New administrative requirements for the provision of medicines and delayed payments for already made deliveries create double uncertainty for people with rare diseases. The National Alliance of People with Rare Diseases warns that the changes should not destroy what is working in the system, but rather make it faster and closer to the patient. In parallel, the organization signals that there are unpaid deliveries from 2025 and 2026, which already puts the continuation of therapies at risk.
For most people, three months, a few weeks or even a few days are administrative deadlines. For a person with a rare disease, this is sometimes the time between two doses of a medicine for which there is no alternative. That is precisely why any change in the rules for access to such therapies must be evaluated not only through the price and control over public funds, but also through a much simpler question – Can the new procedure delay the treatment of a particular patient?
This question is raised by the National Alliance of People with Rare Diseases (NAHRB) in a statement sent to the Ministry of Health on the draft amendments to Regulation No. 10.
The organization recalls that this mechanism is an exception to the general order for the supply of medicines precisely because it should enable patients without another therapeutic option to receive a new and proven effective treatment in a relatively short period of time. According to the Alliance, the current regulatory system has given Bulgaria a good position in terms of access to new therapies.
Therefore, they warn: “The additional procedures envisaged risk slowing down the process“ and compromising the already established mechanism for the treatment of people with rare diseases.
More control - but without new barriers to the medicine
This is precisely where the main dispute surrounding the proposed changes lies. No one disputes the need for control of public funds. The question is whether the control should add new steps before the patient receives their medicine.
Among the problems that arise under the proposed changes to the Regulation are the increased weight of the price criterion when choosing a supplier, additional studies and justifications, minimum deadlines for collecting offers, new requirements for supply documentation, and additional administrative movement of medical and financial information.
Each of these mechanisms may seem technical in itself. Taken together, however, they raise the more substantial question - how many administrative checks can there be between the doctor's decision and receiving the therapy before the procedure itself begins to create a medical risk?
It is precisely this risk that lies behind the general position of the National Alliance that the necessary changes should upgrade the system, not make it more cumbersome.
The opinion recalls a period in which treatment was provided through centralized tender procedures under the Public Procurement Act in the Ministry of Health. The organization's assessment of this model is laconic: “The mechanisms were cumbersome.“
A particularly serious problem, according to the Alliance, was the inability to include new patients in treatment within the current year. It is this previous experience that is one of the reasons why the organization insists that the new rules do not return the system to more difficult and slow procedures.
Price does not make the medicine the best
In rare diseases, the drug market has peculiarities that are difficult to fit into the standard logic of public procurement. For certain therapies, there are few suppliers, sometimes only one, and delivery options are severely limited. Therefore, the lowest price does not exhaust the question of whether the specific offer is the safest for the patient.
The expiration date, whether the medicine will arrive on time, the reliability of the supply chain and ensuring the continuation of therapy are important. When it comes to treatment without an alternative, economic efficiency must be part of the decision, but it cannot be considered in isolation from clinical risk.
The doctor must treat, the administration - to administer
Another fundamental problem is how far the administrative duties of medical teams should go. If additional research, market research and motivated justifications must be carried out for each delivery, the logical question arises whether these functions should be performed by the treating specialists or by structures that have the necessary administrative capacity. This is especially important in rare diseases, where experts are often few and patients - scattered throughout the country.
The issue is similar with deadlines. The minimum administrative deadline may seem short from the institution's perspective. However, in the case of a progressive disease or the need to continue therapy that has already begun, the same deadline may be fatal. Therefore, each procedure must have a sufficiently clear and working expedited mechanism for cases in which the medical situation does not allow waiting.
The medicine should not become hostage to a document
The same principle applies to documentary requirements. Control over the origin, quality and traceability of medications is mandatory. However, in the case of a medicine without a therapeutic alternative, the possibility of treatment being blocked solely because of a document whose issuance depends on a third party and is beyond the control of the patient and his doctor must be minimized.
This is the boundary that the new rules must carefully maintain – between the necessary accountability and the formality that itself begins to create a risk.
Why should a patient from the country travel to Sofia?
The National Alliance also poses another problem - the place where patients can receive treatment. Under the current model, therapy is carried out in the medical institution that has prepared the documents. In practice, this can force families from different parts of the country to travel to Sofia repeatedly. The Alliance believes that the system should also develop towards treatment at the place of residence, when medical conditions allow this, and notes that the proposed project does not solve this problem.
The Alliance also proposes a specific administrative change. Currently, treatment protocols are valid for three months. According to the organization, the treating specialist should be given the opportunity to assess whether the protocol can be issued for a specific patient for a longer period - up to one year inclusive.
The aim is to reduce unnecessary repetition of the same procedure in patients whose treatment is long-term and does not change.
A new patient should not have to wait for the next year
The second specific proposal is related to the list of medicinal products under Art. 266a, para. 2 of the Act on Medicinal Products in Human Medicine. Currently, it is supplemented on a certain date once a year. The National Alliance insists that this can be done at any time when the need arises for a specific patient.
This looks like a technical change, but behind it lies a very real case study – a person can be diagnosed with a rare disease a week after the administrative deadline. However, the disease does not comply with the calendar of the institutions.
Delayed payments – second risk for patients
However, the dispute over the future regulation is not the only problem facing patients with rare diseases. The National Alliance of People with Rare Diseases is filing a complaint with the Ministry of Health and the NHIF about delayed payments for medicines already delivered. According to information received by the organization from treating doctors and medical institutions, there are unpaid deliveries made throughout 2025, as well as deliveries from the beginning of 2026.
When a delivery remains unpaid for months, the issue is no longer just financial. The supplier must decide whether to continue providing the next quantities without having received the funds for the previous ones.
And if it stops - the consequences are transferred directly to the patient. In the complaint, the National Alliance warns of several possible consequences - termination of future supplies due to unpaid debts, inability to start therapy for newly diagnosed patients despite an issued protocol, and interruption of ongoing life-sustaining treatments for people who have been receiving their medications in this order for years.
In a number of rare diseases, this is not a treatment that can simply be postponed and made up for later.
We are talking about chronic, severe, and sometimes life-threatening conditions in which therapy can be long-term or lifelong. According to the complaint, its interruption carries a risk of deterioration, hospitalization, loss of the achieved therapeutic result, and in the most severe cases – and death.
The National Alliance recognizes the fact that the state is operating under a stretched budget, but insists that financial difficulties should not become a reason for interrupting treatment.
The organization wants the Ministry of Health to take action, the NHIF to promptly make the necessary payments to medical institutions, and a sustainable mechanism to be created to ensure that such a situation will not put therapies at risk in the future. The Alliance also demands information on what specific measures will be taken.
Source: kmeta.bg