High-priced Medicines Research Articles

Introduction of managed entry agreements in Korea: Problem, policy, and politics.

Objectives: This study aimed to understand Managed Entry Agreements (MEAs) in Korea through the framework of three streams of the policy window model and its practical management and impact on pricing and reimbursement scheme. Methods: An extensive literature review based on Kingdon's model was conducted. We also performed descriptive analyses of MEA implementation using data on medicines listed in Korea and compared its MEA scheme with four different countries. Results: As per problem streams, patients with rare disease or cancers have considerable difficulties in affording their medicines and this has challenged the drug benefit system and raised an issue of patient's access. Policy streams highlighted that MEAs were introduced as a benefit enhancement plan for four major diseases since January 2014. MEAs have also been strengthened as a bypass mechanism to expand the insurance coverage especially for new premium-priced medicines under Moon Care (Listing all non-listed services). In descriptive analysis of MEAs, a total of 48 medicines were contracted as MEAs from January 2014 to December 2020, accounting for 73.4% of listed medicines for cancer or rare diseases and 97.9% of the cases were finance-based contracts. Meanwhile, outcome-based contracts such as CED accounted for only 2.1%. The application of MEAs differs across countries, resulting in a kappa coefficient of 0.00-0.14 (United Kingdom 0.03, Italy 0.00, Australia 0.14), indicating a lack of consistency compared to South Korea. Conclusion: MEAs, which were introduced as a bypass mechanism, have now superseded the standard process for anticancer agents or orphan drugs. Further studies are needed to evaluate the impact of the confidential agreements and effectiveness of new high-priced medicines with limited clinical data at launch.

Promoting Access to Innovative Anticancer Medicines: A Review of Drug Price and National Reimbursement Negotiation in China.

Providing universal access to high-cost medications like anticancer drugs is not an easy feat. Although basic medical insurance has covered over 95% of China's population since 2012, reimbursement for high-priced medicines is limited. In 2015, the Chinese government proposed establishing an open and transparent price negotiation mechanism for some patented and expensive drugs, where oncology was among the prioritized areas. In 2016, three drugs (gefitinib, icotinib, and tenofovir disoprox) underwent negotiation with the government, eventually reducing their prices by over 50% so that they could be prioritized during reimbursement processes. Focusing on anticancer medicines, this study comprehensively summarizes the progress in drug price and national reimbursement negotiation in China. Furthermore, we investigated the changes and development regarding negotiated anticancer medicines from quantity negotiated, classification, indication coverage, utilization, and procurement spending. Our findings could provide a reference for follow-up negotiations and reimbursement policies for high-value anticancer medications in other countries. From 2016 to 2021, 82 anticancer medicines were newly incorporated into the national reimbursement drug list (NRDL) via 6 rounds of negotiation. The majority of these were innovative pharmaceutics (ie, protein kinase inhibitors (28) and monoclonal antibodies (13)). Drug pricing and national reimbursement negotiation led to a marked decrease in prices and a sharp increase in the utilization of negotiated anticancer medicines. Following negotiations, the defined daily doses (DDDs) of innovative anticancer medicines experienced remarkable growth. Their proportion in total anticancer drugs DDDs also increased from 3.4% in 2014 to 20.9% in 2019. However, although drug prices decreased substantially after the negotiations, insurance spending still showed an upward trend owing to the significant increase in utilization. This calls for the government to carefully monitor the rational use of these expensive medicines and explore innovative payment models.

Enhancing Choices Regarding the Administration of Insulin Among Patients With Diabetes Requiring Insulin Across Countries and Implications for Future Care.

