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interview with Anastasiya Tatarnikova, Chairman at the Centre of Expert Help to Patients with Rare Diseases (House of Patients with Orphan Diseases)

Published on 2 November 2020 by Anna Andriyanova


Adam Smith Conferences: Some experts believe 2020 is a pivotal, game-changing year for patients suffering from orphan diseases. Would you agree with that?


Anastasiya Tatarnikova: It is still too early to draw conclusions. It all depends on the final decisions the government will make on the initiatives proposed in 2020. Over the past two years, we have seen some positive shifts in this area – two more high-cost nosological categories of orphan diseases were added to the list of illnesses funded from the federal budget. What is more, these first steps were declared as the beginning, with the Federation Council pledging to pass the costs for rare diseases from regions on to federal budgets. We expected the process to continue into 2020, but the pandemic broke out. I think this was among the main factors that stood in the way of federalisation of the government support for patients with rare diseases.


Adam Smith Conferences: Did the pandemic deal a serious blow to these patients? Is it still affecting them?


Anastasiya Tatarnikova: The pandemic and the related restrictions indeed sent a wave of worries among orphan diseases patients. In the beginning, when the coronavirus made its first appearance in Russia, many regional healthcare authorities focused on prioritising the industry’s most relevant issues. The rare diseases moved down the list of their priorities. Yet, the support was largely delivered – at least in the first half of the year. It was in summer that worries have started to add up. More and more patients reported disruptions in treatment and the supply of drugs. The situation affected even regular nosological categories that have been defined by the legislation and approved for funding from regional budgets.
The pandemic clearly shook the economy, causing ripples across healthcare. Orphan disease patients have felt the blow. The reasons are twofold. First, these diseases are rare, with patients being fewer in number and social impact being less obvious. Second, orphan drugs are expensive. Given the regions’ struggling budgets, the supply of orphan drugs will never be high on the priority list. In case of any perturbations in the drug supply, patients with rare diseases are always the first to suffer from drug shortages. This is why we are so worried about the suspended federalisation in this area: patients receiving federal support are better protected than those getting treatment under regional programmes.


Adam Smith Conferences: You work with patients directly and know the real state of affairs. Can you share the numbers?


Anastasiya Tatarnikova: Just recently, together with one of the intersectoral association, we organised a patient poll titled Healthcare System through the Eyes of Patients with Rare Diseases. The majority of respondents acknowledged that they lack government support. Nearly 80% reported that they faced problems with drug supplies (69% do not receive drugs or experience disruptions in supplies). Our own work confirms these findings. Some reservations should be noted, however. To resolve this problem, some (11% of respondents) indicated that they had to go to court, which does not accurately reflect the true situation. Say, patients with Morquio syndrome always receive treatment through a court ruling. For people with rare diseases whose treatment requires expensive and unregistered drugs, an out-of-court settlement is not really an option. Dozens of patients with various conditions relating to pulmonary arterial hypertension (e. g. IPAH) and chronic thromboembolic pulmonary hypertension who call our hotline have to await treatment for several months and may access it only if they follow sophisticated recommendations of legal advisors. In effect, almost half of the respondents admitted that they went to orphan disease patient organisations primarily to seek legal aid. Moreover, even after the treatment has been agreed, whether in court or out of court, they still might be exposed to undersupply of drugs. This may at times lead to the effect of treatment being entirely offset and a new threat being posed to health and lives of such patients. For instance, regional media recently reported new cases of serious disruption in treatment of paroxysmal nocturnal hemoglobinuria (PNH) notoriously famous for requiring the most expensive medications. As a result, patients end up in hospitals in need of blood transfusion. The reason for these disruptions is obvious: lack of financing.

The poll is another reminder for the expert community that what regulators and organisers include in the existing programmes (the Federal High-cost Nosologies Programme and the Regional List of Life-threatening Rare Diseases) is not at all an exhaustive picture. Many rare disease patients (given that dozens of new nosologies of rare diseases have been identified since the term “rare disease” first appeared in Russia) are struggling, often in vain, to get treatment because these nosologies are not included in the relevant support programmes.


