Finding the Right Balance for Advanced Therapies

Scientific innovation and regulatory reformations are helping to drive the global biopharma market forward, but a balance between cost and value still needs to be accepted for widespread adoption.

Globally, the biopharmaceuticals market has been experiencing growth and is expected to continue its upwards trajectory in the near future. According to market research, the global biopharmaceuticals market is forecast to grow at a compound annual rate of 7.32% between 2021 and 2026 (1). Drivers for the swell in biopharmaceuticals include the rising aging population, increase in the prevalence of chronic diseases, and expanding acceptance of the complex molecular drugs as treatments for diseases that were previously untreatable (1).

A subset of the biopharmaceutical market is advanced therapeutic medicinal products (ATMPs) or cell and gene therapies (CGT). These regenerative medicines have the potential to provide patients with curative treatments for a host of diseases and, as such, have been piquing the interest of big players in the industry for some years now, which is apparent through the influx in M&A activity and investment in the space (2).

Current market

According to the International Society for Cell & Gene Therapy (ISCT), whose expertise lies in the translation of CGTs, the science surrounding CGTs is currently advancing faster than can be feasibly translated through to regulatory approval of therapies. And, in general, clinical trials are being performed with shorter-than-ideal follow-up intervals and smaller-than-ideal trial participants. However, there continues to be an influx of investment in regenerative medicine due to the success of chimeric antigen receptor (CAR) T-cell and recent gene therapy approvals, ISCT specifies.

As an example of a challenge in translating scientific innovation, Massimiliano Gnecchi, MD, PhD, ISCT regional vice-president, Europe, notes the struggles that are being experienced in Europe. Despite being home to 16 of the top 50 global life sciences universities, a leader in CGT research, and a region from which there were 120,000 CGT papers between 2017 and 2019, Europe has only accounted for 16% of the total CGT patent registrations up until 2018, Gnecchi stresses. This percentage can be compared with 56% and 28% registrations in China and the United States, respectively, even though these countries had less prominence in research papers.

One of the reasons for the performance gap between Europe and other areas can be attributed to a lack of investment, explains Gnecchi. “In 2018, the known value of US biotech deals reached $85.9 billion, some five times higher than the value of European deals, which reached $16.3 billion,” he says (3).

Another reason for the performance gap is a lack of funding in translational medicine, Gnecchi continues. “US government funds go a long way to plugging this funding gap through the Clinical and Translational Sciences Awards Program, which has a budget of more than $550 million a year for translational medicine,” he reveals. “European funding pales in comparison. As part of the Horizon Europe program, the European Union will invest about €7 billion (US$8.21 billion) in all health research between 2021 and 2027, but only a fraction of that will go into translational medicine.”

Furthermore, the European region must contend with a fragmented market and variances in regulations across the European Union member states, Gnecchi confirms. “Though unmet need might be higher than in the US, it is much harder to address at scale given different market regulations in different European countries,” he says.

Asia is being heralded as one of the fastest growing CGT markets; however, ISCT stresses that there are widely varying opinions about the size of growth in that region. For example, one report forecasts that the total Asian CGT market will grow from US$3.3 billion in 2018 to US$72.2 billion in 2030 (4), while another report has predicted a much more modest growth, with the Asia Pacific cell therapy market reaching US$2.9 billion in 2028 (5).

Essentially, the sector is burgeoning and trying to find a suitably comfortable state of equilibrium between scientific advancements and commercial viability. “We are trying to balance the excitement of having new ways to make a permanent and definitive difference to many serious diseases, how to evaluate, value, and pay for [ATMPs], and then make [ATMPs] available in an equitable manner,” adds Sven Kili, MBChB, MRCS (Eng), co-chair, ISCT Business Models and Investment Subcommittee.

Market drivers and hindrances

The rapid scientific advancements—as a result of the imagination of scientists and clinicians in their mission to seek new and unusual ways of treating diseases, and a willingness of patients to try new approaches to treatment in combination with an increased ability to manufacture advanced therapies—are driving the market forward. The combination of these factors has led to significant record-breaking investment (6) and suggests that the growth driving CGT advancement will push the broader biopharma market forward as well, ISCT states.

However, the high costs of goods required for CGTs, directly impacts the cost of the therapies—with CGTs remaining as some of the most expensive therapies available. Unless an accepted and effective cost/value mechanism is taken up by health technology assessment organizations, such as the National Institute for Health and Care Excellence and the Institute for Clinical and Economic Review, adoption of CGTs may be limited, ISCT highlights. To be able to broaden out applications from those that are niche to widespread adoption of CGTs, it is important that manufacturing and cost of goods issues are optimized, while excellent and persuasive clinical efficacy are proven at the same time, ISCT reveals.

