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.

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.

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.

"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.

Policies to Encourage the Use of Biosimilars in European Countries and Their Potential Impact on Pharmaceutical Expenditure.

Introduction: Biosimilar medicines are considered promising alternatives to new biologicals with high price tags. The extent of savings resulting from biosimilar use depends on their price and uptake, which are largely shaped by pricing, reimbursement, and demand-side policies. This article informs about different policy measures employed by European countries to design the biologicals market and explores potential savings from the increased use of biosimilar medicines in Germany. Methods: Policy measures that target the price and uptake of biosimilar medicines were identified based on a prefilled questionnaire survey with public authorities in 16 European countries, who were the members of the Pharmaceutical Pricing and Reimbursement Information network (July 2020). Potential savings that could have been generated in Germany if different measures identified in the surveyed countries had been implemented were calculated for six publicly funded biological molecules. Price data of the Pharma Price Information service and German consumption data for 2018 were used for the calculation of five scenarios. Results: Several countries use a price link policy, setting the biosimilar price as a percentage of the price of the reference biological. Also lowering the price of the reference biological upon market entry of a biosimilar is less frequently used. While tendering of biosimilar medicines in the inpatient setting is the norm, it is rarely employed for biosimilars in outpatient use. Reference price systems and INN prescribing of medicines are the commonly used policy measures in the off-patent market, but some countries define exemptions for biologicals. Substituting biosimilars at the pharmacy level is rather an exception. Potential savings in Germany ranged from 5% (simple price link) to 55% (prices at the level of other countries) for the six studied molecules. Conclusion: Despite some differences, there are discernible tendencies across European countries with regard to their applications of certain policy measures targeting the price and uptake of biosimilar medicines. The potential for savings of some of these policies was clearly demonstrated. Monitoring and evaluation of these rather recent measures is key for obtaining a more comprehensive picture of their impact.

Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?

This article illustrates a consensus opinion of an expert panel on the need and usefulness of a framework for price and reimbursement (P&R) process and managed entry agreements (MEAs) for orphan medicines in Italy. This opinion was gathered in three rounds: an introductory document was sent to the panel and discussed during a recorded online meeting. A second document was sent to the panel for their review. In the third step the final document was validated. Members of the expert panel are the authors of the article. The panel agreed that Italy does not need a specific value framework for orphan medicines, driving the P&R process. Rather, a more structured value framework for all medicines tailored to the specific drugs can be useful. For orphan drugs, the panel advocated for a multidisciplinary approach and the contribution of different stakeholders to value assessment, and acknowledged the importance of addressing, more than for other drugs, unmet needs, equity issues and societal value. The panel raised the need of increasing the importance of patient-reported outcomes. Experts, acknowledging the growing criticisms in implementation of outcome-based agreements in Italy, expressed their position against their abandonment in favour of discounts only and supported orphan medicines as natural candidates for these agreements. Finally, the panel made some recommendations on the appraisal process for orphan medicines, including an early discussion on the uncertainty of the evidence generated and the adoption of a structured approach to identify the agreement, which better responds to the uncertainty.

Impact of changes in the methodology of external price referencing on medicine prices: discrete-event simulation.

BACKGROUND: Several governments apply the policy of external price referencing (EPR), which considers the prices of a medicine in one or more other countries for the purpose of setting the price in the own country. Different methodological choices can be taken to design EPR. The study aimed to analyse whether, or not, and how changes in the methodology of EPR can impact medicine prices. METHODS: The real-life EPR methodology as of Q1/2015 was surveyed in all European Union Member States (where applicable), Iceland, Norway and Switzerland through a questionnaire responded by national pricing authorities. Different scenarios were developed related to the parameters of the EPR methodology. Discrete-event simulations of fictitious prices in the 28 countries of the study that had EPR were run over 10 years. The continuation of the real-life EPR methodology in the countries as surveyed in 2015, without any change, served as base case. RESULTS: In most scenarios, after 10 years, medicine prices in all or most surveyed countries were-sometimes considerably-lower than in the base case scenario. But in a few scenarios medicine prices increased in some countries. Consideration of discounts (an assumed 20% discount in five large economies and the mandatory discount in Germany, Greece and Ireland) and determining the reference price based on the lowest price in the country basket would result in higher price reductions (on average - 47.2% and - 34.2% compared to the base case). An adjustment of medicine price data of the reference countries by purchasing power parities would lead to higher prices in some more affluent countries (e.g. Switzerland, Norway) and lower prices in lower-income economies (Bulgaria, Romania, Hungary, Poland). Regular price revisions and changes in the basket of reference countries would also impact medicine prices, however to a lesser extent. CONCLUSIONS: EPR has some potential for cost-containment. Medicine prices could be decreased if certain parameters of the EPR methodology were changed. If public payers aim to apply EPR to keep medicine prices at more affordable levels, they are encouraged to explore the cost-containment potential of this policy by taking appropriate methodological choices in the EPR design.

How to address medicines shortages: Findings from a cross-sectional study of 24 countries.

Shortages of medicines have become a major public health challenge. The aim of this study was to survey national measures to manage and combat these shortages. A questionnaire survey was conducted with public authorities involved in the Pharmaceutical Pricing and Reimbursement Information (PPRI) network. Reponses relating to measures as of March / April 2020 were received from 24 countries (22 European countries, Canada and Israel). In 20 countries, manufacturers are requested to notify - usually on an obligatory basis - upcoming and existing shortages, which are recorded in a register. Further measures include a regular dialogue with relevant stakeholders (18 countries), financial sanctions for manufacturers in cases of non-supply and/or non-compliance with reporting or stocking requirements (15 countries) and simplified regulatory procedures (20 countries). For defined medicines, supply reserves have been established (14 countries), and legal provisions allow the issuing of export bans (10 countries). Some measures have been introduced since the end of 2019 and countries are planning and discussing further action. While governments reacted by taking national measures, the COVID-19 crisis might serve as an opportunity to join forces in cross-country collaboration and develop joint (e.g. European) solutions to address the shortage issue in a sustainable manner. A practical first step could be to work on a harmonisation of the national registers.

The impact of managed entry agreements on pharmaceutical prices.

Managed entry agreements (MEAs) have been used for several years, with the aim of curbing the growth of pharmaceutical expenditure and enhancing patient access to innovation. Yet, much remains to be understood about their economic implications. This paper studies the impact of MEAs on list prices, that is, prices before the deduction of any discount. Using a theoretical model, we show that, under most price setting regimes, the introduction of an MEA leads to a higher list price. This is confirmed by our empirical analysis of a sample of 156 medicines in six countries, providing a conservative estimate of the increase in price due to the MEA of 5.9%. A relevant policy implication is that payers may overestimate the financial gains that can be achieved through this  tool.