Funding of Orphan Drugs: Discussion

We are joined for this session by representatives of the Alpha One Foundation, an advocacy group for Respreeza, and Muscular Dystrophy Ireland, an advocacy group for Translarna, to discuss the subject of funding for orphan drugs. On behalf of the committee, I welcome Ms Geraldine Kelly and Professor Gerry McElvaney of the Alpha One Foundation and Ms Clair Kelly and Mr. Richard Lodge of Muscular Dystrophy Ireland.

I draw the witnesses' attention to the fact that by virtue of section 17(2)(l) of the Defamation Act 2009, they are protected by absolute privilege in respect of their evidence to the committee. However, if they are directed by the committee to cease giving evidence on a particular matter and they continue to do so, they are entitled thereafter only to qualified privilege in respect of their evidence. They are directed that only evidence connected with the subject matter of these proceedings is to be given and are asked to respect the parliamentary practice to the effect that, where possible, they should not criticise or make charges against any person or entity by name or in such a way as to make him, her or it identifiable. I also advise them that any submission or opening statement they have made to the committee may be published on the committee website after this meeting.

Members are reminded of the long-standing parliamentary practice to the effect that they should not comment on, criticise or make charges against a person outside of the Houses or an official either by name or in such a way as to make him or her identifiable.

The witnesses are all very welcome. I am sure they will have listened to the proceedings of the previous session with interest, as we all did. I thank them for being here with us. I ask Ms Kelly of the Alpha One Foundation to make her opening statement.

Ms Geraldine Kelly

Professor Gerry McElvaney and I thank members for having invited the Alpha One Foundation before the committee. The foundation is here to represent approximately 60 alpha-1 patients, some of whom are present today, who are affected by the preliminary decision of the NCPE and the HSE not to approve reimbursement of Respreeza. I acknowledge and thank these patients for attending today's meeting.

By way of background, alpha-1 antitrypsin deficiency is a genetic disorder that affects the lungs, liver and, in rare cases, the skin. Lung disease is the most common presentation, whereby people with alpha-1 may develop emphysema in their 40s and 50s, with or without a history of smoking. They experience frequent and severe chest infections that may require hospitalisation. They will often require the use of oxygen and may ultimately require a lung transplant as a result of irreversible deterioration in lung health.

Respreeza is a new, life-changing drug developed by CSL Behring which treats the underlying cause of emphysema, rather than the symptoms, in specific alpha-1 patients. Clinical trial results published in The Lancet in 2015 and The Lancet Respiratory Medicine in 2016 conclusively demonstrated a slowing down in the progression of emphysema by 34% in patients with alpha-1. Respreeza was approved by the European Medicines Agency in July 2015 and is now reimbursed in 12 European countries. A survey conducted by the Alpha One Foundation of patients receiving Respreeza found that chest infections dropped by 68% and hospitalisations by 69% per year and reported improvements in patients' ability to work and lead active lives.

The decision to approve or not to approve Respreeza will have a hugely significant impact on the lives of approximately 60 patients in Ireland who fit the prescribing criteria. An estimated 40 people with alpha-1 have never had access to Respreeza but would benefit from the therapy. Meanwhile, their health continues to decline, so it is imperative that this situation be resolved. Another 21 patients were involved in CSL Behring's clinical trial of the drug here and have been given continued access to the therapy by the company following the trial's conclusion in 2014. However, over the past year, these 21 patients have received repeated deadlines from the company advising them that the therapy will cease unless approved for reimbursement. The latest deadline of 31 July is fast approaching. These deadlines are causing huge distress and anxiety to patients, are totally unacceptable and should be withdrawn. The "Vision & Values" section of CSL Behring's website states: "We listen to and address the needs of people with life-threatening disorders and the professionals who serve them." We ask CSL Behring to fulfil this vision and listen to patients, listen to the Alpha One Foundation and listen to all committee members as this country's public representatives. We ask CSL Behring to stop issuing deadlines to patients who have given so much in their research endeavours.

