Innovations in pharmaceutical policies and learnings for sustainable access to affordable medicines.

Sustainable access to affordable medicines remains a public health issue globally, including for high-income countries. To foster the debate on avenues for the future, the fifth PPRI Conference held in Vienna on 25 and 26 April 2024 will offer a forum for the debate on innovating pharmaceutical policymaking to develop and implement futureproof policy options, which are able to address current and future challenges. The Conference invites a broad audience of stakeholders, including researchers, policymakers, payers, patients, industry and health professionals. The conference topics are organised in three strands: Strand 1 on 'Local challenges, global learnings' aims to contribute to lively discussions on the implementation of pharmaceutical policies across the globe. Best-practice examples will be presented, supplemented by case studies of less effective policies which can offer rich learnings. Strand 2 on 'Strengthening the evidence base' is the place for presentations and discussions on topics such as health technology assessments, managed entry agreements and real-world data. Strand 3 'Futureproofing pharmaceutical policies' is particularly dedicated to explore innovation in policymaking to achieve sustainable access to affordable medicines.

Tackling medicine shortages during and after the COVID-19 pandemic: Compilation of governmental policy measures and developments in 38 countries.

In response to increasing shortages of medicines, governments have implemented legislative and non-legislative policy measures. This study aimed to map these policies across high-income countries in Europe and beyond as of 2023 and to analyse developments in governmental approaches since the beginning of the pandemic. Information was collated from 38 countries (33 European countries, Australia, Brazil, Canada, Israel and Saudi Arabia) based on a survey conducted with public authorities involved in the Pharmaceutical Pricing and Reimbursement Information (PPRI) network in 2023. 34 countries requested pharmaceutical companies to notify national registers of upcoming shortages and 20 countries obliged manufacturers and/or wholesalers to stock supply reserves of critically needed medicines. Further common measures included export bans for defined medicines (18 countries), regulatory measures to facilitate import and use of alternative medicines (35 countries) and multi-stakeholder coordination (28 countries). While the legislation of 26 countries allows imposing sanctions, particularly for non-compliance to reporting requirements, fines were rather rarely imposed. Since 2022, at least 18 countries provided financial incentives, usually in the form of price increases of some off-patent medicines. Overall, several policies to address medicine shortages were taken in recent years, in some countries as part of a comprehensive package (e.g., Australia, Germany). Further initiatives to secure medicine supply in a sustainable manner were being prepared or discussed.

Barriers and Facilitators in Pricing and Funding Policies of European Countries That Impact the Use of Point-of-Care Diagnostics for Acute Respiratory Tract Infections in Outpatient Practices.

Antimicrobial resistance is a major global health threat, which is increased by the irrational use of antibiotics, for example, in the treatment of respiratory tract infections in community care. By using rapid point-of-care diagnostics, overuse can be avoided. However, the diagnostic tests are rarely used in most European countries. We mapped potential barriers and facilitators in health technology assessment (HTA), pricing, and funding policies related to the use of rapid diagnostics in patients with community-acquired acute respiratory tract infections. Expert interviews were conducted with representatives of public authorities from five European case study countries: Austria, Estonia, France, Poland, and Sweden. Barriers to the HTA process include the lack of evidence and limited transferability of methods established for medicines to diagnostics. There was no price regulation for the studied diagnostics in the case study countries, but prices were usually indirectly determined via procurement. The lack of price regulation and weak purchasing power due to regional procurement processes were mentioned as pricing-related barriers. Regarding funding, coverage (reimbursement) of the diagnostic tests and the optimized remuneration of physicians in their use were mentioned as facilitators. There is potential to strengthen peri-launch policies, as optimized policies may promote the uptake of POCT.

How to Price and to Reimburse Publicly Funded Medicines in Latin America? Lessons Learned from Europe.

This paper reviews the main pricing policies in Latin American countries, discussing their shortcomings. It also gives an overview of the most common pricing and reimbursement policies in Europe and describes in detail three well-established approaches - international price referencing, value-based pricing, including setting up of health technology assessment, and generic and biosimilar policies - building on country examples.

Shortages of Medicines to Treat COVID-19 Symptoms during the First Wave and Fourth Wave: Analysis of Notifications Reported to Registers in Austria, Italy, and Spain.