The biopharma market is not insulated from the new and innovative challenges companies are facing today, including cybersecurity issues, ISCT continues. Life sciences companies are increasingly susceptible to cyber-attacks on valuable intellectual property, digital assets, and sensitive medical data. Healthcare companies have consistently been rated as the lowest performers in protecting against cybersecurity risks as many of them rely on out-of-date legacy systems and have only recently started investing in this area.

Holistic growth

For ISCT, it is vital that scientific and commercial growth are promoted to ensure the whole sector can prosper. When looking at scientific advancement around the world, government investment has played a major role, ISCT explains.

For example, in the United Kingdom (UK), the government has invested in innovation hubs (Cell and Gene Therapy Catapult), and there has been a massive boom in China due to state investment in new technologies. Additionally, in the US, the National Science Foundation has funded the Engineering Research Center CMaT (Cell Manufacturing Technologies), which is a consortium of universities, industry, and non-profits engaged in the development and translation of cell therapies along with workforce education and training for the sector, ISCT asserts.

ISCT adds that the Australian Federal Government has established an AUS$500 million Biomedical Translation Fund (BTF) with AUS$250 million of government capital and AUS$250 million private sector capital to be used to invest in biomedical discoveries, particularly targeting projects at advanced pre-clinical and Phase I and II stages of development. The BTF is intended to complement the Medical Research Future Fund (MRFF), which is currently valued at AUS$20 billion.

Sector growth is often commercially tied to fast growing economies as these, combined with mature regulatory environments, provide the conditions necessary for extensive expansion, ISCT states. Focusing on Asia, Japan ranks as the most active in market approval of new CGTs after the country’s regulatory reform in 2013–2014 has allowed for a proactive conditional approval of therapies. Japan is also home to big companies, such as Fujifilm and Astellas, which are investing a lot of money in CGTs and appropriate manufacturing technologies, ISCT specifies.

Korea has been a leading country in the cell therapy market having 16 approved products developed by domestic companies (7), including two immune cell therapies and four mesenchymal stem cells therapies, ISCT reports. Korea has also enacted new legislation on CGTs in August 2020 that includes the conditional and fast-track approval process and facilitates clinical research (8).

Reformations of CGT regulations have also been happening in other countries within Asia, such as China, India, Taiwan, and Singapore, ISCT continues. These reformations have provided a favorable landscape for market approvals of CGTs with new products expected to be available soon, ISCT stresses.

Trends for the future

The cost of goods will be improved in the future, particularly as there is continued scale up of cell and gene therapies and the industry gains a better understanding of critical manufacturing components and quality attributes, which can lead to manufacturing products in a more scalable and automated way, ISCT states. Additionally, the rate of innovation and investment in the sector will continue to move in an upwards trajectory, fuelling exponential increase in biopharma clinical trials, especially in the areas of CGT and messenger RNA (mRNA) manufacturing, ISCT continues.

Furthermore, mRNA technology, which has been validated thanks to the success of the COVID-19 vaccination programs, will witness further development in other areas, ISCT explains. In-vivo gene therapy will also continue to expand.

However, industry will also continue to experience challenges and disruptions because of the pandemic, ISCT warns. Supply chain disruptions will continue and raw materials, that are required for CGT products, will still be difficult to obtain by manufacturers.

References

1. Mordor Intelligence, Biopharmaceuticals Market—Growth, Trends, COVID-19 Impact, and Forecasts (2021–2026), Market Report, May 13, 2021.
2. finnCap Group, “Cell and Gene Therapy: The Future of Medicine Attracts Major M&A Activity,” Blog, Aug. 6, 2020.
3. A. Loche, et al., “A Call to Action: Opportunities and Challenges for CGTs in Europe,” McKinsey and Company, Article, Jan. 19, 2021.
4. Visiongain, “Visiongain Publishes Cell Therapy Technologies Market Report 2021–2031,” Press Release, March 15, 2021.
5. Research and Markets, Asia Pacific Cell Therapy Market Size, Share, and Trends Analysis Report by Use-Type (Clinical-Use, Research-Use), by Therapy Type (Autologous, Allogenic), and Segment Forecasts, 2021–2028, Market Report, June 2021.
6. Alliance for Regenerative Medicine, Regenerative Medicine in 2021: A Year of Firsts and Records, Report, August 2021.
7. N. Cuende, et al., Cytotherapy, 20 (11) 1401–1412 (2018).
8. MFDS, Act on the Safety of and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, July 3, 2020.

About the Author

Felicity Thomas is the European editor for BioPharm International.