Performance-based Risk-sharing Arrangements Research Articles

It Takes 2 to Tango. Setting Out the Conditions in Which Performance-Based Risk-Sharing Arrangements Work for Both Parties

ObjectivesFaster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements, to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes.One important reason for risk sharing is to resolve differences of opinion between innovators and payers about a technology’s underlying value. To date, there has been no formal attempt to set out the circumstances in which risk sharing can address these differences. MethodsWe use a value of information framework to understand what a performance-based risk-sharing arrangements can, in principle, add to a reimbursement scheme, separating payer perspectives on cost-effectiveness and the value of research from those of the innovator. We find 16 scenarios, developing 5 rules to analyze these 16 scenarios, identifying cases in which risk sharing adds value for both parties. ResultsWe find that risk sharing provides an improved solution in 9 out of 16 combinations of payer and innovator expectations about treatment outcome and the value of further research. Among our assumptions, who pays for research and scheme administration costs are key. ConclusionsSteps should be undertaken to make risk sharing more practical, ensuring that payers consider it an option. This requires additional costs to the health system falling on the innovator in an efficient way that aligns incentives for product development for global markets. Health systems benefits are earlier patient access to cost-effective treatments and payers with higher confidence of not wasting money. Innovators get greater returns while conducting research.

Personalized Reimbursement Model (PRM) program: A real-world data platform of cancer drugs use to improve and personalize drug pricing and reimbursement in France.

ObjectiveThis article describes the Personalized Reimbursement Model (PRM) program methodology, limitations, achievement and perspectives in using real-world data of cancer drugs use to improve and personalize drug pricing and reimbursement in France.Materials and methodsPRM platform aggregates Electronic Pharmacy Records (EPR) data from French medical centers (PRM centers) to build retrospective cohorts of patients treated with injectable cancer drugs in a hospital setting. Data extracted on January 1st, 2020, from breast cancer (BC) patients who received trastuzumab, trastuzumab emtansin or pertuzumab since January 1st, 2011, and from lung cancer (LC) patients who received bevacizumab or atezolizumab since January 1st, 2015, enabled recovering their injectable cancer drugs history from diagnosis date until December 30th, 2019, and served as dataset for assessment.Results123 PRM centers provided data from 30,730 patients (25,660 BC and 5,070 LC patients respectively). Overall, 20,942 (82%) of BC and 4,716 (93%) of LC patients were analyzed. Completion rate was above 98% for patients characteristics, diagnostic and treatment related data. PRM centers cover 48% and 33% of BC and LC patients in-hospital therapeutic management in France, respectively. Distribution of BC and LC patients therapeutic management, by medical center category and geographic location, was similar in PRM centers to all French medical centers, ensuring the representativeness of the PRM platform.ConclusionPRM Platform enabled building a national database generating on demand Real-World Evidence based on EPR. This enabled the first performance-based risk-sharing arrangements based on PRM data, between the CEPS and Roche, for atezolizumab cancer immunotherapy in metastatic non-small cell lung cancer indication.

Performance-based risk-sharing arrangements for devices and procedures in cardiac electrophysiology: an innovative perspective.

There is an increasing pressure on demonstrating the value of medical interventions and medical technologies resulting in the proposal of new approaches for implementation in the daily practice of innovative treatments that might carry a substantial cost. While originally mainly adopted by pharmaceutical companies, in recent years medical technology companies have initiated novel value-based arrangements for using medical devices, in the form of 'outcomes-based contracts', 'performance-based contracts', or 'risk-sharing agreements'. These are all characterized by linking coverage, reimbursement, or payment for the innovative treatment to the attainment of pre-specified clinical outcomes. Risk-sharing agreements have been promoted also in the field of electrophysiology and offer the possibility to demonstrate the value of specific innovative technologies proposed in this rapidly advancing field, while relieving hospitals from taking on the whole financial risk themselves. Physicians deeply involved in the field of devices and technologies for arrhythmia management and invasive electrophysiology need to be prepared for involvement as stakeholders. This may imply engagement in the evaluation of risk-sharing agreements and specifically, in the process of assessment of technology performances or patient outcomes. Scientific Associations may have an important role in promoting the basis for value-based assessments, in promoting educational initiatives to help assess the determinants of the learning curve for innovative treatments, and in promoting large-scale registries for a precise assessment of patient outcomes and of specific technologies' performance.

