ATMP Issue #2

Challenges and Opportunities in Expanding CAR T-Cell Therapy Access in Czechia

PharmDr. Zora Čechová, Ph.D. Regulatory Specialist, MUNI | CREATIC

CAR T-cell therapies have revolutionised the treatment of haematological malignancies since their introductionto the market in 2018. Czechia is at the forefront of adopting these innovative treatments in Central andEastern Europe, with three out of six authorised CAR T-cell therapies already available to patients. The othertwo therapies, Abecma (idecabtagene vicleucel) and Breyanzi (lisocabtagene maraleucel) have had theirmaximum prices determined and are nearing rollout. However, this progress also introduces significantchallenges, especially with regard to multiple myeloma treatment with Abecma.

Despite substantial advancements, multiple myeloma remains an incurable disease with increasing incidenceand prevalence. Approximately 550 new cases are diagnosed annually in Czechia. While advances in therapyhave extended survival, most patients relapse and require subsequent treatment lines, driving demand forAbecma as a third-line treatment. The estimates approach one hundred patients per year.

Several bottlenecks remain in delivering this highly personalised treatment to patients who need it, startingwith the complex, patient-specific manufacturing process, which requires careful coordination to ensure thetimely availability of manufacturing slots. Additionally, although there are nine specialized centres certified toadminister CAR T-cell products in Czechia, these facilities may still face personnel and capacity constraints asthe number of eligible patients continues to grow.

Pricing and reimbursement procedures present another significant challenge. Although the State Institute ofDrug Control has determined the maximum price for Abecma, a formal reimbursement procedure, includinghealth technology assessment, is not required for medicinal products administered solely in inpatient settings.These products, including CAR T-cell, are available to Czech patients within all EMA-approved indications, theyare fully covered by public health insurance, and patients do not contribute to their financing. However, thismight not be sufficient to cover the full financing from payers to healthcare providers, as it is secured only upto the limit calculated based on the year before last. Therefore, budget planning does not account forunexpected increases in demand for new treatment options, such as Abecma for treating multiple myelomapatients. Further, as the high cost of CAR T-cell treatments adds pressure to the healthcare budget, to mitigatethe financial burden, arrangements between manufacturers and payers are essential. So far, the only type ofagreement used to limit the budget impact of ATMPs is price reduction agreements. Value-based pricingmodels, where financing aligns with clinical outcomes, offer a potential solution but require complexnegotiations. However, the lack of healthcare digitalisation remains a key barrier in Czechia to implementingsuch agreements effectively.

A cohesive strategy involving all stakeholders – clinicians, healthcare providers, regulators, payers, andmanufacturers – is essential to address these challenges. Collaboration will be critical to ensuring broaderpatient access without compromising the quality of care.