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

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.

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?

“…New orphan medicinal products (OMPs), some costing over €1 million per patient per year (20,23,100), are also putting pressure on healthcare budgets, especially with some OMPs achieving blockbuster status (9,100,101). This increases the debate whether pharmaceutical companies should also consider social good alongside financial returns (20), and whether health authorities should be more critical when assessing the value of new OMPs.…”

“…Expenditure on OMPs has been influenced by few marketing authorisation and reimbursement hurdles at requested prices (9,(102)(103)(104)(105)(106)(107). However, this is changing with managed entry agreements increasingly needed for successful reimbursement.…”

“…Unless these problems are addressed, the number of struggling countries will grow driven by factors including changing demographics, increasing prevalence of non-communicable diseases (NCDs), rising patient expectations and the continued launch of new premium priced technologies (10)(11)(12)(13)(14)(15)(16)(17)(18)(19)(20). New medicines increasingly include novel biological drugs (21), often priced at between US$100,000 -US$400,000 (Euro74,000 -296,000) per patient per course or year (8,10,18,22,23). The challenges of funding new premium priced medicines are augmented by lobbying, marketing and other strategies among pharmaceutical companies, including influencing guidelines to accelerate the uptake of new medicines (24)(25)(26)(27)(28)(29)(30)(31)(32), despite the imposition of multi-million dollar fines (33)(34)(35)(36).…”

“…Challenges to be addressed include adequately dealing with demands for premium prices for new medicines with often limited health gain versus current standards including those for cancer and orphan diseases (23,(37)(38)(39). Funding new effective treatments for patients with hepatitis C virus (HCV) is a also growing concern given its prevalence and requested prices for new treatments (6,14,40).…”

A National Approach to Reimbursement Decision-Making on Drugs for Rare Diseases in Canada? Insights from Across the Ponds

“…These schemes facilitate patient access to expensive and typically innovative drugs that do not appear to offer significant benefits over existing treatments by improving their cost-effectiveness. This may be achieved through listing agreements or risksharing arrangements that incorporate outcomes guarantees realized within a defined period (Morel et al 2013). Other countries have implemented "fast-track" mechanisms, where eligible drugs are prioritized by the CDR, bypassing the standard process in which drugs are reviewed Hilary Short et al in the order received (e.g., FRA, the NET, SWI).…”