On the evening on Wednesday 26th April, Dr Joanna Nightingale, Head of Scientific Affairs attended a roundtable meeting hosted by MAP Patient Access Limited on “Harnessing the potential of advanced therapy medicinal products (ATMPs) – the case for change” at the Palace of Westminster.

The event was supported by BioMarin Pharmaceutical, Orchard Therapeutics and PTC Therapeutics. The panel consisted of Liz Twist, Labour MP for Blaydon, Laura Beswick, Head of Health Economics and Market Access, Cell and Gene Therapy Catapult, Jacoline Bouvy, Programme Director for Medicines Evaluation, National Institute for Health and Care Excellence, Steve Bates OBE, CEO, BioIndustry Association (BIA), Sheela Upadhyaya, Life Sciences Consultant – specialising in Rare Disease and Nick Meade, Director of Policy, Genetic Alliance UK.

Each panel member spoke about the opportunities and challenges facing the UK in becoming a world leader in ATMPs and how to process the excellent research into suitable and commercially sustainable treatments for genetic diseases.

The UK is good at life science research and innovation and the NHS is a unique selling point. However, there are issues which need to be addressed to meet the unmet health needs particular in rare diseases. Some of these issues include optimising manufacturing capabilities for a global market, getting potential participants to engage with clinical trials, how patient outcomes are collected, pricing of expensive new therapies and the need for sustainable access to therapies for patients.

Jo asked about how the diagnostic odyssey should change as new treatments become available so that patients receive life saving treatment before there is a chance for the effects to become permanent. It is acknowledged that new-born screening needs to be in place where there is a treatment available. However, this process takes time and money. The panel were asked to summarise what they thought was the most important requirement to move forward. It was money.