There are a number of ongoing developments to improve the care of patients with diabetes across countries given its growing burden. Recent developments include new oral medicines to reduce cardiovascular events and death. They also include new modes to improve insulin administration to enhance adherence and subsequent patient management thereby reducing hypoglycaemia and improving long-term outcomes. In the case of insulins, this includes long-acting insulin analogues as well as continuous glucose monitoring (CGM) systems and continuous subcutaneous insulin infusion systems, combined with sensor-augmented pump therapy and potentially hybrid closed-loops. The benefits of such systems have been endorsed by endocrine societies and governments in patients with Type 1 diabetes whose HbA1c levels are not currently being optimised. However, there are concerns with the low use of such systems across higher-income countries, exacerbated by their higher costs, despite studies suggesting their cost-effectiveness ratios are within accepted limits. This is inconsistent in higher-income countries when compared with reimbursement and funding decisions for new high-priced medicines for cancer and orphan diseases, with often limited benefits, given the burden of multiple daily insulin injections coupled with the need for constant monitoring. This situation is different among patients and governments in low- and low-middle income countries struggling to fund standard insulins and the routine monitoring of HbA1c levels. The first priority in these countries is to address these priority issues before funding more expensive forms of insulin and associated devices. Greater patient involvement in treatment decisions, transparency in decision making, and evidence-based investment decisions should help to address such concerns in the future.

European collaborations on medicine and vaccine procurement.

To ensure equitable access to medicines and vaccines, organizational efforts and purchase volumes have been pooled in joint procurements and negotiations for decades in some regions of the world, as well as globally through supranational procurement mechanisms. In Europe, countries started to collaborate on procurement and negotiations recently when it became increasingly difficult to ensure access to high-priced medicines, even in high-income countries. Two European country collaborations (the Nordic Pharmaceutical Forum and the Baltic Procurement Initiative) have successfully concluded at least one joint tender process for medicines and vaccines and the Beneluxa Initiative has concluded its first successful joint price negotiation. This article describes the experiences of these country collaborations. Challenges observed included: legal barriers; institutional and organizational differences between health-care systems in member countries; and the risk that suppliers will be reluctant to cooperate with country collaborations. Although these collaborations helped improve access to medicines and vaccines for the countries involved, in situations such as a global health crisis, larger-scale, more-inclusive initiatives are needed. In the current coronavirus disease 2019 (COVID-19) pandemic, COVID-19 Vaccines Global Access (COVAX) initiative established a global procurement mechanism to ensure the equitable distribution of COVID-19 vaccines globally. Despite differences in organization and scale, the European country collaborations and COVAX have some similarities: (i) their success depends on the increased purchasing power associated with pooled order volumes; (ii) expert knowledge and previous procurement experience is pooled; (iii) they perform other collaborative activities that go beyond procurement alone; and (iv) they actively involve external partners and stakeholders.

Understanding the trends in international agreements on pricing and reimbursement for newly marketed medicines and their implications for access to medicines: a computational text analysis

BackgroundHealth systems are struggling with unprecedented drug spending and governments have devised various policy options to manage high-priced medicines. Meanwhile, some pricing and reimbursement processes are currently moving under the jurisdiction of international agreements. This study aims to understand trends in international agreements from the perspectives of pricing and reimbursement policies for newly marketed medicines.MethodsWe proposed the framework to interpret the international agreements as code and applied computational text analysis to understand international agreements as data. In particular, we selected the AUSFTA, KORUS, and TPP to assess the progress and evolution in international agreements and investigate the existing relevant content on the pricing and reimbursement of newly marketed medicines.ResultsSimilar to the provisions for intellectual property, the scope of international agreements regarding pricing and reimbursement decisions are broadened and strengthened. Over time, the domain of transparency, re-naming procedural fairness, has changed significantly more than the remaining domains. Pharmaceutical companies will have more opportunities to advocate for their positions, to protect their interests in decision processes, to investigate the decisions on listings and setting the amounts of reimbursement, and to challenge these decisions.ConclusionsRecently signed international agreements favor companies over governments with underscoring procedural fairness and timely access. However, access to affordable medicines is the goal towards which international agreements should aim. In a similar vein, substantial fairness and the accountability of companies should be discussed when negotiating agreements or adopting international agreements through domestic legislation.

Integrative Review of Managed Entry Agreements: Chances and Limitations.

