Rare disease patients go without therapy for up to a month, while the national strategy sits with the government

When a drug does not arrive on time, a patient with a rare disease does not just lose days. They lose functions that never come back. That is why patient associations have gone public again: therapy is late, new medicines wait for years, and the National Strategy for Rare Diseases, they say, has already been delivered to the government in its final version.

The National Alliance for Rare Diseases of Macedonia, the Bitola association "Living with Challenges" and the Association of General and Family Medicine Doctors held a press conference and a symposium under the motto "Think Rare: Recognise and Refer". The message was clear: despite cooperation with the institutions, delays in procurement and delivery leave patients without therapy for anything from a few days to a month.

The demands are not a luxury. A steady supply of medicines. A clear, published plan for when and by what criteria new patients and new therapies will be added. And an end to the practice of patients waiting two years for access to drugs the European Medicines Agency has already approved. According to the Alliance's president, Nataša Anǵeleska, patients are not asking for privileges, but for basic rights to healthcare.

It is hardest for those with muscular dystrophy. The patients' association has 26 members - the youngest is a six-month-old baby, the oldest is 23. For them every delay means an irreversible loss of physical abilities. The associations expect the procurement of the therapy that slows the disease to be completed by the end of the year. If the tender is late, so is everything the drug was supposed to preserve.

There is also a hole in the system that nobody is filling: when a patient turns 18, they leave paediatric care and have nowhere to go. "Once they turn 18, we wander around, nobody accepts them, we don't know where to apply for an examination," said the parent of a child with a rare disease. The associations want a multidisciplinary team, so that patients travelling to Skopje can complete all their check-ups in a single day.

They have also requested a meeting with Health Minister Sašo Klekovski, who took office in July, to agree the budgeting for the therapy in time. The strategy is written, the demands are clear. What exactly is the state still waiting for?