Reforming the World Health Organization's Essential Medicines List: Essential but Unaffordable.

This Viewpoint proposes restructuring the WHO Essential Medicines List to remove consideration of cost and cost-effectiveness from the expert committee reviews of clinical effectiveness, safety, and public health value, and chartering a new framework for pooled global negotiation and procurement of costly medicines included in the list.

Evaluating essential medicines for treating childhood cancers: availability, price and affordability study in Ghana.

INTRODUCTION: Access to childhood cancer medicines is a critical global health challenge. There is a lack of sufficient context-specific data in Ghana on access to essential medicines for treating childhood cancers. Here, we present an analysis of essential cancer medicine availability, pricing, and affordability using the pediatric oncology unit of a tertiary hospital as the reference point. METHOD: Data on prices and availability of 20 strength-specific essential cancer medicines and eight non-cancer medicines were evaluated using the modified World Health Organization (WHO)/Health Action International method. Two pharmacies in the hospital and four private pharmacies around the hospital were surveyed. We assessed their median price ratio using the WHO international reference price guide. The number of days wages per the government daily wage salary was used to calculate the affordability of medicines. RESULTS: The mean availability of essential cancer medicines and non-cancer medicines at the hospital pharmacies were 27 and 38% respectively, and 75 and 84% respectively for private pharmacies. The median price ratio of cancer medicines was 1.85, and non-cancer medicines was 3.75. The estimated cost of medicines for treating a 30 kg child with Acute lymphoblastic leukaemia was GHÈ» 4928.04 (US$907.56) and GHÈ» 4878.00 (US$902.62) for Retinoblastoma, requiring 417 and 413-days wages respectively for the lowest-paid unskilled worker in Ghana. CONCLUSION: The mean availability of cancer medicines at the public and private pharmacies were less than the WHO target of 80%. The median price ratio for cancer and non-cancer medicines was less than 4, yet the cost of medicines appears unaffordable in the local setting. A review of policies and the establishment of price control could improve availability and reduce medicines prices for the low-income population.

Importance of medicine quality in achieving universal health coverage.

OBJECTIVE: To assess the importance of ensuring medicine quality in order to achieve universal health coverage (UHC). METHODS: We developed a systems map connecting medicines quality assurance systems with UHC goals to illustrate the ensuing impact of quality-assured medicines in the implementation of UHC. The association between UHC and medicine quality was further examined in the context of essential medicines in low- and middle-income countries (LMICs) by analyzing data on reported prevalence of substandard and falsified essential medicines and established indicators for UHC. Finally, we examined the health and economic savings of improving antimalarial quality in four countries in sub-Saharan Africa: the Democratic Republic of the Congo (DRC), Nigeria, Uganda, and Zambia. FINDINGS: A systems perspective demonstrates how quality assurance of medicines supports dimensions of UHC. Across 63 LMICs, the reported prevalence of substandard and falsified essential medicines was found to be negatively associated with both an indicator for coverage of essential services (p = 0.05) and with an indicator for government effectiveness (p = 0.04). We estimated that investing in improving the quality of antimalarials by 10% would result in annual savings of $8.3 million in Zambia, $14 million in Uganda, $79 million in two DRC regions, and $598 million in Nigeria, and was more impactful compared to other potential investments we examined. Costs of substandard and falsified antimalarials per malaria case ranged from $7 to $86, while costs per death due to poor-quality antimalarials ranged from $14,000 to $72,000. CONCLUSION: Medicines quality assurance systems play a critical role in reaching UHC goals. By ensuring the quality of essential medicines, they help deliver effective treatments that lead to less illness and result in health care savings that can be reinvested towards UHC.

Newborn Service Readiness of Primary Level Health Facilities of Eastern Mountain Region of Nepal.