The study aimed to investigate medicine shortages of critical relevance in the pandemic. A total of 487 active substances for the treatment of COVID-19-related symptoms and therapeutically similar medicines were reviewed as to whether or not a shortage had been notified in Austria, Italy, and Spain for February 2020, March 2020, April 2020 (first wave of the pandemic), and, in comparison, in November 2021 (fourth wave). Publicly accessible shortage registers managed by the national regulatory authorities were consulted. For 48 active substances, a shortage was notified for at least one of the study months, mostly March and April 2020. Out of these 48 active substances, 30 had been explicitly recommended as COVID-19 therapy options. A total of 71% of the active substances with notified shortage concerned medicines labeled as essential by the World Health Organization. During the first wave, Spain and Italy had higher numbers of shortage notifications for the product sample, in terms of active substances as well as medicine presentations, than Austria. In November 2021, the number of shortage notifications for the studied substances reached lower levels in Austria and Spain. The study showed an increase in shortage notifications for COVID-19-relevant medicines in the first months of the pandemic.

Interface policies bridging outpatient and hospital sectors in Europe: can cross-sectorial collaboration in reimbursement and procurement improve access to affordable medicines?

INTRODUCTION: Pharmaceutical systems are frequently characterized by fragmentation, and competences for outpatient and inpatient sectors sit with different authorities, payers, and purchasers. This fragmentation of responsibilities can incentivize shifting expensive therapies and thus patients from one sector to the other. AREAS COVERED: Reimbursement and procurement policies in Europe addressing unwanted consequences of this fragmentation were identified through literature reviews and surveys with policy-makers. Good practice examples include cross-sectorial reimbursement lists managed by committees with representatives from the outpatient and hospital sectors, specific funding mechanisms, joint procurement involving purchasers from both sectors, actions against procurement contracts prohibiting generic competition, and an extension of Health Technology Assessment to the hospital sector. EXPERT OPINION: Recognizing fragmentation as a major challenge for pharmaceutical systems, policy-makers in some countries reacted by implementing policies to support cross-sectorial collaboration. However, only a handful of good practice examples exist for reimbursement and procurement policies in Europe. Though robust evaluations are lacking, there are indications that pharmaceutical policies which ensure collaboration at the interface of the outpatient and inpatient sectors would likely result in efficiency gains and better use of public budgets and may serve as lever to improve access to medicines.

In several European countries, the decision which medicines are funded by public money (reimbursement) and purchased by public institutions (public procurement) is taken independently for the outpatient sector and the hospital sector. There are different payers and procurers per sector, and even within a sector. Patients may be transferred between the sectors for financial reasons because one payer aims to shift the financial burden for the medication to the other sector.Policy-makers have understood the importance of better collaboration between the sectors, and some European countries introduced policies addressing the issue.The article presents examples of how reimbursement and procurement policies can be designed to improve the collaboration between the outpatient and hospital sectors. Committees that decide whether or not a medicine should be covered may contain representatives from both sectors; they may be mandated to take decisions that apply to medicines for outpatient use and administered in hospitals. Purchasers of both sectors may procure jointly a medicine. Supporting tools, such as the assessment of a medicine to support the decision on coverage and the price, may be used in both sectors. Financing solutions can reduce the incentive for one sector to shift a medicine to the other sector.These measures can help that patients gain improved access to affordable medicines. However, despite the introduction of such interface policies in some countries, policy-makers still need to continue working on overcoming the fragmentation in the pharmaceutical system.

Marketing authorisation and pricing of FDA-approved cancer drugs in Brazil: a retrospective analysis.

Background: Most cancer drugs enter the US market first. US Food and Drug Administration (FDA) approvals of new cancer drugs may influence regulatory decisions in other settings. The study examined whether characteristics of available evidence at FDA approval influenced time-to-marketing authorisation (MA) in Brazil, and price differences between the two countries. Methods: All new FDA-approved cancer drugs from 2010 to 2019 were matched to drugs with MA and prices approved in Brazil by December 2020. Characteristics of main studies, availability of randomised controlled trials (RCTs), overall survival (OS) benefit, added therapeutic benefit, and prices were compared. Findings: Fifty-six FDA-approved cancer drugs with matching indications received a MA at the Brazilian Health Regulatory Agency (Anvisa) after a median of 522 days following US approval (IQR: 351-932). Earlier authorisation in Brazil was associated with availability of RCT (median: 506 vs 760 days, p = 0.031) and evidence of OS benefit (390 vs 543 days, p = 0.019) at FDA approval. At Brazilian marketing authorisation, a greater proportion of cancer drugs had main RCTs (75% vs 60.7%) and OS benefit (42.9% vs 21.4%) than that in the US. Twenty-eight (50%) drugs did not demonstrate added therapeutic benefit over drugs for the same indication in Brazil. Median approved prices of new cancer drugs were 12.9% lower in Brazil compared to the US (adjusted by Purchasing Power Parity). However, for drugs with added therapeutic benefit median prices were 5.9% higher in Brazil compared to the US, while 17.9% lower for those without added benefit. Interpretation: High-quality clinical evidence accelerated the availability of cancer medicines in Brazil. The combination of marketing and pricing authorisation in Brazil may favour the approval of cancer drugs with better supporting evidence, and more meaningful clinical benefit albeit with variable degree of success in achieving lower prices compared to the US. Funding: None.