Managed entry agreements (MEAs) consist of a set of instruments to reduce the uncertainty and the budget impact of new high-priced medicines; however, there are concerns. There is a need to critically appraise MEAs with their planned introduction in Brazil. Accordingly, the objective of this article is to identify and appraise key attributes and concerns with MEAs among payers and their advisers, with the findings providing critical considerations for Brazil and other high- and middle-income countries. An integrative review approach was adopted. This involved a review of MEAs across countries. The review question was 'What are the health technology MEAs that have been applied around the world?' This review was supplemented with studies not retrieved in the search known to the senior-level co-authors including key South American markets. Italso involved senior-level decision makers and advisers providing guidance on the potential advantages and disadvantages of MEAs and ways forward. Twenty-five studies were included in the review. Most MEAs included medicines (96.8%), focused on financial arrangements (43%) and included mostly antineoplastic medicines. Most countries kept key information confidential including discounts or had not published such data. Few details were found in the literature regarding South America. Our findings and inputs resulted in both advantages including reimbursement and disadvantages including concerns with data collection for outcome-based schemes. We are likely to see a growth in MEAs with the continual launch of new high-priced and often complex treatments, coupled with increasing demands on resources. Whilst outcome-based MEAs could be an important tool to improve access to new innovative medicines, there are critical issues to address. Comparing knowledge, experiences, and practices across countries is crucial to guide high- and middle-income countries when designing their future MEAs.

A Comprehensive Study on Availability of Anti-cancer drugs in Dhaka City, Bangladesh

With the increasing rate of people being affected by cancer in Bangladesh, the disease has become a national concern. Anti-cancer drugs play a significant and crucial role in cancer treatment regimen. Inadequate access to essential anti-cancer medicines may impose serious public health problems in Bangladesh. The present study gives an insight on the availability of eleven WHO enlisted essential anticancer drugs that are commonly prescribed for the top five prevalent cancer types (Breast, Oesophagus, Lung, Cervix, Lips and Oral cavity) in Bangladesh. Expert advice on which essential anti-cancer medicines are commonly prescribed were gathered from oncologists of three medical college hospitals (Dhaka Medical College Hospital (DMCH), Bangabandhu Sheikh Mujib Medical University (BSMMU) and National Institute of Cancer Research and Hospital (NICRH). The study was conducted among a total of 151 pharmaceutical drug stores including 119 retail, 27 model and 5 hospital pharmacies. The sample size of 151 pharmacies was calculated for 4547 pharmacies in Dhaka district with 95% confidence interval and 8% margin of error. It was found that 74% of the pharmacy stores surveyed kept no anti-cancer drugs at all. There is a significant negative relationship between price and availability of oncology medicine for which Pearson’s correlation co-efficient ‘r’ is found to be -0.739 (p=0.01). This means that high priced medicines are less available. Pharmacy stores keep anti-cancer drugs from both local (81.9%) and foreign (18.1%) pharmaceutical companies. In order to evaluate sales performance of pharmacies in context of oncology drugs, monthly number of cancer patients buying medicines from these pharmacies has been studied as well. It has been found that supply of anti-cancer drugs by pharmaceutical companies among medicine stores is not homogenous. Availability and sales rate of oncology products in some pharmacies situated within the same area is way more than others and the number of customer (cancer patient) varies by wide margins. A comparative analysis on availability rate of model versus retail pharmacies in different areas of Dhaka city has been carried out. Retail pharmacies near cancer-care hospitals tend to keep more anti-cancer medicines compared to those situated away from cancer-care hospitals. Bangladesh Pharmaceutical Journal 23(2): 155-166, 2020