Performance-based risk-sharing arrangements for devices and diagnostics in the United States: a systematic review.

BACKGROUND: Performance-based risksharing arrangements (PBRSAs) have continued to emerge and evolve over the last 2 decades. To date, most of the attention and available literature have focused on pharmaceuticals. OBJECTIVE: To assess the current status and trends regarding the use of PBRSAs for diagnostics and devices in the United States. METHODS: We reviewed publicly available PBRSAs for diagnostics and devices using the University of Washington Performance Based Risk Sharing Database. We augmented the review using PubMed, Google, and payer and industry websites. Key words and phrases such as outcomes-based, value-based, coverage with evidence development, performance-based, and risk-sharing were used in combination with device or diagnostic. To characterize arrangements in terms of product and market attributes, we extracted data for each product, including arrangement descriptions, arrangement type, year, therapeutic area, product manufacturer, payer, and product type. Arrangements were analyzed using descriptive statistics. RESULTS: Fifty-two arrangements were identified between the years 2001 and 2019, with 30 (57.7%) for devices and 22 (42.3%) for diagnostic tests. Among these, 23 (44.2%) were coverage with evidence development (CED), only in research; 17 (32.7%) were performance-linked reimbursement (PLR); and 12 (23.1%) were CED, only with research. The majority of arrangements for devices were developed in cardiology (12, 40%), endocrinology (4, 13.3%), and radiology (3, 10%). Most of arrangements for identified diagnostic tests were in oncology (17, 77.3%). Over time, there has been a trend towards increasing adoption of PLR and CED, only with research, especially since 2014. CONCLUSIONS: This is the first study to comprehensively review PBRSA arrangements for diagnostics and devices in the United States. Our findings demonstrated that there is substantial PBRSA activity for devices and diagnostics, and the pace of PBRSA adoption appears to be increasing in terms of frequency and variety. These arrangements have implications for managed care into the future as the health care system shifts towards value-based care and value-based pricing to contain cost for payers and ensure value in the patient populations. DISCLOSURES: No funding supported this study. The authors have nothing to disclose.

RE1 Performance-Based Risk-Sharing Arrangements in Diagnostics and Devices in the United States

Performance-based risk sharing agreements (PBRSAs), defined as linking coverage and reimbursement to the future performance of medical products, have continued to emerge and evolve over the last two decades. In the U.S., most arrangements and publications have focused on pharmaceuticals. Our aim was to assess the current status and trends of the uptake of PBRSAs for diagnostics and devices in the U.S. We reviewed publicly available PBRSAs for diagnostics and devices leveraging the University of Washington Performance Based Risk Sharing Database®. We augmented the review using PubMed, Google, and payer and industry websites. Key words including “outcomes-based”, “value-based”, “coverage with evidence development”, “performance-based”, “risk-sharing” were used in combination with “device” or “diagnostic”. To characterize arrangements in terms of product and market attributes, we extracted data for each product including arrangement descriptions, arrangement type, year, therapeutic area, product manufacturer, payer, and product type. Arrangements were analyzed using descriptive statistics. 53 arrangements were identified between the years 2001 and 2019, with 31 (58.5%) for devices and 22 (41.5%) for diagnostic tests. Among these, 23 (43.4%) were coverage with evidence development (CED) - only in research, 18 (34.0%) were performance linked reimbursement (PLR), and 12 (22.6%) were CED - only with research. The majority of arrangements for devices were developed in cardiology (13, 41.9%), endocrinology (4, 12.9%), and radiology (3, 9.7%). While most of arrangements for diagnostic tests identified were in oncology (17, 77.3%) and some in neurology (2, 9.1%). Over time, there has been a trend towards increasing adoptions of PLR and CED – only with research since 2013. This is the first study to comprehensively review PBRSA arrangements for diagnostics and devices in the U.S. Our findings showed that the pace of PBRSA adoption appears to be increasing, with more types of arrangements.

