The health service’s decision to fund a treatment for the rare genetic disorder Friederich’s ataxia can only be welcomed.
As patients and their families have begged for what they have described as their only hope to slow the progressive illness, one would have to be heartless to have wished for any other outcome.
But the decision also points to a growing problem our health system must battle – how to balance the books while also ensuring Irish patients have access to the best innovative treatments.
The State’s medicines bill is climbing. It now stands at between €3 and €4 billion – twice the amount spent on our entire defence budget.
RM Block
One commonly cited reason is the introduction of advanced, high-cost, specialised medicines to treat cancer and rare conditions like Friedreich’s ataxia.
The Government insists significant funding has been made available for new medicines, which between 2021 and June of this year has delivered 280 types – 77 of which were for rare diseases.
But the decision-making process on what to reimburse is complicated and lengthy.
Research published by the Irish Pharmaceutical Healthcare Association in May found the time between European approval and Irish access for drugs for rare conditions was 801 days, or just over two years, compared with an EU average of 614 days.
The calculation faced by the State every time it considers reimbursement for a drug is: what will deliver the greatest benefit for the greatest number of people?
[ The limitations of ‘red flags’ in healthcareOpens in new window ]
That can be particularly challenging when it comes to so-called orphan drugs – those used to treat rare conditions – because of their high price and relatively small number of potential recipients.
Skyclarys is not the first battleground drug. In 2016, cystic fibrosis patients were left devastated when the National Centre for Pharmacoeconomics (NCPE) recommended that “game-changing” drug Orkambi should not be funded as it was not cost-effective.
Following intensive lobbying, in 2017 an agreement was reached between the HSE and pharmaceutical company Vertex to make it available to 900 patients, with access expanded in 2018 to younger children following European approval.
Also in 2017, the HSE decided not to add Respreeza, a drug to treat a rare lung disease, through its reimbursement scheme after assessing its clinical benefits and cost effectiveness. A deal was later reached in 2018.
In 2019, the HSE approved Spinraza to treat the muscle-wasting disease spinal muscular atrophy, overturning a negative recommendation by its drug group in light of a revised price application by the manufacturer, Biogen.
What’s important to note on Skyclarys, however, is the statement from the HSE that had the “substantially improved financial offer” not materialised from the manufacturer, it would have come to the same conclusion as the drugs group, which was to refuse reimbursement.
[ The question changed – from whether I want a child to if I can have oneOpens in new window ]
The executive seemed quick to point out that this was not a case of the system bending to political or public campaigning, of which there was plenty. Though it must be acknowledged that a decision not to reimburse this drug would have been politically unpopular.
Money is finite, particularly in a health service that is consistently criticised for overspending. Every euro spent on one item is one less euro available in another area of healthcare.
A quick search on the NCPE website shows many instances of recommendations against reimbursement, primarily rejected as not cost effective.
The harsh truth is that while this story ended well for Skyclarys patients, many others will not be so fortunate.
As further advanced, innovative treatments come on stream to treat rare conditions, the HSE will inevitably have to refuse some drugs for reimbursement.