The interpretation of China national essential medicines list 2018

ABSTRACTBackground: In 2018, China implemented the latest National Essential Medicines List (NEML) by enhancing the NEML 2012. The goal of our studies is to analyze the changes in the two lists and compared them with the 20th EML issued by WHO in 2017. And then provide suggestions for emerging problems.Method: The overall composition of the categories, specific drugs, characteristics, advantages and disadvantages of the lists were compared by descriptive analysis. The neuropsychiatric disorders system medicines and patented medicines were analyzed to illustrate the changes of NEML.Results: In 20th WHO-EML, the largest increase was the medicines used for children (13 to the core list and 12 to the complementary list). In 2018 NEML, rounding out the top were medicines used for cardiovascular system. Among the 120 new medicines, 30 new medicines were included in 2017 WHO-EML. Eleven patented medicines were new-added in NEML; however, 8 was not included in WHO-EML.Conclusion: China has a large population, and the territorial development is uneven. Although the essence of EMLs is a limited list, NEML should enlarge the choices properly. 2018 NEML provides a comprehensive coverage of diseases. Some of the medicines, including high-priced medicines that were not recommended by WHO.

What Is Possible and What Is Not? The Development of a Legal Framework for Drug Pricing Mechanisms in the EU.

As a result of the constantly increasing number of innovative but exceedingly high priced medicines, many Member States have implemented price-regulating mechanisms to ensure the financial viability of their healthcare systems. However, the European Court of Justice applies strict criteria to these measures - some of them have already been suspended for the purposes of the free movement of goods. The European Union (EU) allowed the development of an ever-stricter case law on one hand, without changing the legal frame on the other hand. Considering the importance and the binding nature of the Court's judgements, this leaves great uncertainty for national legislators about which measures to remedy the pricing problem are legally possible and which are not. In order to provide clarity, this article seeks to analyse the development of the case law and to define the legal scope for pharmaceutical pricing mechanisms in the EU.

Improving patient access to hepatitis C antiviral medicines in Switzerland: Understanding the financial risks for community pharmacies.

As observed in other countries, some patients may experiment difficulties in obtaining their hepatitis C antiviral medicines (HCVm) in Swiss community pharmacies. There is a lack of data related to access to HCVm at the patient level and notably related to the potential financial risks for the community pharmacies. (a) To evaluate the potential financial risks for community pharmacist associated with the delivery of HCVm in the Swiss healthcare system; (b) to explore the attitudes and experiences of community pharmacists related to these risks and their consequences for the patients. A three-step approach was chosen as follows: (a) estimation of costs, incomes, and gross financial results directly related to 3-month treatment with Harvoni based on the drug delivery process (data from 68 patients over 2years); (b) sensitivity analyses; (c) exploration of local community pharmacists' attitudes and experiences related to the delivery of HCVm in the canton of Vaud (Western Switzerland). Two main risks were identified: (a) Incomes do not always cover costs; (b) reimbursement issues could lead to an increase in the requirement for working capital. According to the survey, 23% (14/60) of pharmacies refused to deliver HCVm to at least one patient, and these patients had to find a solution mostly on their own. The scenario analysis clarifies the causes of the possible refusal to deliver HCVm. With the growing number of high-priced medicines, the healthcare systems should have a clear strategy to encourage their delivery by community pharmacies by ensuring seamless and collaborative care for patients. The community pharmacists could be accountable to provide such services-if they get the education, training, and remuneration.

The South Region Cancer Registry: an evaluation of its exhaustiveness in a cohort of lung cancer patients.

The regional cancer registry for Southern Portugal (ROR‐Sul) is a population‐based registry set up in 1988 to observe and monitor disease incidence, prevalence and survival. Recently, the need to monitor real‐life use of early approved and high‐priced medicines led to therapeutic effectiveness becoming an emerging area of interest. We aimed to evaluate the exhaustiveness of the ROR‐Sul database, covering around 4.8 million inhabitants. We have used a retrospective cohort study comprising 3457 lung cancer cases diagnosed during 2014 and 2015 and extracted from ROR‐Sul database. Descriptive analysis of missing data was undertaken using IBM SPSS software, v.24. Exhaustiveness of data registry was classified into high (missing values <1%), medium (missing values {1–15%}) or low (missing values > 15%). High exhaustiveness was found for patients demographic information, date of diagnosis, date of first medical appointment, topography, morphology, cancer differentiation, stage of disease and surgery procedure. Medium exhaustiveness was found for biomarkers (ALK, KRAS, and EGFR) results, and immunotherapy regimens. Low exhaustiveness was found for performance status, chemotherapy regimen, and chemotherapy treatment response. The findings highlight the need to transform treatment variables into compulsory, so that the cancer registry may be used to support effectiveness studies. Education, training and behaviour changes must also be considered to foster the process.

