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Rare Diseases Q1 2023

HSE struggling to meet the needs of rare disease patients

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Pádraig O’Sullivan TD

Cork North Central

There are over 6,000 recognised rare diseases in Ireland, with many more yet to be identified. 

Rare diseases in Ireland are not rare; collectively, over 300,000 of our citizens are affected. So, you would think that the health service of this country would be well-versed in how they scrutinise and evaluate orphan drugs relating to the treatment of these diseases.  

You might also think that a specific pathway for the assessment of these orphan drugs might already be in situ, given the significant pharmaceutical footprint we have in this country. However, this is not the case.  

Model comparison 

In Ireland, an assessment of an orphan drug or an application for reimbursement is undertaken through a rigid assessment process, where transparency is questionable — with little or no patient input.  

In other progressive EU states, models vary. Some of our Nordic counterparts operate a system where the patient is at the heart of the process; they, after all, have been diagnosed with — in many cases — a life-altering or life-limiting condition.  

Countries like Scotland, Sweden, Denmark and most recently the Czech Republic give relatively quick access to drugs for patients. Some operate a ‘risk-sharing’ partnership with the drug manufacturers where you operate on a pay-by-efficacy basis.  

Other countries make drugs available immediately, on a needs basis. All these models have the patients and their wellbeing at the centre.  

The reimbursement system is not responsive to patients’ needs.

System shortcomings 

In Ireland, we operate an archaic model with an inflexible QALY threshold of e45,000. In the case of orphan drugs and rare diseases, this threshold is rarely adhered to thus leading to an initial and inevitable refusal of the drug.  

The system does allow for subsequent assessment and further investigation, but the inevitable consequence of this bureaucracy is time — and time is crucial here. Ireland ranks among the laggards of Europe in terms of the assessment and reimbursement process with decision-waiting times ranging from 600–1,000 days, depending on the drug and circumstances.  

Reimbursement process 

Patients face an agonising wait, on what may be a whim and a prayer, often in pain, often in deteriorating health — sometimes death.  

The reimbursement system is not responsive to patients’ needs. The reimbursement process we have does nothing to encourage further research and clinical trials from the industry; and above all else, the reimbursement process here is primitive — particularly when so many of these highly sought-after drugs are manufactured here, shipped from our ports all over the globe, yet many remain unavailable to citizens who need them most.  

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