OBJECTIVES: This policy perspective presents a unique pricing and reimbursement (P&R) system for orphan medicinal products (OMP) recently adopted in Czechia.

METHODS: The updated legislation follows the recommendations for value assessment and funding processes for rare diseases (ORPH-VAL). It also incorporates additional elements of value defined by ISPOR Special Task Force.

RESULTS: Out of 185 OMP registered by EMA from 2015 to 2021, a mere 110 (59%) were available to Czech patients, and only 54 (29%) were officially reimbursed. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted towards creating a special pathway for the reimbursement of OMP. Thus, a rigorous P&R procedure with strict timelines and elaborated methodology has been established.

The application with clinical evidence, cost-effectiveness and budget impact analyses is submitted to the governmental HTA agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, relevant professional associations and patients organizations are rightful participants in the proceeding, providing evidence and comments. The HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the Assessment Report summarizing available information. The Assessment Report is then forwarded to the Ministry of Health and its Advisory body consisting of four stakeholders: (1) patients, (2) clinical experts, (3) health insurance funds, and (4) State. The Advisory body critically evaluates the documents and issues a binding opinion based on following decision-making criteria: a) therapeutic effectiveness/safety, b) disease severity, c) reimbursed comparators, d) societal impact, e) quality of life, f) specialised medical centers network, g) guidelines, h) managed entry agreements, i) cost-effectiveness, j) budget impact. Finally, the Decision is issued by the HTA agency.

CONCLUSIONS: We strongly believe that this novel approach may inspire many countries worldwide that are struggling to offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision-making.