Drug Prices and Value of Oncology Drugs in Italy

ObjectiveThe main objective of this study was to evaluate the potential role of efficacy data and other information available at the time of price and reimbursement (P&R) decision-making process within the definition of oncology treatment costs in Italy.MethodsThe study included all P&R dossiers submitted to the Italian Medicines Agency between July 2015 and December 2017. It prospectively collected the data of the P&R process starting from dossier submission up to the Italian Health Service reimbursement decision. The cost of treatment per patient was estimated using both the list price (“gross cost”) and the confidential net price (“net cost”) of drug packages and applied to the median duration of treatment. A 2-sample stage Heckman decomposition model was used to evaluate the potential role of efficacy data and other information available at the time of P&R decision making on the gross and net cost.ResultsA total of 37 oncology drugs related to 58 therapeutic indications were analyzed. The multivariate model showed that the variation of progression-free survival is the only variable predictor statistically associated with treatment cost, but this effect was observed only when confidential net prices were used (P=.026).ConclusionsConsidering the perspective of a developed country having a public healthcare service with a central reimbursement negotiation is determined a relevant reduction in the treatment cost purchased by public payers. This is a useful approach to guarantee the affordability of innovative oncology drugs and to contain public expenditures on healthcare. Furthermore, the negotiation of confidential discounts and agreement clauses in managed entry agreements seemed to reward oncology drugs displaying an added therapeutic benefit.     

Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia

ObjectivesMexiletine is a long-known drug used for the treatment of arrhythmias and repurposed in the 1980s for patients with nondystrophic myotonia (NDM). Recently, the price of mexiletine in Europe increased significantly after registration as an orphan drug for NDM. This led to international discussions on affordability and willingness to reimburse mexiletine in the absence of background information that would justify such a price. Our objective was to calculate a cost-based price for mexiletine for adult patients with NDM based on detailed information on development costs.MethodsWe calculated a fair price based on a cost-based pricing model for commercial mexiletine to treat adults with NDM using a recent European drug-pricing model as a framework to include actual costs incurred. Three scenarios were applied: 1 with minimum estimated costs, 1 with maximum estimated costs, and 1 with costs as if mexiletine was innovative.ResultsThe calculated fair price of mexiletine per patient per year (PPPY) is €452 for the minimum scenario and €1996 for the maximum scenario. By using hypothetical R&D costs used for innovative drugs, the price would be €6685 PPPY. In Europe, the list price of mexiletine ranges from €30 707-60 730 PPPY, based on 600 mg daily.ConclusionsThe current list price for mexiletine in Europe is manifold higher than any scenario of the cost-based models. Accounting for the reduced costs for clinical development in a repurposing scenario, the cost-based pricing model provides a fair commercial price range, which can be used as benchmark for pricing negotiations and/or reimbursement decisions.     

药物经济学在医保药品谈判中的角色 ——以丙型肝炎直接抗病毒药物为例

目的:以参加2019年国家医保药品谈判的丙型肝炎治疗药物为例,探讨药物经济学在医保药品谈判中的作用,为医保药品目录调整和同类药品谈判提供参考.方法:通过成本效用分析和阈值分析,测算丙肝治疗药物的降价幅度并与实际降价幅度进行对比,结合具体谈判方式探讨相关影响因素.结果:2019年丙肝药品谈判采用了竞争性谈判和比价磋商谈判...     

A comparative study of European rare disease and orphan drug markets

ObjectivesThis article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom.MethodsInformation was derived from the international literature, analysis of legal texts, and a survey completed by national experts.ResultsThese countries adopted varying approaches towards regulating rare disease and orphan drug markets and, hence, the availability, pricing and reimbursement of orphan drugs vary between countries. Strategies to keep down prices include public procurement in Sweden, profit controls in the United Kingdom, and price comparisons with other countries. To gain reimbursement, the cost-effectiveness and/or budget impact of orphan drugs is considered in some countries. Other societal considerations, such as whether the drug treats a life-threatening disease, are sometimes taken into account.ConclusionsExtensive government intervention exists in rare disease and orphan drug markets in the countries studied. Our recommendations are to define priorities for research on rare diseases and orphan drugs at the European level, to set up disease and patient registries with a view to investigating the long-term effectiveness and cost-effectiveness of orphan drugs, to assess the profitability of orphan drugs, and to take into account societal considerations when evaluating orphan drugs.     

