The family of the child, known as Merilin, was waiting for the drug to be added to the list of EHIF covered items. Whilst this will happen by or on 1 January 2019, her family says it is too late, forcing them to leave the country to seek alternative treatment. Another minor, still in Estonia, is reportedly in a similar situation regarding a form of nerve cancer.

The EHIF promised the drug, Eculizumab, sold under the trade name Soliris and used to treat paroxysmal nocturnal hemoglobinuria, a rare, acquired, life-threatening disease of the blood characterised by destruction of red blood cells by the complement system, a part of the body's innate immune system, amongst other illnesses, would be added to the list on 1 January 2019.

Very expensive drug

However, the family, who have not been named, had been desperate for a response since spring this year, reportedly being rebuffed several times by the authorities.

''What both the law and the heart says on the matter is clear to all, but you have to ask yourself, did Merilin deserve this?'' said the child's mother to ETV.

Manufactured by Connecticut-based Alexion Pharmaceuticals in the US, In 2015, Soliris was reportedly the fourth priciest medication in the US on a per-person-treated basis.

The case rases the issue of the coverage of expensive drugs used in rare illnesses, and concentrates minds on reforming the process of adding covered drugs to the EHIF list, something which currently only happens on an annual basis.

"It is a very dear medication and we relied on the recommendation of the medicines committee,'' said Rain Laane, chair of the EHIF, adding that it has long been in negotiations with Alexion.

System due for a shake-up

Health minister Riina Sikkut (SDE) said in July that required medicines will soon be added to the health fund's covered list several times per year, though this has not happened yet.

''The system of adding new drugs to the list covered by the fund is getting more flexible, so people won't have to wait a whole year for required medicines to be added,'' said Ms Sikkut at the time.

A paper signed on Friday is to add medicines to a value of €20 million to the covered list including drugs use to treat neuroblastoma, a form of cancer which another young girl, known as Annabel, suffers from and had previously been unable to get the drug via the EHIF.

EHIF chief regrets delays

As noted, the negotiations process had been blamed for holding up supplies of Soliris to date.

''We regret that these processes take such a long time,'' Mr Laane said.

''We have had on the one hand protracted negotiations with the pharmaceutical firm which makes the drug, but on the other, it is still on the paid list so far as the insurance fund goes since it is a hospital-administered drug, as confirmed annually by the government,'' he continued.

''We hope that our negotiations will continue with the pharmaceutical company, since even the figure we have arrived at is unsatisfactory, but at the same time, there are virtually no alternatives,'' Mr Laane continued, adding the existing system for financing medicines for rare diseases is not sensible and finding a workable solution is essential.

Family grateful for support so far

For their part Merilin's family have said when Soliris does arrive and gets covered by the EHIF, it should go to other needy cases since they are out of the country now. Soliris will reportedly be free for blood disorders such as that suffered by Merilin, but those requiring it for kidney disorders, which it also treats and which two people are known to require it for, will not get it for free.

The family were keen to stress they were still extremely grateful to the University of Tartu Hospital, and its childrens' fund, plus all the donors, thanks to whom Merilin is still alive, and that she and her smaller brother will miss school (she was in 11th grade) now that they are no longer in Estonia.

The EHIF is due to meet in a week with Riina Sikkut to discuss the system and the use of such drugs.

The original article (in Estonian) and link to the ''Aktuaalne kaamera'' clip is here.

Download the ERR News app for Android and iOS now and never miss an update!