BACKGROUND: Newborn service readiness is facility's observed capacity to provide newborn services and a pre-requisite for quality. Newborn services are priority program of government and efforts are focused on infrastructure and supplies at peripheral health facilities. Study describes health facility readiness for newborn services in four domains of general requirements, equipment, medicines and commodities, and staffing and guidelines. METHODS: Convergent parallel mixed method using concurrent triangulation was done in public health facilities providing institutional deliveries of two randomly selected districts- Taplejung and Solukhumbu of Eastern Mountain Region of Nepal. Face to face interview and observation of facilities were done using structured questionnaire and checklist; in-depth interviews were done using interview guideline from November 2016 to January 2017. Ethical clearance was taken. Descriptive analysis and deductive thematic analysis were done. RESULTS: Mean score of newborn service readiness was 68.7±7.1 with range from 53.3 to 81.4 out of 100. Domains of general requirement, equipment, medicine and commodity, supervision, staffing and guideline were assessed. The gaps identified in general requirements were availability of uninterrupted power supply, means of communication and referral vehicle. Clean wrappers and heater for room temperature maintenance were identified during interviews to be part of the readiness. All health facilities had trained staff while retention of skill was of concern. There was felt need of enforcing adequate training coverage to suffice the need of human resources in remote. CONCLUSIONS: Efforts of improving transportation, heater for room temperature maintenance, trainings with skill retention strategy, utilization of guidelines, availability of skilled birth attendance could result increased and improved newborn service readiness.

Access to medicines through health systems in low- and middle-income countries.

Nearly 2 billion people globally have no access to essential medicines. This means essential medicines are unavailable, unaffordable, inaccessible, unacceptable or of low quality for more than a quarter of the population worldwide. This supplement demonstrates the implications of poor medicine access and highlights recent innovations to improve access to essential medicines by presenting new research findings from low- and middle-income countries (LMICs). These studies answer key questions such as: Can performance-based financing improve availability of essential medicines? How affordable are cardiovascular treatments for children? Which countries' legal frameworks promote universal access to medicines? How appropriately are people using medicines? Do poor-quality medicines impact equity? Answers to these questions are important as essential medicines are vital to the Sustainable Development Goals and are central to the goal of achieving Universal Health Coverage. Access to affordable, quality-assured essential medicines is crucial to reducing the financial burden of care, preventing greater pain and suffering, shortening the duration of illness, and averting needless disabilities and deaths worldwide. This supplement was organized by the Medicines in Health Systems Thematic Working Group of Health Systems Global, a membership organization dedicated to promoting health systems research and knowledge translation. The five studies in the supplement further our understanding by showcasing recent successes and challenges of improving access to quality-assured medicines through health systems in LMICs.

Acceptability and feasibility of a national essential medicines list in Canada: a qualitative study of perceptions of decision-makers and policy stakeholders.

BACKGROUND: Policy approaches have been considered to address inconsistent and inequitable prescription drug coverage in Canada, including a national essential medicines list. We sought to explore key factors influencing the acceptability and feasibility of an essential medicines list in Canada. METHODS: We conducted semi-structured interviews with decision-makers and other key stakeholders from government or pan-Canadian institutions, civil society and the private sector across Canada. We analyzed data using inductive thematic analysis and by applying Kingdon's Multiple Streams Framework to analyze the emergent themes deductively. RESULTS: We conducted 21 interviews before thematic saturation was achieved. We categorized emergent themes to describe the problem, the essential medicines list policy (including content and process), and politics. There was consensus among participants that prescription drug coverage was an important problem to address. Participants differed in their views on how to define essential medicines and concerns about what would be excluded from an essential medicines list. There was consensus on important features for a process to develop an essential medicines list: an independent decision-making body, use of defined selection criteria based on quality evidence, and clear communication of the purpose of the essential medicines list. Federal government financing and the broader pharmacare model, engagement of various interest groups and changing political agendas emerged as core political factors to consider if developing a Canadian essential medicines list. INTERPRETATION: Although stakeholders' views on the content of a Canadian essential medicines list varied, there was consensus on the process to formulate and implement an essential medicines list or common national formulary, including choosing medicines based on best evidence. Greater understanding is now needed on how patients, clinicians and the public perceive the concept of an essential medicines list.

