This study examines patient access to orphan medicinal products (OMPs) in Central and Eastern Europe (CEE) over the past five years, focusing on seven countries: Bulgaria, Czechia, Hungary, Poland, Romania, Slovakia, and Slovenia. While these jurisdictions have undergone rapid healthcare transformations, significant disparities in OMP access persist compared to Western Europe. This study aimed to address this gap by identifying barriers and enablers to optimize patient access to OMPs in a sustainable and equitable manner. A mixed-methodology approach was utilized, combining systematic literature reviews, in-depth interviews, and advisory board insights. Perspectives were gathered from a wide range of stakeholders, including policymakers, payers, academia, industry associations, and patient advocacy groups. Additionally, the study incorporated data from CEE-specific initiatives to triangulate findings and evaluate barriers, enablers, and best practices in OMP access. The analysis identified sub-optimal OMP access across most CEE countries, marked by prolonged delays and lower reimbursement rates compared to Western Europe, with Slovenia and Czechia as notable exceptions. Key barriers include limited awareness, inadequate health technology assessment (HTA) frameworks, insufficient financing mechanisms, underutilization of novel access schemes, and fragmented patient engagement. Conversely, enablers include the presence of rare disease policies, OMP-specific HTA frameworks, and patient-inclusive decision-making processes.

ACESO Healthcare Consulting 03 719 Warsaw Poland

Business School Warsaw University of Technology 02 008 Warsaw Poland

Chiesi Pharmaceuticals GmbH 1010 Vienna Austria

Department of Epidemiology and Preventive Medicine Jagiellonian University 31 007 Cracow Poland

Department of Pharmacoeconomics Institute of Mother and Child 01 211 Warsaw Poland

Faculty of Mathematics and Physics University of Ljubljana 1000 Ljubljana Slovenia

Faculty of Pharmacy Medical University of Sofia 1431 Sofia Bulgaria

iHETA 120 00 Prague 2 Czech Republic

Institute of Mathematics Physics and Mechanics 1000 Ljubljana Slovenia

Media Kompass 023808 Bucharest Romania

Syreon Research Institute 1142 Budapest Hungary

Value Outcomes 120 00 Prague 2 Czech Republic

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Klimova B., Storek M., Valis M., Kuca K. Global View on Rare Diseases: A Mini Review. Curr. Med. Chem. 2017;24:3153–3158. doi: 10.2174/0929867324666170511111803. PubMed DOI

Delaye J., Cacciatore P., Kole A. Valuing the ‘Burden’ and Impact of Rare Diseases: A Scoping Review. Front. Pharmacol. 2022;13:914338. doi: 10.3389/fphar.2022.914338. PubMed DOI PMC

Adachi T., El-Hattab A.W., Jain R., Crespo K.A.N., Lazo C.I.Q., Scarpa M., Summar M., Wattanasirichaigoon D. Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges. Int. J. Environ. Res. Public Health. 2023;20:4732. doi: 10.3390/ijerph20064732. PubMed DOI PMC

Vogler S. Payer Policies to Support Innovation and Access to Medicines in the WHO European Region. Oslo Medicines Initiative Technical Report. World Health Organization Regional Office for Europe. 2022. [(accessed on 21 January 2025)]. Available online: https://iris.who.int/bitstream/handle/10665/361753/9789289058247-eng.pdf?sequence=1. PubMed

Korchagina D., Jaroslawski S., Jadot G., Toumi M. Orphan Drugs in Oncology. In: Walter E., editor. Regulatory and Economic Aspects in Oncology. Volume 213. Springer International Publishing; Cham, Switzerland: 2019. pp. 109–142. Recent Results in Cancer Research. PubMed DOI

Szegedi M., Zelei T., Arickx F., Bucsics A., Cohn-Zanchetta E., Fürst J., Kamusheva M., Kawalec P., Petrova G., Slaby J., et al. The European challenges of funding orphan medicinal products. Orphanet J. Rare Dis. 2018;13:184. doi: 10.1186/s13023-018-0927-y. PubMed DOI PMC

