Myasthenia gravis – a chance for a normal life still beyond the reach of many patients
Myasthenia gravis, although considered the most common of rare diseases, is still undertreated – despite groundbreaking changes in therapy and the availability of reimbursed biological drugs. Experts warn that most patients cannot benefit from modern therapies that can change their lives and restore their independence. Prof. Anna Kostera-Pruszczyk, chairwoman of the Rare Diseases Council, talks about hopes, barriers and necessary changes in the system.
Myasthenia gravis , or rather pseudoparalytic myasthenia , is a chronic autoimmune disease that affects neuromuscular function . Its characteristic symptom is excessive muscle fatigue , which can involve various muscle groups - from the eyelid and eyelid muscles , to the muscles responsible for speech , swallowing , breathing or movement . In the most severe cases, a so-called myasthenic crisis can occur, a life-threatening condition resulting from the involvement of the respiratory muscles .
Myasthenia is a disease that significantly limits independence in many patients – explains prof. dr hab. n. med. Anna Kostera-Pruszczyk , head of the Department and Clinic of Neurology at the Medical University of Warsaw and chairwoman of the Council for Rare Diseases at the Ministry of Health . Every third patient needs constant help from a caregiver. This is a huge burden not only for the patient, but also for their family and the entire social system – she adds.
Myasthenia affects the lives of patients to varying degrees. For more than half of patients, symptoms are mild, and some achieve remission without treatment. However, for many people, the disease means serious limitations - difficulty walking , preparing meals, dressing or taking up professional work.
We estimate that half of myasthenia patients have to give up work not of their own free will, but because of the physical inability to do it. It is worth adding that as many as half of patients are of working age - emphasizes prof. Kostera-Pruszczyk .
In recent years, significant progress has been made in the treatment of myasthenia . Until now, treatment has been based mainly on symptomatic therapy and immunosuppression , which, although effective, were burdened with numerous side effects in long-term use. Since 2024, the B.157 drug program has been in operation, under which three biological drugs are reimbursed - including two new-generation therapies , targeted at causes .
These drugs are much more effective, safer and better tolerated – explains the expert. – For patients who have struggled with troublesome symptoms for years and have been excluded from family and social life, modern therapy means a real chance to return to normality. – he adds.
Although reimbursement is already formally available, real access to treatment with modern drugs remains very limited. According to data, only about 60 patients have been included in the drug program, although up to 150 may qualify.
This means that two thirds of patients do not have access to therapy that can save their lives or improve their quality of life, says Prof. Kostera-Pruszczyk . – As a result, patients lose the chance for independence, and their caregivers – for returning to work and normal duties, he notes.
There are several reasons for limited access to treatment. The expansion of the program in the regions was stretched over time, as were the procedures for purchasing medicines. Another major problem is the underfunding of the so-called service of medicine programs, which translates into a lack of adequate staff.
Drug programs are very demanding – it's a lot of bureaucracy and administrative work. Hospitals should be able to hire additional staff to implement them. We should feel proud that we are treating the most seriously ill patients in a modern way – but also satisfied that these are procedures that are profitable for hospitals – says the expert.
Experts hope that the drug program will finally be fully implemented and access to innovative therapies will become widespread.
I would like every patient with myasthenia who qualifies for treatment to have real access to modern medicines – emphasizes Prof. Kostera-Pruszczyk . – Today, we are too often faced with a choice: to administer an outdated medicine with severe side effects or to administer nothing. And yet we have therapies that can give these people back their lives, work, and sense of security. I believe that step by step we will move towards treatment that meets the standards of modern medicine. This is what our patients expect and need.
Updated: 01/07/2025 07:30
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