DÁIL QUESTION addressed to the Minister of State at the Department of Health (Kathleen Lynch) Orkambi for treatment of Cystic Fibrosis

 To ask the Minister for Health to fast track the approval of the new drug Orkambi for the treatment of Cystic Fibrosis; and if he will make a statement on the matter.


REPLY.
I am aware that there is a lot of concern and worry among cystic fibrosis (CF) sufferers, and families of children who have CF, about the current debate on the cost and availability of this medicine.

Orkambi (lumacaftor/ivacaftor) is a combination drug for the treatment of CF in patients with two copies of a specific CF gene mutation. The drug was licensed by the U.S. Food and Drug Administration in July 2015 and by the European Medicines Agency in November 2015. To date, it has not been approved for reimbursement by any public health service in Europe. 

Each country has its own process for approval. The Irish process is laid down in law in the Health (Pricing and Supply of Medical Goods) Act 2013. Under this law, the HSE has statutory responsibility for decisions on pricing and reimbursement of medicinal products under the community drug schemes in accordance with the provisions of the law. It is appropriate that these should not be political decisions and that a scientific and evidence based approach is taken to determine the extent to which patients would benefit from treatment with expensive new drugs.

Prior to reimbursing any medicine, the HSE considers a range of statutory criteria, including clinical need, cost-effectiveness, and the resources available to the HSE. The process for approval of high-tech new drugs in Ireland involves three steps. The first step involves a Rapid Review, which has been completed. The second involves a full Health Technology Assessment by the National Centre for Pharmacoeconomics (NCPE). This expert assessment is scientific and objective and will evaluate whether the claims being made about the benefits of a new drug are valid, and to what extent, in terms of its impact on life expectancy, quality of life, reducing hospital admissions, reduced need for transplants etc. The NCPE will also consider which patient groups might benefit, how the drug compares with existing treatments and if it is cost-effective at the price being sought by the manufacturer. The Minister for Health has requested that the NCPE expedite this part of the process. The Centre hopes to be in a position to issue its recommendation to the HSE in early June, and the HSE may then engage in price negotiations with the manufacturer. 

The process being followed is the same as that for other CF drugs like Kalydeco (ivacaftor), new drugs to treat cancer and other rare diseases. Orkambi will be treated in the same way as these were, and has to be, under the law. This process is designed to secure the best value for the HSE, and the savings that accrue from this process can be used to fund other health service developments, for example isolation rooms for CF and cancer patients, better ambulance services, home supports for the elderly and safer maternity services.

I hope that it will be possible for Orkambi to be approved for patients who will benefit from it, and at a fair price, but it must go through the normal approval procedure first, like all other high-tech medicines.