Performance-Based Risk-Sharing Arrangements (PBRSA): Is it a Solution to Increase Bang for the Buck for Pharmaceutical Reimbursement Strategy for Our Nation and Around the World?

Due to the risks involved in not achieving desired health outcomes for the dollar spent on drugs, healthcare decision makers, including payers, providers, drug manufacturers, and patients, need a mechanism to share this financial risk among the involved parties. Performance-based risk-sharing arrangements (PBRSAs) are agreements that can potentially reduce the ‘drug lag’ in which patients wait for an unknown amount of time until a particular drug is covered under their health plan. In addition, PBRSAs can mitigate the risk of investing heavily in drugs that are ineffective or do not deliver good value or “bang for the buck”. This review describes and evaluates PBRSAs for drugs in the USA and juxtaposes to other developed nations (i.e. Germany) that adopted PBRSAs in their healthcare model. There are different types of outcomes-based health schemes, namely conditional coverage, which can be further broken down into coverage with evidence development (CED), conditional treatment continuation (CTC), and performance-linked reimbursement, which includes outcomes guarantees. Both CED and CTC are ‘conditional’ on the collected evidence of the new drug’s effectiveness, offering discount only if the drug delivers desirable results. The outcomes guarantee scheme offers discount or even a full refund if the outcome is less than expected, forcing the drug to meet the expected effectiveness. The USA can follow the German reference pricing model in which the assessment of new drugs is centralized and done collectively by representatives from a group of healthcare decision makers. In any shape or form, PBRSA is a clever mechanism to cope with uncertainty if drug price is scaled appropriately based on value.

International experience with performance-based risk-sharing arrangements: implications for the Chinese innovative pharmaceutical market

Various forms of outcomes-based or risk-sharing agreements have been implemented since early 2000s as a way of access to innovative medicinal products. This study aims to summarize the international experience of performance-based risk-sharing arrangements (PBRSAs) and identify the preconditions for a successful implementation of such schemes. Their implications for the Chinese healthcare market are discussed. A systematic literature review (in PubMed) was conducted to review the evidence on the nature and performance of PBRSAs in the past 10 years. Grey literature was searched for reports in government websites of the countries in scope. The search identifies 463 records from PubMed and 3 additional records from other sources. Thirty-one publications are included in the final review. The following preconditions were identified to support a successful implementation of PBRSAs: (1) Identify meaningful and feasible outcome measurements; (2) Establish an effective and efficient data collection infrastructure; (3) Control of the implementation costs; (4) Develop governance and administrative infrastructure to allow delisting and rebate/refund; (5) Clarify personal data protection issues. The implementation of PBRSAs has proven to be challenging. Although the Chinese healthcare system is not yet well equipped to implement such schemes, some recent changes may pave the way to successful PBRSAs for particular innovative products.

Performance-Based Risk-Sharing Arrangements for Pharmaceutical Products in the United States: A Systematic Review.

