Thanks to major scientific and technological breakthroughs, in recent years the world has seen a surge in novel complex and personalised therapies. The most popular of these is Cell and Gene Therapies (CGT), where the real complexities surrounding their manufacturing, regulation, and commercialisation increase the need for leading biopharma organisations to take innovative strategic decisions.
In this blog post we take an overview of the current CGT market landscape touching on the challenges associated with manufacturing and administering CGTs, the regulatory framework, as well as issues surrounding the commercialisation and cost.
The CGT market today
The history of CGT has shown an industry with incremental growth, and over the medium term, the prospects for the global cell therapy market are promising. Expectations are that the CGT market will exceed USD 93.78 billion by 2030, growing at a CAGR of 22.41%.[1] Right now, more than 1,000 cell and gene therapy candidates are being examined as potential treatments for a myriad of disease indications. This implies a steep rise in the number of CGTs that will be developed clinically and made commercially available over the next few years, putting pressures on biopharma organisations and payers to improve patient access to treatment and ensure affordability.
To ensure that these novel therapies can maintain and extend their success, a number of efforts are currently underway to address the many complications associated with these treatments; specifically, their manufacturing and production, their regulation, and their cost models and commercialisation.
Manufacturing and administering CGTs
At present, the CGT market involves more than 240 Contract Development and Manufacturing Organisations and in-house players.[2] The manufacturing processes involved make CGTs particularly challenging to scale up commercially in order to meet increased clinical demand, particularly due to the lack of standardization. In cases when a CGT is expanding its eligible population due to an additional indication or increasing its geographical presence, the process can be complicated and lengthy. In addition, CAR T therapies, in particular, are difficult to administer, especially for certain patients in oncology, such as those who might have come out of chemotherapy and/or are invariably frail.
To address these challenges, several large pharma companies are looking to enhance their cell therapy manufacturing capabilities. Recent moves include undertaking expansion projects and establishing strategic partnerships with other major players in the sector.
Initiatives such as these to mitigate the practical complexities of CGT underpin the market’s optimism that it can support significant growth in the foreseeable future. A forthcoming blog will discuss the manufacturing complexities of cell therapies, along with the different ways that industry stakeholders can invest to enhance their production chains.
The regulatory framework
The rapidly evolving nature of cell therapy, and the need for them to become viable clinical treatments in a timely manner, have put pressure on regulatory bodies to improve the support system available for sponsors.
Significant initiatives throughout 2022, led by the FDA, are now changing the regulatory landscape. In March 2022, the FDA released two draft guidance documents aimed at supporting companies that develop CGT products
Human Gene Therapy Products Incorporating Human Genome Editing, and Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products. The FDA also announced the Gene Therapy Pilot Program, in which regulators give real-time feedback to sponsors during their clinical development process.
Beyond individual initiatives, a key priority is to increase harmonisation between different regulatory bodies. This is particularly important for surrogate end-points where greater international consistency will help streamline the clinical development process so that it becomes more efficient and cost-effective for sponsors.
Nevertheless, the recent energy shown by the FDA in this area is critically important. Combined with the rising number of CGT products that are moving closer towards clinical trials, it is expected to drive significant developments in the CGT arena.
The commercial implications of high-cost CGT
While significant advances have been made to streamline the patient journey and reduce burden post-infusion, high pricing of CGTs remains a complex system for both healthcare professionals (HCPs) and patients to navigate, especially as a formalised guidance is still evolving and payment models are yet to mature. A future blog in this series will delve deeper into gene therapies reimbursement models and the current challenges faced in the industry.
In addition, due to the high cost of CGTs, HCPs and patients quite naturally expect to receive a high-quality, holistic service and we see treatment centres that invest in complex therapies become a direct client of the sponsor company. Until recently, providing direct and efficient customer service to hospitals and clinics was an unexplored area for pharma companies.
The learning curve has been steep, and it is being climbed. Recent years have seen a dramatic evolution of complex therapy support solutions as these treatments become more seminal and we can learn how to better manage them, to the point where the future of CGTs looks extremely exciting.
Look out for a blog within this series that will discuss how biopharma organisations can set new industry standards for customer service associated with complex therapies
Blog written by Ioanna Ferekidou & Chrystalla Constantinou
Resources
[1] Business Wire; 26th Jan 2023
[2] Roots Analysis; Cell Therapy – The Revolutionary Therapeutic Modality: All Set To Obliterate Oncological Disorders; 3rd Feb 2023