Mazzucato M., Minichiello C., Vianello A., Pozza L.V.D., Toto E., Facchin P. Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study. Front. Pharmacol. 2022;13:940010. doi: 10.3389/fphar.2022.940010. PubMed DOI PMC

Adkins E., Nicholson L., Floyd D., Ratcliffe M., Chevrou-Severac H. Oncology drugs for orphan indications: How are HTA processes evolving for this specific drug category? ClinicoEcon. Outcomes Res. 2017;9:327–342. doi: 10.2147/CEOR.S134230. PubMed DOI PMC

Hren R. Impact of the Pharma Economic Act on Diffusion of Innovation and Reduction of Costs in the Hungarian Prescription Drug Market (2007–2010) Value Health Reg. Issues. 2013;2:290–299. doi: 10.1016/j.vhri.2013.06.013. PubMed DOI

Tambor M., Klich J., Domagała A. Financing Healthcare in Central and Eastern European Countries: How Far Are We from Universal Health Coverage? Int. J. Environ. Res. Public Health. 2021;18:1382. doi: 10.3390/ijerph18041382. PubMed DOI PMC

Lipska I., Di Bidino R., Niewada M., Nemeth B., Bochenek T., Kukla M., Więckowska B., Sobczak A., Iłowiecka K., Zemplenyi A., et al. Overcoming Barriers in Hospital-Based Health Technology Assessment (HB-HTA): International Expert Panel Consensus. Healthcare. 2024;12:889. doi: 10.3390/healthcare12090889. PubMed DOI PMC

Csanádi M., Kaló Z., Molken M.R.-V., Looman W., Huic M., Ercevic D., Atanasijevic D., Lorenzovici L., Petryszyn P., Pogány G., et al. Prioritization of implementation barriers related to integrated care models in Central and Eastern European countries. Health Policy. 2022;126:1173–1179. doi: 10.1016/j.healthpol.2022.08.012. PubMed DOI

Godman B., Novakovic T., Tesic D., Oortwijn W., Martin A.P., Parker M., Haycox A. Addressing challenges for sustainable healthcare in Central and Eastern Europe. Expert. Rev. Pharmacoecon. Outcomes Res. 2016;16:685–687. doi: 10.1586/14737167.2016.1165610. PubMed DOI

Eurostat Healthcare Expenditure Statistics by Function, Provider and Financing Scheme. Nov, 2024. [(accessed on 21 January 2025)]. Available online: https://ec.europa.eu/eurostat/statistics-explained/index.php?title=Healthcare_expenditure_statistics_by_function,_provider_and_financing_scheme.

Latos-Bieleńska A. Plan dla chorób rzadkich (Rare Diseases Plan) [(accessed on 21 January 2025)];2024 November; Available online: https://chorobyrzadkie.gov.pl/pl/choroby-rzadkie/plan-dla-chorob-rzadkich. (In Polish)

Czech M., Baran-Kooiker A., Atikeler K., Demirtshyan M., Gaitova K., Holownia-Voloskova M., Turcu-Stiolica A., Kooiker C., Piniazhko O., Konstandyan N., et al. A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries. Front. Public Health. 2020;7:416. doi: 10.3389/fpubh.2019.00416. PubMed DOI PMC

Zelei T., Molnár M.J., Szegedi M., Kaló Z. Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries. Orphanet J. Rare Dis. 2016;11:72. doi: 10.1186/s13023-016-0455-6. PubMed DOI PMC

Bhattacherjee A., Toleman M., Rowling S., Frederiks A., Andersen N. Social Science Research: Principles, Methods and Practices. University of Southern Queensland; Darling Heights, Australia: 2019. DOI

Hussein A. The use of Triangulation in Social Sciences Research: Can qualitative and quantitative methods be combined? J. Comp. Soc. Work. 2009;4:106–117. doi: 10.31265/jcsw.v4i1.48. DOI

GAP The GAP (GEARING UP ACCESS PROPOSAL FOR V4) Tool. [(accessed on 21 January 2025)]. Available online: https://gapv4.eu/gapv4/

Newton M., Stoddart K., Travaglio M., Troein P. EFPIA Patients W.A.I.T. Indicator 2023 Survey. Jun, 2024. [(accessed on 21 January 2025)]. Available online: https://rtl.ee/wp-content/uploads/2024/06/EFPIA-2023-Patient-W.A.I.T.-Indicator-Final12.06.2024.pdf.