Value for money is a growing necessity in today's U.S. health care system in which drug spending is expected to increase by an average rate of 6.7% yearly through 2025. In response to uncertainty about real-world clinical and economic outcomes for many drugs, health insurers and pharmacy benefit managers (PBMs) have implemented various contracts and arrangements with drug manufacturers that can collectively be described as performance-based risk-sharing arrangements (PBRSAs). Little is known about U.S.-specific PBRSAs for drugs. To conduct a systematic review of U.S.-specific PBRSAs for drugs to describe (a) trends over time and (b) key aspects including outcome measures and terms of arrangements between stakeholders. A systematic review was conducted in MEDLINE (January 1, 1946-April 1, 2017), Embase (January 1, 1988-April 1, 2017), and the grey literature (up to April 1, 2017) to identify publicly disclosed PBRSAs. Articles and conference abstracts were included if they were published in English and described a U.S.-specific PBRSA for a drug. Articles and conference abstracts were excluded if they only described a PBRSA similar to a money-back guarantee to patients. They were also excluded if they only described a PBRSA between a PBM and a health insurer in which the latter would receive a discount for patients nonadherent to a drug. Results were summarized as counts and percentages. From the literature review, 26 publicly disclosed PBRSAs were identified. Of these, 16 (62%) were announced or initiated from 2015 to 2017, and 10 (38%) were announced or initiated from 1997 to 2012. Thirteen (50%) PBRSAs involved cardiometabolic indications; 5 (19%) involved oncology indications; and 8 (31%) involved other indications. Categorized by health insurer or PBM, 10 (38%) PBRSAs involved large multistate insurers; 5 (19%) involved the Centers for Medicare & Medicaid Services; 7 (27%) involved regional insurers; 3 (12%) involved PBMs; and 1 (4%) involved multiple unspecified insurers. Regarding the most active drug manufacturers, Amgen initiated 5 (19%) PBRSAs and Novartis initiated 4 (15%). Relative to the initial FDA approval of a treatment, 15 (58%) PBRSAs were announced or initiated within 5 years, and 11 (42%) were announced or initiated more than 5 years later. For data collection, electronic medical record (EMR) data would have been an appropriate source for 12 (46%) PBRSAs; claims data would have been an appropriate source for 11 (42%) PBRSAs; and EMR and claims data would have been appropriate sources for 2 (8%) PBRSAs; no description of the outcome measures was available for 1 (4%) PBRSA. The number of publicly disclosed U.S.-specific PBRSAs for drugs has increased over the years. This review's findings confirm the interest of stakeholders in such arrangements and their confidence in the use of the selected outcome measures. Each PBRSA represents a timely collaboration among stakeholders to provide access to a drug while generating evidence to better elucidate its clinical and economic value. No funding supported this systematic review. Yu is an employee and shareholder of Allergan. Chin reports personal fees from Formulary Resources. Oh and Farias have nothing to disclose. Study concept and design were primarily contributed by Yu, along with the other authors. All authors contributed to the collection and interpretation of the data. The manuscript was written by Yu, Chin, Oh, and Farias and revised by Yu and Chin, along with the other authors.

Performance-Based Risk-Sharing Arrangements: U.S. Payer Experience.

