Patients with rare conditions: “Without innovative drugs the little Panagiotis – Raphael would not live”

The risk bell is struck by patients after the Ministry of Health decided to put a “knit” on import costs from abroad. In Greece, patients with rare diseases are about 500,000 and are at a time when they are in a difficult position not to have many therapeutic options and medications, such as patients suffering from more widespread diseases. The gap between the lack of “orphan” medicines – as called medicines for “rare” patients – has so far covered the process of introducing experimental treatments from abroad through the EOF and the Institute for Pharmaceutical Research and Technology (IFET). With the early access process and the intervention of former Minister of Health, Vassilis Kikilia, the innovative Trikafta treatment came in Greece in April 2020, which saved 10 Greek patients with the rare cystic fibrosis disease. Speaking to iatropedia.gr, the president of the Association of Rare Diseases Greece (ESA) Dimitris Athanasiou, carries the agony of 500,000 patients, who from now on may – as he says – need early access to some new treatment that exists abroad and not have it. “The Panayiotis – Raphael was a child with a neuromuscular condition from our union. If he hadn’t had access to treatment in America with the money we had collected then, he would have been lost along with the other six children lost 16 months ago. He still lives. His life expectancy without treatment was 16 months. So if you don’t have early access to drugs, every day she spends—especially our own diseases—you have casualties. It’s that simple. Let’s say the people with cystic fibrosis who got the approval in 2020, if they didn’t have the early access to IFT we would have lost half. Until he got the drug honor. So it is a channel that needs to be protected,” he notes. The failure of RELYVROP experimental therapy and the measures “knit” at IFET To date, the accessibility of patients with rare diseases to new innovative treatments has been quite satisfactory in Greece. The recent failure of RELYVROP therapy, however, introduced by IFT for about 20 to 30 patients suffering from rare side amyotrophic sclerosis (ALS), opened the Aeolus pouch. Speaking recently at a conference, Health Minister, Adonis Georgiadis, revealed that for this expensive innovative treatment – which ultimately proved ineffective in phase III study and was withdrawn from the manufacturing company – the Greek state spent 15m euros. He even rejected responsibilities to the doctors whom he accused of sub-subscribing, and made it clear that from now on no medicine will be introduced into Greece which is approved by the US FDA only and not by the European Medicines Agency (EMA). He also tabled an urgent amendment setting up a non-paid Electronic Pre-authorisation Control Committee (EOPY), to contain the “derailment” of the cost of medicines imported via IFT. The work of the new Commission will be to check all requests for the import of expensive medicinal products from abroad through the Institute for Pharmaceutical Research and Technology (IFET). These preparations should be noted that EOPY fully compensates them. Patients with rare conditions: “Early access must not be cut” The president of the Association of Rare Diseases of Greece, Dimitris Athanasiou, states that he chooses to keep the Minister of Health’s promise at a Patient Conference on 29 February, that “the patients who want their medicines will have them” “In the case of RELYVROP there was no waste or sub-subscribing. People suffering from ALS and receiving this medicine live one to two years. When you don’t have medicine – because the last medicine that came out for this condition was 30 years ago – you necessarily go to medicine that is in clinical trials. When there is such a great need, the legislator provides for the possibility of access even from positive indications, phase II study. And a doctor must have the option to give a drug that has been granted early approval in the United States to keep a patient alive. The recipe is passed by EOPY, and by a special committee and pre-authorisation is required. He does not write what the doctor wants” Patients are calling for an improvement in how to monitor the effectiveness and compensation of innovative and experimental treatments in Greece, based on the standards of foreign countries and for horizontal cuts in valuable treatments. “The wrong way is ‘I cut everything and let people die’. It’s that simple. There’s nothing in between. We ask that the “channels” of early access be not closed. But the Rare Disease Registers have not been created. The National Action Plan for Rare Diseases has not been fixed. We need to negotiate with companies and monitor whether the drugs ‘work’ and pay only what they ‘work’. Create a structured monitoring and evaluation system for medicines in which there will be no leaks. This is the right thing to do, the other is a “knife”, notes the president of the Spanish Patients. Association of Patients in Greece: To be a representative of patients in the New Commission The Association of Patients of Greece also took place. By its announcement the EAE asks the Ministry of Health to include Patient Representatives in the composition of the new Electronic Pre-authorisation System Control Committee of the National Health Services Agency. “This Commission, which will check requests for medicines for diseases rare and life-threatening, diseases that cause serious disability and diseases for which there are no treatments in the country, but have been approved by the competent European and international bodies, will judge issues decisive for access to treatments necessary for their survival. The Association of Patients of Greece, aware of the need for rational management of the limited resources of the Health System, must be a guarantor of transparency and efficiency in every action in this direction. Therefore the voice of patients, i.e. those directly concerned, cannot be absent from such an instrument of key importance, not only for treatments, but also for the sustainability of the Health System, a policy inherent to modern developments at European level,” the Patients end up. By Gianna Soulaki / Source: iatropedia.gr