Building Europe’s gene therapy ecosystem

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Gene therapy is a transformative field of medicine that could halt diseases before patients become symptomatic, saving lives and improving quality of life for millions of people who suffer from conditions caused by genetic abnormalities1. But these breakthrough treatments require new approaches to pricing and reimbursement2

While the science behind gene therapy has existed for decades, the first generation of treatments is now emerging for debilitating conditions including spinal muscular atrophy3 and inherited retinal diseases4 5. Although Europe was at the forefront6 of science a decade ago, other regions have invested heavily in research and Europe has work to do to re-gain7 its leadership role. 

A cell is the fundamental biological building block of all living things8. Genes, found within cells, are small sections of DNA that carry the cell’s genetic information. Genes are the cell’s blueprint for making proteins that support functions such as muscle strength, and mutations in genes contribute to a range of diseases including those caused by recessive gene disorders, like hemophilia and sickle cell anemia, acquired genetic diseases such as certain cancers, and some viral infections like AIDS9.

Gene therapies improve and/or replace mutated genes, most often via the technique of recombinant DNA technology in which a molecular ‘carrier’ known as a vector is used to carry a healthy copy of the gene and introduce the new genetic material into the cell11. Most commonly, gene therapy allows cells to add an additional genetic unit, but new engineering techniques now exist to switch off, allow modification to or correct the problematic DNA sequence12

Europe’s gene therapy leadership 

Academic researchers, biotech and pharmaceutical companies in Europe have been instrumental in developing the field of gene therapy13. Between 2017 and 2019, the lead author of 120,000 papers in cell and gene therapy (CGT) publications was affiliated with a European institution, compared to 72,000 and 100,000 in the U.S. and China respectively14. Professor Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin was a joint winner of the Nobel Prize in Chemistry 2020, a recognition of her contribution to the development of tools for gene editing15

The European Union’s medical regulatory institution, the European Medicines Agency (EMA), has been a world leader in assessing advanced therapy medicinal products (ATMPs), a class of innovative biological products of which gene therapy is part, approving a tissue-engineering product in 2009, a year before the U.S.’s first ATMP approval16. It also approved the world’s first gene therapy in 201216. The EMA has granted orphan drug status to the majority of CGT drugs currently in development and performed accelerated assessments for a number of cell and gene therapies14

But the region needs to build on its momentum to take gene therapy from the lab to patients. The total number of gene-, cell- and tissue-based therapeutic developers worldwide lags behind the North American and Asia-Pacific regions17.

Europe’s gene therapy sector could be boosted by introducing more flexibility and agility to the methodologies that are used to evaluate and reimburse breakthrough treatments.

Because the diseases that cell and gene therapies are aimed at are rare, trials are conducted in a far smaller number of patients than is normal for a new medicine. Decisions to license the drug will be based on the data from one small trial7.

Health Technology Assessment (HTA) bodies, who ultimately determine whether a new therapy is reimbursed, expect to review randomized controlled trials against a standard of care18. Yet gene therapies are a new approach that can effectively intercept, rather than manage, a disease — and there is therefore no standard of care to compare against19

Cost-effectiveness is commonly evaluated based on the direct financial burden of a disease on the health system18. Yet many genetic diseases’ biggest impact is indirect — inherited blindness, for instance, is costly for the individual, their family and carers, and most costs are borne in the social and welfare system, rather than in clinics and hospitals20. One review by Deloitte estimated the total cost attributable to inherited retinal diseases at £523.3 million in 2019, across economic and wellbeing costs (see chart). Health system costs were the lowest share20

Gene therapies that have the potential to intercept a disease entirely, are more difficult to quantify in terms of benefits19. Conventional HTA methods discount a benefit over time as a patient’s quality of life deteriorates due to aging and its other attendant health consequences21. This makes little sense in the case of a gene therapy that can avoid blindness emerging in a young person, say, thereby allowing them to lead a full and active adult life21.

