Yanko Popstoilov is the manager of “Reimex Pharma“ OOD. The company specializes in the supply of medicinal products for the treatment of rare diseases. It is built on the principles of international and national best practices in the industry.
Popstoilov has a master's degree in health management. Founder and member of the Board of Directors of the Association of Importers of Unregistered Medicinal Products for the Treatment of Rare Diseases.
Mr. Popstoilov, the science of rare diseases has never progressed so quickly. Why then does access to treatment remain so difficult in the world?
The development of science is outpacing access. Over 300 million people in the world live with rare diseases. There are thousands of these diseases, and only about 5% of them have approved treatments. At the same time, therapies are emerging that seemed like fantasy a decade ago - by the end of 2025, the US Food and Drug Administration (FDA) has already approved 26 gene therapies.
However, these drugs are produced in small quantities, often by a single manufacturer in the world, and are expensive because the costs of their development are spread over a small number of patients. Therefore, the decision of where and when to launch them is primarily an economic one. Large and solvent markets come first. Small ones are often last - if they are reached at all.
Along with economic considerations, geopolitical ones should not be underestimated. Since May 2025, the US has been pursuing a policy of matching drug prices in its country with the lowest prices in comparable developed countries. The logic for companies is simple: a low price somewhere in Europe can cost them dearly in America. Initial analyses show that in the first year after the announcement of this policy, over a third fewer new drugs were launched on the market in eleven EU countries than a year earlier. The decline is not only due to the American policy – other factors also play a role, including the new common European rules for evaluating drugs. For drugs for rare diseases, the decline is so far much smaller – around 10%. However, the conclusion is clear: companies have a serious reason not to rush to launch new drugs in countries with lower prices. For a country like Bulgaria, this is a serious warning.
And another alarming signal. Several gene therapies approved in Europe were withdrawn from the market for commercial reasons – not because they do not work. The discovery is just the beginning, then there is ensuring a working system: who pays, how and when, who will monitor the patient for years – in the case of gene therapies up to 15 years. Systems work with annual budgets, and a gene therapy is usually applied once, while the benefits, risks and need for follow-up continue for years. Therefore, today, access to treatment depends not only on the scientific discovery, but also on the readiness of the health system to implement it, pay for it and monitor the result.
How is Europe responding - and will the patient in Bulgaria feel the European changes?
The good news is that rare diseases are now a global priority. In May 2025, the World Health Assembly declared them as such with a separate resolution, and in 2028 it must consider a draft of a ten-year global action plan.
In the European Union, between 27 and 36 million people live with rare diseases, and Europe itself has its own paradox: it creates medical discoveries, but often loses them. About a quarter of scientific publications in biotechnology are European, and only about 10% of the venture capital for the sector remains in Europe. Discoveries are made in Europe, but they are turned into medicines and successful businesses elsewhere - often in the USA.
Europe is opening doors, but each country goes through them at its own speed. Price and payment remain a national responsibility. There is also the European path for planned treatment in another country - for some of the most complex therapies it is the only one, but it is often slow and administratively difficult. And something is missing: Europe has a plan to fight cancer, but there is still no common plan for rare diseases. The European Parliament is working on such a request and I hope it becomes a reality.
Where is Bulgaria in this picture?
Judging by official statistics - in the middle. According to the latest study by the European Federation of Pharmaceutical Industries and Associations (EFPIA), out of 168 new medicines approved in the EU in 2021-2024, 77 will be available in Bulgaria by the beginning of 2026 - at the EU average. But while in Germany the time to access is about two months, in our country it is over 600 days.
However, these statistics measure the inclusion of medicines in the public payment system – in our country this is the Positive Medicines List. In the case of rare diseases, the picture is different: a large part of the therapies are prescribed and dispensed outside the list – as medicines that are not authorized or not distributed in Bulgaria. Many of them are not officially released on the Bulgarian market, but nevertheless reach patients – on an individual basis.
Your company supplies medicines from different parts of the world to individual patients in a small country. What are the advantages and disadvantages of being a trader with such a model?
The numbers speak for themselves – the population of Bulgaria is about 6.4 million people and is decreasing – in 2025 alone, the natural increase is minus 49 thousand people. Nearly a quarter of people are 65 years of age and older. The gross domestic product per capita in purchasing power standards is 32% below the EU average - on a par with Greece, the lowest in the Union. And 36% of health care costs in our country are paid directly from the patients' pockets - the highest share in the EU. If we were influenced only by these figures, no business plan would choose rare diseases in Bulgaria.
The disadvantages are clear. In a specific rare disease, patients are often single. Each patient is a separate project - a separate protocol, a separate order, a separate delivery, often at a controlled temperature. And the costs of licenses, qualified people, a cold chain and documentation are almost the same, regardless of whether you deliver one package or a thousand. When quantities are limited, the small market is last in line. The financial risk is not to be underestimated - we often pay manufacturers in advance, and we wait months for payment from the system. Last but not least are the predictable rules - for a business that plans deliveries months in advance, this is the most expensive risk.