Added therapeutic value of new drugs approved in Brazil from 2004 to 2016 / Valor terapêutico acrescentado de novos medicamentos aprovados no Brasil de 2004 a 2016 / Valor terapéutico añadido de los nuevos medicamentos aprobados en Brasil desde 2004 a 2016

Abstract: This study aimed to assess the level of therapeutic innovation of new drugs approved in Brazil over 13 years and whether they met public health needs. Comparative descriptive analysis of therapeutic value assessments performed by the Brazilian Chamber of Drug Market Regulation (CMED) and the French drug bulletin Prescrire for new drugs licensed in Brazil, from January 1st 2004 to December 31st 2016. The extent to which new drugs met public health needs was examined by: checking inclusions into government-funded drug lists and/or clinical guidelines; comparing Anatomical Therapeutic Chemical Classification (ATC) codes and drug indications with the list of conditions contributing the most to the national disease burden; and assessing new medicines aimed to treat neglected diseases. 253 new drugs were approved. Antineoplastics, immunossupressants, antidiabetics and antivirals were the most frequent. Thirty-three (14%) out of 236 drugs assessed by the Brazilian chamber and sixteen (8.2%) out of 195 assessed by the French bulletin Prescrire were considered innovative. Thirty-six drugs (14.2%) were selected for coverage by the Brazilian Unified National Health System (SUS), seven of which were therapeutically innovative, and none were aimed to treat neglected disease. About 1/3 of the drugs approved aimed to treat conditions among the top contributors to Brazil's disease burden. Few therapeutically innovative drugs entered the Brazilian market, from which only a small proportion was approved to be covered by the SUS. Our findings suggest a divergence between public health needs, research & development (R&D) and drug licensing procedures.

Resumo: O objetivo foi avaliar o nível de inovação terapêutica de novos medicamentos aprovados no Brasil ao longo de 13 anos e se eles atendem a necessidades de saúde pública. Foi feita uma análise comparativa descritiva da avaliação de valor terapêutico realizada pela Câmara de Regulação do Mercado de Medicamentos (CMED) e pelo boletim de medicamentos francês Prescrire para novos medicamentos licenciados no Brasil entre 1º de janeiro de 2004 e 31 de dezembro de 2016. Examinamos em que medida os novos medicamentos atendem a necessidade de saúde pública por meio de: checagem da inclusão em listas de medicamentos financiados pelo governo e/ou diretrizes clínicas; comparação de códigos da Classificação Anatômica Terapêutica Química (ATC, em inglês) e indicações de medicamentos com a lista de condições que mais contribuem para a carga de doença nacional; e avaliação de se os novos medicamentos tinham por objetivo tratar doenças negligenciadas. Foram aprovados 253 novos medicamentos. Antineoplásicos, imunossupressores, antidiabéticos e antivirais foram os mais frequentes. Trinta e três (14%) dos 236 medicamentos avaliados pela Câmara brasileira e 16 (8,2%) dos 195 avaliados pelo boletim francês Prescrire foram considerados inovadores. Trinta e seis medicamentos (14,2%) foram selecionados para cobertura no Sistema Único de Saúde (SUS), sete dos quais eram inovadores do ponto de vista terapêutico e nenhum dos quais tinha por objetivo tratar uma doença negligenciada. Em torno de 1/3 dos medicamentos aprovados tinha por objetivo o tratamento de doenças que figuram entre as principais contribuidoras da carga de doença no Brasil. Poucos medicamentos inovadores do ponto de vista terapêutico entraram no mercado brasileiro, dos quais apenas uma pequena proporção foi aprovada para ser coberta pelo SUS. Nossos resultados sugerem uma divergência entre necessidades de saúde pública, pesquisa e desenvolvimento (P&D) e procedimentos de licenciamento de medicamentos.