Of course, this does not remove obligations on the NCPE, the HSE and the Department of Health to acknowledge the evidence that this therapy does work, as clearly understood and appreciated by the European Medicines Agency and our European neighbours. I ask the committee to remember that this is about not just the 21 patients already on the therapy but also approximately 40 others who continue to be deprived of its benefits. The NCPE, in making its original determination, did not consider a second study which further demonstrated the clinical effectiveness of the therapy nor did it take into account the results of the Alpha One Foundation patient survey. It is a cause of further distress for patients that there is a complete lack of transparency and communication with the approvals process. Since the first determination was made by the NCPE, patients have continued to fret and worry, with no formal notification from the HSE as to the current status. It was only when patients grew increasingly and publicly frustrated and upset that a meeting was quickly arranged with the HSE in February of this year. However, since then, little information has been forthcoming, only occasional updates gleaned from media statements.

Patients have pointed out to us how bizarre it is that the Government funds a national screening programme for alpha-1 but then fails to fund a new treatment that tackles it. Patients also highlight that there are savings to be made in keeping them healthy for longer, keeping them out of expensive hospitals and enabling them to contribute to our economy and our society.

These are important considerations.

It is obvious from our experience that the current approvals system is not fit for purpose and there is a clear need for appropriate patient engagement structures. We urge the implementation of the technical review group for orphan drugs as recommended in the national rare disease plan.

Finally, it is important to state that the Alpha One Foundation and the patients we represent have no interest in supporting "big pharma" but we do support big therapies - therapies that make life-changing differences to people's lives.

We urge all parties to put patients first and to agree a deal. Yes, taxpayers should get maximum value for money but our patients are entitled to a maximum quality of life too. Would the members here not agree?

I welcome the representative of Muscular Dystrophy Ireland, Mr. Lodge, and invite him to make his opening remarks.

Mr. Richard Lodge

My name is Richard Lodge and I am the chief executive officer of Muscular Dystrophy Ireland and I am here with our information specialist Ms Clair Kelly. I thank the committee for taking the time to hear our representations today. I speak on behalf of the 800 members of Muscular Dystrophy Ireland and the five children currently known to us who could benefit from the drug Translarna. I especially speak on behalf of the two children aged five and seven years who are currently losing valuable treatment time, children of parents who are forced to watch as a narrow window of opportunity closes permanently.

Duchenne muscular dystrophy is a debilitating condition resulting in the progressive weakening and wasting of the muscles. A child with Duchenne can expect to lose the ability to walk by the age of ten years, to develop cardiac and respiratory problems in their teens and will have an average life expectancy of 27 years. Translarna is the first ever treatment for Duchenne to receive European Medicines Agency, EMA, approval. European specialists believe it has the potential to change the course of the disease and improve the overall life expectancy of patients. Since 2014, more than 400 children in 22 European countries have been receiving this medication. This leaves Ireland as one of the last remaining countries yet to facilitate treatment. It appears as though our decision not to reimburse for Translarna is at odds with experts across Europe. Would this imply that the European Medicines Agency and the 22 other European countries have all got it wrong or it is the case that we are behind the curve?

Muscular Dystrophy Ireland has made several observations regarding the assessment of Translarna including an excessively long process, little apparent understanding of the progressive nature of the condition, no meaningful engagement with patient groups and no evidence of expert clinical input despite the clear unmet clinical need. There is also the decision not to include into the equation the compelling evidence from a phase 3 clinical study. Delays to date have included the failure to discuss Translarna at the most recent drugs group meeting despite it being an item on the agenda. It is now almost a year since the HSE leadership team stated it was eager to review Translarna again. One requirement of treatment is the ability to walk at least ten steps unaided. These delays in access will directly result in some of these children missing the opportunity for treatment as their condition deteriorates and they lose ambulation. This drug may not be cheap but it may be cheaper than the cost of caring for a non-ambulatory child for a year. Evidence indicates that the earlier the drug is prescribed, the more muscular integrity is retained. Under the UN Convention on the Rights of the Child, children have an explicit right to achieve their developmental potential and sustain the highest possible standards of health. Is there a danger that we are jeopardising that right? For the first time ever, we have treatments in development for Duchenne, spinal muscular atrophy, SMA, alpha-1, cystic fibrosis, cystinosis and a number of other rare diseases. Ireland does not appear to be ready for this and Irish patients are falling behind their European Counterparts. In the case of access to Translarna, Ireland is now three years behind France and Germany and a full year behind England, Scotland and Northern Ireland.

