Thomas Pringle TD

PQ: PKU drug KUVAN rejected by Government


*  295.  To ask the Minister for Health the reason kuvan, the only drug available to those diagnosed with PKU, was refused by the HSE in view of the fact that it radically improves the quality of life for those with the condition; if he has given consideration to providing funding in order that the drug can be offered to persons here; and if he will make a statement on the matter.

– Thomas Pringle T.D.

For WRITTEN answer on Tuesday, 10th October, 2017.

Details Supplied: . It is the only drug available for this condition and can vastly improve the quality of life of the users. This drug is available throughout the EU in: Austria, Belgium, Bulgaria, Czech Republic, Estonia, France, Germany, Hungary, Italy, Netherlands, Luxembourg, Norway, Portugal, Romania, Russia, UK, areas of Sweden, Lithunia and Denmark. It is also available worldwide and other parts of Europe




The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drug schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.


In reaching its decision, the HSE examines all the evidence which may be relevant in its view for the decision and will take into account such expert opinions and recommendations which may have been sought by the HSE, including, for example, advice from the National Centre for Pharmacoeconomics (NCPE).


Sapropterin (Kuvan) was previously considered under the national pricing and reimbursement processes in 2009. At that time insufficient evidence was available to support the pricing and reimbursement application submitted by Merck Serono for Kuvan.


In December 2015 Merck Serono advised the HSE that the market authorisation for Kuvan was transferring to Biomarin in 2016. The HSE met with the new market authorisation holder Biomarin in May 2016 and was advised that it would be submitting a health technology assessment dossier in relation to Kuvan.


The NCPE assessment of the Biomarin dossier was completed on the 15 September 2017 and the NCPE did not recommend Kuvan for reimbursement as it was not deemed cost effective.


The HSE assessment process is ongoing and the HSE will take into account any relevant expert advice when making its decision, in line with the Health (Pricing and Supply of Medical Goods) Act 2013.


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