[Twenty years since the publication of medication price regulation in Brazil: time to update?] / Vinte anos da publicação da regulamentação de preços de medicamentos no Brasil: momento de atualizar?

Criteria for setting medication prices in Brazil are set forth in CMED Resolution n. 2/2004 of the (Medicines Market Regulation Chamber). The stipulated prices influence the private and public markets, which makes it challenging to review pricing policies due to the need to harmonize social and economic interests. A proposal for reviewing this Resolution was made available through the SEAE Public Consultation n. 2/2021 of the Competition and Competitiveness Advocacy Secretariat/Brazilian Ministry of Economy; however, so far without publication of the consolidated results. Recent recommendations from the World Health Organization regarding the adoption of different thresholds for setting medication prices are adopted in this Resolution, although it was published 20 years ago. To interpret and describe the alignment, possible advances and setbacks between the legal texts related to medication price regulation, we conducted an analytical-descriptive and exploratory documentary research. As a result, the list of reference countries for international price verification and the thresholds for internal and external price referencing were maintained. The normative omissions of the Resolution remain in the Public Consultation, such as the absence of criteria for pricing radiopharmaceuticals, advanced therapies and medication without international and comparator prices in the Brazilian market, to revise prices and transpose provisional to definitive prices. A critical point was the creation of a 35% bonus above the stipulated price for medication that present additional clinical benefit without, however, defining clear contours as to the acceptable scientific evidence to prove such benefit. In short, few advances were noticed in the Public Consultation.

Os critérios para definir os preços de medicamentos no Brasil estão previstos na Resolução CMED nº 2/2004 da Câmara de Regulação do Mercado de Medicamentos. Os preços estipulados influenciam o mercado privado e público, o que torna desafiador a revisão de políticas de preços devido a necessidade de harmonizar interesses sociais e econômicos. Uma proposta de revisão dessa Resolução foi disponibilizada por meio da Consulta Pública SEAE nº 2/2021 da Secretaria de Advocacia da Concorrência e Competitividade/Ministério da Economia, porém, até o momento sem publicação dos resultados consolidados até o momento. Recomendações recentes da Organização Mundial da Saúde em relação à adoção de diferentes limiares para definição de preços de medicamentos são adotadas nessa Resolução, embora essa tenha sido publicada há 20 anos. Com o objetivo de interpretar e descrever o alinhamento e os possíveis avanços e retrocessos nos textos legais relacionados à regulação de preços de medicamentos, foi utilizado o método da pesquisa documental analítica-descritiva, de cunho exploratório. Como resultado, foram mantidas a lista de países referência para conferência de preço internacional e os limiares de referenciamento interno e externo de preços. As omissões normativas da Resolução permanecem na Consulta Pública, como a ausência de critérios para precificar radiofármacos, terapias avançadas e medicamentos sem preço internacional, e sem comparadores no mercado brasileiro para revisar preços e transpor preço provisório para definitivo. Um ponto crítico foi a criação de bônus de 35% acima do preço estipulado para medicamentos que apresentem benefício clínico adicional sem, contudo, definir contornos claros quanto às evidências científicas aceitáveis para a comprovação desse benefício. Em suma, poucos avanços foram percebidos na Consulta Pública.

Los criterios para definir los precios de los medicamentos en Brasil están establecidos en la Resolución CMED nº 2/2004 de la Cámara de Regulación del Mercado de Medicamentos. Los precios estipulados influyen en el mercado público y privado, lo que dificulta la revisión de las políticas de precios debido a la necesidad de armonizar los intereses sociales y económicos. Una propuesta para revisar esta Resolución se puso a disposición mediante la Consulta Pública SEAE nº 2/2021 de la Secretaría de Competencia y Promoción de la Competitividad/Ministerio de Economía, sin embargo, hasta el momento no se han publicado los resultados consolidados. En esta Resolución se adoptan recomendaciones recientes de la Organización Mundial de la Salud sobre la adopción de diferentes umbrales para fijar los precios de los medicamentos, aunque fue publicada hace 20 años. Con el objetivo de interpretar y describir el alineamiento, posibles avances y retrocesos, entre los textos legales relacionados con la regulación de precios de medicamentos, se utilizó el método de investigación documental analítica-descriptiva, de carácter exploratorio. Como resultado, se mantuvieron la lista de países de referencia para la verificación de precio internacional y los umbrales para la referenciación interna y externa de precios. Quedan en Consulta Pública las omisiones normativas de la Resolución, como la ausencia de criterios de fijación de precios de radiofármacos, terapias avanzadas y medicamentos sin precio internacional y comparadores en el mercado brasileño, para revisar precios y transponer el precio provisional al definitivo. Un punto crítico fue la creación de una bonificación del 35% sobre el precio estipulado para los medicamentos que presenten un beneficio clínico adicional sin definir, sin embargo, contornos claros sobre las evidencias científicas aceptables para demostrar dicho beneficio. En definitiva, se percibieron pocos avances en la Consulta Pública.