Trends in Sales Volumes of Cancer Medicines in Six Asian Countries Working Toward Universal Health Coverage

Background: In low- and middle-income (LMIC), medicines represent an important proportion of total out-of-pocket expenditures on health care and should therefore be a key target for financial protection through inclusion in benefit packages under universal health coverage (UHC). For antineoplastic medicines, this need will become even more urgent with the increasing incidence of cancers in LMIC and the availability of new, highly priced cancer. Previous studies have shown that countries have listed cancer medicines recommended in the World Health Organization (WHO) essential medicines list (EML) in their national EMLs. However, little is known about use of these medicines at country level and changes in use over time as countries work toward UHC. Aim: To analyze trends in sales volumes of cancer medicines in China, Indonesia, Kazakhstan, Malaysia, the Philippines and Thailand between 2006 and 2017. Methods: Using quarterly data from IQVIA (former IMS and Quintiles) on sales volumes in 6 middle-income Asian countries of cancer medicines (ATC-L01/02) included in the 2017 WHO EML, we estimated annual sales by international nonproprietary name, year and country. Sales volumes were estimated in total milligrams and total defined daily doses (DDD) per new cancer case. The reference DDD were taken from the German Institute of Medical Documentation and Information. We adjusted total mg and DDD by the total number of new cancer cases per country in 2012 ( Globocan 2012 ). Results: The 2017 WHO EML contains 49 medicines in the antineoplastic section; 40 of them were used in 1 or more countries included in the study. Availability of these 40 medicines ranged from 33 in Indonesia to 40 in the Philippines. The median percentage increase in sales volumes between 2006 and 2017 ranged from 42% in Thailand to 94% in Indonesia. Use of new, high-priced medicines such as trastuzumab and rituximab increased more than 90% and 72% in all countries, respectively, during the study period. Limitations: National sales volumes are extrapolated from standardized audits of procurement and sales data in sample facilities and/or distribution data supplied by wholesalers in each country and may underestimate actual sales. Given different indications for which a cancer medicine can be used and highly individualized antineoplastic treatment regimens and dosages, standardized DDD do not represent actual prescribed daily doses. Growth rates will reflect the place of the medicine within the national treatment guidelines and healthcare delivery capacity. Conclusion: This study provides evidence that use of WHO-recommended essential medicines for cancers increased in 6 Asian countries working toward UHC. Many factors are likely to have contributed to this outcome, including improvements in diagnosis and treatments. The next step in this study will be to assess the role of insurance coverage changes as a factor contributing to changes in utilization of cancer medicines.

PCN25 - Evaluating Availability, Prices and Affordability: Case Study of Selected Oncology and Innovator Medicines in Malaysia

