Patient access to Slovak market’s most expensive drug remains blurry

In Slovakia, a dispute has resurfaced over who should foot the bill for highly expensive drugs – with patients and their caregivers struggling under the unclear regulatory system.

Between six and nine children are diagnosed with Spinal Muscular Atrophy (SMA) in Slovakia every year. This hereditary neuromuscular disease affects the nervous system that controls muscle movements. While intellect stays intact, children gradually lose the ability to walk, stand, or sit, and occasionally, breathing and swallowing problems can occur.

Although the disease is incurable, a one-time gene therapy can stop its progression.

Zolgensma is an orphan medicine with conditional marketing authorisation by the European Medicines Agency. It is the most expensive drug registered in Slovakia – with the current market price ranging around €1.9 million.

Who covers the costs is dealt with on a case-by-case basis since Zolgensma is not officially categorised as a reimbursable medicine and the rules remain blurry.

Currently, parents or legal guardians have to request a reimbursement exception from their health insurance company.

In a recent case, the health insurance company refused to allow this exception and cover the amount. The health insurance company, struggling under heavy debt, argued that the reimbursement would constitute irresponsible management of public finances.

Whether such an amount of public money should be used for the treatment of one individual has also divided the public.

The dispute ended up in front of the District Court, which ordered the state-controlled health insurance company to reimburse the family.

This case underlined a serious concern brought up by the Slovak Healthcare Surveillance Authority and National Health Technology Assessment Institution in March: “The absence of transparent rules for prescribing and authorising special medicines on an exception basis poses risks and can lead to serious consequences. The need for a Court intervention only reinforces the acute need for clearer rules.”

Health insurance companies appeal

In mid-October, the departing Minister of Health Michal Palkovič approved a proposal submitted by the Categorisation Committee to reimburse Zolgensma under certain conditions, starting from January 2024.

Health insurance companies jointly objected to the proposal, however, stating that the approval did not take into account insurance companies’ budgets, and the Ministry of Health has made no guarantees that it would allocate more money for these instances.

In 2023, the Ministry of Health allocated €55 million for exception-related medicine, most of which was already spent in the first quarter of the year.

Due to insurance companies’ objections, the January 2024 target of adding Zolgensma to the reimbursement list is unlikely to be met.

Lack of transparency 

The market price for Zolgensma is estimated at around €1.9 million, but the health ministry revealed that the amount paid is lower than the amount officially listed.

“If a drug is included in the categorisation list, the price is not unknown, but confidential,” Iveta Pálešová, the executive director of AIFP Slovakia, told Euractiv.

Minister Palkovič added: “We cannot reveal the price due to competitive pressures in other countries.”

Confidential pharmaceutical agreements are not an uncommon strategy among the member states to secure a more favourable price. This hints at diverging prices for Zolgensma in different member states and revealing the procurement price would threaten the deal.

Currently, only a small circle of people working on the deal and the health insurance companies know the confidential price and the terms negotiated. The process, therefore, cannot be adequately evaluated and lacks transparency.

Rules remain unclear

SMA is categorised into five types, each varying in occurrence rate, life expectancy, symptoms, and age of onset. The Categorisation Committee has outlined rules in order to receive reimbursement starting January 2024, only for the most common type 1 SMA.

The conditions are the following: a maximum age of six months (or 12 months if a neurologist concludes at least a 70% chance that the child will be able to sit unassisted); the child must be born between 35-42 weeks of pregnancy; at the time of administering the medicine, the child must not weigh more than 13.5 kilograms; and the child cannot have non-invasive ventilation support.

For other SMA types, there is no guideline or reimbursement guarantee. As a result, the legal guardians are limited to requesting an exception, just as in the latest instance.

“If the drug is not part of the categorisation list, or if the patient’s criteria are not met, it is up to the health insurance company to decide if and to what extent the medicine will be reimbursed, with the price subject to negotiations,” explained Pálešová.

Oftentimes, administering the gene therapy against the SMA is time-sensitive.

The lack of transparency and no clear rules in assigning exceptions remain without a solution and can delay the treatment. Despite the current dispute culminating in a District Court intervention, the case-by-case nature for the other four SMA types seems to remain unresolved for the time being.

The Categorisation Committee must evaluate the objections made by the health insurance companies and issue a new recommendation to the health minister by November 15.

With the new government and health minister sworn in on Wednesday, it is yet unclear what their stance on this issue will be.

(Filip Áč – Edited by Vasiliki Angouridi/Zoran Radosavljevic | Euractiv.com)

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