As a result of global concern about rising drug costs, many U.S. payers and European agencies such as the National Health Service have partnered with pharmaceutical companies in performance-based risk-sharing arrangements (PBRSAs) by which manufacturers share financial risk with health care purchasing entities and authorities. However, PBRSAs present many administrative and legal challenges that have minimized successful contract experiences in the United States. To (a) identify drug and disease characteristics and contract components that contribute to successful PBRSA experiences and the primary barriers to PBRSA execution and (b) explore solutions to facilitate contract negotiation and execution. A 37-item, web-based survey instrument (Qualtrics), approximately 20 minutes in duration, was open during July and August 2016. The survey was emailed to 90 pharmacy and medical directors of various health care organizations. Statistical analysis included the Kruskal-Wallis test and chi-square tests to examine differences among payer responses. Survey responses were anonymized and data were aggregated. Twenty-seven individuals completed the survey (30% completion rate). The majority of respondents worked for regional health plans (52%, n = 14), covering at least 1 million lives (63%, n = 17), with at least 7 years of managed care experience (81%, n = 22). A total of 51 PBRSAs were active among respondents at the time of the survey. Easily obtainable and evaluable drug data and medical data were the most important drug and disease attributes for successful PBRSAs, respectively. Pharmacy claims and patient demographic data were assessed as "very easy and inexpensive" to collect. Type and amount of manufacturer payment for drug outcome performance failure, endpoint measurement, and necessary clinical data for drug performance measurement were all critical factors for successful PBRSAs. Standardized contract templates and transparent contract financial risk evaluation and modeling ranked highest among methods of manufacturer facilitation of PBRSAs. This study was limited by sample size and survey questions were limited to explanation of PBRSAs at the disease state level. On the basis of PBRSA experiences, respondents noted that drug use in chronic medical conditions and objective drug outcome performance measurements were favorable drug characteristics and serve as the primary source of satisfaction for these types of contracts. Third parties and manufacturers can facilitate the uptake and success of PBRSAs by developing standardized contracting templates in addition to other methods that increase their stake in the arrangement. Looking forward, mounting perceptions of success in this realm of contracting for pharmaceuticals may contribute in the quest for value-based payments in the U.S. health care system. The construction of the survey and payment for survey respondents were supported by Charles River Associates. Parece is an employee of Charles River Associates. Goble and Ung are completing fellowship training sponsored by Novartis and Celgene, respectively, but do not have any conflicts of interest and did not receive any funding related to this study. Navarro reports consulting fees from Analysis Group, TEVA, and Amgen, unrelated to this study. Van Boemmel-Wegmann declares no conflict of interest. Study concept and design were contributed by Navarro, Goble, Ung, and Parece. Navarro took the lead in data collection, along with Goble and Ung, and data interpretation was performed by van Boemmel-Wegmann, Goble, and Ung. The manuscript was written by Goble, Ung, Navarro, and van Boemmel-Wegmann and revised by all of the authors.

Using Performance-Based Risk-Sharing Arrangements to Address Uncertainty in Indication-Based Pricing.

The rise in pharmaceutical expenditures in recent years has increased health care payer interest in ensuring good value for the money. Indication-based pricing (IBP) sets separate, indication-specific prices paid to the manufacturer according to the expected efficacy of a drug in each of its indications. IBP allows payers to consistently pay for value across indications. While promising, a limitation of IBP as originally conceived is that efficacy estimates are typically based on clinical trial data, which may differ from real-world effectiveness. An outcomes guarantee is a type of performance-based risk-sharing arrangement that adjusts payments according to prospectively tracked outcomes. We suggest that an outcomes guarantee contract, which has been used by some payers, may be adapted to achieve indication-based prices supported by real-world effectiveness. To illustrate the potential of an outcomes guarantee to achieve indication-based prices aligned with real-world value, using a case study of trastuzumab for the treatment of metastatic breast and advanced gastric cancers. We estimated costs and outcomes under traditional IBP (i.e., expected value IBP) and outcomes guarantee frameworks and calculated incremental cost-effectiveness ratios (ICERs) comparing treatment with and without trastuzumab. Efficacy data came from pivotal trials, whereas effectiveness data came from observational studies. We adjusted trastuzumab prices in order to achieve target ICERs of $150,000 per quality-adjusted life-year under each framework and for each indication. To achieve the ICER target under traditional IBP, the unit price of trastuzumab using efficacy evidence was adjusted for metastatic breast and advanced gastric cancers from an average sales price of $9.17 per mg to $3.50 per mg and $0.93 per mg, respectively. Under an outcomes guarantee, the unit price of trastuzumab using effectiveness evidence was adjusted for metastatic breast cancer and advanced gastric cancer to $8.66 per mg and $0.20 per mg, respectively. Like expected value IBP, outcomes guarantee contracts can also vary payment based on indication. In addition, an outcomes guarantee can also reduce uncertainty regarding effectiveness and better align payment with the actual value of a treatment. No funding supported this study. Carlson reports consulting fees from Genentech, Pfizer, and Seattle Genetics. The other authors have no conflicts of interest to disclose. Study concept and design were contributed by Carlson, Yeung, and Li. Yeung, Carlson, and Li collected and analyzed the data. The manuscript was written primarily by Yeung, along with Carlson and Li, and revised by all the authors.

Performance-Based Risk-Sharing Arrangements: An Updated International Review.

Enthusiasm for performance-based risk-sharing arrangements (PBRSAs) continues but at variable pace across countries. Our objective was to identify and characterize publicly available cases and related trends for these arrangements. We performed a review of PBRSAs from 1993 to 2016 using the University of Washington PBRSA Database. Arrangements were categorized according to a previously published taxonomy. Macro-level trends were identified related to the timing of adoption, countries involved, types of arrangements, and disease areas. Our search yielded 437 arrangements. Among these, 183 (41.9%) were categorized as currently active, while 58.1% have expired. Five main types of arrangements have been identified, namely coverage with evidence development (149 cases, 34.1%), performance-linked reimbursement (104 cases, 23.8%), conditional treatment continuation (78 cases, 17.8%), financial or utilization (71 cases, 16.2%), and hybrid schemes with multiple components (35 cases, 8.0%). The pace of adoption varies across countries but has renewed an upward trend after a lull in 2012/2013. Conditions in the USA may be changing toward a more favorable environment of PBRSAs. Interest in PBRSAs remains high, suggesting they are a viable coverage and reimbursement mechanism for a wide range of medical products.

Emerging roles for pharmacists in performance-based risk-sharing arrangements.

The changing landscape of healthcare payment models is providing new opportunities for pharmacists. Traditional models were focused on paying for a product or a service. Evolving models are focused on paying for positive healthcare outcomes. One of the emerging payment models, the performance-based

PCN263 - Role of Clinical Pharmacist in Optimizing Reimbursement Originating from Performance-Based Risk-Sharing Arrangements: The Experience of the University Hospital “G. Martino” from Messina, Italy

PCN263 - Role of Clinical Pharmacist in Optimizing Reimbursement Originating from Performance-Based Risk-Sharing Arrangements: The Experience of the University Hospital “G. Martino” from Messina, Italy

Current Status and Trends in Performance-Based Risk-Sharing Arrangements Between Healthcare Payers and Medical Product Manufacturers

Our objective was to identify and characterize publicly available cases and related trends for performance-based risk-sharing arrangements (PBRSAs). We performed a review of PBRSAs over the past 20 years (1993-2013) using available databases and reports from colleagues and healthcare experts. These were categorized according to a previously published taxonomy of scheme types and assessed in terms of the underlying product and market attributes for each scheme. Macro-level trends were identified related to the timing of scheme adoption, countries involved, types of arrangements, and product and market factors. Our search yielded 148 arrangements. From this set, 65 arrangements included a coverage with an evidence development component, 20 included a conditional treatment continuation component, 54 included a performance-linked reimbursement component, and 42 included a financial utilization component. Each type of scheme addresses fundamental uncertainties that exist when products enter the market. The pace of adoption appears to be slowing, but new countries continue to implement PBRSAs. Over this 20-year period, there has been a consistent movement toward arrangements that minimize administrative burden. In conclusion, the pace of PBRSA adoption appears to be slowing but still has traction in many health systems. These remain a viable coverage and reimbursement mechanism for a wide range of medical products. The long-term viability and growth of these arrangements will rest in the ability of the parties to develop mutually beneficial arrangements that entail minimal administrative burden in their development and implementation.

A New Model for Reimbursing Genome-Based Cancer Care

Molecularly targeted medicine poses substantial challenges for incorporation into the existing U.S. health care system. It is argued here that performance-based risk-sharing arrangements may be a promising approach to address the competing needs of patients, clinicians, and payers.

