In France, gene therapy suffers from weak economic model

France is one of the most advanced countries for gene therapies, both in Europe and worldwide. However, the lack of funding and the economic fragility of the sector are holding back the development of these innovative treatments.

In 2016, a 13-year-old boy suffering from sickle cell anaemia, an inherited genetic disorder affecting red blood cells, was successfully treated with gene therapy by doctors at Necker Hospital in France. It was a world first.

“France is a major country for the invention of gene therapies, and many of them were born in France, notably at the Necker hospital,” said Eric Baseilhac of the professional organisation of pharmaceutical companies operating in Leem, France, in an interview with Euractiv.

Gene therapies, which form part of innovative therapies, involve introducing genetic material into cells to treat a rare disease. This treatment is used in particular to treat cancers and paediatric diseases.

Worldwide, around 2,000 clinical trials have been conducted or are underway since 1989, 65% of which involve cancer. In France, around 30 gene therapy trials have taken place or are underway, according to the French National Institute for Health and Medical Research (INSERM).

However, despite this success, it is difficult to make the development of gene therapies economically viable for pharmaceutical laboratories.

“France is finding it very difficult to raise the funds needed to support start-ups developing gene therapies all the way,” Baseilhac said.

If gene therapies are not very profitable for the companies that market them, it is mainly because of their high price, which is explained by the complexity of their manufacturing.

“Production costs for gene therapies can currently amount to several hundred thousand euros per patient. The challenge is to bring them down,” Frédéric Revah, a specialist in the research and development of gene therapies, told Le Monde newspaper.

According to Baseilhac, the high cost of gene therapies must be understood “in terms of cure”, i.e. lifelong efficacy.

Health Innovation Plan 2030

In June 2021, French President Emmanuel Macron announced the launch of the Health Innovation Plan 2030, which aims to make France the leading European nation in terms of innovation and sovereignty in healthcare.

“We are currently experiencing a real revolution in the fields of health and life sciences,” Macron said.

With a budget of €7 billion, the plan was intended to finance “major transformations” in healthcare, such as gene therapies and the use of artificial intelligence (AI).

However, according to Baseilhac, the plan alone will not be enough to accelerate France’s development and commercialisation of gene therapies.

“The plan does not solve the problem of the economic model for gene therapies and their funding,” he highlighted.

In France, the majority of companies working in such treatments are start-ups – and face a complicated business model.

According to the Leem expert, marketing a treatment that cures a rare genetic disease means “resolving to have nothing left to sell after a few years”.

The only way for these small companies to continue to be economically viable would be to create new gene therapies every five years or so, but this is a real challenge.

“So they are often bought up by larger laboratories,” said Baseilhac, such as Novartis.

Europe lags behind the US

Access to gene therapies for patients is restricted not only in France but throughout Europe, not least for economic reasons.

“In Europe, we are still looking for a sound economic model that will enable us to offer access to these therapies on our territory”, said Baseilhac.

Christian Anastasy, chairman of strategic healthcare consultancy firm Persan Conseil, said in a press release: “While the unit cost of the treatment is significant, let’s not forget that this investment is sustainable and that it will generate savings on the various forms of care that will no longer be necessary.”

Gene therapies are mainly used to treat rare diseases. “Up to 36 million people in the EU live with a rare disease. There are more than 6000 distinct rare diseases in the EU. So, whilst one rare disease may affect only a handful of patients, another may touch as many as 245 000. Around 80% of rare diseases are of genetic origin and, of those, 70% already start in childhood”, according to the Commission data.

“For the time being, there is no great pressure from European patients because these therapies affect so few people. This is unfair compared with patients based in the United States,” added Baseilhac.

In fact, there are dozens of gene therapies in the United States, compared with just a handful in Europe and one in France. “The EU is out of step with the United States. This is not acceptable”, he concluded.

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.

[Edited by Giedrė Peseckytė/Nathalie Weatherald]

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