[The metamorphoses of denationalization of 1993. Report-I. The market fight for medications import].

In the history of the Russian pharmaceutical market the year of 1993 became the year of formation of rules of market relations and changing under them of market landscape. The significant segment of state-centralized purchases moved under responsibility of regional authorities and their health care authorities. At that, powers of three former state organizations being occupied with purchases of imported medications were distributed between new state companies of Ministry of Health and commercial organizations and firms that entered pharmaceutical market. This diversity rather soon gave rise to competitive fight for budget funds and experiments of Government with market regulation. The traces of these clashes and attempts to make market regulated can be found in Ministry of Health archives and journal publications. The Report I reveals circumstances of entrance of Ministry of Health into market relationships and its acquisition of market player identity.

Evaluating Unmet Needs in Large-Volume Subcutaneous Drug Delivery: US Payer Perspectives on a Novel, Large-Volume On-Body Delivery System.

This research sought to evaluate payer perspectives around OBDS's through semi-structured interviews with 17 US payers representing approximately 227 million covered lives. When asked about the perceived advantages of the novel on-body delivery system (OBDS), 70.6% of payers independently cited simplicity, ease-of-use, and convenience as the most beneficial features, with the potential to replace IV infusion by facilitating at-home HCP- or self-administration being the second most frequent response. Most payers (88.2%) believed that the novel OBDS could fulfill unmet needs such as cost of IV infusion, convenient administration, and improved patient compliance in large-volume SC delivery by enabling safe, easy, self-administration. Nearly all payers (88.2%) stated they would consider covering and managing pharmaceutical products packaged with the novel OBDS, with the remaining payers considering the total cost compared to other routes. A significant proportion of payers (82.4%) acknowledged that a drug delivered via the novel OBDS could warrant a price premium above the cost of the standalone SC drug vial, with half stating the premium would range from 5% to 20% and the other half citing an unspecified premium dependent on the individual drug situation.Conclusions: Given the ability to help address critical unmet needs for the patient and healthcare system, a large proportion of the payers stated that the novel OBDS would fulfill unmet needs and warrant a price premium versus the cost of the standalone SC vial and certainly over the IV counterpart. Future research to quantify the value that OBDS efficiencies could bring to healthcare delivery is warranted.

Predicting pharmaceutical prices. Advances based on purchase-level data and machine learning.

BACKGROUND: Increased costs in the health sector have put considerable strain on the public budgets allocated to pharmaceutical purchases. Faced with such pressures amplified by financial crises and pandemics, national purchasing authorities are presented with a puzzle: how to procure pharmaceuticals of the highest quality for the lowest price. The literature explored a range of impactful factors using data on producer and reference prices, but largely foregone the use of data on individual purchases by diverse public buyers. METHODS: Leveraging the availability of open data in public procurement from official government portals, the article examines the relationship between unit prices and a host of predictors that account for policies that can be amended nationally or locally. The study uses traditional linear regression (OLS) and a machine learning model, random forest, to identify the best models for predicting pharmaceutical unit prices. To explore the association between a wide variety of predictors and unit prices, the study relies on more than 200,000 purchases in more than 800 standardized pharmaceutical product categories from 10 countries and territories. RESULTS: The results show significant price variation of standardized products between and within countries. Although both models present substantial potential for predicting unit prices, the random forest model, which can incorporate non-linear relationships, leads to higher explained variance (R2 = 0.85) and lower prediction error (RMSE = 0.81). CONCLUSIONS: The results demonstrate the potential of i) tapping into large quantities of purchase-level data in the health care sector and ii) using machine learning models for explaining and predicting pharmaceutical prices. The explanatory models identify data-driven policy interventions for decision-makers seeking to improve value for money.

The impact of centralized band purchasing of pharmaceuticals on innovation of Chinese pharmaceutical firms: an empirical study based on double difference models.

