Rare disease committee has still to be set up despite three-year wait

A committee to evaluate drugs for rare diseases has yet to be set up three years after a Government report recommended its establishment.

The HSE confirmed the Rare Disease Technology Review Committee has a chair, but no members, and is not expected to be in place before the first quarter of 2018. The setting up of the committee was a key recommendation of the national rare disease plan for Ireland 2014-2018, launched by then health minister James Reilly.

The need for such a committee was highlighted recently at a hearing of the joint Oireachtas health committee where politicians pointed to the difficulties of obtaining state reimbursement for orphan drugs.

They include therapies to treat extremely rare conditions such as rare metabolic disease PKU (decision pending on reimbursement of the drug Kuvan); and Translarna (currently subject of a court case) to treat Duchenne muscular dystrophy.

The State recently refused to fund Respreeza, used to treat genetic emphysema.

Orphan drugs are currently assessed through the same mechanism as all pharmaceutical products. Politicians and patients argue the odds are stacked against them because the numbers affected are so small.

The HSE said the committee “is in the process of being established”; that terms of reference have been agreed and that it will include patient representatives, technology assessment experts, clinical experts and the HSE.

Prof Michael Barry, clinical director of the National Centre for Pharmacoeconomics (NCPE), is chairman. The NCPE is tasked with deciding if drugs represent value for money.


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