Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries.

Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022. Methods: A survey was distributed in July 2022 to representatives of 46 countries. Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs. Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe's Access to Novel Medicines Platform.

Trends in cost and consumption of essential medicines for non-communicable diseases in Azerbaijan, Georgia, and Uzbekistan, from 2019 to 2021.

BACKGROUND: Access to medicines is a global priority. Azerbaijan, Georgia, and Uzbekistan have different approaches to pricing policies for pharmaceuticals. The aim of this study was to analyze recent trends in the consumption and prices of non-communicable disease (NCD) medicines in Azerbaijan, Georgia, and Uzbekistan, in the outpatient setting. METHODS: We included medicines for asthma and COPD, cancer, cardiovascular disease, diabetes, epilepsy, and mental disorders. Sales data for pharmaceutical products in community pharmacies were extracted from a commercial database. Changes in consumption and prices were analyzed across all included NCD medicines, by disease category and pharmacological group. RESULTS: Consumption of NCD medicines was highest in Georgia, at twice the levels in Azerbaijan, and four times levels in Uzbekistan. Average prices of NCD medicines, weighted by consumption, increased by 26% in Georgia, but decreased by 3% in Azerbaijan and by 0.1% in Uzbekistan. Prices increased for all disease groups in Georgia (from +13% for epilepsy medicines to +86% for cancer), varied by group in Uzbekistan (from -22% for epilepsy medicines to +47% for cancer), while changes in Azerbaijan were smaller in magnitude (from -4% for medicines for cardiovascular disease to +11% for cancer). Cancer medicines had markedly higher prices in Uzbekistan, and asthma and COPD medicines had markedly higher prices in Azerbaijan and Uzbekistan. CONCLUSIONS: Georgia showed the highest outpatient consumption of NCD medicines, suggesting the broadest access to treatment. However, Georgia also saw marked price increases, greater than in the other countries. In Georgia, where there was no price regulation, widespread price increases and increases in consumption both contribute to increasing pharmaceutical expenditures. In Azerbaijan and Uzbekistan, increases in outpatient pharmaceutical expenditures were primarily driven by increases in consumption, rather than increases in price. Comparing trends in consumption and pricing can identify gaps in access and inform future policy approaches.

The role of intellectual property rights on access to medicines in the WHO African region: 25 years after the TRIPS agreement.

BACKGROUND: It is now 25 years since the adoption of the Agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPS) and the same concerns raised during its negotiations such as high prices of medicines, market exclusivity and delayed market entry for generics remain relevant as highlighted recently by the Ebola and COVID-19 pandemics. The World Health Organization's (WHO) mandate to work on the interface between intellectual property, innovation and access to medicine has been continually reinforced and extended to include providing support to countries on the implementation of TRIPS flexibilities in collaboration with stakeholders. This study analyses the role of intellectual property on access to medicines in the African Region. METHODS: We analyze patent data from the African Regional Intellectual Property Organization (ARIPO) and Organisation Africaine de la Propriété Intellectuelle (OAPI) to provide a situational analysis of patenting activity and trends. We also review legislation to assess how TRIPS flexibilities are implemented in countries. RESULTS: Patenting was low for African countries. Only South Africa and Cameroon appeared in the list of top ten originator countries for ARIPO and OAPI respectively. Main diseases covered by African patents were HIV/AIDS, cardiovascular diseases, cancers and tumors. Majority countries have legislation allowing for compulsory licensing and parallel importation of medicines, while the least legislated flexibilities were explicit exemption of pharmaceutical products from patentable subject matter, new or second use of patented pharmaceutical products, imposition of limits to patent term extension and test data protection. Thirty-nine countries have applied TRIPS flexibilities, with the most common being compulsory licensing and least developed country transition provisions. CONCLUSIONS: Opportunities exist for WHO to work with ARIPO and OAPI to support countries in reviewing their legislation to be more responsive to public health needs.

Impact of the COVID-19 pandemic on blood supply and demand in the WHO African Region.

