PHP372 - Managed Access Agreements: A New Model Pathway for The Reimbursement of Non-Oncology Drugs in England Approved Under European Adaptive Pathways?

Abstract

Managed Access Agreements (MAAs) are agreements between NHS England and manufacturers to enable a drug to become available for a limited time period at a discounted price. This allows patients to access the drug whilst further evidence is gathered on its real-world effectiveness. The National Institute of Health and Care Excellence (NICE) subsequently re-issues guidance on whether this drug should be funded. In December 2015, elosulfase alfa (Vimizim), which treats Morquio A syndrome became the first therapy to be recommended by NICE with an associated MAA. In April 2016, NICE issued draft guidance recommending ataluren (Translarna) in Duchenne muscular dystrophy, conditional on an MAA. MAAs can provide an access route for drugs for very rare diseases, which typically have a limited supporting clinical evidence base and significant associated uncertainties alongside high treatment costs. Indeed, elosulfase alfa (costing £395,000 per patient annually 3 people are born with this condition every year in the UK) and ataluren (costing £222,000 per patient annually; 50 patients are estimated to receive ataluren under the 5-year MAA) had previously been rejected by the Scottish Medicines Consortium (SMC). The number of therapies approved in Europe supported by a clinical data package lacking large multicentre, randomized, comparative Phase 3 trials will likely increase, driven by the EMA Adaptive Pathways pilot alongside very promising new pipeline therapy classes (e.g. CAR-T and gene therapies). For cancer therapies lacking supportive Phase 3 data, under new arrangements for the Cancer Drugs Fund (CDF), promising drugs for which the evidence is not strong enough can temporarily enter the CDF to collect real-world evidence to help inform subsequent NICE appraisals. MAAs therefore can similarly provide a model that allows temporary patient access whilst additional evidence is generated for non-oncology drugs that treat severe, rare diseases.