Access to CAR-T therapies in Central and Eastern Europe in “catch-up” mode

Some EU member states in central and eastern Europe are still struggling to access chimeric antigen receptor (CAR) T-cell therapies compared to their Western counterparts despite regulatory approvals, oncologists say.

In June 2018, the European Medicines Agency (EMA) recommended approval of the first CAR-T treatments in Europe – Kymriah (tisagenlecleucel) from Novartis and Yescarta (axicabtagene ciloleucel) from Gilead Sciences. When it comes to the deployment of these therapies, EU states like Poland and Romania lag behind larger European countries like Spain, which have seen greater uptake. While access to these treatments improves in some countries as more CAR-Ts are approved, their high price could prove difficult for health systems with tighter budgets, according to the researchers. experts. Therefore, high prices could push pharmaceutical companies to focus on larger countries, potentially slowing the rollout of these treatments in smaller countries.

Despite EU efforts to tackle variable access across the bloc with initiatives such as the European Cancer Inequalities Registry, CAR-T therapies could deepen inequalities, says Center President Marius Geantă for Innovation in Medicine from Bucharest. Due to their high costs and logistical complexity, these treatments may require changes in healthcare models, especially in cases where they are underfunded, he adds.

Central and Eastern Europe at different stages of catching up

While CAR-T centers have expanded to multiple locations in countries like Czechia and Poland, which have also launched funding programs, other states like Romania and Slovakia are still in the early stages of development. ‘adoption. Clinical sites capable of administering CAR-T therapies are being added in countries like Spain, expanding access to treatment, says Dr. Fermín Sánchez-Guijo, professor in the Department of Medicine at the University of Salamanca, in Spain.

According to the Organization for Economic Co-operation and Development, Central and Eastern European countries include Albania, Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Slovakia and Slovenia. With the exception of Albania, all the countries listed are members of the EU.

In the Czech Republic, Dr Petr Cetkovský, director of the Institute of Hematology and Blood Transfusion in Prague, says the country’s CAR-T program has caught up with its Western counterparts. News of Yescarta’s first administration emerged in late 2019, and Kymriah was first used in a pediatric patient in April 2020. Compared to the Czech population of over 10.5 million, the number of patients who have been treated with CAR-T therapies are not “significantly worse” than in Western countries, he says.

Although there is some delay between when treatments are first available in the US and when they arrive in the EU, availability then depends on each country and its own processes, Cetkovský explains. The FDA approved Kymriah in August 2017. Different governments, reimbursement agencies, pharmaceutical companies and states offer treatments at different prices, he explains.

The Czech CAR-T program also benefits patients from neighboring Slovakia. Since Slovakia does not yet have access to CAR-T therapies, patients in need of such treatments are sent to Czechia, noted by email Dr. Andrej Vranovský, a doctor at the National Institute of Oncology in Bratislava. . There has been no particular delay in obtaining CAR-T therapies in the Czech Republic. They are now available at six centers across the country, including five for adult patients and one for children. Even Slovakia will soon see the launch of its own program, with the first patients able to be treated with CAR-T therapy at the National Institute of Oncology this summer, writes Vranovský.

However, elsewhere in the region, the use of CAR-T therapy is just beginning, as is the case in Romania, says Geantă. Romania approved reimbursement for Kymriah in December 2020. However, the country is still waiting for its first patient to be successfully treated with the therapy, he says. In southern Romania, at the Medical University of Sofia, Guenka Petrova, a professor in the Department of Social Pharmacy and Pharmacoeconomics, writes that there is currently no access to CAR-T technologies in Bulgaria, citing problems prices and a consequent lack of interest from companies.

Cost remains at the forefront of CAR-T deployment, with some governments taking this into account. This is the case in Poland, says Dr Maciej Niewada, a professor in the Department of Clinical and Experimental Pharmacology at the University of Warsaw Medical School, where the government has included innovative treatments in its new $4 billion medical fund. of PLN ($895.2 million). which was signed by the President of Poland in October 2020. While Niewada says CAR-T treatments are more likely to be covered as they are currently limited to relatively rare indications, access to these therapies in Poland is at “very beginning” of availability. The first Yescarta CAR-T treatment was administered at the end of 2019. In addition, six treatment centers in the country offer Kymriah. Kymriah will be reimbursed for adults from next month, while it has been covered for pediatric patients since September 2021.

Meanwhile, Western countries like Spain are seeing a “second wave” of new centers that are treating patients with CAR-T therapies, Sánchez-Guijo says, adding this is being seen in other EU states. like France and Germany.

Despite this, Niewada says there could be a backlog in access to these treatments in the region. However, this is the case for all expensive treatments, rather than just CAR-T therapies, he says, adding that “catch-up” and uptake are “generally slower.” According to Niewada, this is due to the high costs of treatments, which are more “settled” to do business in Western European countries.

Sánchez-Guijo, who is also vice president-elect for Europe at the International Society for Cell & Gene Therapy, says the introduction of CAR-T treatments is challenging not only because of cost, but also the need to obtain approval from each centre. by the pharmaceutical company. Cetkovský confirms that potential CAR-T centers need to be audited by companies to ensure they are able to offer such treatments, which is delaying their rollout. At the same time, it’s important to note that auditing and preparing potential centers adds a “certain burden” on pharma companies themselves, says Matthew Lakelin, co-founder of TrakCel, a provider of software solutions for the management. delivery of cell and gene therapy products. As such, the company may not want to launch its product in all countries, he adds.

Potential inequalities in the future

Any cost hurdles may become an even bigger issue as CAR-T therapies are approved in more mainstream indications or earlier treatment lines, Cetkovský says. This problem will not just be confined to Central and Eastern Europe, and expensive treatments for multiple myeloma or chronic lymphoblastic leukemia could potentially strain budgets even further. According to the clinical practice guidelines of the European Society for Medical Oncology, the the overall estimated incidence of acute lymphoblastic leukemia and lymphoblastic lymphoma is 1.28 per 1 million individuals per year, while multiple myeloma has an estimated incidence of 4.5 to 6 per 100,000 patients per year in Europe.

Cetkovský cites a 2017 paper co-authored by Dr. Mohamad Mohty, the former president-elect of the European Group for Bone Marrow and Transplantation (EBMT), to summarize this challenge, as it compared CAR-T therapy to a “narrow path between hope and failure”.

Different reimbursement approaches, such as the pay-for-performance model, could bring change to the industry, says Romain de Rauville, vice president of business development at Exothera, a Belgian contract development and manufacturing company specializing in manufacturing. viral vectors for gene therapy. .

That said, “harmonising” treatment access and pursuing reimbursements remains a challenge, which could ultimately lead to differences in patients’ ability to obtain treatment across the EU, says Sánchez-Guijo . According to the spokesperson for the European Commission, the agency has several plans and strategies to tackle these inequalities, including the launch of a pilot project on the marketing of centrally authorized products (CAPs). This project aims to improve the knowledge of regulators on the planned commercialization of CAPs and the reasons for the delayed marketing, specifies the spokesperson. Still, Sánchez-Guijo says it’s important to see initiatives in motion, rather than just being written down.

Coverage of cell and gene therapy on pharmaceutical technology supported by Cytiva.

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