David Stanton (Cork East, Fine Gael) | Oireachtas source

The Minister is unable to respond because he can only speak once in the debate. That is unusual and it happens on Tuesdays and Wednesdays. He can speak twice on a Thursday. It is bizarre but that is the way it is. He has listened carefully to what has been said. He has informed me that he will take note of this debate. It has been sincere and important. I commend my colleagues on the quality of the debate, to which I have listened. I thank Deputies on all sides for their contributions to this important debate on the Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018. I welcome the opportunity to contribute.

I appreciate that this Bill represents a well-meant endeavour on the part of Deputy Brassil to allow patients timely access to new and innovative orphan medicinal products. I acknowledge the considerable work which Deputy Brassil and members of the Joint Committee on Health have done in recent months in respect of orphan drugs. In February, the joint committee published a report - cited on a number of occasions this evening - on evaluating orphan drugs. The Minister and the HSE have given detailed consideration to the recommendations contained in that report. As a result, a number of recent changes have been introduced to the assessment and reimbursement process within the confines of the Health (Pricing and Supply of Medical Goods) Act 2013. This provides a greater balance and transparency to the assessment process where orphan drugs are concerned.

As every Deputy is aware, a rare disease diagnosis poses particular difficulties for patients who are affected and for the people who care for them. Living with a rare disease is a constant daily challenge which places enormous pressure on patients and their families. Deputy O'Reilly mentioned people outside of the gates of Leinster house and we can understand why they are there. It is vital that these patients receive the best evidence-based, cost-effective treatments our health service can provide. I completely understand that access to potentially beneficial medicines for the treatment of rare diseases is a crucial issue for these patients and their families. I join Deputy Gino Kenny in stating that nobody has a monopoly on compassion. The Deputy is correct and we feel that on all sides of the House.

The Minister is aware of campaigns by parents and advocacy groups for access to orphan medicinal products and he wishes to assure them that he, his Department and the HSE continuously work to address issues around access for patients to new and innovative medicines. The Government will be opposing this Bill on the basis that the statutory process for assessing medicines as contained in the Health (Pricing and Supply of Medical Goods) Act 2013 is fit for purpose where orphan drugs are concerned. In 2013, following on from substantial deliberations, Members of the Oireachtas put in place a thorough legal framework in the 2013 Act. The latter accords full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinion as appropriate. The Act specifies the criteria to be applied, including the clinical effectiveness and cost-effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE. While the Health (Pricing and Supply of Medical Goods) Act 2013 did not establish specific criteria to be used by the HSE in reaching decisions on the reimbursement of orphan medicinal products, the HSE and the NCPE consider issues such as the small patient numbers and the rarity of the condition to be treated when evaluating these medicines. There is an acute awareness of the vulnerability of these patients and the chronically debilitating or life threatening nature of many of these rare diseases. It is the Government's view that the criteria that apply to the evaluation process allow ample scope for the HSE to consider the particular circumstances that pertain to orphan drugs including that oftentimes there is a high level of unmet need for these rare disease conditions.

Deputies have correctly identified that increasingly orphan medicinal products are being developed to target very rare conditions. In view of the small number of patients affected by rare diseases, the pharmaceutical industry has been reluctant to invest in the research and development of medicinal products to treat them. In 2000, the EU introduced new legislation with the aim of providing incentives for the development of medicines for rare diseases. This intervention has proven very successful and has stimulated the development of many orphan drugs. The Government is fully aware and appreciative of the potential which these new orphan medical products can have in creating better health outcomes for Irish patients living with a rare disease diagnosis. We, of course, want all of our citizens to have access to the newest, most innovative medicines out there and as quickly as possible. Increasingly, the orphan medicinal products being developed to target very rare conditions are coming with extremely high list prices, prices which in many cases go well beyond what our health service can afford.

Health financial systems throughout the developed world are grappling with the challenges which these new and very often expensive orphan medicinal products pose. In many cases, the main driver of the price is an estimate of the maximum amount that health care systems can pay and is not connected to the health benefit of the treatment. Deputy Kelly is correct. It is expected that the trend will be towards more applications for orphan drugs in the years ahead. It is estimated that in the region of 45 new molecules, many of which will be orphan drugs, are due to receive market authorisation in Europe each year over the next five years. There is, therefore, no end-point whereby the pressure on the system to fund new products will abate. That is why it is essential that Ireland retains a threshold incremental cost-effective ratio so as to ensure that decisions in relation to the reimbursement of medicines are made on an objective and scientific basis, recognising the health needs of the entire population and other factors.

Affordability and sustainability remain major challenges for the health service in the context of our budget-limited health service. A less rigorous set of rules for orphan products will not change this. The HSE is required under the 2013 Act to consider the funding challenges that orphan drugs represent. It does this by drawing on the criteria contained in the Health Act including the potential or actual budget impact of the drug in question and the cost-effectiveness of meeting health needs by supplying a particular item rather than providing other health services. These are difficult decisions that must be made in the context of the availability of finite resources in the face of ever-competing demands. Since the signing of the four-year framework agreement on the supply and pricing of medicines in 2016, a significant number of orphan drugs have been reimbursed by the State.

In terms of the availability of orphan medicinal products in Ireland, the 2013 Act is delivering and patients with rare diseases are receiving access to new and innovative medicines. The Government is opposing this Bill because it is satisfied that the current criteria contained in Act are fit for purpose when assessing orphan drugs. There is sufficient flexibility contained within the current legislation to allow ample scope for the HSE to consider all relevant evidence that pertains to orphan drugs.

The Government is of the view that the 2013 Health Act is delivering on its objectives to provide new and innovative medicines to Irish patients, including those with a rare disease diagnosis.

The Bill as drafted would disregard any threshold incremental cost-effective ratio in the assessment of orphan medicines, thereby removing an accepted, validated economic evaluation system for assessing medicines. In doing so, it would dilute considerations around affordability and sustainability. The health service must look to care for the entire population. Accordingly, every decision, including the reimbursement of orphan medicinal products, must be based on scientific assumptions, clinical evidence and cost-effective analysis as provided for by the 2013 Health Act.

The Minister for Health is aware of the human side of this entire process. Patients who are living with a rare disease diagnosis face enormous challenges in their day-to-day life. Such a diagnosis has significant repercussions for the whole family. These diseases can be acute or chronic in nature. While there has been remarkable progress in advancing innovation for rare diseases, there still remain significant challenges in that there are many rare conditions which still do not have a treatment option.

It is right that Deputies assess and question the way that we deliver health services to our citizens. The Minister for Health and I appreciate the conviction and sentiment behind the Bill. The Minister for Health looks forward to further positive engagement with Deputies in seeking solutions to address the challenges which we all face around securing more timely access to new and innovative medicines for all our citizens in an affordable and sustainable manner. I will bring the Deputies’ comments this evening to the Minister’s attention. This is an important and sincere debate. It is one which will add to our thinking and progress in the future. I thank Deputy Brassil for the work he has done on this matter. The Minister is looking forward to continuing to engage on this important issue.

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