BACKGROUND AND OBJECTIVES: The coronavirus disease 2019 (COVID-19) affected millions of people worldwide and caused disruptions at the global level including in healthcare provision. Countries of the WHO African region have put in place measures for the COVID-19 pandemic containment that may adversely affect blood system activities and subsequently reduce the supply and demand of blood and blood components. This study aims to assess the impact of the COVID-19 pandemic on blood supply and demand in the WHO African Region and propose measures to address the challenges faced by countries. MATERIALS AND METHODS: A survey questionnaire was sent to all 47 countries in the WHO African Region to collect information on blood supply and demand for the first 5 months of 2019 and 2020, respectively, and on COVID-19 Convalescent Plasma therapy in September 2020. RESULTS: Thirty-seven countries provided responses. The total number of blood donations dropped in 32 countries while it increased in five countries. The proportion of blood drives also decreased in 21 countries and increased in nine countries. The blood requested and issued for transfusion decreased for blood demand and for blood issued for transfusion in 30 countries. Ten countries reported some activities of convalescent plasma. However, very few units of this product collected have been transfused to COVID-19 patients. CONCLUSION: The COVID-19 pandemic has led to a reduction of blood related activities in the region, including the supply and demand. Countries preparedness plans for health emergencies need more emphasis to maintaining blood stock.

Prevention, Detection and Response to incidences of substandard and falsified medical products in the Member States of the Southern African Development Community.

BACKGROUND: Medical products are an integral and pivotal part of health care delivery. They need to be available, affordable and quality-assured. The SADC region is prone to threats arising from the availability and use of substandard and falsified (SF) medical products. This is something that needs to be actively addressed. METHOD: A survey, constructed around four themes, was carried out between September 2018 and January 2019. The National Medicines Regulatory Authorities (NMRAs) of the 16 Member States within the SADC region were asked to respond to the survey questionnaire. The objective was to map existing fameworks, mechanisms and approaches to prevention, detection and response (PDR) to SF medical products. RESULTS: Responses were received from twelve out of the sixteen NMRAs. Only three of the twelve respondents had included elements for PDR for SF medical products in their national medicine policies. Regardless of the status in terms of policies, legislation is however in place for the majority of NMRAs. The mandate for regular sampling, an important detection mechanism, was enshrined in the legislation of nine of the twelve respondents. In terms of response mechanisms, six of the respondents had both inter-agency and intra-agency co-ordination for responding to SF products. CONCLUSION: Though findings point to some deficiencies in terms of policies and implementation plans, the majority of countries have the mandate and legislation to deal with substandard and falsified medical products. Effective enforcement requires more investments into human resources, infrastructure, stakeholder coordination and public outreach. WHO has an important source of actionable information about incidience of substandard and falsified medical products. It needs to be leveraged to improve outreach to stakeholders and to raise awareness about SF problem and mechanisms available to address it. The extent, to which mechanisms and procedures are in place, varies. Some elements of the desired approach exist in the region; however, they will benefit from targeted strengthening to ensure a holistic approach across 12 action areas recommended by WHO.

A review of policies and programmes for human organ and tissue donations and transplantations, WHO African Region.

Several resolutions, endorsed by the World Health Assembly and the United Nations General Assembly, articulate the need to improve the availability, quality and safety of organ and tissue donation and transplantation, as well as to prevent and combat trafficking in human organs. Here we assessed the implementation of these resolutions pertaining to organ and tissue donations and transplantations by sending out a questionnaire to all 47 countries in the World Health Organization African Region. From 33 countries that provided data, we identified several obstacles and challenges. Compared to other regions, there are very limited data on organ donation and transplantation. Most countries are lacking legal and regulatory frameworks, since they did not yet establish a specific or comprehensive legislation covering donation and transplantation of human organs and tissues. Countries also have a poor national capacity to perform organ and tissue transplantations and the organization and management of national programmes are weak. Funding, both from domestic and external sources, is insufficient to implement effective transplantations programmes and patients have inadequate financial protection. To address these challenges, we propose that countries and partners should develop and implement policies, strategies, plans and regulatory frameworks for all aspects of organ and tissue donations and transplantations, including fighting against organ trafficking and transplant tourism. Where donation and transplantation programmes exist, stakeholders should develop the skills of human resources, adopt technical standards and quality management procedures to improve donation and transplantation of human organs and tissues.