In 2018, China began to gradually promote the pilot policy of centralized band purchasing of medicines. Implementing this policy has resulted in a significant decrease in drug prices. However, there needs to be a clear consensus on the impact and mechanism of action on the innovation of pharmaceutical companies. Therefore. Taking the data of Chinese Shanghai and Shenzhen A-share pharmaceutical listed companies from 2016 to 2022 as a sample, this paper empirically investigates the impact of the centralized banded purchasing policy of drugs on the innovation of pharmaceutical enterprises by using a double difference model and further analyzes the mechanism of its action. The results show that implementing the centralized banded purchasing policy can promote pharmaceutical enterprises' innovation input and output, which is robust under the parallel trend and placebo tests. Further exploring the impact mechanism of the centralized band purchasing policy on pharmaceutical enterprises' innovation, it can be found that it promotes innovation inputs through three channels: government subsidies, enterprise profits, and operating income. In addition, the impact of centralized band purchasing on enterprise innovation is heterogeneous in terms of region, enterprise nature, and scale. Therefore, the positive effects of the centralized band purchasing policy on promoting innovation in pharmaceutical enterprises should be fully recognized, and enterprise heterogeneity should be taken into account when implementing the policy. This study provides empirical evidence on the implementation effect of the centralized banded purchasing policy and provides lessons for continuously optimizing the policy to promote the high-quality development of pharmaceutical enterprises.

Coping with drug shortages: A study of government-enterprise option cooperation stockpiling strategies for drugs in shortage considering API surrogate stockpiling subsidies.

Drug shortage is a global problem, and the development of government-enterprise cooperative stockpiles of drugs in shortage, combining physical and production capacity, has become one of the most important means of coping with drug shortages. However, existing studies have tended to overlook the fact that shortages of Active Pharmaceutical Ingredients (APIs) have become an important constraint on production capacity stockpiling and that the lack of incentives and provisions for coordination of benefits have led to a double marginal effect of joint stockpiling by government and enterprises of drugs in shortage. Accordingly, this study introduced the option contract to the drug supply system composed of government and pharmaceutical enterprises and used the subsidy of API storage in lieu as an important initiative to incentivize the reserve of APIs, to construct a model of shortage drug reserve under the government's leadership. This study aims to improve the effect of government-enterprise joint stockpiling of drugs in shortage, which is of great theoretical and practical significance. According to the classification of production license types of pharmaceutical enterprises, this study established a three-level supply chain decentralized decision-making model consisting of the government, formulation enterprises, and API enterprises, and a two-level supply chain centralized decision-making model consisting of the government and API Formulation (API-F) integrated enterprises, respectively. By solving the inverse order derivation, the government-enterprise option cooperation conditions and optimal decision-making strategy were derived. The study results showed that: (i) The addition of enterprise API stockpiling mode can help the government conventional reserves, and enterprise production capacity reserves, broaden the way of drug reserves, and improve the effect of government-enterprise option cooperation; (ii) when the probability of drug shortages is high, the government should prefer the cooperation of API-F integrated enterprises, which is conducive to reducing intermediate links and government costs and improving the supply responsiveness to shortages of medicines; (iii) Setting appropriate government subsidies for API storage can incentivize enterprises to stockpile APIs and improve drug production capacity and physical supply response capability. This study took the problem of socialized stockpiling of drugs in shortage as an entry point and explored the problems and solution strategies in the government-enterprise cooperative stockpiling of drugs in shortage, which not only made some theoretical contributions to the application of options contract in the government-enterprise cooperative stockpiling of drugs in shortage but also provided new ideas and theoretical basis for the improvement of the stockpiling work of drugs in shortage.

Cheaper is not always better: Drug shortages in the United States and a value-based solution to alleviate them.

Drug shortages threaten patients' access to medications and are associated with adverse health outcomes and increased costs. Drug shortages disproportionately occur among generic drugs of limited profitability, most notably drugs administered by injection. In this perspective, we discuss how reimbursement and purchasing practices that were meant to create an efficient marketplace for generics have generated strong price pressure that threatens profitability in certain markets. We further explain how, faced with limited profitability, manufacturers lack incentives to invest in resilient supply chains, and in some cases, engage in cost-containment strategies or decide to exit the market, ultimately contributing to shortages. We propose the development and implementation of value-based reimbursement to provide needed incentives for drug purchasers and manufacturers to establish a more reliable supply chain as part of the policy solution to reduce the number and extent of drug shortages. This reimbursement model would necessitate the development of a rating system that measures supply chain resilience and maturity for each generic product. This rating would then be applied as a value-based modifier to reimbursement rates for generic products. The proposed model would result in higher reimbursement rates for generic products from more dependable supply chains, generating incentives for manufacturers to invest in supply chain resiliency. We propose the application of this reimbursement system originally in Medicare given Congressional interest on reforming Medicare payment to prevent drug shortages.

Misaligned Pharmacy Incentives in Value-Based Care.

This Viewpoint explains some ways in which the alignment of incentives between payers and clinicians in value-based care (VBC) arrangements can be interrupted and proposes a multifaceted approach to realigning incentives for drug spending within VBC contracts to better provide value-based care and improve patient outcomes.

A conceptual framework to build effective medicine pricing policies for low and middle-income countries (LMICs).

Building effective medicine pricing policies is a challenging task in all high-, middle- and low-income countries. In high-income countries have stable health systems in place, trained workforce, as well as specialised agencies to evaluate innovative drugs for reimbursement. However, in low- and middle-income countries (LMICs) the challenges are manifold. A large majority of low- and middle-income countries lack technical expertise either to evaluate new medicines or to set efficient medicine prices. The countries also lack comparator reference pricing data to set prices. Also, there are significant out-of-pocket expenditures as people pay for medicines by themselves. An effective medicine pricing policy is vital in the context that it can be used as a tool to improve access and affordability among the masses. This discussion paper presents a conceptual framework to build effective medicine pricing policies for low and middle-income countries (LMICs). The enablers to build effective medicine pricing policies are also discussed. This includes (a) data and statistics on the pharmaceutical situation of the country (b) Having a national medicine policy in the country (c) The availability of the medicine pricing data and (d) Human resources and technical capacity. WHO has recommended several strategies including External Price Referencing (EPR), Internal Reference pricing, Value-based pricing, Cost-plus pricing, and Generic medicine policies to build a pricing policy. However, this information is generic and it's a complex task for countries to tailor to their needs, hence a critical analysis is provided on these policies. The concepts related to fair medicine pricing, providing information to consumers and price transparency are also discussed.

A proposed analytical framework for qualitative evaluation of access to medicines from a health systems perspective.

OBJECTIVE: Despite global recognition that access to medicines is shaped by various interacting processes within a health system, a suitable analytical framework for identifying barriers and facilitators from a system's perspective was needed. We propose a framework specifically designed to find drivers to access to medicines from a country's health system perspective. This framework could enable the systematic evaluation of access across countries, disease areas and populations and facilitate targeted policy development. This framework is the byproduct of a larger study on the barriers and facilitators to childhood oncology medicines in South Africa. RESULTS: Eight core (pharmaceutical) functional processes were identified from existing frameworks: (I) medicine regulation, (II) public financing and pricing, (III) selection, (IV) reimbursement, (V) procurement and supply, (VI) healthcare delivery, (VII) dispensing and (VIII) use. National contextual components included policy and legislation and health information systems. To emphasize the interlinkage of processes, the proposed framework was structured as a pharmaceutical value chain. This framework focusses on national processes that are within a country's control as opposed to global factors, and functional mechanisms versus a country's performance or policy objectives. Further refinement and validation of the framework following application in other contexts is encouraged.

The Impact of Serialisation on Operational Efficiency and Productivity in Irish Pharmaceutical Sites.

Technology enabling drug serialisation technology was introduced by regulators to enhance security in pharmaceutical supply chain and protect drugs from infiltration by falsified and substandard medicines. The introduction of systems for serialisation required huge financial outlays manufacturers of pharmaceuticals. This study investigated the impact of serialisation on the operational efficiency and productivity in Irish pharmaceutical sites. A qualitative study was conducted with 11 manufacturing sites in Ireland. The participating companies operated a total of 114 pack-lines, representing approximately 65% of the automated packing lines in the country. The study found that serialisation had a negative effect on packaging production line OEE and line availability and on the individuals cost per unit of packaged pharmaceuticals. The research results estimated that the capital costs of serialisation were four times greater than those estimated by the regulators. There was a 4.1 cents average cost per pack for serialisation with high volume sites reporting an annual cost of serialisation of up to €4.5 m per annum and a 2.7% increase in the average cost of goods sold. A pattern whereby where many pharmaceutical manufacturers are transitioning from smaller batch production and moving toward larger batch production sizes in order to increases efficiencies was identified. The research also proposed the use of a serialisation depreciation factor as a method to determine the impact of serialisation on the cost of goods sold. This is the first study of its kind into the cost of serialisation from a manufacturer's viewpoint and studying the effects of serialisation on productivity, line availability and operational efficiency.

Citizens' views on prices of medicines reimbursed by the National Health Service: Findings from Italian online focus groups.

