Dáil debates

Wednesday, 15 November 2017

Ceisteanna - Questions (Resumed) - Priority Questions

Medicinal Products Supply

3:10 pm

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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25. To ask the Minister for Health the status of the HSE's approval process for the health technology assessment report from the National Centre for Pharmacoeconomics, NCPE, on the drug Kuvan, which is used to treat persons with phenylketonuria; when he expects the drug to be approved for treatment of these persons; and if he will make a statement on the matter. [48283/17]

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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The Minister is very familiar with the rare genetic disease phenylketonuria where patients have difficulty processing the amino acid phenylalanine and therefore must live on very restricted, low-protein diet. We have had the report of the NCPE, which the HSE was considering. We have had two recommendations against sapropterin dihydrochloride, Kuvan, in the past nine years. A meeting was scheduled with the relevant drug company before Storm Ophelia occurred. Has a meeting happened and has information from the meeting been forwarded to the HSE drugs group? When will we get approval for the drug?

Photo of Simon HarrisSimon Harris (Wicklow, Fine Gael)
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I thank the Deputy for raising this issue. The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs scheme, under powers this House gave to the HSE in the Health (Pricing and Supply of Medical Goods) Act 2013. In reaching a 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 it has sought including, for example, advice from the NCPE.

Kuvan was considered under the national pricing and reimbursement process in 2009. At that time, insufficient evidence was available to support the pricing and reimbursement application submitted. In 2016, the HSE was advised that a new health technology assessment dossier would be submitted on the drug. The NCPE completed its assessment of the new dossier in September 2017 and did not recommend the drug for reimbursement as it was not deemed cost-effective at the price sought by the company. I highlight that point. The HSE assessment process is ongoing and the HSE will take into account any relevant expert advice when making its decision.

I have been assured by the HSE that commercial discussions are ongoing. I cannot answer the Deputy's question about whether the meeting has taken place. I do not know but I am told commercial discussions are ongoing. I am also told the HSE has received additional submissions from patient groups, via a Member of this House, which will be considered as part of the process. Both the commercial negotiations and the consideration of the patients' submissions will take place before the drugs group finally reviews the matter and takes a decision to the HSE leadership.

I have heard parents campaigning for this drug make a very valid point. They are concerned the system we have in place for assessing drugs for rare diseases is not adequate. A new committee called the rare diseases medicinal product technology review committee has been established by the NCPE. The terms of reference of the committee have been finalised and its responsibilities include the review of proposals for funding of new products for rare diseases or expanded indications for existing products for rare diseases. It will also contribute to the development of clinical guidelines. It is a valid point. We have quite a good assessment process.

Parents argue about the need for a better review when it comes to rare diseases. This committee will carry out a review that is in addition to and separate from the assessment process.

3:20 pm

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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I welcome the comments on the quality-of-life criteria for all rare diseases. The National Centre for Pharmacoeconomics report seems to make it clear that on clinical grounds there is a positive approach. As the Minister has said, it seems to come down to the economics and the difficulty of measuring costings for a very small cohort.

Like the Minister, I am very familiar with this disease and with the needs of families with experience of PKU - my family is one of those concerned. From listening to the PKU Association of Ireland and the various families involved, the Minister will know of the ongoing struggle of trying to live on a low-protein diet. It is a very small cohort. One in 4,500 babies is born with PKU in Ireland with about 20 referrals to the National Centre for Inherited Metabolic Disorders at Temple Street every year. Most Deputies will have received very valuable information from the PKU Association of Ireland.

Photo of Pat GallagherPat Gallagher (Donegal, Fianna Fail)
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Go raibh maith agat.

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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Many parents have outlined their daily struggle and the greater freedom-----

Photo of Pat GallagherPat Gallagher (Donegal, Fianna Fail)
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Go raibh maith agat. The Minister has one minute.

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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----- having access to a wider protein diet would have-----

Photo of Pat GallagherPat Gallagher (Donegal, Fianna Fail)
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The Deputy will have another minute.

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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----- if Kuvan were authorised. I know the Minister is sympathetic, but we need to act soon.

Photo of Simon HarrisSimon Harris (Wicklow, Fine Gael)
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I agree with pretty much everything the Deputy has said. While the PKU Association of Ireland is absolutely campaigning, as would any of us, for their loved ones to have access to this drug, it is also making the broader point about the need to review the equality in the assessment process for rare diseases. I assure the House that process is under way with a new working group of the NCPE.

As I did in my original answer, I assure people, who are wondering about the availability of Kuvan, that this process has not ended. The HSE has informed me that commercial negotiations are ongoing and it is also considering information it has received from patient groups, presumably including the PKU Association of Ireland.

When it comes to drug negotiations pressure is rightly put on me and on the HSE. It is important in a negotiation that companies also negotiate in the interests of patients in terms of a fair price for this country.

As the Deputy probably knows better than I do, PKU is an extraordinarily challenging condition. We have recently expanded the list of products available to PKU patients through our pharmacy and we need to do more in that regard. I will keep the Deputy informed on the process.

Photo of Tommy BroughanTommy Broughan (Dublin Bay North, Independent)
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The cost outlined in the report for 100% uptake is relatively tiny in the health budget at €12.7 million over five years or €2.5 million a year. The European PKU society which has strongly supported the PKU Association of Ireland has made the basic point that the EU regulatory agencies approved the drug in 2009 for use in 20 European countries at the time. A range of our European partners, including France, Austria, Germany, Bulgaria and Romania as well as the US have access for parents of children with PKU. Given that there is a genetic aspect to our community, it is extraordinary that Ireland is the one country where this medicine is not available. It is potentially life changing for those 800 individuals and their families. I know the Minister is sympathetic. Many of the advocates for rare diseases have forward their cases to him and I have supported those also. In this instance it should be approved.

Photo of Simon HarrisSimon Harris (Wicklow, Fine Gael)
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I wish to be very factual. The decision to approve or not is a matter for the HSE leadership team, which will hear from the drugs group under the legislation passed by this House. There may be a view that the process has come to an end and that is it. The NCPE has made its views known and they are a matter of public record. However, commercial negotiations are ongoing. In my 18 months as Minister for Health I have stood in this House quite a few times discussing drugs that patients have sought. Even though it looked extraordinarily difficult to obtain them, through negotiations we have managed to make drugs available. I hope we will continue to make new and innovative medicines available.

The international aspect the Deputy raised is something on which I have been working quite hard. Ireland is a very small country. While he is right in highlighting the comparative incidence of PKU, it is important that we collaborate internationally and share information with other countries on challenges we are experiencing. We need co-operate and scan the horizon jointly. In the coming weeks I hope to be able to make an announcement about how Ireland could formalise its involvement in working with other countries to get better access to new and innovative medicines and better value at the same time.