However, the advantages are just as real – and it is precisely because of them that this model exists. The regulation currently allows treatment with a drug approved for use in another country, including outside the EU, when treatment with drugs approved in our country is impossible or ineffective. This is important because for the period 2001 - 2024, the FDA has approved over three times more orphan drugs than are approved in the EU. There is no minimum order volume requirement – we can ensure delivery for a single patient. Specialized knowledge – regulatory, logistical, commercial – and connections with manufacturers from a number of countries, including small biotech companies without representation in Europe. For them, we are the channel through which the medicine can be delivered when Bulgarian doctors prescribe it.
And there is a paradox that explains our mission better than anything. When a medicine is authorized and released on the Bulgarian market, the individual order no longer applies to it – and we lose that supply. We consider this a success. Our role is to be the bridge until the road is built. A small market is a disadvantage for business. It should not be a disadvantage for the patient.
Beyond the specific patient – how does the provision of these therapies contribute to the development of medicine in Bulgaria?
With much more than it seems. The medicine of the future often starts with rare diseases. The first gene therapy authorized in the European Union was for a hereditary disease that affects no more than one or two people per million. The first CRISPR gene-editing drug approved in the EU is for two inherited rare blood diseases - beta-thalassemia and sickle cell anemia. Many of the new technologies, flexible regulatory approaches and patient participation models are first applied to rare diseases and then enter other areas of medicine. A country that provides these therapies to its patients also develops its medicine. A country that stands aside is lagging behind.
Every modern treatment implemented in Bulgaria is an experience for Bulgarian doctors, pharmacists and nurses - to work with the most complex products, to monitor the effect and safety, to build teams of different specialists. When treatment is only possible abroad, knowledge and money flow out. When it is here - they stay and accumulate.
Diagnosis follows treatment: when there is therapy, early diagnosis takes on a new meaning. From March 2026, newborn screening in our country covers six diseases instead of three - spinal muscular atrophy, severe combined immunodeficiencies, and cystic fibrosis have been added. This is a big step, and its value comes from the fact that there is now something to be done for these children even after diagnosis.
Over 1,600 specialized centers from 375 hospitals participate in the 24 European Reference Networks for Rare Diseases. From Bulgaria, seven centers will be full members by the end of 2024, and the National Center for Public Health and Analysis defines our participation as “very limited“. To enter these networks, a center must have experience and patients. And experience and patients come when the treatment is available in our country.
Every treated patient can provide data on the real effect of the therapy – for registries, for European assessments, for subsequent studies, if we collect them systematically. Bulgaria already has more clinical trials per capita than the EU average. Experienced centers attract more trials, and they mean earlier access to new therapies for participating patients.
About 70% of rare diseases begin in childhood. In a country that is losing population and aging, every child who receives treatment on time and then studies and works is an investment. Every parent who is not forced to quit their job to care for a seriously ill child – too. That is why I do not accept the treatment of rare diseases as an expense for a few cases. It is an investment in people – the resource that Bulgaria has less and less of.
What should the state do – and what can Bulgaria achieve in the next three to five years?
Bulgaria cannot become a large market. But it can become a fast, predictable and reliable system – an advantage that does not depend on the size of the population. This requires six things.
- A national plan for rare diseases – with measurable goals, clear responsibilities, predictable financing and a working patient registry;
- Reliable rules for individual order. Legally, it is an exception, but in practice it is a permanent and necessary part of the system. The supplier must be chosen not only by price, but also by the security of the chain, delivery time and the ability to provide the entire course of treatment. The budget must be planned according to the actually approved treatment, and payments must be made on time;
- Active use of European instruments: joint clinical assessment - so as not to duplicate analyses and to decide faster; the right to request delivery from the holder of the marketing authorization; joint purchases and joint negotiations with countries with similar markets;
- New payment models for the most expensive one-time therapies - in installments and according to the achieved result. The budget is annual, and the benefits and risks of a gene therapy are monitored for years;
- More Bulgarian centers in the European reference networks - with targeted support, because membership brings knowledge, consultations with leading specialists and best practice;
- Early diagnosis – continuing the expansion of neonatal screening and genetic diagnostics.
And finally – dialogue. The rules for these drugs affect people for whom time is crucial, and must be written together with doctors, hospitals, patient organizations, institutions and suppliers – from the very beginning, and not when the project is already ready.
If you had to summarize?
In a small and aging country, timely treatment of every patient with a rare disease is an investment – in a family, in Bulgarian medicine and in the future of the country. Our job is to get the drug – with proven origin, traceable and on time.
Source: www.economic.bg