EU needs to step up to unleash gene therapies potential, stakeholders say

While gene therapies have the potential to transform the approach to rare diseases and improve the quality of life for patients, stakeholders and patients complain that they are very expensive to develop and difficult to access.

Gene therapies are innovative treatments that try to address the genetic cause of a disease by replacing the malfunctioning gene, inactivating it, or introducing a new or modified gene into the body to help fight the disease. 

Also referred to as Advanced Therapy Medicinal Products (ATMPs), these treatments are mostly used to treat rare diseases which does not come as a surprise as around 80% of rare diseases are of genetic origin.  

There are currently 6,000 different rare diseases in Europe affecting 36 million people. 

Gene therapies have been investigated by the pharmaceutical sector since the 1990s. However, it was only in 2012 that the first gene therapy – an in vivo treatment targeting an ultra-rare disease – was approved for the EU market.

Since then, there have been 18 gene therapies authorised by the European Medicines Agency. However, not all of them are still available. In May 2023 there were only 15, after three withdrawals of marketing authorisation as the companies did not find the commercialisation of their product profitable. 

The small number of therapies that reach marketing authorisation is worrying for the industry as the development of the treatments is exceptionally expensive, but also for the patients, who see their opportunities highly reduced.

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Need for a common approach

To guarantee access for all patients to these kinds of therapies, Simone Boselli from Eurordis, an NGO working for rare diseases in Europe, stressed the need for cross-border care and harmonisation of the framework across the EU. 

“Collating the evidence generated from one country to another leads to potential better decision making, also in pricing and reimbursement,” he said. 

Due to the complexity of the diseases and the low prevalence, expertise and capacities are often missing within a region or country of the European Union. 

François Houÿez from Eurordis recognised that it is clear that not all countries need to have specialised centres able to administer these therapies for a small number of patients, but patients, wherever they live, should be able to travel to the specialised centre and receive treatment there.

That is why stakeholders criticised the incentive scheme set by the European Commission in the new proposal for pharmaceutical legislation, aimed to encourage investment in Europe and address the current industry challenges.

The Commission’s proposal includes extra regulatory protection for those companies that place their products in the 27 member states. This tool, aiming to ensure accessibility across Europe, is being questioned by stakeholders, in the case of rare diseases, for being unachievable. 

“These incentives are not attractive enough for gene therapies”, explained Boselli. Eurordis maintains that there is no need for a product to be placed in the market in all member states but it should be available only for a few centres to serve the entire population. 

“A common European pathway would help in this situation to get to resolve this conundrum that we have, that we have more scientific innovation, but less patient access to those therapies,” he added. 

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Long way ahead

One of the tools the EU could use to harmonise the gene therapies environment across member states is the new regulatory framework for Health Technology Assessments (HTA). 

The legislation, adopted in 2021, will enter into force in 2025 when all cell and gene therapies, among others, will undergo a single EU assessment instead of the current 27 for each country. 

The HTA regulation will help EU countries determine the effectiveness and value of new technologies and decide on pricing and reimbursement by health insurers or health systems. 

Novartis, one of the few companies with a gene therapy authorised in Europe, also told Euractiv that the fragmentation of the framework was one of the main challenges they encountered when commercialising their product. 

“We were talking to HTAs in Europe and we were looking at nearly 30 different systems that all required a tailored approach,” a spokesperson explained. 

The spokesperson added that their future hope is for a greater alignment of regulatory and HTA evidence requirements, adding that without payers and HTA bodies creating pathways for ATMPs, the true impact of innovation may not reach patients. 

Houÿez told Euractiv that they have “good hopes” as it seems the EU has taken the initiative to address this situation. 

However, he recognised that small fixes will not solve the current situation.

“We will need a huge change in the organisation of the pharmaceutical market.”

[Edited by Giedrė Peseckytė/Zoran Radosavljevic]

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