Performance-Based Risk-Sharing Arrangements—Good Practices for Design, Implementation, and Evaluation: Report of the ISPOR Good Practices for Performance-Based Risk-Sharing Arrangements Task Force

There is a significant and growing interest among both payers and producers of medical products for agreements that involve a “pay-for-performance” or “risk-sharing” element. These payment schemes—called “performance-based risk-sharing arrangements” (PBRSAs)—involve a plan by which the performance of the product is tracked in a defined patient population over a specified period of time and the amount or level of reimbursement is based on the health and cost outcomes achieved. There has always been considerable uncertainty at product launch about the ultimate real-world clinical and economic performance of new products, but this appears to have increased in recent years. PBRSAs represent one mechanism for reducing this uncertainty through greater investment in evidence collection while a technology is used within a health care system. The objective of this Task Force report was to set out the standards that should be applied to “good practices”—both research and operational—in the use of a PBRSA, encompassing questions around the desirability, design, implementation, and evaluation of such an arrangement. This report provides practical recommendations for the development and application of state-of-the-art methods to be used when considering, using, or reviewing PBRSAs. Key findings and recommendations include the following. Additional evidence collection is costly, and there are numerous barriers to establishing viable and cost-effective PBRSAs: negotiation, monitoring, and evaluation costs can be substantial. For good research practice in PBRSAs, it is critical to match the appropriate study and research design to the uncertainties being addressed. Good governance processes are also essential. The information generated as part of PBRSAs has public good aspects, bringing ethical and professional obligations, which need to be considered from a policy perspective. The societal desirability of a particular PBRSA is fundamentally an issue as to whether the cost of additional data collection is justified by the benefits of improved resource allocation decisions afforded by the additional evidence generated and the accompanying reduction in uncertainty. The ex post evaluation of a PBRSA should, however, be a multidimensional exercise that assesses many aspects, including not only the impact on long-term cost-effectiveness and whether appropriate evidence was generated but also process indicators, such as whether and how the evidence was used in coverage or reimbursement decisions, whether budget and time were appropriate, and whether the governance arrangements worked well. There is an important gap in the literature of structured ex post evaluation of PBRSAs. As an innovation in and of themselves, PBRSAs should also be evaluated from a long-run societal perspective in terms of their impact on dynamic efficiency (eliciting the optimal amount of innovation).

Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries

BackgroundNational payers across Europe have been increasingly looking into innovative reimbursement approaches – called managed entry agreements (MEAs) – to balance the need to provide rapid access to potentially beneficial orphan medicinal products (OMPs) with the requirements to circumscribe uncertainty, obtain best value for money or to ensure affordability. This study aimed to identify, describe and classify MEAs applied to OMPs by national payers and to analyse their practice in Europe.MethodsTo identify and describe MEAs, national health technology assessments and reimbursement decisions on OMPs across seven European countries were reviewed and their main characteristics extracted. To fill data gaps and validate the accuracy of the extraction, collaboration was sought from national payers. To classify MEAs, a bespoke taxonomy was implemented. Identified MEAs were analysed and compared by focusing on five key themes, namely by describing the MEAs in relation to: drug targets and therapeutic classes, geographical spread, type of MEA applied, declared rationale for setting-up of MEAs, and evolution over time.Results42 MEAs for 26 OMPs, implemented between 2006 and 2012 and representing a variety of MEA designs, were identified. Italy was the country with the highest number of schemes (n=15), followed by the Netherlands (n=10), England and Wales (n=8), Sweden (n=5) and Belgium (n=4). No MEA was identified for France and Germany due to data unavailability. Antineoplastic agents were the primary targets of MEAs. 55% of the identified MEAs were performance-based risk-sharing arrangements; the other 45% were financial-based. Nine of these 26 OMPs were subject to MEAs in two or three different countries, resulting in 24 MEAs. 60% of identified MEAs focused on conditions whose prevalence is less than 1 per 10,000.ConclusionsThis study confirmed that a variety of MEAs were increasingly used by European payers to manage aspects of uncertainty associated with the introduction of OMPs in the healthcare system, and which may be of a clinical, utilisation, or budgetary nature. It remains unclear whether differences in the use of MEAs reflect differences in how ‘uncertainty’ and ‘value’ are perceived across healthcare systems.