Greek legal framework fails to address complexities of access to gene therapies

The Greek legislative framework lacks concrete solutions to advance gene therapies and improve access for patients, with several challenges – including financial support, treatment availability and stakeholder cooperation – remaining.

In Greece, over 500,000 patients have been diagnosed with rare diseases. A glimmer of hope for some rare disease sufferers is the option of gene therapy, which can be used to treat and manage illnesses for which there are no other treatment options.

However, Greece has not yet established any legal framework for gene therapies, Olga Katsarou, Director of the Blood Unit and National Reference Center for Congenital and Bleeding Disorders, told Euractiv.  

Moreover, it will “take more than 1 or 2 years [for such a framework] to be established”, Katsarou said.

After Euractiv’s research, it was found that no reference is made in the Greek bills to gene therapies and  no specific framework about conducting and accession to them for patients’ convenience has been established yet.

One positive step has been made for patients’ access with the Personalised Cellular & Gene Therapies next to the Greek Patient (POCare) Model investment programme, launched in 2021, but the scheme’s results are yet to be seen.

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.

A multifactor process with limited financial support 

Currently, access to gene therapies is a “complex process and depends on many factors”, Dimitrios Athanasiou, Chair of Rear Diseases Greece, told Euractiv.

In many cases, the cost of gene therapy is “as high as one, two or more million euros”, Athanasiou said, adding that it is certain that “no family or patient could cover this therapy with own resources”. 

Therefore, they need to find other sources of financing for their treatment. The availability of financial tools for patients in Greece varies depending on the type of condition, the patient’s situation and available coverage by the public health system or private insurance.  

He added that in Greece some of these therapies “are not always guaranteed covered by the National Health System”. 

The Greek National Health System (NHS) provides medical care to patients with rare diseases and they can receive medical treatment through the system. However, in some cases, this treatment needs to be reimbursed by private insurance as the NHS cannot fully cover it.

Some patients resort to charities and foundations which provide financial support or enforce the development of gene therapies in Greece. One typical example is Marianna Vardinogianni Foundation, which established the first Cellular and Gene Therapy Center for children and adolescents in Greece. 

Indeed, according to reports in Greek media, a new plan by the Ministries of Health and Finance, for a separate fund is being worked on, to which some wealthy Greeks could contribute with sponsorships, with special provisions to prevent the costs of gene therapies.

Yet, free access to gene therapies is still difficult to ensure for Greek patients. 

According to the Chair of Rare Diseases Greece Athanasiou, there are only special early access programmes in which “the state can come to an agreement with the pharmaceutical company in order to give access in gene therapies to a small number of patients and for a limited period”.

However, the “details and prerequisites” for patients’ eligibility “may vary depending on patient’s disease and the treatment process the patient can undergo”, Athanasiou told Euractiv.  

Italy’s advanced therapy journey

Advanced Medicinal Therapies (ATMPs), including gene and cell therapies, offer treatment options for patients suffering from rare or ultra-rare diseases. Italy is learning to see expenditure on advanced therapy drugs as an investment but the EU’s pharmaceutical revision is causing concerns among Italian stakeholders.

Restrictions in therapy availability 

Another important issue regarding gene therapies is their limited availability.

“95% of rare diseases have no cure since, due to the complexity of the diseases and the limited number of patients, no specific treatments have been efficiently developed,” Athanasiou told Euractiv, referring to recently published data.

This can lead to further difficulties in accessing gene therapies, since patients may not have access to effective treatments and pharmaceutical products which save their lives.

In Greece, the problem is bigger due to the lack of appropriate infrastructure. 

Athanasiou explained that the “small number of specialised laboratories and infrastructures for carrying out gene therapies” put additional constraints on gene therapy development as well as on patients’ access to them. 

He added that while there has been an increased interest by the scientific community in Greece regarding these therapies in the last years, “there is still a lot of work to be done to ensure safe and universal access”.

Another issue Greek patients face when accessing gene therapies is delayed or incorrect diagnosis. Due to their scarce nature, rare conditions are difficult to diagnose. 

Almost 50% of patients with rare diseases are undiagnosed or remain with incorrect diagnoses from five to 30 years, if not their entire lives, according to data derived from the Rare Disease Greece Association. Therefore, access to gene therapies and treatments becomes more complicated for patients and any delays in treatment could lead to further deterioration of their condition and quality of life. 

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.

Cooperation among Greek stakeholders is imperative

Technology has made big advances during the last decade regarding gene therapies and rare disease treatments, however, cooperation among all EU member states and stakeholders is necessary to ensure patients’ access to therapies.

Athanasiou also highlighted the need to develop national access strategies with the cooperation of all stakeholders following the technology and scientific developments and new advances in gene therapies.  

He added that while reimbursement systems for gene therapies and the timing of access to them, are improving as the benefits of gene therapies are increasingly being recognised, “Greece has not yet reached the general average of the European Union”. 

According to him, it is important to encourage cooperation between stakeholders such as pharmaceutical companies, research institutions, scientists and patient organisations. 

“Concerted efforts” are required to improve patient accessibility to gene therapies in Greece, Athanasiou said.

There is also “the need to develop policies that will provide, inter alia, for the adoption of a National Action Plan for Rare Diseases and the creation of specialized Reference Centres for Rare Diseases”, Athanasiou told Euractiv, stressing out the need for concrete and collective action.

[Edited by Giedrė Peseckytė/Nathalie Weatherald]

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