How do hospital characteristics and ties relate to the uptake of second-generation biosimilars? A longitudinal analysis of Portuguese NHS hospitals, 2015-2021.

BACKGROUND: There is limited evidence on within-country discrepancies in biosimilar uptake. This study analyzes differences in timing and diffusion of biosimilar uptake across Portuguese NHS hospitals and explores possible determinants. RESEARCH DESIGN AND METHODS: We analyzed publicly accessible consumption data of originator biologic and biosimilar drugs for adalimumab, etanercept, infliximab, rituximab, and trastuzumab, by hospital and month for the years 2015-2021 (N = 9,467). We modeled the time to biosimilar adoption using survival regression models and the share of biosimilar consumption using generalized estimated equations with random hospital effects. RESULTS: Academic hospitals were characterized by a quicker uptake of adalimumab and infliximab biosimilars but lower shares for other drugs. A higher total consumption of biologics was related to a lower share of biosimilar uptake. A stronger participation in randomized controlled trials was linked to higher biosimilar shares and quicker uptake, except for rituximab. If all NHS hospitals had biosimilar shares equal to the highest ones, potential annual savings could reach 13.9 million euros. CONCLUSION: The findings suggest a need for capacity-building on biosimilar prescribing, including for doctors of academic hospitals and those working in settings where high biosimilar use would be expected.

Prices of new medicines: International analysis and policy options.

Equitable and sustainable access to medicines is a global challenge, including for high-income countries. Over the last two decades, launch prices of new medicines have considerably increased, making some medicines unaffordable for many countries. This article is based on a presentation held by the author at the IQWiG Autumn Symposium in November 2021. The medicine price situation in European countries and beyond is presented informed by international evidence. Medicine prices vary across countries, and for some therapeutic groups (e.g., cancer drugs) medicines are largely unaffordable and/or unsustainable for most countries. Selected policy options to improve affordability, which are commonly applied and considered as potential solutions, are discussed. The article stresses the importance of price regulation, and of transparency of prices and further components across the pharmaceutical value chain. Many governments opted for concluding managed-entry agreements (e.g., pay-for-performance) with confidential discounts to make new medicines with high price tags affordable and/or to manage uncertainty about their performance. However, this policy is linked to limitations, particularly due to its secrecy of discounts. Patent expiry of biological medicines can contribute to affordable access; however, it is important to strategically exploit the biosimilar potential by implementing a mix of appropriate policies to foster competition and enhance the uptake of biosimilar medicines.

Assessing, Pricing and Funding Point-of-Care Diagnostic Tests for Community-Acquired Acute Respiratory Tract Infections-Overview of Policies Applied in 17 European Countries.

Point-of-care diagnostic tests for community-acquired acute respiratory tract infections (CA-ARTI) can support doctors by improving antibiotic prescribing. However, little is known about health technology assessment (HTA), pricing and funding policies for CA-ARTI diagnostics. Thus, this study investigated these policies for this group of devices applied in the outpatient setting in Europe. Experts from competent authority responded to a questionnaire in Q4/2020. Information is available for 17 countries. Studied countries do not base their pricing and funding decision for CA-ARTI diagnostics on an HTA. While a few countries impose price regulation for some publicly funded medical devices, the prices of CA-ARTI diagnostics are not directly regulated in any of the surveyed countries. Indirect price regulation through public procurement is applied in some countries. Reimbursement lists of medical devices eligible for public funding exist in several European countries, and in some countries these lists include CA-ARTI diagnostics. In a few countries, the public payer funds the health professional for performing the service of conducting the test. Given low levels of regulation and few incentives, the study findings suggest room for strengthening pricing and funding policies of CA-ARTI diagnostics to contribute to increased acceptance and use of these point-of-care tests.

Centralised Pharmaceutical Procurement: Learnings from Six European Countries.