De nombreuses résolutions approuvées par l'Assemblée mondiale de la Santé et l'Assemblée générale des Nations Unies ont souligné le besoin d'améliorer la disponibilité, la qualité et la sécurité des dons et de transplantations d'organes et de tissus humains, de même que celui d'assurer la prévention et la lutte contre le trafic d'organes. Dans ce document, nous avons étudié la mise en œuvre de ces résolutions relatives aux dons et transplantations d'organes et de tissus, par le biais d'un questionnaire envoyé aux 47 pays appartenant à la région Afrique de l'Organisation mondiale de la Santé. Nous avons identifié de nombreux obstacles et défis dans les 33 pays qui nous ont transmis des données. Par rapport à d'autres régions, il existe très peu d'informations à ce propos. La plupart des pays ne possèdent pas de cadres juridiques et réglementaires car ils n'ont pas encore établi de législation spécifique ou exhaustive couvrant les dons et transplantations d'organes et de tissus. Certains manquent également de moyens au niveau national pour réaliser des greffes d'organes et de tissus, tandis que leur organisation et leur gestion des programmes nationaux sont inadaptées. Tant les fonds provenant de l'intérieur que ceux fournis par l'extérieur ne permettent pas d'instaurer des programmes de transplantation efficaces. Enfin, les patients ne bénéficient pas d'une protection financière suffisante. Afin de pouvoir relever ces défis, nous proposons que ces pays et leurs partenaires développent et appliquent des politiques, stratégies, projets et règles pour tous les aspects liés aux dons et transplantations d'organes et de tissus, y compris la lutte contre le trafic d'organes et le tourisme de la transplantation. Et là où des programmes de dons et de transplantations existent, les intervenants devraient acquérir des compétences en ressources humaines, mais aussi adopter des normes techniques et des procédures de gestion de la qualité afin d'optimiser les dons et transplantations d'organes et de tissus.

Diversas resoluciones que la Asamblea Mundial de la Salud y la Asamblea General de las Naciones Unidas aprobaron articulan la necesidad de mejorar la disponibilidad, la calidad y la seguridad de la donación y el trasplante de tejidos y órganos, así como de prevenir y combatir el tráfico de órganos humanos. En el presente documento se evalúa la implementación de estas resoluciones relacionadas con la donación y el trasplante de tejidos y órganos por medio del envío de un cuestionario a los 47 países de la Región de África de la Organización Mundial de la Salud. De los 33 países que suministraron los datos, se identificaron varios obstáculos y desafíos. En comparación con otras regiones, existen muy pocos datos sobre la donación y el trasplante de órganos. La mayoría de los países carecen de marcos normativos y legales, ya que todavía no han establecido una legislación específica o integral que aborde la donación y el trasplante de tejidos y órganos humanos. Los países también tienen una capacidad nacional deficiente para realizar los trasplantes de tejidos y órganos, además de que la organización y la gestión de los programas nacionales son débiles. El financiamiento, tanto de fuentes nacionales como internacionales, es insuficiente para implementar programas de trasplantes efectivos y los pacientes tienen una protección financiera inadecuada. Para hacer frente a estos desafíos, se propone que los países y los socios elaboren e implementen políticas, estrategias, programas y marcos normativos de todos los aspectos de la donación y el trasplante de tejidos y órganos, incluida la lucha contra el tráfico de órganos y el turismo de trasplantes. Cuando existan programas de donación y trasplante, las partes interesadas deberían desarrollar las habilidades de los recursos humanos y adoptar estándares técnicos y procedimientos de gestión de calidad para mejorar la donación y el trasplante de tejidos y órganos humanos.