PND91 - HOW ADVANCED THERAPY TREATMENT CENTERS IN THE UK CAN IMPROVE MARKET ACCESS FOR SPECIALISED TECHNOLOGIES

An increasing number of gene therapies are assessed by the European Medicines Agency (EMA)'s Committee for Advanced Therapies. These treatments, which are expected to shift the treatment paradigm in numerous therapeutic areas, are viewed with concern by payers, as the maturity of their clinical data and cost of these treatments remain a critical hurdle for their successful reimbursement. This paper aims to evaluate the potential role of the three Advanced Therapy Treatment Centres (ATTCs) established in the UK in easing market access of advanced therapy medicinal products (ATMPs). Primary and secondary research was employed to collect information. Official secondary sources were consulted to identify ATMPs currently under review by the National Institute for Health and Care Excellence (NICE) and the role and the funding available to the three ATTCs. The ATTCs network focuses on several activities, including creating the necessary infrastructure for the long-term collection of clinical data, ensuring safe and effective delivery and distribution of innovative therapies, plus the necessary frameworks to facilitate potential innovative payment systems essential for super high-cost technologies. There are currently three cell-based ATMPs that are under review Novartis’ Kymriah (tisagenlecleucel-t), Gilead Science’s Yescarta (axicabtagene ciloleucel) and Takeda’s Alofisel (darvadstrocel), while Spark Therapeutics’ Luxturna (voretigene-neparvovec) has been proposed for assessment. Should these treatments receive positive recommendation, they may benefit from the support provided by the ATTC network. The introduction of ATTCs offers early access to advanced therapies delivered in highly specialised centres, bridging the gap between development and commercialisation of advanced therapies. Through this network, manufacturers will have the opportunity to build stronger clinical data sets, to reduce administrative burden and to introduce new financial models to address pricing and reimbursement challenges. This is expected to have a positive impact on market access opportunities for ATMPs in the UK market.