Regenerative therapies have ushered in a new era in medicine. Cell and gene therapies can now be used to effectively treat what were previously incurable diseases.
However, are we yet in a position to produce them economically on an industrial scale to deliver those therapies to a broad mass of patients?
The mass production of drugs for the global population is still in high demand; but, increasingly, the patient is in the focus of current research.
The application of individualised or personalised medicine is a trend that will continue to have a massive impact on the pharmaceutical industry in the future.
As such, novel therapies such as advanced therapies, including cell and gene therapies, play a major role. Today, most of those therapies focus on the treatment of rare diseases; however, there are many efforts under way to extend this to widespread diseases such as cancer and diabetes.
A rare disease is typically identified as an illness that affects fewer than five in 10,000 people worldwide.
Despite the infrequency of each specific condition, more than 300 million people worldwide are living with one of 7000 currently identified rare diseases.
At the same time, only about 5% have an FDA-approved treatment.1 Current therapeutic approaches in the field of advanced therapies have the potential to treat and even cure many of these.
Although the costs of these specialty therapies are immense, the IQVIA, the Institute for Human Data Science, anticipates that these expenses will not drastically escalate growth in spending as these specialty products only apply to small patient populations and are therefore produced in small batch sizes.2
Cell and gene therapies on the rise
Besides the fact that there will be many specialty therapies in the marketplace, new approaches to treat widespread diseases are already in early clinical trials in humans — and their approval and subsequent availability will take place progressively.
Last year alone (2020), 17 breakthrough therapies were approved by the US FDA by 30 June … and more are expected.3 The development of these novel products is mainly driven by oncology therapies.
Last year, 30% of the total expenditure on specialty drugs was attributed to oncology in developed markets, with a forecast increase of 51% by 2024.2
Hence, the market for specialty therapies, including advanced therapies, is taking up an increasing share of our current pharmaceutical industry.
Built on decades of diligent research, advanced therapies, or as pharma giant Novartis likes to call it, “a new area in medicine,” is the next step in personalised treatment.
Genes play an extraordinary role in our life — when they are changed or missing, they cause disease. The approach of Advanced Therapy Medicinal Products (ATMPs) is to treat the root causes of diseases and disorders.
This includes gene therapies, cell therapies and tissue-engineered products that are intended to augment, repair, replace or regenerate organs, tissues, cells, genes and metabolic processes in the body.
Unlike conventional medicine that often needs to be taken for long periods of time, most cell and gene therapies are designed to be a one-time treatment for extremely ill patients.
Through the use of groundbreaking scientific discoveries and technologies, ATMPs are creating transformative, permanent treatments and potential cures for some of humankind’s most devastating diseases, of which many are untreatable with conventional methods at the moment.
This is why, in medical circles, there are high hopes for cell and gene therapeutics.
A distinction is made here between in vivo gene therapeutics, which can specifically repair genetic defects (viral vectors, for example) and cell therapeutics such as CAR-T cells, which are genetically modified cells that are able to cure cancer.
The first CAR-T cell products — such as Kymriah (Novartis) and Yescarta (Kite/Gilead) and viral vector products such as Zolgensma (AveXis/Novartis) and Luxturna (Spark Therapeutics/Roche) — have been approved … and the results are promising.
There is an abundant pipeline for pharmaceutical and biotech companies, and cur-rent market research data is showing that a new era in medicine has begun.
Annual market growth of 33%
According to the Alliance for Regenerative Medicine, an international multistake-holder network with more than 350 members, there were in excess of 1000 cell and gene therapies undergoing clinical trials, at the end of 2019, most of them at Phase II clinical stage.4
The FDA expects that 10–20 new regenerative remedies, including cell and gene therapies, will be approved every year during the next 5 years.5
This massive rise in new registrations will lead to an increase in the market share of cell therapeutics, from €1.3 billion in 2017 to approximately €13 billion by 2025.
Between 2017 and 2025, the robust compound annual growth rate (CAGR) is thus 33.2%.6 This dramatic growth calls for innovative technologies that make it possible to produce small-to-medium batch sizes industrially and cost-efficiently with technologies that are not available at present.
Challenges ahead
This industry is currently facing many challenges. Research and development (R&D) in clinical studies usually takes years, as does approval by the FDA or EMA, and requires immense costs.
A lack of funding in the early stages prevents innovative products from gaining access to the market, and irregularities in government regulations for R&D restrict research activities, which could thus hinder growth.
Additionally, production technologies in the early clinical phases are not well suited for commercial production at higher scales. In the field of cell therapies, especially for autologous therapies, we talk about a scale-out functionality rather than a scale-up.
Small batch sizes or even one single patient batch has to be produced individually and incurs high costs.
For instance, novel genetically modified cell therapies such as Kymriah and Yescarta, which represent some of the few currently approved CAR-T cell therapy products available on the market, are priced between $300,000 and $500,000.
As drug manufacturers are moving towards allogeneic stem cells — that is, stem cells from a donor rather than the patient’s own cells as in the autologous procedure — the lack of sufficient donors might impede cell cultivation and subsequent drug development.
At present, producing these valuable drugs is still very expensive and time-consuming … and is largely done at laboratory scale.
“We have received a growing number of enquiries about automated production and filling solutions,” reports Juergen Rothbauer, Managing Director of OPTIMA pharma GmbH, a German solu-tion provider for the production and fill/finish of cell and gene therapeutics.
References
- https://alliancerm.org/indication-data/rare-disease-2019-pdf.
- www.iqvia.com/-/media/iqvia/pdfs/institute-reports/globalmedicine-spending-and-usage-trends.pdf?_=1593529881375.
- www.fda.gov/media/97001/download.
- https://alliancerm.org/sector-report/2019-annual-report.
- www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics.
- https://store.frost.com/growth-opportunities-in-the-global-cell-therapy-market-forecast-to-2025.html.