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O’Reilly calls for Dedicated Criteria for Assessment of Orphan Medicines

8 November, 2017 - by Louise O'Reilly TD

Speaking at this morning’s Joint Oireachtas Committee on Health, Sinn Féin Health Spokesperson Louise O’Reilly TD called on the Minister for Health Simon Harris and the HSE to review the approval and reimbursement policy for medicines, specifically for orphan and ultra-orphan medicines like Respreeza and Translarna, and to bring forward dedicated criteria for the assessment of orphan and ultra-orphan medicines to treat rare diseases. 

Teachta O’Reilly said:

“Rare diseases collectively affect up to around 300,000 people in Ireland. There are between 6,000 and 8,000 known rare diseases, but just 5% of these have a licensed treatment option. The medicines for treating these rare diseases are known as an orphan or ultra-orphan medicine. 

“The National Rare Disease Plan for Ireland 2014-2018 recommended the bringing forward of: …appropriate decision criteria for the reimbursement of orphan (rare) medicines and technologies. 

“Indeed, in a recent appearance before the Health Committee, the HSE acknowledged the difficulties they face in determining an orphan medicine’s because the evidence they review on efficiency, cost, and budget impact is ‘often less than sufficient’.

“However, over the past number of years, it has become increasingly apparent that when it comes to approving orphan medicines for rare diseases the current system has a significant blind spot, in that the existing cost-effectiveness thresholds are not appropriate for medicines to treat rare conditions. 

“The problem in many ways is twofold; the metrics used by the HSE to evaluate effectiveness and approve orphan drugs has a blind spot in that it does not take into account the distinct differences between drugs for rare diseases and drugs for non-rare diseases and secondly, because there is a lack of dialogue and discussion with patients, advocacy groups, and clinicians. 

“Given the circumstances and the blind spot of the current system when it comes to orphan drugs, a dedicated, transparent, patient centric process which involves a broad decision-making framework is necessary. 

“Therefore, it is imperative that the Government recognise that the methods used to assess orphan drugs cannot be the same as those for non-orphan drugs and that the Minister for Health needs to ensure the full implementation of the National Rare Disease Plan for Ireland 2014-2018, specifically the bringing forward of appropriate decision making criteria for the reimbursement of orphan medicines and technologies.” 

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