High-priced medicines such as patented innovators and oncology items are great burden to healthcare budgets and barriers to treatment access worldwide. This study investigates availability, medicine price and affordability of selected oncology and on-patent medicines (medicines that still have patent protection) in Malaysia’s public and private sectors. This cross sectional study adapted the validated methodology developed by the World Health Organization/Health Action International. Procurement and patient prices were collected from public and private facilities nationwide for a case study of four oncology and four on-patent medicines. Availability was reported as the percentage of medicine outlets with the medicine. For international comparison, median prices were compared against International Reference Price (IRP). Mark-up was calculated as the percentage of patient price to procurement price. Affordability was determined by the number of days’ wages required for selected treatment courses. The average availability of the selected basket of oncology and on-patent medicines were 53.9% and 51.0%, respectively. There were wide variations between public and private procurement prices of the medicines studied. Compared to international benchmarks, oncology drugs were procured at 0.2 to 3.2 times the IRP. Median retail mark-ups of originator and generic oncology medicines were 20.7% and 130.2%, respectively. High mark-ups of 41.4% were observed for on-patent medicines. Although generics were affordable, originators were unaffordable. To illustrate, low-income patients need to work for about three days to afford on-patent medicines and up to a year to afford one course of cancer treatment. Since there are no generic alternatives, patients that are not able to afford the needed treatment may not have other treatment alternatives. Sustainable financing for expensive medicines is needed to safeguard medicines access and prevent catastrophic outcomes. Policies to reward innovation should be balanced with affordability, as people from different socio-economic groups deserve the same treatment.

The effect of group purchasing on prices hospitals pay for medicines.

I estimate the impact of group purchasing on medicine prices in French hospitals, taking advantage of the entry of hospitals into regional purchasing groups between 2009 and 2014. Based on a new database providing the average annual prices paid for all innovative and high-priced medicines in public hospitals, I use a two-way fixed effects model that controls for hospitals' medicine-specific bargaining power and medicine-specific price trends. I find that group purchasing slightly reduces the overall prices of medicines but has no impact on the prices of medicines that have no alternative on the market. On the contrary, prices of medicines in oligopoly markets are extensively impacted.

How Can Pricing and Reimbursement Policies Improve Affordable Access to Medicines? Lessons Learned from European Countries.

This article discusses pharmaceutical pricing and reimbursement policies in European countries with regard to their ability to ensure affordable access to medicines. A frequently applied pricing policy is external price referencing. While it provides some benchmark for policy-makers and has been shown to be able to generate savings, it may also contribute to delay in product launch in countries where medicine prices are low. Value-based pricing has been proposed as a policy that promotes access while rewarding useful innovation; however, implementing it has proven quite challenging. For high-priced medicines, managed-entry agreements are increasingly used. These agreements allow policy-makers to manage uncertainty and obtain lower prices. They can also facilitate earlier market access in case of limited evidence about added therapeutic value of the medicine. However, these agreements raise transparency concerns due to the confidentiality clause. Tendering as used in the hospital and offpatent outpatient sectors has been proven to reduce medicine prices but it requires a robust framework and appropriate design with clear strategic goals in order to prevent shortages. These pricing and reimbursement policies are supplemented by the widespread use of Health Technology Assessment to inform decision-making, and by strategies to improve the uptake of generics, and also biosimilars. While European countries have been implementing a set of policy options, there is a lack of thorough impact assessments of several pricing and reimbursement policies on affordable access. Increased cooperation between authorities, experience sharing and improving transparency on price information, including the disclosure of confidential discounts, are opportunities to address current challenges.

A human rights-based approach to the reimbursement of expensive medicines.