Several European countries have introduced centralised procurement for all or some medicines. This article comparatively describes key features of national centralised pharmaceutical procurement (CPP) systems of six European countries (Cyprus, Denmark, Estonia, Italy, Norway and Portugal). Additionally, it aims to identify benefits, challenges and prerequisites for successful CPP, with a view to offering learnings for other countries. Information was collected based on literature and interviews with national procurement experts. While all countries studied established a designated procurement entity, other institutional and organisational features of the CPP systems vary across the countries. All CPP systems apply a mix of procurement procedures depending on the type of medicine (mainly tendering and negotiations). Reported benefits of CPP include lower purchase prices, stronger bargaining power of the public purchasers, enhanced transparency and governance, improved equity, and eventually improved access to medicines. Challenges that are to be met particularly in the starting phase are opposition of some users and the management of stakeholders with different expectations. Issues such as limited competition and non-availability of medicines may continue in the CPP setting. Compliance with good procurement principles is indispensable for any procurement, including CPP. Further prerequisites for successful CPP include a consistent, comprehensive and regularly updated procurement strategy, sufficient funding and appropriate staffing of the procurement entity, efficient processes including contract management and logistics, data collection and monitoring as well as interaction with users and suppliers.

"Ready for the future?" - Status of national and cross-country horizon scanning systems for medicines in European countries.

Background: Horizon scanning aims to systematically identify upcoming health technologies and thus allows policy-makers to be better prepared for the entry of new medicines with possibly high price tags into the national health system. The aim of this study is to survey the existence of national and cross-national horizon scanning systems for medicines in European countries. Methods: Experts working in public authorities (members of the Pharmaceutical Pricing and Reimbursement Information/PPRI network) in the WHO European region participated in surveys in 2014 and 2019 and informed about the status of horizon scanning in their country (response rate: 14 and 44 countries, respectively). Identified advanced horizon scanning systems as of 2019 were further investigated based on a literature review. Results: In 2019, six countries (Iceland, Italy, the Netherlands, Norway, Sweden, United Kingdom) reported systematic use of horizon scanning for some new medicines, and four countries (Austria, Denmark, France, Ireland) had some horizon scanning activities ongoing. No systematic use of horizon scanning was reported from the remaining 34 countries. The findings of the survey undertaken five years earlier were similar, with even fewer systems in place. A recent development is the establishment of cross-country initiatives of governments that aim, among others, to jointly perform horizon scanning; the International Horizon Scanning Initiative (IHSI) initiated by the Beneluxa collaboration is the most advanced undertaking in this respect. Countries with systematic use tend to have horizon scanning fully integrated in a system for the management of new medicines, and they use horizon scanning outcomes to inform decisions as to whether or not a Health Technology Assessment will be conducted and price negotiations be started. Differences between existing horizon scanning systems mainly concern the timings of scanning and reporting, the sources for the inputs and the accessibility of the findings. Conclusion: There appears to be a discrepancy between the perceived importance of horizon scanning based on some eye-opening examples in the past and its actual implementation in European health systems. The latter is likely attributable to horizon scanning being resource-intensive. The establishment of new national and international horizon scanning systems offers the opportunity to investigate their impact on sustainable access to affordable medicines from the start.

Purchasing medicines for the public sector: Evaluation of the performance of centralised procurement in Portugal.

OBJECTIVE: The study aimed to evaluate centralised procurement of medicines (CPM) in Portugal. METHODS: Data were collected through different methods, including a review of the literature and (procurement) documents and an analysis of selected bids. Thirty-seven face-to-face interviews with representatives of public authorities, users (hospitals and regional health administrations), patient associations and pharmaceutical industry were held in Portugal in Q1/2020. RESULTS: CPM has contributed to improved transparency in processes and governance, to increased equity in access to medicines across the country and to lower workload for some users. The findings of the impact on medicine prices and availability are mixed. The benefits of CPM are undermined by some gaps: Lengthy, bureaucratic processes have resulted in delayed availability of medicines at the beginning of a year and in coping strategies of hospitals such as parallel individual procurements. The list of active ingredients under CPM has not been updated since 2016. The procurement agency does not routinely perform market consultations. Key performance indicators for CPM are lacking. CONCLUSIONS: Portuguese policy-makers are urged to develop an updated procurement strategy to provide guidance and clarity on the objectives of CPM, the role of the procurement agency and further authorities and key performance indicators.

Payer policies to support innovation and access to medicines in the WHO European Region

This Oslo Medicines Initiative technical report presents existing policy options for payers that support innovation and access to medicines in the WHO European Region. It identifies innovation incentives, such as early assessment schemes, managed entry agreements and dedicated budgets (so-called innovation funds), across 48 countries in the Region. These incentives are supplemented by supporting assessment tools to generate evidence for informed decision-making (such as horizon scanning and health technology assessments) and access policies for innovation, such as value-based pricing, pooled procurement and subscription fee-based procurement. The report also points to possible limitations of the identified policies; some innovation policies can challenge the financial sustainability of health-care systems, and there are trade-offs between incentivizing innovation and principles of evidence generation, transparency and budget impact. Case studies demonstrate how two countries apply a mixture of policies to support innovation and access to medicines.

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.

Depuis des décennies, certaines régions du monde ont uni leurs efforts pour s'organiser, négocier et effectuer des achats groupés de grandes quantités afin d'assurer un accès équitable aux médicaments et vaccins. Des mécanismes d'acquisition supranationaux ont fait de même à l'échelle planétaire. En Europe, des États ont récemment commencé à collaborer en matière d'achat et de négociation lorsqu'il est devenu de plus en plus difficile de garantir l'accès à des médicaments coûteux, y compris dans les pays à haut revenu. Deux collaborations entre pays européens (le Forum pharmaceutique nordique et l'Initiative d'acquisition de la Baltique) ont mené à bien au moins un processus d'offre conjoint pour des médicaments et vaccins, tandis que l'Initiative Beneluxa a conclu sa première négociation tarifaire conjointe. Cet article décrit les expériences liées à ces collaborations entre nations. Plusieurs défis se sont posés, notamment des obstacles juridiques; des différences institutionnelles et organisationnelles entre les systèmes de santé des États membres; et enfin, le risque que les fournisseurs soient peu enclins à accepter ces collaborations entre pays. Bien que ces collaborations aient amélioré l'accès aux médicaments et vaccins pour les pays impliqués, des initiatives plus globales et à plus grande échelle sont nécessaires dans des situations telles qu'une crise sanitaire mondiale. Durant l'actuelle pandémie de maladie à coronavirus 2019 (COVID-19), l'initiative COVAX (COVID-19 Vaccines Global Access) a abouti à un dispositif d'approvisionnement mondial pour veiller à distribuer équitablement des vaccins contre la COVID-19 dans le monde. Malgré des variations d'organisation et d'échelle, les collaborations entre États européens partagent des similitudes avec le COVAX: (i) le succès de ces deux démarches dépend d'un accroissement du pouvoir d'achat combiné à des volumes de commande groupés; (ii) elles mettent en commun les connaissances approfondies et expériences passées; (iii) elles mènent d'autres activités collectives qui dépassent le simple cadre de l'acquisition; et enfin, (iv) elles impliquent activement une série d'intervenants et de partenaires externes.

Para garantizar un acceso equitativo a los medicamentos y las vacunas, los esfuerzos organizativos y los volúmenes de compra se han unido en adquisiciones y negociaciones conjuntas durante décadas en algunas regiones del mundo, así como a nivel mundial a través de mecanismos de adquisición supranacionales. En Europa, los países empezaron a colaborar en las adquisiciones y negociaciones recientemente, cuando se hizo cada vez más difícil garantizar el acceso a los medicamentos con precios altos, incluso en los países de renta alta. Dos colaboraciones de países europeos (el Foro Farmacéutico Nórdico y la Iniciativa de Adquisición del Báltico) han concluido con éxito al menos un proceso de licitación conjunta de medicamentos y vacunas, y la Iniciativa Beneluxa ha concluido con éxito su primera negociación conjunta de precios. Este artículo describe las experiencias de estas colaboraciones entre países. Entre los retos observados se encuentran: las barreras legales, las diferencias institucionales y organizativas entre los sistemas sanitarios de los países miembros y el riesgo de que los proveedores se muestren reacios a cooperar con las colaboraciones entre países. Aunque estas colaboraciones ayudaron a mejorar el acceso a los medicamentos y las vacunas para los países implicados, en situaciones como una crisis sanitaria mundial, se necesitan iniciativas a mayor escala y más inclusivas. En la actual pandemia de la enfermedad por coronavirus (COVID-19), la iniciativa Acceso global a las vacunas de la COVID-19 (COVAX, por sus siglas en inglés) estableció un mecanismo de adquisición mundial para garantizar la distribución equitativa de las vacunas contra la COVID-19 en todo el mundo. A pesar de las diferencias de organización y escala, las colaboraciones de los países europeos y COVAX tienen algunas similitudes: i) su éxito depende del mayor poder adquisitivo asociado a los volúmenes de pedidos mancomunados; ii) se ponen en común los conocimientos de los expertos y la experiencia previa en materia de adquisiciones; iii) realizan otras actividades de colaboración que van más allá de la mera adquisición; e iv) implican activamente a socios y partes interesadas externas.