Our current system of assessment for rare diseases is not fit for purpose and we are concerned about the lack of development of strategies for the provision of high technology and orphan therapies. As it stands, no orphan drugs will get through the current health technology assessment, HTA, process and therefore no people with a rare disease will be treated until a solution is put in place. One such solution is the implementation of the recommendations of the national rare disease plan, approved by the Government in 2014. The HSE has committed to the development of a working group to bring forward appropriate decision criteria for the reimbursement of orphan medicines and technologies. The promised technical review committee for orphan drugs, while in progress, was without a chair until this morning. It may be that this meeting has prompted that appointment. It remains unresourced and has yet to meet.

We also need to consider other avenues for accessing orphan drugs, including fast track, managed access and managed risk programmes. We need an immediate fix for the current situation while the oil tanker that is the present process alters course. Our request as a patient organisation is for a timely conclusion of the review of Translarna and a fair and transparent process that will not leave rare diseases behind. We also ask for our children with muscular dystrophy to attain the same opportunities for health as their European counterparts, especially those in adjoining jurisdictions, without the need to move there. Currently, we are failing these children.

I thank Mr. Lodge. I call Deputy Durkan, followed by Deputy Brassil.

I thank the witnesses for giving their time to come before us this morning. The drugs require Government, ministerial and HSE approval and so on. On the drugs which were referred to, what do the witnesses envisage as the shortest, quickest route that would short-circuit that process for drugs in the future? Each time the question arises, we go through the same process we discussed earlier. Having direct knowledge, the witnesses are in a good position to tell us what we can do to short-circuit the delays and as a consequence, the patients' concerns. I will not refer to the drugs, but the witnesses will know them.

I welcome the witnesses and apologise to Mr. Lodge and Ms Clair Kelly. Had I known that people with Duchenne muscular dystrophy were being represented here this morning, I would have pursued the same line of questioning as I had done with Respreeza for alpha-1. I am a substitute on this committee for Deputy Kelleher and I had not seen that but I will try to follow up by email on the questioning on their behalf when the decision is being made.

It probably has been interesting for Professor McElvaney, Ms Geraldine Kelly, Ms Clair Kelly and Mr. Lodge to have been present and to have heard the discussion earlier. Those are the answers that we have been given. We asked thorough questions and received fairly comprehensive answers but I would be interested to hear from the witnesses, as representatives of advocacy groups, whether what members have been told is factual or partly factual and we might return to challenge some of the answers given to us. The issue of understaffing of the NCPE is highly significant. Its representative gave us the impression that the timeframe was quite tight but that does not seem to be the experience that I have had in dealing with groups such as those represented here. I have campaigned on the Respreeza issue since my election and hopefully we will have the answer we have sought in three or four weeks, and I hope for the same with Translarna.

Having worked with the groups here from the start, I am familiar with the issues. There can be no argument about the need to sanction these drugs as their clinical efficacy has been proven beyond doubt. The question is how do we get to the final step and have the costs reimbursed. A number of people with alpha-1 are on clinical trials. Are sufferers of muscular dystrophy on such trials or are they relying on data from other jurisdictions?

On companies' responsibility, it is welcome in one respect that companies opt for clinical trials and put patients on them. However, I am concerned that having proven the efficacy of a drug at a subsequent stage, a company could threaten its withdrawal. Either the companies should not start at all or if they start and the drug is proving to be effective, there is a moral responsibility on them to continue. Are there any examples of companies that have started clinical trials, proven efficacy and then stopped giving the drug to the patients because of not being reimbursed or is it just a stick they use to beat us with?

I have some questions and then I will come to Deputy Murphy O'Mahony and Senator Colm Burke. Ms Kelly spoke about deadlines. Could she provide an insight on behalf of the people she is here to represent of the kind of impacts those deadlines have on families and the individuals themselves? It must be horrendous for them. I have met people in my constituency who were affected and the manner in which some of the process is conducted is nothing short of cruel.