INTRODUCTION: Access to medicines is one of the biggest challenges to health systems, affecting society and individuals. This study aims to explore citizens' opinions, perceptions and attitudes on the model of medicines' research and development (R&D) and price setting of medicines reimbursed by the Italian National Health Service. MATERIALS AND METHODS: We run four online focus groups, analysed through thematic analysis. INCLUSION CRITERIA: people aged 30-70 years, who had completed at least compulsory schooling (8-10 years), with no specialised knowledge about the subject. EXCLUSION CRITERIA: healthcare workers, pharmaceutical and device industry employees, researchers and medicine policy board members. We aimed to include a purposive sample of 20 participants, variable in terms of age, educational level and place of residence. RESULTS: Eleven women and six men participated. The mean age was 53 years (range: 28-73). Most (n = 15) had a university degree or attended secondary schools. Eight had a job, five were not employed, and four were retired. In general, participants supported the role of the public health service. Almost all had limited knowledge of medicines' R&D and price setting. Most asked for transparency on medicine prices and negotiation criteria. Participants considered revenues of pharmaceutical companies disproportionate and most called for containment measures of profits. Most were in favour of a stronger public intervention in R&D and prices' negotiations. Few were sceptical of the public sector's ability to play this role. DISCUSSION: Medicines' prices were discussed as a health matter. Increasing citizens' awareness of these topics is needed by providing spaces and conditions to participate in the discussion, including different perspectives and interests. PATIENT OR PUBLIC CONTRIBUTION: Members of BEUC-the European consumer organisation-proposed the project. Altroconsumo, an independent consumer organisation and OCU, a Spanish consumer organisation, participated in developing the project and the main topics to discuss. The Mario Negri Institute and Aplica cooperative-the Spanish methodological team-were involved by BEUC and their national organisations to define the methodology, organisational aspects and contents and conducted the focus groups.

Why are our medicines so expensive? Spoiler: Not for the reasons you are being told .

Often described as a natural economic trend, the prices that pharmaceutical companies charge for new medicines have skyrocketed in recent years. Companies claim these prices are justified because of the 'value' new treatments represent or that they reflect the high costs and risks associated with the research and development process. They also claim that the revenues generated through these high prices are required to pay for continued innovation.This paper argues that high prices are not inevitable but the result of a societal and political choice to rely on a for-profit business model for medical innovation, selling medicines at the highest price possible. Instead of focusing on therapeutic advances, it prioritises profit maximisation to benefit shareholders and investors over improving people's health outcomes or equitable access.As a result, people and health systems worldwide struggle to pay for the increasingly expensive health products, with growing inequities in access to even life-saving medicines while the biopharmaceutical industry and its financiers are the most lucrative business sectors.As the extreme COVID-19 vaccine inequities once again highlighted, we urgently need to reform the social contract between governments, the biopharmaceutical industry, and the public and restore its original health purpose. Policymakers must redesign policies and financing of the pharmaceutical research and development ecosystem such that public and private sectors work together towards the shared objective of responding to public health and patients' needs, rather than maximising financial return because medicines should not be a luxury.

Portuguese Global Medicines Access Index 2021: An Indicator to Measure Access to Hospital Medicines.

OBJECTIVES: Access to innovative and effective medication is a citizen's right. The main objectives of this study were to build an indicator to measure access to medicines within hospitals, the Global Medicines Access Index, and to identify the main existing barriers. METHODS: Cross-sectional study carried out in Portuguese National Health Service hospitals. A consensus methodology (expert panel of 7 members) was used to define which dimensions should be included in the index and the weighting that each should take. The panel identified 6 dimensions: access to innovative medicines, proximity distribution, shortages, access to medicines before financing decision, value-based healthcare, and access to medication depending on cost/funding. Data were collected through an electronic questionnaire (September 2021). RESULTS: The response rate was 61.2%. Most hospitals used medicines with and without marketing authorization before the funding decision. Monitoring and generating evidence of new therapies results is still insufficient. The identified barriers were the administrative burden as the major barrier in purchasing medicines, with a relevant impact on shortages of medicines. Most respondents (87%) had a proximity distribution program, mainly implemented in the pandemic context, and the price/funding model was only identified by 10% as a barrier to access. The 2021 Global Medicines Access Index was 66%. Shortages and value-based use of medicines were the dimensions that had more influence in lowering the index value. CONCLUSIONS: The new formula used to obtain a unique and multidimensional index for access to hospital medicines seems to be more sensitive and objective and will be used to monitor access.