A few days ago, the Minister of He alth, Dr. Petar Moskov, announced that as part of the planned reform, drugs for rare diseases will be transferred for funding to his ministry. The argument - "we are talking about therapies that reach 800-900 thousand per patient per year, and this cannot be an obligation of the solidarity fund".
His idea is not new, but once again it stirred spirits in the circles of patients with rare diseases. Their medicines were already part of the ministry's portfolio and they remember that time with large periods of missing medicines, restrictions and hardships.
At the same time, according to a report by the Institute of Rare Diseases, since 2011by 2015, the number of drugs and medical devices for rare diseases paid by the NHIF increased by about 40%, but the costs remained between 7 and 8% of the total budget of the Medicines Fund. In addition, there are therapies for rare diseases that are carried out with ordinary drugs at a price of BGN 50-60 per month, but the Fund does not pay for them. These two facts make patients question the ministry's arguments and demand that the idea be reconsidered and debated again. Vladimir Tomov, chairman of the National Alliance of People with Rare Diseases, told more about these issues.
Mr. Tomov, why don't you want your medicines to "move" to the Ministry of He alth?
- We don't want that because of the results we've seen from the treatment done through the MH. There were annual auctions there that resulted in limited access. This was a barrier to the treatment of new patients every year, and new therapies were difficult to enter, as the fund itself was limited, with a tight budget. We have both witnessed and suffered from cases of treatment delays. Now that we're at the Treasury, patients receive their therapy up to two months after they're diagnosed. Incorporating new diagnoses is still difficult, but since 2011 the number has increased from 28 to 36. This is extremely important for rare diseases, as few - only about 5% - are treatable at all and it is unacceptable to for these 5% patients, let's put additional restrictions, such as a limited fund to the Ministry of He alth.
What have you discussed with representatives of the Ministry of He alth on the subject?
- Their main argument is that the state should have its share in providing he alth care. Insurance for rare diseases is a very small part of the overall he alth budget. With over BGN 2 billion disbursed by the NHIF in 2014, almost half of which - for drugs for home treatment - our medicines cost BGN 74 million. So if the state really wants to significantly participate in providing the treatment of Bulgarians citizens - let the hospital care take over! Here we are talking about a billion and something BGN, and so it will be able to take its leading place, as it is according to the Constitution.
But - kidding aside - we would like the institutions to first clarify what rare diseases are. This will be done after drawing up a list of rare diseases, as laid down in the latest regulatory changes. After that a resource can be planned and only then could we discuss where it is
their place in our he althcare system
and in what way to help these people. Because outside of the conversation about the source of the money, the important question remains that we focus on medicines, but people with rare diseases also need timely diagnosis, they need rehabilitation, social adaptation, which is missing.
If I understand correctly, you want the state to first recognize the rare diseases in its territory and determine which ones it will pay for treatment. As far as I remember, at the moment, about 1/3 of the existing orphan drugs on the pharmaceutical market are imported into our country, right?
- Regarding orphan drugs, their number is unsatisfactory. What encourages us is that in recent years, however, the number of reimbursed drugs has been increasing.
We hope to reach European level
in this respect as we have achieved it in terms of the regulatory framework. Since last year, we have a definition in the He alth Act of what a rare disease is, we also have an ordinance that regulates the registration of rare diseases, patients and expert centers where they can receive adequate care. All this will pay off once we see the list of rare diseases ready, on the basis of which we can now plan, judge and seek the best for patients and their families.
It should be clear that according to WHO data on the prevalence of rare diseases, there are 400,000 people in Bulgaria who are affected by some rare disease.
At the moment it is impossible to assess what will happen if rare diseases are mechanically "moved" before we know how many rare diseases are registered in our country, how many patients there are with each of them, what are the ways of treatment and does the state support them.
We are fortunate that more and more drugs are being discovered for rare diseases and they can then be used for widespread diseases. I don't use the term "socially significant" because I don't understand why significance is measured by quantity. After all, we are not talking about a kilogram of potatoes, but about human life.
Iliana Tonova, with sarcoidosis: Every person is a person, the efforts for his life are worth it
Ms. Tonova, what difficulties do patients like you most often encounter?
- Our first problem is diagnosis, since the symptoms of the disease are not specific and it is very difficult to recognize. Our second problem is follow-up - last year we applied to be dispensary, as it was until 1990. The disease is not new, it has been known for more than 150 years. There is no cure, but there is maintenance, which reduces the damage to the patient and postpones his disability. That is why regular visits to a specialist are extremely important.
How are you being treated?
- We don't have an orphan medicine, we don't have a new and very expensive medicine, but we have the chance - if the patients are regularly monitored and the medicines that support them are dosed correctly, they won't lose their ability to breathe normally or walk. And to be able to work, pay taxes, and not wait on the state for meager social assistance. That is why we have been fighting for years to be recognized by the Bulgarian he alth system. This can now be done thanks to this list of rare diseases to be drawn up. But here too we have a stumbling block - the diseases that until now were in Ordinance No. 38 and received treatment from the NHIF will automatically enter the list. And we still have to prove that this disease is rare, that there are only a few patients and a certain therapy.
That is, your medicines are not paid for by the NHIF?
- That's right. We applied last year to be included in Ordinance No. 38. We submitted documents in May, in January this year the opinion of the national pulmonary consultant was still not received. Now changes are being planned in the procedure and we will have to start the whole battle all over again - to have them admit that we exist, to have at least some of our medicines taken over by the NHS…
What kind of money are we talking about in the end?
- In the case of sarcoidosis, depending on the doses and exact medications that the respective patient needs to take, the monthly therapy costs between BGN 60 and 200. Considering that there are around 30 patients, the amount is extremely modest for an institution as NHIF.
If it is not a lot of money, how do you explain the absence of your therapy from the list of those reimbursed by the NHIF?
- I will tell you a real case. When I got sick and the GP said to me: "Well, yes, your illness is chronic and you will have to take your medicines for life, but the National He alth Insurance Fund does not pay for them", I decided that this was some kind of mistake. I went to the RHOC and asked why there is no reimbursement for our drugs. One employee told me: "We are not interested in you. A lot of bureaucratic energy will go into dealing with your illness, you are so few people.”
That's why I think that often the administration, dealing with numbers, sees only the "big" groups. But I'm an engineer, and as someone who works well with numbers, I want to tell you that it would be a lot cheaper for the state if a guy like me got regular checkups, treatment, and work instead of paying for the consequences of his disability. Not to mention that every person is a person, their life is worth the effort.