I propose to take Questions Nos. 421 and 457 together.
I am aware that there is a lot of concern and worry among cystic fibrosis (CF) sufferers and parents and families of children who have CF about the current debate on the cost and availability of this medicine. I met with Cystic Fibrosis Ireland on Monday 25th January to discuss the matter.
Orkambi 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 on the 19th of November 2015. In other words, Orkambi has only been licensed for use in the European Union within the last few months. 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 done. 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 quoted by the manufacturer. This usually takes about 90 days. Orkambi's manufacturer, Vertex Pharmaceuticals, submitted an application to the HSE on 3 February 2016. I have requested the NCPE to expedite this part of the process to less than 90 days, and the Director has confirmed that he believes this can be achieved. However, it is not possible to provide status updates on the NCPE assessment while the assessment is still ongoing.
Once the assessment is complete, a recommendation is made to the HSE, which may then engage in price negotiations with the manufacturer.
A similar process is carried out in England by the National Institute for Health and Care Excellence (NICE), which does not expect to make a recommendation until July.
The process being followed is the same as that for other CF drugs like Kalydeco, 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.