Medicinal Products for Rare Diseases in Europe. Orphanet Report Series, Orphan Drugs Collection. Apr, 2024. [(accessed on 21 January 2025)]. Available online: https://www.orpha.net/pdfs/orphacom/cahiers/docs/GB/Medicinal_products_for_rare_diseases_in_Europe.pdf.

Rais C., Kaló Z., Csanádi M., Negulescu V. Current and future perspectives for the implementation of health technology assessment in Romania. Health Policy Technol. 2020;9:45–52. doi: 10.1016/j.hlpt.2019.11.007. DOI

Ivanova H., Macaulay R. PMU74 Health Technology Assassment in Bulgaria—3 Year Report Card. Value Health. 2019;22:S262. doi: 10.1016/j.jval.2019.04.1235. DOI

Decker B., Mlcoch T., Pustovalova A., Dolezal T. Novel approach to decision making for orphan drugs. Int. J. Technol. Assess. Health Care. 2023;39:e10. doi: 10.1017/S0266462323000053. PubMed DOI PMC

HAS Press Department Early Access Authorisation: A Positive Initial Report and Refined Assessment Methods. May 20, 2022. [(accessed on 21 January 2025)]. Available online: https://www.has-sante.fr/jcms/p_3340090/en/early-access-authorisation-a-positive-initial-report-and-

Ádám I., Callenbach M., Németh B., Vreman R.A., Tollin C., Pontén J., Dawoud D., Elvidge J., Crabb N., Doorn-Khosrovani S.B.v.W.v., et al. Outcome-based reimbursement in Central-Eastern Europe and Middle-East. Front. Med. 2022;9:940886. doi: 10.3389/fmed.2022.940886. PubMed DOI PMC

Dimitrova M., Jakab I., Mitkova Z., Kamusheva M., Tachkov K., Nemeth B., Zemplenyi A., Dawoud D., Delnoij D.M.J., Houýez F., et al. Potential Barriers of Patient Involvement in Health Technology Assessment in Central and Eastern European Countries. Front. Public Health. 2022;10:922708. doi: 10.3389/fpubh.2022.922708. PubMed DOI PMC

Jakab I., Dimitrova M., Houÿez F., Bereczky T., Fövényes M., Maravic Z., Belina I., Andriciuc C., Tóth K., Piniazhko O., et al. Recommendations for patient involvement in health technology assessment in Central and Eastern European countries. Front. Public Health. 2023;11:1176200. doi: 10.3389/fpubh.2023.1176200. PubMed DOI PMC

Mela A., Lis D., Rdzanek E., Jaroszyński J., Furtak-Niczyporuk M., Drop B., Blicharski T., Niewada M. AOTMiT reimbursement recommendations compared to other HTA agencies. Eur. J. Health Econ. 2024;25:1291–1310. doi: 10.1007/s10198-023-01655-x. PubMed DOI PMC

Fontrier A.-M., Kamphuis B., Kanavos P. How can health technology assessment be improved to optimise access to medicines? Results from a Delphi study in Europe: Better access to medicines through HTA. Eur. J. Health Econ. 2024;25:935–950. doi: 10.1007/s10198-023-01637-z. PubMed DOI PMC

Pitter J.G., Nagy L., Nagy B., Hren R. Development Perspectives for Curative Technologies in Primary Demyelinating Disorders of the Central Nervous System with Neuromyelitis Optica Spectrum Disorder (NMOSD) and Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD) at the Forefront. J. Pers. Med. 2024;14:599. doi: 10.3390/jpm14060599. PubMed DOI PMC

Andreu P., Karam J., Child C., Chiesi G., Cioffi G. Rare Disease Burden of Care and the Economic Impact on Citizens in Germany, France and Italy. 2024. [(accessed on 21 January 2025)]. Available online: https://chiesirarediseases.com/assets/pdf/rare-disease-burden-of-care-and-the-economic-impact-on-citizens.pdf.