Resumen: El objetivo fue evaluar el nivel de innovación terapéutica de los nuevos medicamentos aprobados en Brasil durante 13 años y si cumplen con las necesidades sanitarias. Llevamos a cabo un análisis comparativo descriptivo acerca del valor terapéutico presente en las evaluaciones realizadas por la Cámara de Regulación del Mercado de Medicamentos (CMED) y la revista francesa Prescrire sobre los nuevos medicamentos autorizados en Brasil, desde el 1º de enero 2004 hasta el 31de diciembre de 2016. Su alcance, es decir, hasta qué punto los nuevos medicamentos cumplían con las necesidades de salud pública se comprobaron revisando las inclusiones en listas de medicamentos subvencionados por el gobierno y/o directrices clínicas; comparando los códigos de la Classificación Anatómicos Terapéuticos Químicos (ATC por sus siglas en inglés) y las indicaciones de los medicamentos respecto a la lista de enfermedades que contribuían a la mayor carga de morbilidad nacional; y asesorando si los nuevos medicamentos tenían como objetivo tratar enfermedades desatendidas. Se aprobaron 253 nuevos medicamentos. Los antineoplásicos, inmunosupresores, antidiabéticos y antivirales fueron los más frecuentes. Treinta y tres (14%), aparte de los 236 medicamentos evaluados por la Cámara Brasileña, y 16 (8,2%), aparte de los 195 evaluados por la revista francesa Prescrire, se consideraron innovadores. Treinta y seis medicamentos (14,2%) se seleccionaron para que tuvieran cobertura por el Sistema Único de Salud (SUS), siete de ellos eran terapéuticamente innovadores, y ninguno tenía como meta tratar enfermedades desatendidas. Alrededor de 1/3 de las medicinas aprobadas tenían como meta tratar problemas de salud entre las enfermedades con mayor carga de morbilidad en Brasil. Pocos medicamentos terapéuticamente innovadores accedieron al mercado brasileño y de éstos sólo una pequeña parte fueron aprobados para que fueran cubiertos por el SUS. Nuestros resultados sugieren una divergencia entre las necesidades públicas de salud, investigación & desarrollo (I&D) y los procedimientos para la autorización de medicamentos.

How the Uruguayan Judiciary Shapes Access to High-Priced Medicines: A Critique through the Right to Health Lens.

Uruguay has witnessed an ever-increasing number of domestic court claims for high-priced medicines despite its comprehensive universal coverage of pharmaceuticals. In response to the current national debate and development of domestic legislation concerning high-priced medicines, we review whether Uruguayan courts adequately interpret the state's core obligations to provide essential medicines and ensure non-discriminatory access in line with the right to health in the International Covenant on Economic, Social and Cultural Rights. Using a sample of 42 amparo claims for the reimbursement of medicines in 2015, we found that the circuits of appeal fail to offer predictable legal argumentation, including for nearly identical cases. Moreover, the judiciary does not provide an interpretation of state obligations that is consistently aligned with the right to health in the International Covenant on Economic, Social and Cultural Rights. These findings illustrate that medicines litigation in Uruguay offers relief for some individual claims but may exacerbate systemic inequalities by failing to address the structural problems behind high medicines prices. We recommend that the judiciary adopt a consistent standard for assessing state action to realize the right to health within its available resources. Moreover, the legislature should address the need for medicines price control and offer a harmonized interpretation of the right to health. These transformations can increase the transparency and predictability of Uruguay's health and legal systems for patients and communities.

Insights from the WHO and National Lists of Essential Medicines: Focus on Pediatric Diabetes Care in Africa.