Adam Smith Conferences: If the problem is so obvious, why then the announced federalisation plans have failed to materialise?


Anastasiya Tatarnikova: I have my own explanation. Recently the financing of the Fourteen High-cost Nosologies programme has become a matter of public discussion. Sixteen patient organisations issued a statement in early October calling for urgent extra financing of the programme in the amount of RUB 21 bn as it was already experiencing shortage of funds. Therefore, I would be very careful in saying that this programme is the only effective instrument to provide medicines for patients with orphan diseases. Besides, we should realise that both federalisation waves had a very specific logic in a sense that the programme covered only those diseases that it could pay for within the Finance Ministry’s funding limits rather than those requiring federalisation the most. Some RUB 11 bn was provided in the first instance and RUB 2 bn in the second. In other words, they covered what they were able to rather then what had to be covered. Therefore, the process of federalisation is increasingly raising questions. And at this critical, I would say, moment (23 June 2020) the president comes forward with his unprecedented initiative to create a targeted mechanism to fund the treatment of children with rare diseases.


Adam Smith Conferences: Do you support this initiative? Can it help solve the problems we are facing?


Anastasiya Tatarnikova: I do welcome it. The proposed Fund can become a milestone in treating rare diseases. Until now, we lacked a comprehensive approach to dealing with them. The high-cost disease programme we mentioned covers only 11 orphan diseases, whereas currently we have 258 rare diseases registered in Russia. Seventeen orphan diseases remain at the regional level, some of them creating a serious burden on regional budgets with patients having difficulties in receiving relevant medicines. For example, phenylketonuria (PKU) patients need a court ruling to receive a targeted therapy. There are 16 more rare diseases with a registered pathogenetic therapy in Russia that are not included in either the first or the second group. Plus, there are a number of rare diseases without a registered therapy in Russia. Experience shows that federalisation of orphan disease related expenses is the right approach. It allows for centralised purchases and helps save significant resources. However, so far we have not seen any federal decision targeting rare diseases exclusively. Even the subsidy allocated as part of the intergovernmental transfer in 2015 provided funding “including for rare diseases.” As a result, the regions used it at their discretion.
Therefore, we believe the president’s initiative to create a funding mechanism for orphan diseases is a very useful and promising approach to treating children suffering from rare diseases. In fact, it may also help adult patients, as regions will be able to reallocate the resources freed by the federal funding of children to treating rare adult diseases. Especially considering the fact that children with orphan diseases increasingly live to adulthood and longer thanks to the availability of medicines. What makes the need for this children’s orphan disease Fund even more urgent is the absence today and in the foreseeable future of a special charitable foundation funding the treatment of rare children’s and adult diseases. Unfortunately, as experience shows, even major charities turn down requests by parents to assist with the treatment of rare children’s diseases. And there are objective reasons for that. First, the charters of many charitable foundations specifically mention the medical conditions they can assist with, which mostly include oncological and hematological diseases. Secondly, children suffering from orphan conditions need constant therapy, becoming a multimillion-dollar burden on the foundations that mainly focus on end-result cases and are unable to continue funding indefinitely. Therefore, the government Fund with predictable and stable financing could successfully fill this niche.


Adam Smith Conferences: It is believed that among other things the fund can help with financing of unregistered medicines as you mentioned. 

Anastasiya Tatarnikova: This is true. Today, you need a court ruling to receive an unregistered medicine. On average, legal proceedings last for about nine months. During this time the patient’s condition significantly deteriorates and in many cases it becomes critical. And the reason for that is the fact that the law does not name the financing source for unregistered medicines. It includes a procedure for prescribing these medicines and outlines an imports mechanism, but fails to identify a financing source. Naturally, this loophole creates different interpretations of the law in the regions, with patients suffering as a result. The Fund can emerge as an effective tool legally assigned to finance unregistered medicines. Of course, any such financing by the Fund should be based on well-defined criteria to avoid accusations in mismanagement.

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