Louise O'Reilly (Dublin Fingal, Sinn Fein) | Oireachtas source

I will not need all that time. I thank Deputy Brassil for bringing this Bill forward. Deputy Brassil and I are members of the Joint Committee on Health. We have had occasion to attend many presentations and hearings on the issue of access to orphan medicines. We have also been approached, as every person in this House probably has, by patients, their families and clinicians on this very issue. In recent years it has become increasingly apparent that when it comes to approving drugs for rare diseases, the system is simply not fit for purpose. While this Bill is not a panacea for every issue surrounding access to orphan drugs and the approval process, it does go some way towards addressing a fundamental problem. It allows for the establishment of specific criteria applicable to orphan medicinal products for the purposes of the Health Service Executive's decision on adding an item to the reimbursement list.

The HSE estimates that 300,000 people in the State will develop a rare disease at some stage in their lives. There are between 6,000 and 8,000 known rare diseases. Only 5% of these have a licensed treatment option and there is a high level of unmet need. It is that unmet need that we meet at the gates of this institution when we are going in and out. It is that unmet need that causes people to get out of their sickbed, or people to come here on behalf of their children, to protest outside the Dáil. That unmet need that must be addressed. It puts sometimes unfair pressure on members of the Government, but people feel that they have no option. The pressure is put on all of us. Deputies encounter people waving pieces of paper asking what price is a life. That is no way to do business. That is no way for sick people to have to behave, but they are not doing it because it is their first option. They are doing that because they feel they have been left out of the process. Which of us would not do that? If it was our children, our loved ones, someone in our family or our neighbour, which of us would not do it? Of course we would if we thought it might work. Every time we see a protest outside here, it is evidence that the system for assessment is not fit for purpose.

The metrics used by the Health Service Executive to evaluate the effectiveness of and approve orphan drugs are unfit for purpose. They do not take into account the distinct differences between drugs for rare diseases and drugs for non-rare diseases. Therefore, we see it as imperative that the HSE changes the way it evaluates medicines to treat these very rare conditions. We believe that when it comes to the approval of orphan drugs, the process needs to be more transparent as well as giving patient groups and clinicians a stronger voice in the National Centre for Pharmacoeconomics.

We are also aware that the pricing strategy adopted by some pharmaceutical companies in recent years presents significant challenges and this is leading to serious affordability problems for the health service in Ireland and internationally. It is exactly that to which I was referring. It is that pricing strategy adopted by some of the pharmaceutical companies that puts pressure on the campaign groups, which, in turn, put pressure on politicians who, in turn, find themselves asking the Minister what is the price of a life. That is no way for this to be run. None of us wants to be in that position. That is why I welcome the Bill. I would urge the Minister and his colleagues to reconsider support for this. I am sure Deputy Brassil would be open to working with them, if the belief is that amendments are needed. We all have acknowledged that a change is needed and that is what we should be working for.

The national rare disease plan, the generic policy framework for rare diseases compiled by the Department of Health, recommended the bringing forward of appropriate decision criteria for the reimbursement of orphan rare medicines and technologies. However, to date, no dedicated decision-making criteria have been forthcoming. Instead orphan drugs have to be assessed in this narrow confine where matters of cost almost immediately rule out their approval for any patient.

The HSE has traditionally been an early adopter of new medicines where clear evidence of clinical benefits to patients can be demonstrated and value for money assured. This remains the case when it comes to non-orphan drugs. However, when it comes to approval and adoption of new orphan medicines for rare diseases, it is failing. It is important that the Bill seeks to establish a new set of criteria for consideration of orphan medicinal products mitigating against low qualitative data and guaranteeing that the HSE will consider qualitative data presenting on an orphan medicinal product for patients, clinicians and others.

We are all aware that a fundamental problem with the current approval process is that there are no dedicated criteria for the approval of orphan drugs. Unfortunately, unlike many other health systems across Europe, there are no specific protocols and processes to evaluate orphan medicines, meaning that orphan medicines are assessed under the same methodology intended to be applicable to all other medicines. Essentially, a drug that treats a patient with muscular dystrophy is appraised using the same cost-effectiveness process that is used to assess and approve an aspirin to treat headaches. We agree with experts, including the Irish Pharmaceutical Healthcare Association, that the quality system and the threshold are fair in assessing the value for money of innovative non-rare disease medicines brought forward by companies, but not for rare disease medicines.

Furthermore, the current decisions regarding the cost-effectiveness of a new drug for a rare disease do not take into account the wide-ranging savings that could be made from a drugs approval. For instance, it does not factor in savings from a patient not needing to return to hospital or require a more expensive treatment down the line, or, indeed, time taken off work or time committed by a family carer.

We need to change the way we evaluate medicines to treat very rare conditions. As well as the passing of this Bill, we also need to see a new assessment system and approval process which guarantees input from patients and clinicians and gives them a stronger voice in the HSE decision-making. Such a system works extremely well in Scotland where they have a patient and clinician engagement assessment process. If we had such a process here, it would give patient groups and clinicians a stronger voice in the HSE decision-making and the assessment process. It would also allow the HSE to gather detailed information which would allow a discussion on the benefits of a medicine, including how it can impact on the quality of a patient's life. This information is not currently captured within the present assessment process and is why patient groups tell us that they feel left out of the decision-making process. As I stated, that manifests itself at the gates. That is where that ends up. People do not feel that they have been part of the process. They feel that they have been shut down, that the answer is "No" before anyone ever looks at the assessment and, more importantly, that nobody is taking their child, their loved one, the person they represent, into account. I am not suggesting that is how it is. I am telling the Minister that is how they feel. That is how they end up outside. They feel that they are locked out of the process or that the process is not fit for purpose and cannot adapt to assess properly what the needs are for their loved one.

The drug approval process I have referred to works incredibly well in Scotland. It ensures that all those who are affected by decisions, such as approval for reimbursement, feel that their opinion and evidence, most especially the patient's, have been heard.

The current situation we have regarding orphan medicines is clearly not working. That is why Deputy Brassil has brought forward this Bill and why, I believe, there will be cross-party support for it. I commend this Bill which Sinn Féin will support. We hope that it is the start of a significant movement on this issue.

We all have listed off the cases. We know the names of the drugs. We usually know the first names of those who are campaigning because we meet them but we should not have to meet them and they should not have to feel that they have to come here. The truth is the pressure they apply often works. When they get the initial "No", sometimes when sufficient pressure is applied it works. That should not be the criterion. It should not be the case that the sickest people who travel the furthest and who stand outside the longest are the ones who can get the medicines. It has to be the case that there is a process that is fit for purpose, that does not always give every person exactly what he or she believes it should, that is based on clinical evidence and that involves those patients. I would urge the Minister to reconsider and join with us, Sinn Féin, and the other parties in ensuring that the Bill is passed.

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