In recent years, pharmaceutical companies have introduced new expensive medicines, some of which target only small patient populations. For example, trastuzumab (Herceptin*), imatinib (Glivec[R]) and sofosbuvir (Sovaldi[R]) are high-priced medicines that have been shown to be effective and safe for treating cancer or hepatitis C, diseases for which no effective treatment existed previously. In 2015, World Health Organization included several such expensive medicines in their model list of essential medicines, (1) despite current prices of 60000-100 000 United States dollars per treatment. The initial public enthusiasm for these medicines' therapeutic value is now tempered by practical concerns about how patients and health systems can afford them. Even in high-income countries affordability is a concern. Ethical and economic dilemmas arise when decision-makers must ration an expensive, life-saving or life-extending medicine. We contend that not funding an effective essential medicine contradicts Article 12 in International Covenant on Economic, Social and Cultural Rights, which declares the States Parties to present Covenant recognize right of everyone to enjoyment of highest attainable standard of physical and mental By September 2016, over 160 countries have ratified or acceded to Covenant. (2) In many countries, such as Argentina, Brazil, Latvia, New Zealand and Uruguay, patients have used Article 12 to litigate through domestic courts for reimbursement of expensive medicines. (3) In Netherlands, advocates of patients with Pompes disease --a rare muscle disease--based part of their arguments on fact that it was patients' right to receive continued reimbursement of an expensive treatment with marginal health benefits. Decision-makers decided to continue reimbursement, despite fact that many other patients in country were not reimbursed for more cost-effective treatments. How can governments with finite public health budgets ensure fair access to expensive, new and essential medicines as part of right to health? Is right to health equally important for all patients? Governments' efforts to prioritize access to essential medicines could be supported by a human rights approach and specifically by principle of progressive realization of right to health. The United Nations Committee on Economic, Social and Cultural Rights describes progressive realization as States Parties' obligation to use maximum of its available resources to move as expeditiously and effectively as possible towards achieving highest attainable standard of health for everyone. (2,4) The approach does not necessarily create an immediate right for everyone to receive treatment at any cost. Instead, progressive realization means that access to health care is gradually fulfilled for all patients. All Parties have an immediate obligation to take deliberate, concrete and targeted steps towards progressive realization. (3) However, providing immediate universal health care with all possible treatments for all people is not a legal obligation. (5) For prioritization, a transparent and independent reimbursement ranking process of medical treatments, largely based on estimates of comparative cost-effectiveness, presents a concrete and practical tool for decision-makers. Progressive realization justifies such ranking of treatments by ensuring that governments use available resources as effectively as possible and thereby progressively realizing right to health for largest number of people at lowest possible cost and without discrimination against patients. This duty of efficiency is also in line with authoritative interpretation by Committee on Economic, Social and Cultural Rights of governments' legal obligations under human rights treaties in General Comment 3, as well as a well-founded set of principles adopted by a group of scholars and policy-makers in Limburg Principles. …

Price comparison of high-cost originator medicines in European countries

ABSTRACTBackground: In recent years, high-cost medicines have increasingly been challenging the public health budget in all countries including high-income economies. In this context, this study aims to survey, analyze and compare prices of medicines that likely contribute to high expenditure for the public payers in high-income countries.Methods: We chose the following 16 European countries: Austria, Belgium, Denmark, Finland, France, Germany, Greece, Hungary, Ireland, Italy, the Netherlands, Portugal, Sweden, Slovakia, Spain and United Kingdom. The ex-factory price data of 30 medicines in these countries were collected in national databases accessible through the Pharmaceutical Price Information (PPI) service of Gesundheit Österreich GmbH (Austrian Public Health Institute).Results: The ex-factory prices (median) per unit (e.g. per tablet, vial) ranged from 10.67 cent (levodopa + decarboxylase inhibitor) to 17,000 euro (ipilimumab). A total of 53% of the medicines surveyed had a unit ex-factory price (median) above 200 Euro. For two thirds of the medicines, price differences between the highest-priced country and lowest-priced country ranged between 25 and 100%; the remaining medicines, mainly low-priced medicines, had higher price differential, up to 251%. Medicines with unit prices of a few euros or less were medicines for the treatment of diseases in the nervous system (anti-depressants, medicines to treat Parkinson and for the management of neuropathic pain), of obstructive airway diseases and cardio-vascular medicines (lipid modifying agents). High-priced medicines were particularly cancer medicines.Conclusion: Medicine prices of Greece, Hungary, Slovakia and UK were frequently at the lower end, German and Swedish, as well as Danish and Irish prices at the upper end. For high-priced medicines, actual paid prices are likely to be lower due to confidential discounts and similar funding arrangements between industry and public payers. Pricing authorities refer to the higher undiscounted prices when they use price data from other countries for their pricing decisions.

A pill too hard to swallow: how the NHS is limiting access to high priced drugs

A joint investigation by The BMJ and Cambridge and Bath universities uncovers how NHS England tried to limit access to expensive new drugs for hepatitis C. Jonathan Gornall , Amanda...

