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

Thomas Morel,Francis Arickx,Gustaf Befrits,Caroline Van Der Meijden,Entela Xoxi,Steven Simoens,Paolo Siviero

doi:10.1186/1750-1172-8-198

Abstract

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.

Highlights

National 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
Their clinical evidence package is often associated with uncertainty at product launch due to the difficulty of recruiting a sufficient number of patients, patient population is often heterogeneous, many trials of approved OMPs are only based on surrogate endpoints, traditional study designs are sometimes not feasible, and the assessment of the observed clinical improvement may be difficult as little is usually known about the natural history of the disease
Many OMPs are associated with relatively high treatment costs [8,9,10,11,12,13,14], which adds to the budgetary uncertainty dimension and/or to the financial risk to the payer in the event the treatment does not work in real life as well as anticipated

Summary

Introduction

National 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. As a new orphan medicinal product (OMP) receives European marketing authorisation across the European Union (EU), national health technology assessment (HTA) bodies and national healthcare payers subsequently determine its value to inform or decide reimbursement This is usually not a straightforward exercise as there is often considerable uncertainty about the ultimate real-world clinical and economic performance of that new OMP. Findings show that in those countries both expenditure and utilisation increased in the year 2010 compared to 2009, ranging from 13 to 28% and 7 to 17% respectively [16]

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