Первая терапия атаксии Фридрейха столкнулась с отказами в возмещении

In Ireland, reimbursement has been recommended for refusal for omaveloxolone, the first medicinal product for the treatment of Friedreich's ataxia. Similar decisions or refusal of standard reimbursement have already been made in a number of European countries.

Where the drug is funded, access is generally provided under conditions that allow for cost containment and regular assessment of treatment outcomes.

🧬 What Is Friedreich's Ataxia

Friedreich's ataxia (FA) is a rare inherited neurodegenerative disease.

Frataxin deficiency impairs mitochondrial function and energy metabolism, leading to progressive loss of coordination and ambulation, cardiomyopathy, and other systemic complications.

Skyclarys – omaveloxolone by Biogen – is authorised in the European Union for the treatment of patients aged 16 years and older. The drug is taken orally once daily.

Omaveloxolone activates the transcription factor Nrf2, which regulates antioxidant defence, cellular detoxification, and energy metabolism. It is hypothesised that enhancing this signalling pathway reduces the consequences of mitochondrial dysfunction and oxidative stress.

📊 Clinical Data

In the 48-week Phase II MOXIe trial involving 103 patients, the difference between omaveloxolone and placebo was approximately 2.4 points on the mFARS scale in favour of the drug.

However, no convincing benefits were demonstrated for walking speed, fall frequency, or quality of life. Data on the effect of therapy on loss of ambulation, development of cardiomyopathy, and survival are currently lacking.

💶 Why Reimbursement Challenges Arose

The Irish National Centre for Pharmacoeconomics (NCPE) estimated the incremental cost at approximately €1.75 million per QALY compared with the national threshold of €45,000.

The probability of the drug being cost-effective at this willingness-to-pay threshold was 0%.
The annual cost of treatment reaches approximately €280,000 per patient per year, and five-year healthcare expenditure could exceed €134 million.

On this basis, reimbursement was recommended for refusal in Ireland at the price proposed by the manufacturer.

Negative decisions or refusal of standard reimbursement for omaveloxolone have already been made not only in Ireland, but also in Denmark, Sweden, Norway, Belgium, and the Czech Republic.

In the Netherlands, a modest clinically meaningful effect was acknowledged, but it was calculated that to achieve an acceptable cost-effectiveness ratio, the actual price would need to be reduced by at least 84%. Until such a pricing agreement is concluded, the drug is not recommended for reimbursement.

🌍 Where Access Has Been Granted

In Germany, Spain, Italy, Austria, Poland, Slovakia, Portugal, and France, access to the drug has been granted, though in most countries – under certain conditions.

Key mechanisms include:
— establishment of a budget cap;
— confidential price reductions;
— manufacturer rebates;
— outcomes-based payment elements.

📌 Pay-for-Performance and Registries

In several countries, continuation of funding depends on clinical dynamics.

Patient condition is regularly assessed using the mFARS scale. In the absence of the required effect or confirmed progression, further payment may be discontinued.

Additionally, mandatory patient registries, prescribing only in specialised centres, and subsequent reassessment of accumulated data are applied.

For example, in Italy, patients are included in the Italian Medicines Agency (AIFA) registry, and the drug is used through a monitoring system.

📌 Why This Matters

The situation with omaveloxolone shows that differences between national decisions are not solely related to the assessment of clinical effect.

The central question is how much a healthcare system is willing to pay for a modest slowing of neurological progression in the absence of robust data on long-term outcomes.

For orphan drugs, this is becoming an increasingly typical dilemma: innovative therapy becomes available before comprehensive data on long-term efficacy, impact on quality of life, and cost-effectiveness have been accumulated.

This is precisely why managed entry agreements, registries, regular reassessment of outcomes, and payment models linked to real-world clinical outcomes are gaining increasing importance.

📎 Source: Official Technical Summary of the Irish National Centre for Pharmacoeconomics