Orphan Drugs: Building the Regulatory Framework in the Czech Republic


A cross-industry push for better and broader access to innovative treatments is underway in the Czech Republic. Ripe for reform is the country’s orphan drug legislation as no standard pathway for their assessment, pricing and reimbursement currently exists.


[For cancer treatment the country has been] quite successful in moving the needle, but we can do a lot better in small niche-indications

Robin Turner, Roche

Companies have to apply for exceptional reimbursement under the ominous ‘Paragraph 16,’ which takes up considerable time and energy for local market access teams, with the State Institute for Drug Control (SUKL), the Czech drug regulator, offering a simple “accept or deny” result. Roche’s general manager Robin Turner indicates that a change to this process “will be beneficial to increase access to orphan drugs for which it is almost impossible to demonstrate cost-effectiveness,” which is standard criteria for an approval from the regulatory body.


This ambitious reform, called the Act on Public Health Insurance, is set to become law in late 2020. “It is not just about cost-effectiveness” explains Minister of Health Adam Vojtěch, adding that “through this amendment insurance companies, industry associations, patient groups, and experts will evaluate how impactful a given orphan drug is and how it will improve the quality of life of patients.” Takeda’s general manager for the Czech Republic and Slovakia, Kieran Leahy adds “Our goals are aligned with those of the Minister of Health, which are for patients to have access to innovative life-changing medicines as quickly as possible in an affordable way.”


Increased dialogue between SUKL and insurance companies has enhanced patient access for highly innovative, but extremely costly CAR-T treatments. In 2019, VZP, the country’s largest health insurance fund, announced it had negotiated access to two breakthrough CAR-T therapies: Gilead’s Yescarta for large B-cell lymphoma and Novartis’ Kymriah for B-cell acute lymphoblastic leukaemia. “We have special proceedings for early access to modern treatments such as cell and gene therapies and orphan drugs” shares David Šmehlík, the VZP’s deputy director. To achieve this, VZP and the Czech Haematology Society signed a memorandum to grant funding to these therapies for the next twelve months, defined patients with a strong medical need for treatment and developed recommendations for both products.


Martin Puchwein, country president & general manager pharma for market leader Novartis, celebrates “Novartis is now at the finish line!” Roche’s Turner agrees that for cancer treatment the country has been “quite successful in moving the needle, but we can do a lot better in small niche-indications.” He feels it is near impossible to gather enough clinical trial data, claiming “the whole model is not suited to the advent of personalized medicine based on genomic profiling.” If the Czech Republic wants to close the gap on Western Europe, “the country will have to change its approach to modern treatments.”


Turner quips, “the government is trying to fix the symptoms rather than treat the cause” and is less enthusiastic about other market access regulations for innovative pharmaceuticals in general. Pavel Brezina, managing director of the Czech Republic & Slovakia for Gilead Sciences agrees, sharing his opinion that “these so-called soft criteria that orphan drugs will be assessed against should be taken into consideration for regular prescription drugs as well.”

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