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Rare Disease Research in Central and Eastern Europe

By Renee Butler
On March 3, 2021

There are more than 7,000 rare diseases in existence right now and together they impact millions of people around the world. However, individually, a rare disease can be defined as impacting fewer than 5 in 10,000. When indications are that sparse, developing treatments for rare diseases is challenging. Choosing the right location can make a difference. While the right site location will vary depending on the indication, some parts of the world are particularly well-suited to rare disease research.

Central and Eastern Europe (CEE) is a popular location in rare disease clinical trials for several reasons.

 

Standard of Care

Access to medical care is unequal around the world, particularly where rare diseases are concerned. Awareness of rare diseases, public health initiatives, diagnostic screening, rehabilitation opportunities, and clinical management options are problematic within the CEE as well as access to rare disease treatments.

Orphan medicinal products (OMPs) exist for roughly 3% of rare diseases. To receive those medicines, rare disease patients need to be registered and often can only get the (frequently expensive) OMP through reimbursement. Some countries, like the Netherlands, reimburse almost all OMPs whereas other countries only reimburse a limited number.

Many countries within the CEE fall on the lower side. Latvia and Poland reimburse 25 and 48 OMPs respectively, while, In Armenia, there is no reimbursement at all. They have limited options for accessing expensive treatments. For people in these countries, alternative methods of accessing rare disease treatments are critical to addressing their medical needs.

 

Patient Associations

In some cases, rare disease patients in the CEE have access to patient associations and advocacy groups that help inform them about their conditions, treatment options, and upcoming studies. This includes those with cystic fibrosis, neuromuscular dystrophies, and thalassemia, but there are few patient associations for rare diseases in general. In 2009, only four countries in the CEE had developed national organizations devoted to rare disease – Bulgaria (NAPRD), Hungary (HUFERDIS), Romania (RONARD), and Russia. In 2019, another review found that number had increased but many countries still lack a national plan for rare diseases.

 

Centralized Medicine

Centralized medicine helps. In countries with government-sponsored healthcare, there is a centralized database of citizen health data so finding people with specific indications is easy, even rare conditions. In contrast, locating a viable patient population in a country without centralized healthcare makes it impossible to identify study candidates en masse. Furthermore, many of these countries use federal clinics to treat their citizens and those facilities often contain equipment that can support even the most complex clinical trials. (Read: Benefits of Conducting Clinical Research in Russia)

 

Creating a Healthier World

Pharm-Olam has spent decades working in the rare disease research space and we understand what it takes to produce a successful study. Proper site selection and understanding the complications around conducting a rare study can go a long way to completing study enrollment on-time and delivering endpoints that meet study goals. To learn more about how Pharm-Olam can help your clinical research efforts in Central and Eastern Europe, speak with our team today.

 

Bibliography

Abbas, A., Vella, J., Azzopardi, L.M. & Serracino-Inglott, A. (2019). Orphan drug policies in different countries. Journal of Pharmaceutical Health Services Research, Volume 10, Issue 3, September 2019, Pages 295–302, https://doi.org/10.1111/jphs.12305

Czech, M., Baran-Kooiker, A., Atikeler, K., Demirtshyan, M., Gaitova, K., Holownia-Voloskova, M., Turcu-Stiolica, A., Kooiker, C., Piniazhko, O., Konstandyan, N., Zalis'ka, O., & Sykut-Cegielska, J. (2020). A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries. Frontiers in public health, 7, 416. https://doi.org/10.3389/fpubh.2019.00416

Gammie, T., Lu, C. Y., & Babar, Z. U. (2015). Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PloS one, 10(10), e0140002. https://doi.org/10.1371/journal.pone.0140002

Kamusheva, M., Manova, M., Savova, A. T., Petrova, G. I., Mitov, K., Harsányi, A., Kaló, Z., Márky, K., Kawalec, P., Angelovska, B., Lakić, D., Tesar, T., Draganic, P., Geitona, M., Hatzikou, M., Paveliu, M. S., & Männik, A. (2018). Comparative Analysis of Legislative Requirements About Patients' Access to Biotechnological Drugs for Rare Diseases in Central and Eastern European Countries. Frontiers in pharmacology, 9, 795. https://doi.org/10.3389/fphar.2018.00795

Pejcic, A. V., Iskrov, G., Raycheva, R., Stefanov, R., & Jakovljevic, M. M. (2017). Transposition and implementation of EU rare disease policy in Eastern Europe. Expert review of pharmacoeconomics & outcomes research, 17(6), 557–566. https://doi.org/10.1080/14737167.2017.1388741

Puiu, M., & Dan, D. (2010). Rare diseases, from European resolutions and recommendations to actual measures and strategies. Maedica, 5(2), 128–131. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3150005/

Stefanov, R. & Taruscio, D, (2009). Rare diseases and orphan drugs in Eastern European countries. Italian Journal of Public Health, 7 6 (4). https://www.researchgate.net/publication/290276242_Rare_diseases_and_orphan_drugs_in_Eastern_European_countries

 

Useful Resources

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