The House of Lords Regenerative Medicine Report - Clearing the hurdles for cell therapy clinical translation and commercialisation
Cell therapy is slowly emerging as a global industry likely to equal (or exceed) the economic successes of big pharma, biotech and medical device companies (1). There are approximately 500 cell therapy companies spread across Asia, Europe and North America (2) with combined annual revenues in excess of £1 billion in 2012 and predicted to double by 2014, with even greater growth expected to follow. Around one million patients have received cell-based therapies and well over 1,000 clinical trials are underway (3), and yet the total number of regulatory agency approved products is still only 20. Stem cells are great science. However, if their legacy is to be significant health and wealth benefits this will require substantial funding, appropriate infrastructure and lots of patience – unfortunately there will be setbacks along the path to success.
Cell therapies have the potential to transform the lives of many millions of patients and their carers. They have been hailed as the next big healthcare technology, but unless governments step-in with both adequate funding streams and the necessary infrastructure changes to produce cell therapy-friendly environments for late stage development and commercialisation, cell therapies will always remain the next big opportunity. Although governments have injected billions of pounds into research and early clinical trials, creating cell therapy friendly environments has not been a priority and without coordinated change, the majority of the funding will be wasted. The recently published House of Lords (HoL) Regenerative Medicine Report (4) has fully recognised the urgent need for the UK to leverage its first-rate clinical translation and commercialisation assets, including the NHS and the City, in order to capture the healthcare and commercial benefits from our outstanding stem cell and regenerative medicine research.
In London 2012, when Olympic gold medalist Aries Merritt, USA (5), heard the starting pistol for the final of the men's 100 meters hurdles race he knew exactly what lay ahead of him – ten hurdles measuring 1.067 meters high. The first hurdle placed at 13.72 meters from the start line with 9.14 meters between hurdles and 14.02 meters from the final hurdle to the finish line. Getting to the finish first would result in the gold medal and an assured place in the history books. But when it comes to developing life-changing cell therapies, there is no such certainty. The overall time course, the number and height of the hurdles are all variable (and often change on route) and even if the finish line is reached, the final outcome (adoption into routine clinical practice) may remain elusive.
Business and investors avoid uncertainty, thus stalling the progress of cell therapy, instead preferring 'sprint races' with few if any hurdles, such as social media where backing an idea to return on investment may take only months or at most a few years. Cell therapy will always need appropriate hurdles to ensure patient safety, clinical efficacy and product cost effectiveness – but not uncertainty including needless delays. The aim of the HoL's report is to greatly reduce the uncertainty from the clinical translation and commercialisation pathway. If the UK Government robustly backs this comprehensive set recommendations, Britain will become the first country to have both availability of R&D funding (Biomedical Catalyst, MRC, NIHR, TSB and charities including UKSCF and Wellcome) and a supportive infrastructure to accelerate the delivery of cell therapies into routine clinical practice.
Speed of adoption of the recommendations is also required. Other nations, including Japan (6) and Korea (7), are already lowering their infrastructure hurdles. The comprehensive approach that is proposed in the HoL report is way ahead of our competitors, but we cannot be complacent as they too will read the report and take notice. Just as the UK Stem Cell Initiative (the 'Pattison Report') (8) in 2005 quickly became the global blueprint for stem cell research, so too will this HoL report become an international exemplar for cell therapy translation and commercialisation.
Stream-lining regulation and creating innovative funding mechanisms, deploying the market pull of 63 million NHS patients and appropriate reimbursement rates, will provide British cell therapy companies with the much needed boost and also attract inward investment resulting in a globally competitive UK cell therapy cluster. The biggest challenge will be obtaining and maintaining enthusiastic buy-in from all the government departments and organisations involved along the critical development pathway following the initial scientific breakthrough. This will not be easy, but the enormity of the potential health and wealth benefits to the UK will far outweigh the effort required to fine-tune, leverage and integrate our world-class assets including the Cell Therapy Catapult, MHRA, NHS, NICE, NIHR and TSB. The wide-reaching recommendations in this Regenerative Medicine Report are the urgent call to action essential for making Britain the permanent home to a prosperous multi-billion pound cell therapy industry.
The UK from the outset has been a world-leader in stem cell and regenerative medicine research, but is today underprepared for the next steps - clinical translation and commercialisation, despite having an enviable line up of existing healthcare infrastructure. Meanwhile the rest of the world is catching up and overtaking us. In an interview with Aries Merritt after winning the Olympic gold medal (9) he said, 'The hardest thing to deal with as an athlete is watching people you used to beat do phenomenal things… Talent isn't enough to get to the top… I decided I didn't want to be a shoulda-woulda-coulda – I wanted to be something. It was a wake-up call'. Shoulda-woulda-coulda is the current destiny of the UK cell therapy industry. This HoL Regenerative Medicine Report is our wake-up call – the opportunity is ours to lose, it is now, it is urgent, and there is no second chance.