BACKGROUND: Access to essential medicines in pediatric endocrinology and diabetes is limited in resource-limited countries. The World Health Organization (WHO) maintains two non-binding lists of essential medicines (EMLs) which are often used as a template for developing national EMLs. METHODS: We compared a previously published master list of medicines for pediatric endocrinology and diabetes with the WHO EMLs and national EMLs for countries within the WHO African region. To better understand actual access to medicines by patients, we focused on diabetes and surveyed pediatric endocrinologists from 5 countries and assessed availability and true cost for insulin and glucagon. RESULTS: Most medicines that are essential in pediatric endocrinology and diabetes were included in the national EMLs. However, essential medicines, such as fludrocortisone, were present in less than 30% of the national EMLs despite being recommended by the WHO. Pediatric endocrinologists from the 5 focus countries reported significant variation in terms of availability and public access to insulin, as well as differences between urban and rural areas. Except for Botswana, glucagon was rarely available. There was no significant relationship between Gross National Income and the number of medicines included in the national EMLs. CONCLUSIONS: Governments in resource-limited countries could take further steps to improve EMLs and access to medicines such as improved collaboration between health authorities, the pharmaceutical industry, patient groups, health professionals, and capacity-building programs such as Paediatric Endocrinology Training Centres for Africa.

Parents' and guardians' perceptions on availability and pricing of medicines and healthcare for children in eThekwini, South Africa - a qualitative study.

BACKGROUND: Inadequate access to affordable essential medicines poses a challenge to achieving Universal Health Coverage. Access to essential medicines for children has been in the spotlight in recent research. However, information from the end users of medicines, i.e. patients is scarce. Obtaining information at a household level is integral to understanding how people access, obtain and use medicines. This study aimed to gather opinions and perceptions from parents/guardians on availability, affordability and quality of medicines and healthcare for children in SA. METHODS: Eight Focus group discussions were held with 41 individuals in eThekwini, South Africa (SA), from September-November 2016. Participants were parents/guardians of children up to 12 years from different ethnicities, ages, gender, and socio-economic backgrounds. Key informants identified by the principal researcher recruited participants using snowball sampling. Focus group discussions were recorded, transcribed verbatim, coded by the first author, verified by the second author, reconciled for consensus and imported into NVIVO for data analysis. RESULTS: Medicines and healthcare facilities are accessible in urban and peri-urban areas in eThekwini. Medicines may not always be available in public sector facilities due to medicine shortages, compelling parents to purchase medicines from private sector pharmacies. Common medicines were perceived as affordable for most socio-economic groups except the 'Poor' group. Quality of medicines was perceived as 'good' especially if obtained from the private sector but sometimes perceived as 'poor' and viewed with suspicion when received from public sector clinics. Quality of healthcare was perceived as 'good' but requires improvement for both sectors. CONCLUSIONS: This is the first study in SA to report on parent/guardian perceptions on availability, affordability and quality of medicines and healthcare for children. It has the potential to be up-scaled to a country-wide investigation to paint a national picture of parents' opinions of healthcare for children. This will allow for patient input into pharmaceutical and healthcare policy governing access to and availability of essential medicines and services within the country. The study recommends that patient input be sought to assess impact of policies on the intended target group in the country to ensure that the policy objectives are achieved.

Is Compulsory Licensing Bad for Public Health? Some Critical Comments on Drug Accessibility in Developing Countries.

As one of the flexibilities provided by the agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPS) establishing minimum standards for the protection of property rights, compulsory license (CL) represents a means towards the protection of public health issues within a context of stringent protection of intellectual property rights (IPRs), most notably in poor-resource settings. However, recent literature asserts that CL constitutes a serious limitation to the full enjoyment of property rights by innovators and may therefore threaten drug accessibility in developing countries. This paper outlines the impact of CL on drug accessibility in developing countries by addressing the three main dimensions of accessibility (availability, affordability and quality) and proceeding to a literature survey of key arguments for and against CL. It concludes that CL inhibits neither the availability of essential drugs nor the affordability of life-saving treatments or the supply of high-quality drugs in developing countries, in particular antiretroviral drugs.

Essential Medicines in National Constitutions: Progress Since 2008.

