The first gene and cell therapies have been approved for use in Europe and many more are expected to enter European markets in the near future. These state-of-the-art techniques can revolutionise medicine as we know it. Bringing high hopes for thousands of patients in Europe as they become available in some countries, they also spark a lot of questions.

The history of medicine counts many chapters from early medical traditions to recent inventions that shaped the way humans approach diseases. The next chapter may very well be written as we speak. Gene and cell therapies are the latest addition to a series of technologies that have expanded the realm of medicine. Researchers around the world are actually making it happen.

Having some of the pioneers in gene and cell therapy research as member companies, EUCOPE plays an important role in raising awareness of these new treatments and in ensuring an appropriate framework is in place for patients to reap the benefits of gene and cell therapies.

With this in mind, EUCOPE has recently published a set of the frequently asked questions about gene and cell therapies. The list, available on our website, aims at providing the key answers to better understand this complex topic.

What is a gene therapy?

Gene therapy is the use of genetic material to treat genetic diseases. This may involve adding a wild type copy of the gene (gene addition) or altering a gene with mutation to the wild type gene (gene editing). The treatment may take place outside of the body (ex vivo) or inside the body (in vivo).[1]

What is a cell therapy?

A cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells). The cells used in cell therapy can be classified by their potential to transform into different cell types. Pluripotent cells can transform into any cell type in the body and multipotent cells can transform into other cell types, but their repertoire is more limited than that of pluripotent cells.[2]

“A promising alternative or adjunct treatment for symptoms of many diseases”.

Gene and cell therapies have the potential for high therapeutic gain for a broad range of diseases, improving the quality of life of persons living with severe or chronic diseases such as cancer, rheumatoid arthritis, diabetes, Parkinson’s disease, Alzheimer’s disease, haemophilia, and neurodegenerative diseases.

However, these new therapies require adequate and agile health systems to adjust and provide a balanced framework, leading to an equal access for patients.

Evidential Uncertainty

The current health technology assessment (HTA) models are not fit-for-purpose. Flexible HTA processes should recognise the limited number of patients and the absence of comparators, whilst taking into account the highly positive short term and best available scientific evidence. Those measures can be strengthened by the alignment of registries requirements across Member States, facilitating data collection to demonstrate ongoing safety and efficacy and improve evidence base.

“A proper application of the Directive on Cross-border healthcare (2011/24/EU) can help ensuring no patient is barred from accessing those revolutionary treatments”.

Gene and cell therapies are highly personalised treatments with complex manufacturing and distribution processes. Many of these therapies will be available in only a small number of treatment centres in Europe with the critical expertise in manufacturing and the administration of therapy.

Affordability

It is necessary to consider the affordability issues for health systems in a multi-year longer-term deal structure. Gene and cell therapies have the potential to be transformative, by bringing benefits to the patients, but also ultimately savings on medicines budget on the long term. Only a collaborative approach to funding/reimbursement/payment mechanisms can ensure appropriate pricing systems, with adequate incentives for research and innovation are in place.

These are right now the most compelling questions all stakeholders, including decision makers at the EU and national level must take into consideration when examining the opportunities offered by gene and cell therapies.

It is therefore highly relevant for stakeholders to get together and find solutions for European patients to gain access to these revolutionary therapies.

EUCOPE and its members work hand in hand in raising awareness of these new therapies and provide a trusted platform to elaborate solutions in terms of access, payment models and delivery challenges.

For further reading, please see our position paper on gene and cell therapies and our FAQs.

[1] European Society of Gene & Cell Therapy, https://www.esgct.eu/Useful-Information/Gene-and-cell-therapy-glossary.aspx

[2] American Society of Gene & Cell Therapy, https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs