John Brassil (Kerry, Fianna Fail)
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As the Minister of State, Deputy Catherine Byrne, will be aware there is great anguish and concern about the availability of the drug, Respreeza, for the treatment of Alpha-1 antitrypsin deficiency, which is a genetic lung disorder affecting approximately 350 people in Ireland. The drug Respreeza is suitable for treatment for approximately 40 more patients than the 21 currently in receipt of it on a compassionate ground. As campaigners, we welcome that the company has made the drug available for a further two months, in March and April, but this means that late next month we will be again faced with a deadline set to expire, leaving patients facing the same anguish. This matter must be resolved. We are also concerned to learn that price is not the only reason for the non-reimbursement of Respreeza. It is claimed that Professor Barry from the National Centre of Pharmacoeconomics, the NCPE, believes that Respreeza does not work. Professor Gerry McElvaney has been doing research in the US on Alpha-1 antitrypsin deficiency since the early 1980s. He has continued that research here since then. He is the leading authority here and one of the top experts in Europe on Alpha-1. He has set up a research centre and done all of the work for the clinical trials. He also worked very hard on the purified protein that was used but all of this work appears to have been dismissed and disregarded by someone who has not even looked at the positive results of the 21 people who are being treated with Respreeza.

The United States Food and Drug Administration and the EU have not only approved Respreeza, the EU is reimbursing it for patients. It defies logic that Respreeza works everywhere else in the world but not in Ireland, apparently. We know from the clinical trial conducted in Ireland and in over 12 other countries that Respreeza has been life-changing for Alpha-1 patients. Not only has it been conclusively shown significantly to slow down the progression of the disease by 34% and, therefore, increase life expectancy, we know from research of patients in Ireland in receipt of it that it has made a huge difference in their daily lives. Irish people on Respreeza strongly believe that the drug has stabilised their condition, with almost 84% reporting an improvement in general symptoms. This was supported by a reduction in the frequency and severity of chest infections and associated hospital admissions. Chest infections per annum dropped by 68% while hospitalisations as a result of chest infection decreased by 69% per annum. People also reported improvements in their ability to work and to lead an active and fulfilled family and social life.

This form of therapy is already available for patients in eight European countries, including Portugal, Italy and Spain and in the United States. When will it be available for patients in Ireland?

Catherine Byrne (Dublin South Central, Fine Gael)
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I am taking this issue on behalf of the Minister for Health, Deputy Harris, who sends his apologies for not being here. I thank Deputy Brassil for raising the issue.

The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drug schemes in accordance with the provisions of the Health (Pricing and Supply of Medical Goods) Act 2013. In reaching its decision, the HSE examines all of the evidence which may be relevant in its view to the decision, including the dossier submitted by the company. The HSE will take into account such expert opinions and recommendations which may have been sought by it at its sole discretion, for example, from the National Centre for Pharmacoeconomics, the NCPE. Under section 19(4) of the Act, the HSE is prohibited from making a decision to reimburse a medicine "except in accordance with the criteria specified in Schedule 3" of the 2013 Act. In the interest of time, I do not propose to read out the statutory criteria.

In line with the statutory assessment process the HSE has confirmed it has received a request from the manufacturer of Respreeza for maintenance treatment of emphysema in adults. The NCPE, following a health technology assessment, determined that the manufacturer failed to demonstrate cost-effectiveness of the drug at the price submitted and did not recommend it for reimbursement. Respreeza was also considered by the HSE drugs group and it did not make a recommendation for reimbursement. The HSE considers the NCPE assessment, the drugs group recommendation and other expert advice as part of its decision-making process. The HSE leadership team has accepted the drugs group recommendation. In line with the 2013 Act, the HSE is required to provide formal written notice of its proposed decision to the company.

Following receipt of the notice, the company may make further representations to the HSE if it so wishes and the latter will consider any such representations in advance of a formal decision. As the statutory process is still ongoing, the company will have the opportunity to have further discussions with the HSE.

I note the decision by the manufacturer, CSL Behring, to extend the compassionate access scheme by a further two months. It is important to point out that the operation of such schemes is at the discretion of manufacturers. However, the Minister, Deputy Harris, has previously stated that any attempt by manufacturers to link continued access for patients already being treated with a new medicine with decisions under the statutory reimbursement process is both inappropriate and unethical. Manufacturers should operate such schemes in a compassionate manner and not on the basis of commercial motivation. There should be no link between compassionate use schemes and reimbursement decisions, and manufacturers should be upfront with patients and clinicians from the outset.

John Brassil (Kerry, Fianna Fail)
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I can only express my extreme disappointment at the weakness of the Minister of State's reply. The only aspect I welcome is the news that the HSE has agreed to re-engage with CSL Behring to seek to find a resolution. It is clear from what we have heard today that the Minister and the HSE accept the NCPE claim that the drug does not provide value for money and is not effective. However, every other source of information, including recognised experts such as Professor McElvaney, have found to the contrary. There seems to be a disconnect in terms of how the NCPE assesses drugs. We had the same situation with Orkambi and it is difficult to understand. There may be a broader discussion to be had as to how we reimburse high-tech drugs in general. Consideration might be given, for example, to funding research on the basis that any breakthrough drugs would be subsequently provided at a low cost base. However, that is a discussion for another day.

We are talking in this instance about 21 people who are being denied the lifestyle to which they, like everybody else, are entitled. The company is taking a humanitarian approach by making the drug available for a further period. I reject any suggestion its actions are being done on some sort of lobbying basis. The people who are suffering from this debilitating condition are living from month to month and it is not acceptable. Will the Minister of State relay to the Minister, Deputy Harris, and the NCPE the need for an urgent resolution of this matter? We are dealing with people's lives here and their suffering must not continue.

Catherine Byrne (Dublin South Central, Fine Gael)
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I will convey the Deputy's concerns to the Minister. As I outlined in the reply, the Minister has no control over how drugs are given out on a compassionate scheme basis. That is a matter for the company in question. I agree with the Deputy that people are suffering as a consequence of being denied access to this lifesaving drug. However, the manufacturers have a responsibility to their customers and it does not all come down to money. I will ask the Minister to reply to the Deputy regarding the concerns he has raised.