丙型肝炎创新药医保准入支付政策对医药费用的影响研究

目的:分析天津和成都两地丙肝创新药医保准入支付政策对人均医药费用产生的影响,探究两地政策效果的差异及原因。方法:收集天津市2014年5月—2018年10月某定点医院的全部丙肝患者病历记录,以及成都市2016年1月—2019年6月医保数据。采用中断时间序列对政策实施前后人均医药费用的变化进行评估。结果:创新药准入支付政策实施后,天津人均医药总费用水平瞬时下降了7 753.43元,其中人均药费下降了5 718.34元,人均其他诊疗费用下降了2 035.09元(P0.01)。成都人均医药总费用水平瞬时上升了6 680.78元(P0.01),其中人均药费上升了6 917.20元(P0.01),人均其他诊疗费用长期趋势每月下降318.88元(P0.01)。结论:创新药医保政策的实施效果受到多个因素的影响,医保准入谈判极大降低了创新药费用,按人头付费政策的实施降低了其他诊疗费用。建议:应积极推进创新药国家准入谈判;在入围创新药支付标准全国统一的情况下,地方医保部门应积极推进实施门诊慢性病患者医药费用按人头支付或基于价值/疗效支付,进一步降低其他诊疗费用;因地制宜制定并落实创新药配套政策。     

Modifying the Criteria for Granting Orphan Drug Market Exclusivity

ObjectivesTo examine policy options to deny orphan drug exclusivity after drugs exceed a target population of 200 000 across all orphan indications (combined prevalence threshold) or once drugs receive a nonorphan approval (market approval threshold).MethodsRetrospective analysis of drugs with 2 or more orphan approvals from 1983 to July 01, 2017 examining prevalence of orphan indications and approval years of orphan and nonorphan indications. Characteristics of drugs crossing either threshold are described. A budget impact analysis of Medicare and Marketscan® claims databases estimated potential savings from generic or biosimilar entry as a result of foregone market exclusivity periods determined by these policies.ResultsOut of 86 drugs with 2 or more orphan approvals, 21 drugs would be denied orphan drug exclusivity periods under the prevalence threshold and 18 drugs would be denied orphan drug exclusivity periods under the market approval threshold. Drugs with orphan approvals after 2010 were more likely to be denied orphan drug exclusivity. In 2017, Medicare could have saved about $2 billion on 8 drugs under the prevalence threshold policy and $1.3 billion on 12 drugs under the market approval threshold policy). Private insurers could have saved $814 and $919 million, respectively. Over half of the savings would come from 9 drugs that first entered the market for a nonorphan indication.ConclusionsModifying the criteria for granting orphan drug exclusivity would affect a small number of orphan drugs but could generate large savings through increased competition. Other incentives such as grants or tax credits for clinical trials could be explored to incentivize research for new orphan indications for drugs that crossed either threshold.     

我国公立医院药品价格形成机制的演变及未来政策选择

首先分析公立医院药品采购中的销售合同及采购合同,并指出两个合同中的"市场失灵"。而政府定价、行政降价、顺加作价、集中招标采购等行政手段实施后,药品采购相关利益方采取各种博弈行为,公立医院药品采购和价格形成出现"政府失灵"。未来的公立医院药品价格形成应该为政府有限调控下的市场定价,在保持集中招标采购和零差率两个行政手段的基础上,提高医疗保险机构的价格谈判和公立医院在招标过程中的参与度。     

An Analysis of Accessibility of Representative Psychotropic Medicine From the World Health Organization Model List of Essential Medicines in Developing Countries With Different Income Levels

ObjectivesThe objective of this study was to analyze the accessibility of psychotropic medicine in developing countries based on the availability, price, and affordability indicators to create international evidence to guide the development of policies on the accessibility of medicines.MethodsThis study included 5 types of psychotropic medicines listed in the 22nd edition of the World Health Organization Model List of Essential Medicines published by the World Health Organization in 2021. Derived from 84 surveys in 59 countries, this study summarizes the availability, price, and affordability of originator branded drugs (OBs) and lowest-price generic drugs (LPGs) in the public and private sectors and compares them based on income levels in different countries.ResultsThe average availability of psychotropic medicine was 45% in low- and lower-middle-income countries (LLMICs) compared with 49% in high- and upper-middle-income countries (HUMICs) whereas the availability of LPGs was higher than that of OBs in all country groups. The average patient price for OBs and LPGs was 94.0 and 23.2, respectively, and the overall patient price of psychotropic medicine in LLMICs was higher than that in HUMICs. The affordability of psychotropic medicine in LLMICs was lower than that in HUMICs.ConclusionsPsychotropic medicines in lower-middle-income countries have lower availability, a higher average patient price, and lower average affordability than in HUMICs, which requires lower-middle-income countries to take effective and various measures to improve the accessibility of psychotropic medicine.     

