approval relative to non-ERP settings. In addition, while ERP was associated with statistically significant reductions in annual drug price changes, such policies did not impact launch price. In addition, no single ERP feature (e.g., number of countries referenced, ERP calculation) was materially associated with the outcomes of interest. We conclude that ERP policies do not appear to impact drug launch price and may delay access to new therapies, raising questions about the utility of such policies in the US and potential consequences abroad.