Unlike rare disease treatments that are regularly administered over decades, gene therapy would be administered only once, providing many years, if not a lifetime, of biological activity and clinical benefit2. Under current reimbursement systems, this therapy would be paid once at the time it is administered. To encourage investment in the development of these therapies, payments in excess of $1 million may be needed2. The fact that one intervention can provide a cure, replacing long-term costs of treatment, makes investment in gene therapy a problem that multiple stakeholders need to work together to solve2. The industry is developing new approaches to pricing and reimbursement, such as outcome-based payment models22, or annuity payments23 based upon duration and efficacy, which could help payers accommodate the one-time nature of gene therapies.

Europe is already moving towards a coordinated response to review the challenges of breakthrough therapies. In December 2021, Europe’s Regulation on Health Technology Assessment was adopted to help member states to make timely and evidence-based decisions on patient access to innovative health technologies24. Gene therapies are amongst the medical breakthroughs that European reform dialogue is focused on to address unequal access across the continent25

Building on this momentum can bring forth the required change to value assessment methodologies and help to realize the full potential of gene therapies. It will ensure gene therapy, long confined to research laboratories, can finally reach patients, and open a new chapter in medical history21.

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1Mayo Clinic Staff. (2017). Gene Therapy. Available at:

2Brennan, T. and Wilson, J. (2014). The special case of gene therapy pricing. Available at:

3Vamshi K. et al. (2018). Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease. Available at:

4EMA Europe. (2018). Luxturna, European Medicines – summary of product characteristics. Available at information_en.pdf  

5Maldonado, R. et al. (2020). Curative gene therapies for disease. Available at:

6Cressey, D. (2012). Europe Nears First Approval for Gene Therapy Treatment. Available at: treatment/

7Collis, H. (2022). Patients with rare diseases hope for Brexit – hastened treatments. Available at: diseases/

8NCBI. (1989). The Fundamental Biological Unit of All Living Organisms is the Cell. Available at:

9NCBI. (2017). Gene therapy: advances, challenges and perspectives. Available at:

10American Society of Gene + Cell Therapy. (2021). Gene, Cell, & RNA Therapy Landscape. Available at:

11Britannica. (2022). Recombinant DNA – Gene therapy. Available at:

12Medline Plus. (2022). What is gene therapy?. Available at:,existing%20DNA%20in%20the%20cell

13AuWerter, T. et al. (2020). Biopharma portfolio strategy in the era of cell and gene therapy. Available at: strategy-in-the-era-of-cell-and-gene-therapy

14Loche, A. et al. (2021). A call to action: Opportunities and challenges for CGTs in Europe. Available at: opportunities-and-challenges-for-cgts-in-europe

15Rogers, K. (2020). Emmanuelle Charpentier. Available at:

16NCBI. (2019). Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States. Available at:

17Alliance for Regenerative Medicine. (2022). Regenerative Medicine: Disrupting the status quo. Available at:

18EUPATI. (2022). HTA systems in Europe. Available at:

19Pochopień M et al. (2021). An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models. Available at:

20Deloitte Access Economics & Retina International. (2019). The socio-economic impact of inherited retinal dystrophies (IRDs) in the United Kingdom. Available at: economics-cost-illness-irds-uk-030919.pdf

21Salzman, R. et al. (2018). Addressing the value of gene therapy and enhancing patient access to transformative treatments. Available at: 

22Jørgensen, J. and Kefalas, P. (2019). The use of innovative payment mechanisms for gene therapies in Europe and the USA. Available at:

23Cook, F. et al. (2018). Regenerative Medicine is Here: New Payment Models Key to Patient Access. Available at:

24HTA Regulation Press. (2021). Health Technology Assessment: Commission welcomes the adoption of new rules to improve access to innovative technologies. Available at:

25Cornetta, K. et al. (2022). Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India. Available at:

CP-330123 July 2022