Medicare Drug Price Negotiation in the United States: Implications and Unanswered Questions

The United States is a relatively free-pricing market for pharmaceutical manufacturers to set list prices at the product launch. Few drug price controls exist, and federal price negotiation as a policy has historically been politically untenable. After decades of debate on whether the federal government, specifically the Medicare program, should more actively manage drug prices, the US Congress passed legislation authorizing Medicare to directly negotiate prices with manufacturers. The purpose of this article is to describe elements and implementation of the price negotiation provisions and then comment on the potential impacts on payers, innovations, and the pharmaceutical industry. While impacting only a few drugs each year in the beginning, price negotiation in the Medicare program will have secondary and long-term effects in the US market and beyond. It is clear that in the United States, the Medicare market for drugs will no longer be a free-pricing environment in the industry.     

International differences in patient access to ultra-orphan drugs

Objectives

Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient access to ultra-orphan drugs between countries. Furthermore, we describe how reimbursed and non-reimbursed orphan drugs differ with respect to pharmacoeconomic properties.

Are Drugs Priced in Accordance With Value? A Comparison of Value-Based and Net Prices Using Institute for Clinical and Economic Review Reports

ObjectivesThe Institute for Clinical and Economic Review (ICER) is an independent organization that reviews drugs and devices with a focus on emerging agents. As part of their evaluation, ICER estimates value-based prices (VBP) at $50 000 to $150 000 per quality-adjusted life-year (QALY) gained thresholds. We compared actual estimated net prices to ICER-estimated VBPs.MethodsWe reviewed ICER final evidence reports from November 2007 to October 2020. List prices were combined with average discounts obtained from SSR Health to estimate net prices. If a drug had been evaluated more than once for the same indication, only the more recent VBP was included.ResultsA total of 34 ICER reports provided unique VBPs for 102 drugs. The net price of 81% of drugs exceeded the $100 000 per QALY VBP and 71% exceeded the $150 000 per QALY VBP. The median change in net price needed to reach the $150 000 per QALY VBP was a 36% reduction. The median decrease in net price needed was highest for drugs targeting rare inherited disorders (n = 15; 62%) and lowest for cardiometabolic disorders (n = 6; 162% price increase). The reduction in net prices needed to reach ICER-estimated VBPs was higher for drugs evaluated for the first approved indication, rare diseases, less competitive markets, and if the drug approval occurred before the ICER report became available.ConclusionNet prices are often above VBPs estimated by ICER. Although gaining awareness among decision makers, the long-term impact of ICER evaluations on pricing and access to new drugs continues to evolve.     

Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis

ObjectivesThree hundred million people living with rare diseases worldwide are disproportionately deprived of in-time diagnosis and treatment compared with other patients. This review provides an overview of global policies that optimize development, licensing, pricing, and reimbursement of orphan drugs.MethodsPharmaceutical legislation and policies related to access and regulation of orphan drugs were examined from 194 World Health Organization member countries and 6 areas. Orphan drug policies (ODPs) were identified through internet search, emails to national pharmacovigilance centers, and systematic academic literature search. Texts from selected publications were extracted for content analysis.ResultsOne hundred seventy-two drug regulation documents and 77 academic publications from 162 countries/areas were included. Ninety-two of 200 countries/areas (46.0%) had documentation on ODPs. Thirty-four subthemes from content analysis were categorized into 6 policy themes, namely, orphan drug designation, marketing authorization, safety and efficacy requirements, price regulation, incentives that encourage market availability, and incentives that encourage research and development. Countries/areas with ODPs were statistically wealthier (gross national income per capita = $10 875 vs $3950, P < .001). Country/area income was also positively correlated with the scope of the respective ODP (correlation coefficient = 0.57, P < .001).ConclusionsGlobally, the number of countries with an ODP has grown rapidly since 2013. Nevertheless, disparities in geographical distribution and income levels affect the establishment of ODPs. Furthermore, identified policy gaps in price regulation, incentives that encourage market availability, and incentives that encourage research and development should be addressed to improve access to available and affordable orphan drugs.     

