BACKGROUND: Out of 185 orphan medicinal products (OMPs) registered in 2015-2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. METHODOLOGY: The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients' organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. CONCLUSION: We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of "value-based" decision making.

Cogvio Prague Czechia

Department of Pharmacology Faculty of Medicine Masaryk University Brno Czechia

Value Outcomes Prague Czechia

See more in PubMed

Regulation (EC) No 141/2000 of the European Parliament and of the Council on orphan medicinal products [Internet]. [Cited 2022 Jan. 12]. Available from: https://ec.europa.eu/health/sites/default/files/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf.

Gammie T, Lu CY, Babar ZUD. Access to orphan drugs: A comprehensive review of legislations, regulations and policies in 35 countries. PloS One. 2015;10(10):e0140002. PubMed PMC

Czech M, Baran-Kooiker A, Atikeler K, et al. A review of rare disease policies and orphan drug reimbursement systems in 12 Eurasian countries. Front Public Health. 2019;7:416. PubMed PMC

Annemans L, Aymé S, Le Cam Y, et al. Recommendations from the European Working Group for value assessment and funding processes in rare diseases (ORPH-VAL). Orphanet J Rare Dis. 2017;12(1):50. PubMed PMC

Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011;71(4):488–496. PubMed PMC

Drummond M, Towse A. Orphan drugs policies: A suitable case for treatment. Eur J Health Econ HEPAC Health Econ Prev Care. 2014;15(4):335–340. PubMed

Lakdawalla DN, Doshi JA, Garrison LP, et al. Defining elements of value in health care-A health economics approach: An ISPOR special task force report [3]. Value Health. 2018;21(2):131–139. PubMed

Logviss K, Krievins D, Purvina S. Rare diseases and orphan drugs: Latvian story. Orphanet J Rare Dis. 2014;9:147. PubMed PMC

Iskrov G, Miteva-Katrandzhieva T, Stefanov R. Challenges to orphan drugs access in Eastern Europe: The case of Bulgaria. Health Policy Amst Neth. 2012;108(1):10–18. PubMed

Act No. 48/1997 Coll., on public health insurance [Internet]. Zákony pro lidi. [Cited 2022 Jan. 25]. Available from: https://www.zakonyprolidi.cz/cs/1997-48.

Skoupá J. Drug policy in the Czech Republic. Value Health Reg Issues. 2017;13:55–58. PubMed

Vostalová L, Mazelová J, Samek J, Vocelka M. Health technogology assessment and evaluation of pharmaceuticals in the Czech Republic. Int J Technol Assess Health Care. 2017;33(3):339–344. PubMed

Public Health - European Commission. Union register of medicinal products [Internet]. [Cited 2022 Feb 7]. Available from: https://ec.europa.eu/health/documents/community-register/html/.

State Institute for Drug Control. List of reimbursed medicinal products [Internet]. [Cited 2022 Feb 7]. Available from: https://www.sukl.eu/sukl/list-of-reimbursed-medicinal-products.

State Institute for Drug Control. DIS-13 | Open data [Internet]. [Cited 2022 Feb 7]. Available from: https://opendata.sukl.cz/?q=katalog/dis-13.

Ornstová E, Sebestianova M, Paris M, et al. PSY147 – Availability and reimbursement on orphan drugs in the Czech Republic. Value Health. 2018;21:S461.

Malinowski KP, Kawalec P, Trąbka W, et al. Reimbursement legislations and decision making for orphan drugs in Central and Eastern European countries. Front Pharmacol. 2019;10:487. PubMed PMC

Garau M and Mestre-Ferrandiz J. Access mechanisms for orphan drugs: A comparative study of selected European countries. OHE (Office of Health Economics) [Internet]. [Cited 2022 Jan 11]. 2009. Available from: https://www.ohe.org/publications/access-mechanisms-orphan-drugs-comparative-study-selected-european-countries.

Zamora B, Maignen F, O’Neill P, Mestre-Ferrandiz J, Garau M. Comparing access to orphan medicinal products in Europe. Orphanet J Rare Dis. 2019;14(1):95. PubMed PMC

Szegedi M, Zelei T, Arickx F, et al. The European challenges of funding orphan medicinal products. Orphanet J Rare Dis. 2018;13(1):184. PubMed PMC

Lasalvia P, Prieto-Pinto L, Moreno M, et al. International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: A scoping review. Expert Rev Pharmacoecon Outcomes Res. 2019;19(4):409–420. PubMed

Robinson LA, Hammitt JK, Chang AY, Resch S. Understanding and improving the one and three times GDP per capita cost-effectiveness thresholds. Health Policy Plan. 2017;32(1):141–145. PubMed

State Institute for Drug Control. Regulation of prices and reimbursements for pharmaceuticals [Internet]. [Cited 2022 Jan 25]. Available from: https://www.sukl.eu/medicines/regulation-of-prices-and-reimbursements-for-pharmaceuticals?highlightWords=sp-cau.

Ornstova E, Sebestianova M, Mlcoch T, Lamblova K, Dolezal T. Highly innovative drug program in the Czech Republic: Description and pharmacoeconomic results—Cost-effectiveness and budget impact analyses. Value Health Reg Issues. 2018;16:92–98. PubMed

Morel T, Arickx F, Befrits G, et al. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: A comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis. 2013;8:198. PubMed PMC