A constitutional guarantee of access to essential medicines has been identified as an important indicator of government commitment to the progressive realization of the right to the highest attainable standard of health. The objective of this study was to evaluate provisions on access to essential medicines in national constitutions, to identify comprehensive examples of constitutional text on medicines that can be used as a model for other countries, and to evaluate the evolution of constitutional medicines-related rights since 2008. Relevant articles were selected from an inventory of constitutional texts from WHO member states. References to states' legal obligations under international human rights law were evaluated. Twenty-two constitutions worldwide now oblige governments to protect and/or to fulfill accessibility of, availability of, and/or quality of medicines. Since 2008, state responsibilities to fulfill access to essential medicines have expanded in five constitutions, been maintained in four constitutions, and have regressed in one constitution. Government commitments to essential medicines are an important foundation of health system equity and are included increasingly in state constitutions.

An evaluation of the Essential Medicines List, Standard Treatment Guidelines and prescribing restrictions, as an integrated strategy to enhance quality, efficacy and safety of and improve access to essential medicines in Papua New Guinea.

The World Health Organization (WHO) has advocated the development and use of country specific Standard Treatment Guidelines (STGs) and Essential Medicines Lists (EML) as strategies to promote the rational use of medicines. When implemented effectively STGs offer many health advantages. Papua New Guinea (PNG) has official STGs and a Medical and Dental Catalogue (MDC) which serves as a national EML for use at different levels of health facilities. This study evaluated consistency between the PNG Adult STGs (2003 and 2012) and those for children (2005 and 2011) with respect to the MDCs (2002, 2012) for six chronic and/or acute diseases: asthma, arthritis, diabetes, hypertension, pneumonia and psychosis. Additionally, the potential impact of prescriber level restrictions on rational medicines use for patient's living in rural areas, where no medical officer is present, was evaluated. Almost all drugs included in the STGs for each disease state evaluated were listed in the MDCs. However, significant discrepancies occurred between the recommended treatments in the STGs with the range of related medicines listed in the MDCs. Many medicines recommended in the STGs for chronic diseases had prescriber level restrictions hindering access for most of the PNG population who live in rural and remote areas. In addition many more medicines were listed in the MDCs which are commonly used to treat arthritis, high blood pressure and psychosis than were recommended in the STGs contributing to inappropriate prescribing. We recommend the public health and rational use of medicines deficiencies associated with these findings are addressed requiring: reviewing prescriber level restrictions; updating the STGs; aligning the MDC to reflect recommendations in the STGs; establishing the process where the MDC would automatically be updated based on any changes made to the STGs; and developing STGs for higher levels of care.

Essential Medicines in a High Income Country: Essential to Whom?

OBJECTIVE: To explore the perspectives of a diverse group of stakeholders engaged in medicines decision making around what constitutes an "essential" medicine, and how the Essential Medicines List (EML) concept functions in a high income country context. METHODS: In-depth qualitative semi-structured interviews were conducted with 32 Australian stakeholders, recognised as decision makers, leaders or advisors in the area of medicines reimbursement or supply chain management. Participants were recruited from government, pharmaceutical industry, pharmaceutical wholesale/distribution companies, medicines non-profit organisations, academic health disciplines, hospitals, and consumer groups. Perspectives on the definition and application of the EML concept in a high income country context were thematically analysed using grounded theory approach. FINDINGS: Stakeholders found it challenging to describe the EML concept in the Australian context because many perceived it was generally used in resource scarce settings. Stakeholders were unable to distinguish whether nationally reimbursed medicines were essential medicines in Australia. Despite frequent generic drug shortages and high prices paid by consumers, many struggled to describe how the EML concept applied to Australia. Instead, broad inclusion of consumer needs, such as rare and high cost medicines, and consumer involvement in the decision making process, has led to expansive lists of nationally subsidised medicines. Therefore, improved communication and coordination is needed around shared interests between stakeholders regarding how medicines are prioritised and guaranteed in the supply chain. CONCLUSIONS: This study showed that decision-making in Australia around reimbursement of medicines has strayed from the fundamental utilitarian concept of essential medicines. Many stakeholders involved in medicine reimbursement decisions and management of the supply chain did not consider the EML concept in their approach. The wide range of views of what stakeholders considered were essential medicines, challenges whether the EML concept is out-dated or underutilised in high income countries.