Drugs for rare diseases: Influence of orphan designation status on price

The literature indicates that the expenditure on orphan drugs will be increasing over the coming years. The market for orphan drugs has inherent market characteristics that sometimes result in high prices. The aim of this study was to analyse whether awarding orphan designation status has an influence on the price setting of drugs for rare disease indications. To this effect, prices of designated orphan drugs were compared with other non-designated drugs for rare disease indications. We identified 28 designated orphan drugs and 16 comparable non-designated drugs for rare disease indications for which we collected official hospital prices (per defined daily dose) in Belgium in 2010. Orphan-designated drugs had a higher median price (138.56 Euros [interquartile range; IQR 406.57 Euros]) than non-designated drugs (16.55 Euros [IQR 28.05 Euros]) for rare disease indications (p < 0.01). In conclusion, our results suggest that awarding orphan designation status in itself is associated with higher prices for drugs for rare disease indications. In order to gain full insight into orphan drug pricing mechanisms, future research should focus on collecting information about the different factors influencing orphan drug pricing.     

Do Reimbursement Recommendations by the Canadian Agency for Drugs and Technology in Health Translate Into Coverage Decisions for Orphan Drugs in the Canadian Province of Ontario?

ObjectivesUnlike other high-income countries, Canada has no national policy for drugs treating rare diseases (orphan drugs). Nevertheless, in 2022, the Canadian government committed to creating a national strategy to make access to these drugs more consistent. Our aim was to study whether recommendations made by the Canadian Agency for Drugs and Technology in Health (CADTH) translated into coverage decisions for orphan drugs in Ontario, the largest Canadian province. This study is the first to look at this question for orphan drugs, which are at the center of policy attention.MethodsWe included 155 orphan drug-indication pairs approved and marketed in Canada between October 2002 and April 2022. Cohen’s kappa was used to test the agreement across health technology assessment (HTA) recommendations and coverage decisions in Ontario. Logistic regression was used to test which factors, relevant to decision-makers, might be associated with funding in Ontario.ResultsWe found only fair agreement between CADTH’s recommendations and coverage decisions in Ontario. Although a positive and statistically significant association between favorable HTA recommendations and coverage was found, more than half of the drugs with a negative HTA recommendation were available in Ontario, predominately through specialized funds. Successful pan-Canadian pricing negotiations were a strong predictor of coverage in Ontario.ConclusionsDespite efforts to harmonize access to drugs across Canada, considerable room for improvement remains. Introducing a national strategy for orphan drugs could help increase transparency, consistency, promote collaborations, and make access to orphan drugs a national priority.     

Price and Consumer Cost Responsibility Effects on Quality Perceptions and Price Negotiation Likelihood for Healthcare Services

This paper presents the results of an experimental study that assessed potential differences in consumer quality perceptions and price negotiation likelihood for three healthcare procedures: a routine physical, rhinoplasty, and a root canal, based on varying levels of price and consumer cost responsibility. Results of this study did not support a general positive price-perceived quality relationship for any of the three procedures. However, several significant effects were observed for price negotiation likelihood. First, price negotiation likelihood was found to be higher for more expensive services (i.e., rhinoplasty) than less expensive services (i.e., routine physical). In addition, consumers were more likely to negotiate price when they were both responsible for the entire cost of an expensive procedure and not accustomed to paying the full cost. Lastly, people who likely perceived a relationship between price and quality were less likely to negotiate pricing at high price levels vis-à-vis low price levels.     

Affordability Challenges to Value-Based Pricing: Mass Diseases,Orphan Diseases,and Cures

Objectives

To analyze how value-based pricing (VBP), which grounds the price paid for pharmaceuticals in their value, can manage “affordability” challenges, defined as drugs that meet cost-effectiveness thresholds but are “unaffordable” within the short-run budget.

Consistency of new drug pricing in Korea: Bridging variations among personnel in price negotiations

ObjectivesThis study aims to explore the performance of price negotiations, over a period of 10 years, and identify individual variations in pricing that directly affect the consistency of pricing decisions.MethodsExisting literature and relative regulations were reviewed, and data provided by the National Health Insurance Service (NHIS) was analysed to evaluate the performance of price negotiations. To examine individual variations in pricing, hypothetical scenarios of four types of drugs were developed. These were subsequently given to the reviewers who have been actually conducting price negotiations at the NHIS.ResultsOver the last decade, negotiations succeeded at a rate of 85.5 %, with a higher agreement rate in the more recent years. The reduction rate, via price negotiations, from the price approved for reimbursement by the Health Insurance Review and Assessment Service (HIRA), was 12.2 %. The survey results confirmed a significant individual variation in pricing. Especially, in the case of Drug N1, the higher-grade personnel and non-pharmacists tended to give higher prices with a significant difference.ConclusionsThe individual variations that would greatly affect the financial results were confirmed when making decisions on price of new drugs.For the consistency of drug pricing, some measures to minimise the variation of reviewers’ judgement are needed.