{"id":600949,"date":"2026-08-22T06:50:10","date_gmt":"2026-08-22T06:50:10","guid":{"rendered":"https:\/\/www.newsbeep.com\/ie\/600949\/"},"modified":"2026-08-22T06:50:10","modified_gmt":"2026-08-22T06:50:10","slug":"a-problematic-child-irelands-rare-drugs-record-casts-doubt-over-skyclarys-decision","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/ie\/600949\/","title":{"rendered":"‘A problematic child’: Ireland’s rare drugs record casts doubt over Skyclarys decision"},"content":{"rendered":"

Ireland\u2019s role as a “problematic child” for rare drugs has cast a reimbursement decision over Friedrich\u2019s ataxia drug Skyclarys<\/a> in doubt, according to politicians, campaign groups and healthcare experts.<\/p>\n

Skyclarys is an oral medication produced by biotech firm Biogen. It is the first known drug<\/a> that can slow the neurological deterioration associated with the rare genetic disorder Friedrich\u2019s ataxia.<\/p>\n

Around 200 people in Ireland suffer from the disease.<\/p>\n

HSE senior management will decide on Tuesday, August 25, whether to publicly reimburse the drug.<\/p>\n

Earlier this month, the HSE drugs group<\/a> recommended against publicly reimbursing Skyclarys, citing a lack of clinical data and a high \u201copportunity cost\u201d.<\/p>\n

The HSE has cited a figure of \u20ac280,000 per patient per year for the drug. That would cost the State around \u20ac134m over a five-year window. Biogen has disputed that figure while\u00a0the HSE refused to comment on negotiations with Biogen.<\/p>\n

As the highly anticipated decision looms, advocates in the sector have called for an overall reform in Ireland\u2019s management of rare drugs.<\/p>\n

Rare Diseases Ireland chief executive Vicky McGrath said Ireland\u2019s framework for approving rare drugs is antiquated and rife with red tape compared to European counterparts.<\/p>\n

\u201cGermany accepts stuff pretty much immediately and sets a price a year or two later after they get some real-world evidence as to the effectiveness of the drug,\u201d she said.<\/p>\n

\u201cWe have such a difficult reimbursement system; companies are delaying making applications in Ireland.<\/p>\n

\u201cWe’d wait five years before we start providing it in this country until we get all of that data. But in the interim, people are deteriorating\u2026 people are dying.\u201d<\/p>\n

Skyclarys first authorised in 2024<\/p>\n

Skyclarys was first authorised by the European Medicines Agency in February 2024. The HSE has since dealt with Biogen in formal pricing and reimbursement negotiations, while the National Centre for Pharmacoeconomics reviewed the clinical data and cost-effectiveness of the drug.<\/p>\n

The drug is available in 11 other European countries, according to Biogen.<\/p>\n

An analysis by the European Federation of Pharmaceutical Industries shows Ireland has approved 53 rare drugs from 2021-2024, in comparison to Germany\u2019s 156.<\/p>\n

According to the Irish Pharmaceutical Healthcare Association, a robust analysis for the reimbursement of a new medicine can comprise 20-30 steps in Ireland.<\/p>\n

The IPHA said the HSE drugs group meets on average once a month for two hours, while equivalent bodies in England and Wales delegate their work across four committees and meet for eight hours once a month.<\/p>\n

Fianna F\u00e1il TD Padraig O\u2019Sullivan said that Ireland was viewed as \u201ca problematic child\u201d by pharmaceutical companies when it comes to rare drugs.<\/p>\n

\u201cIf you look at bigger countries like Germany, France, who have far more progressive reimbursement in the rare disease space, those countries I would assume get preferential rates because they reimburse more drugs,\u201d he told the Irish Examiner.<\/p>\n

Mr O\u2019Sullivan called for more State funding to be directed towards rare drugs.<\/p>\n

\u201cThe existing budget annually for rare diseases and new cancer medicines is \u20ac30m. \u20ac30m of (the healthcare budget)\u2026 it represents less than 1%.<\/p>\n

\u201cAnd until such time as there\u2019s a more even distribution of that budget, you’re always going to have rare diseases and new innovative drugs at the back of the queue.\u201d\u00a0<\/p>\n

Government policy<\/p>\n

Current government policy on rare drugs approval stems from 2013 legislation.<\/p>\n

In June, health minister Jennifer Carroll MacNeill said she had signed off on a plan for a fresh review of Ireland’s rare drugs reimbursement framework.\u00a0<\/p>\n

Ms McGrath is among those who have warned that a review commissioned by Ms Carroll MacNeill’s predecessor, Stephen Donnelly, failed to bring about meaningful change.<\/p>\n

As the decision looms, patients, and advocates raised alarm at the consequences of an unfavourable decision.<\/p>\n

\u201cI think with something as serious as this, I hope they can look beyond some of the trial data and kind of realise the human side of it,\u201d said Helen Carney, company director of Friedrich’s Ataxia Research Ireland.\u00a0<\/p>\n

Ms Carney was diagnosed with the condition in 2002.<\/p>\n

\u201cThere’s no services and there’s no symptom management going forward if this disease-modifying treatment doesn’t become available,\u201d said Maureen Sweeney, head of operations at Ataxia Foundation Ireland.<\/p>\n

\u201cThere’s no neurorehabilitation centres around the country. Ataxia is not on the long-term illness list. There’s no age-appropriate care facility for progressive neurological conditions.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Ireland\u2019s role as a “problematic child” for rare drugs has cast a reimbursement decision over Friedrich\u2019s ataxia drug…\n","protected":false},"author":2,"featured_media":600950,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[2],"tags":[2120,61,60,43],"class_list":["post-600949","post","type-post","status-publish","format-standard","has-post-thumbnail","category-ireland","tag-hse","tag-ie","tag-ireland","tag-news"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts\/600949","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/comments?post=600949"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts\/600949\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/media\/600950"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/media?parent=600949"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/categories?post=600949"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/tags?post=600949"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}