An assessment of a drug for a rare muscular condition was removed from the website of the National Centre for Pharmacoeconomics, at the request of the Department of Health.
The new drug is for a condition called Duchenne Muscular Dystrophy (DMD), which can lead to scoliosis.
The recommendation from the NCPE is that the drug should not be covered by the HSE for the public unless its cost-effectiveness can be improved.
The NCPE provides independent assessments for the HSE on whether new drugs are cost-effective enough to be provided to the public.
NCPE Clinical Director Professor Michael Barry told RTÉ News that he agreed to remove the written assessment following an approach by the Department of Health yesterday on the understanding that the findings could be published in the near future.
The assessment was on the cost-effectiveness of the drug Givinostat, brand name Duvyzat for DMD.
The rare condition mostly affects young boys and it is estimated that around 50 could be eligible for treatment with the drug here if it were approved for use by the HSE.
This singular finding remains on the NCPE website but not the detailed technical summary and a plain English summary for the public which was online for a time yesterday.
The study found the estimated cost of the drug for each patient each year ranges from €92,000 to €334,000, depending on the weight of the patient.
The estimated five-year gross budget impact for the HSE would be over €26m and could be as high as €58m depending on whether all eligible patients were treated, the study found.
It compares to the current established estimated treatment of between €10,000 to €15,000 a year.
Read more: Families call for approval of new ‘game changer’ Muscular Dystrophy drug
The current treatment includes steroids, physiotherapy, occupational therapy and speech and language therapy.
The development comes as Minister for Health Jennifer Carroll MacNeill is due to address the annual conference of the Irish Pharmaceutical Healthcare Association (IPHA) in Dublin later this morning.
A new framework four-year drugs deal was announced by the IPHA and the Government last month.
The NCPE are a team of clinicians, pharmacists, pharmacologists and statisticians who evaluate the benefit and costs of medical technologies and provide advice to the HSE.
The full NCPC assessment of the drug, which has been seen by RTÉ News, said the drug is for patients aged six years and older along with steroid treatment.
It finds that it “not be considered for reimbursement for this indication unless cost-effectiveness can be improved”.
Children with DMD exhibit delayed gross motor development and at ages 10-12 years many require the use of a wheelchair.
As muscle weakness progresses, scoliosis and joint contractures develop, which may be associated with restrictive lung disease.
There is also an increased risk of pneumonia and life-threatening illnesses.
The drug is classified as an orphan medicine – drugs to treat rare diseases.
The Department of Health said that the NCPE’s role is vital in evaluating the cost-effectiveness and clinical benefits of medicines.
It said it is an important step to inform the consideration of the reimbursement of the medicine.
The Department said it has been engaging with the NCPE on matters relating to the publication of assessments and that it is conscious of the need to appropriately update the many patient advocates and families advocating on behalf of their loved ones.
It said that officials from the Department received an update yesterday from the NCPE that the assessment on Givinostat had been completed.
Officials asked for time to appropriately brief the Minister for Health, as there had been significant engagement on the application.
It said the assessment was shared with the HSE yesterday who immediately contacted the pharmaceutical company to schedule a commercial engagement.