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Cell and gene therapy (CGT) also sometimes referred to as advanced therapy medicinal products is now being heralded as the future of medicine and rightly so since numerous successful trials have shown the potential of this innovative therapy to perhaps allow medicine to go from illness management to curative.
There are many different players in the CGT arena, most research and development of ATMPs is conducted by academics, academic spin-offs, not-for-profit organisations, and SMEs. Until recently, only a few larger pharmaceutical companies have engaged in the investigational phases of ATMP development, due to the perception that the early investigational phases of the development of ATMPs is a high-risk activity.1
The main ATMP therapeutic areas are oncology and regenerative medicine, particularly in the field of cardiovascular conditions and haematology. ATMPs offer revolutionary new prospects for the treatment of diseases or injuries, such as skin in burns victims, Alzheimer’s, Parkinson’s disease and cancer or muscular dystrophy.
Research from 2019, revealed that there were over 930 companies making advancements within the CGT arena2 and projections by BIS Research has estimated the value of the global industry to be worth over $12 billion by 2021.3
So what's the story pre-COVID pandemic? CGT is not new having been around for decades, but it was only officially recognized and introduced into the EU regulatory framework as a new classification of biological medicinal products in 2003. However, despite this introduction into EU legislation there still has been an uphill battle characterized by a myriad of not only legal but also ethical obstacles on the pathway from lab to patient, from; clinical trials and recruitment of patients, lack of investment, to the obscure regulations that have potential to slow the commercialization of these products.
Currently, the CGT market is dominated by North America as the healthcare systems there due to willingness to reimburse providers has made for a better commercial market for these treatments. This has arguably been the greater issue rather than regulation. Whether countries are willing to pay producers for the treatments. CGT comes at a great upfront cost, even though it can prove significant value for money when compared with the alternative long-term treatment options. For instance, in the FT Health’s special report Regenerative Medicine,4 it stated that gene therapy Zolgensma, for spinal muscular atrophy (SMA), is priced at $2.1 million one-off treatment but the existing non-ATMP treatment for SMA costs around $4 million over the space of 10 years.
The structure of healthcare systems therefore allowed North America to gain a considerable edge, despite Europe having a proud heritage for innovation in CGT. Notably, France has a rich history of trailblazing in this area with an acclaimed successful trail for immune-deficient babies back in the 1990s.
The UK did received a positive signal from the NHS over the possibility of reimbursements, with the deal that was struck with Novartis that allowed a baby to receive ground-breaking gene therapy treatment Zolgensma.
As discussed above the main issue is reimbursement, so how will the COVID-19 pandemic impact this?
While the pandemic did slow and even grind some actives within CGT to a halt with companies invested in ATMP research and production jumping into the fight against COVID-19, diversifying, adapting, collaborating, partnering, acquiring, to switch focus to vaccine production.
A huge amount can be esteemed by the industry (and regulators) about the vaccine response, and the truncated timelines for product development of what are being shown to be world-changing medicinal products.
Furthermore, the life sciences sector as a general received a flurry of positive attention from key stakeholders from the press to investors. Even those making strides towards innovation for efforts not directly related to COVID-19 vaccine production, such as within MedTech like improving online doctors and veterinary services received an uptick in attention. Research from the UK BioIndustry Association found that a record £1.6 billion was raised for the life sciences sector in just three months.5
The pandemic exposed inefficiencies with how current healthcare practices operated. But now, all eyes are on the life sciences sector and what innovation it can bring to healthcare. Investors that never considered the life sciences sector as an option are now clued up on the activities of the industry.
With this in mind it's likely that the pandemic has set a new precedent for the industry — with stakeholders engaged in serious discussions around how to make under 12-months development nearer the norm, rather than the often-quoted 12 years — not just for emergency vaccines, but also for life-changing treatments for smaller populations than a global pandemic.
Overall, it's becoming clearer that this pandemic is likely to open up the route to market for cell and gene therapy, meaning there will be opportunities for all the players involved from academic institutions to investors and pharmaceutical and biotechnology companies.
Cliodhna McDonough-Stevens is a life sciences regulatory lawyer at Fieldfisher.
1. P. Hourd, A. Chandra, N. Medcalf and D.J. Williams, “Regulatory challenges for the manufacture and scale-out of autologous cell therapies,” (June 30, 2014), StemBook, The Stem Cell Research Community.