Introduction and Utilization of High Priced HCV Medicines across Europe; Implications for the Future.

Background: Infection with the Hepatitis C Virus (HCV) is a widespread transmittable disease with a diagnosed prevalence of 2.0%. Fortunately, it is now curable in most patients. Sales of medicines to treat HCV infection grew 2.7% per year between 2004 and 2011, enhanced by the launch of the protease inhibitors (PIs) boceprevir (BCV) and telaprevir (TVR) in addition to ribavirin and pegylated interferon (pegIFN). Costs will continue to rise with new treatments including sofosbuvir, which now include interferon free regimens.Objective: Assess the uptake of BCV and TVR across Europe from a health authority perspective to offer future guidance on dealing with new high cost medicines.Methods: Cross-sectional descriptive study of medicines to treat HCV (pegIFN, ribavirin, BCV and TVR) among European countries from 2008 to 2013. Utilization measured in defined daily doses (DDDs)/1000 patients/quarter (DIQs) and expenditure in Euros/DDD. Health authority activities to influence treatments categorized using the 4E methodology (Education, Engineering, Economics and Enforcement).Results: Similar uptake of BCV and TVR among European countries and regions, ranging from 0.5 DIQ in Denmark, Netherlands and Slovenia to 1.5 DIQ in Tayside and Catalonia in 2013. However, different utilization of the new PIs vs. ribavirin indicates differences in dual vs. triple therapy, which is down to factors including physician preference and genotypes. Reimbursed prices for BCV and TVR were comparable across countries.Conclusion: There was reasonable consistency in the utilization of BCV and TVR among European countries in comparison with other high priced medicines. This may reflect the social demand to limit the transmission of HCV. However, the situation is changing with new curative medicines for HCV genotype 1 (GT1) with potentially an appreciable budget impact. These concerns have resulted in different prices across countries, with their impact on budgets and patient outcomes monitored in the future to provide additional guidance.

The 19th EML as a global standard: an innovative approach or just an opportunity for new and effective medicines?

In 1977, the World Health Organization (WHO) published its first Model List of Essential Medicines including just over 200 medicines. This year, the 20th Expert Committee for the Selection and Use of Medicines has included several new high priced medicines in the areas of cancer (N=16), hepatitis C (N=6) and multidrug-resistant tuberculosis (N=4). The large cancer update was prepared with the support of UICC by an ad-hoc expert working group that reviewed the overall consistency of 29 applications to WHO EML. This large update challenges the traditional perception of essential medicines emphasizing the role of EML as a global standard and as an important tool to prioritise medicines reimbursement, as part of UHC programs in countries. The Expert Committee's main criteria for inclusion in the List were public health relevance, the magnitude of clinical benefit and a favourable risk–benefit profile determined through a systematic method of evidence synthesis and appraisal. Estimates of the magnitude of the benefit are particularly pertinent for medicines for cancer, given the small gains in life expectancy offered by some new and expensive treatments that led to the decision to select only a limited range of new cancer medicines. The Expert Committee could not suggest a minimum threshold of benefit for cancer medicines because this is out of its role and mandate, but suggested the need for a medium-term strategy to re-discuss existing mechanisms in particular to present approaches and thresholds for both clinical relevance and cost-effectiveness. The inclusion in EML of several highly effective and high priced medicines is raising important questions of how access and affordability can be addressed at global and country levels. The existing EML decision-making rules in place since the EB resolution of 2001 state that absolute treatment cost should not be a reason to reject a proposed addition to the Model List if criteria for benefit and public health relevance are met. In practice, affordability has been changed from a precondition for listing an essential medicine to a challenge that must be addressed once an effective and expensive medicine has been added to the list. The 20th Expert Committee advocated for new and better-coordinated actions at a global level to address issues of thresholds for clinical benefit and cost-effectiveness. Prioritization according to the magnitude of benefit was the guiding principle for EML decision-making and could be relevant to countries in their reimbursement decisions and in developing universal health coverage programs.