If the witnesses were to score the national rare disease plan in terms of strategy could they say it is good? Could they also score it on implementation? I would be interested in seeing if the scores are different. Reference was made to savings and the cost to the taxpayer of a non-ambulatory child versus the cost of the drug. When we questioned the HSE and the NCPE earlier, they were very clear that they took absolutely everything into account in terms of any potential savings. Could the witnesses give us their perspective on whether absolutely every saving is in fact taken into account?

Mr. Lodge spoke about the process and the need for fairness and transparency. If we were to redesign the process we would design a process whereby the people who needed the drugs got the drugs but life is not that simple. In terms of the current process and the constraints under which we are operating, do the witnesses have any suggestions about how that can be improved specifically to inject some fairness and transparency?

My final question is also for Mr. Lodge. He spoke about the drugs group meeting recently and that Translarna was on the agenda but was not discussed. Could he give us his view as to why that happened? I will leave it there. We will take the questions in a batch. I ask the witnesses to respond and we will come back then to the other members who are indicating.

Mr. Richard Lodge

I am happy to kick off on a couple of those questions. Deputy Durkan asked if there is a better way. We heard this morning that there has to be a better way and there has to be a better process.

One of the issues is the recommendations in 2014 for the promised technical review committee for orphan drugs. That touches on the questions from the Vice Chairman also. I think it is three years since that was proposed and nothing has yet happened. That is not strictly true because a chair has been appointed. To address what we could do better, we could implement the recommendations. It is as simple as that. To answer the question from the Vice Chairman about how I would score the strategy, it would score quite highly. It is definitely the way to go. It is definitely what needs to happen. Scoring it on implementation is easy. It is zero, because three years later we are not aware of anything that happened. We were not aware until this morning that a chair had been appointed.

I have some experience with the process for oncology. There is a lot that can be learnt and borrowed from the oncology systems. My biggest concern about the current process relates to the delays. I will ask Ms Kelly to update the committee on some of the delays regarding Translarna in a moment but I must say that communication is a serious concern. There is a simple reason for that. At present, the drug companies put in applications to have their drugs licensed. Therefore, the communication is between those committees and back to the drug companies. We are not aware of the mechanisms by means of which patient input or expert clinical opinion is fed into that process. Unfortunately, we are in a situation whereby patients are aware of the trials. They are aware of the drugs that the companies are seeking to be licensed, so they approach the drug companies directly. The information the patients receive comes via the drug companies, from the HSE, which is not ideal because they will have a completely different agenda and the information we receive as patient representatives comes then from the patients who met with the drug companies. There has got to be a way of short-circuiting that route and going direct.

I do not want to put words into anyone's mouth but it was less than an hour ago so I think I remember what was said. The HSE seemed to be indicating that it does listen to patients and that patients and advocacy groups have a mechanism for input.

Professor Gerry McElvaney

Unfortunately, the HSE does not listen too much to patient groups. One of the things I really appreciate in being here today is to hear from people. I echo what Deputy Brassil said. It was an education for us to see democracy in action. Bureaucracy is being held accountable by our public representatives. It is obvious that things are being said here today that we had not been told. It was out of the blue for us to hear about a decision on Respreeza taking three to four weeks. We have been asking for a timeline for the past three to four months. The Vice Chairman's about getting a house renovated hit the nail on the head.

I was not trying to compare the two.

Professor Gerry McElvaney

How long is a piece of string? We were asking for a long time. Our patients are on a one month by one month timeline. We were asking a very simple question, namely, when will we know and we did not get a single answer to that but we have got it today. If we accomplish nothing else today, at least we have accomplished that.

I will turn to Deputy Durkan's question about short-circuiting the route. The drugs have been approved by the EMA and the US Food and Drug Administration, FDA. Why are we seeking to approve them again? That does not make any sense whatsoever. Our role should be to find out if we can get a good price for drugs that work. That is the only question. The way to get a good price is to go in with a European group and use our strength in numbers. We should not go in as a country of 4.5 million, but as a big bloc of 500 million and tell them this is what we need. It is almost intuitive that that is what we should do but we are not doing it. I do not think we got the answers on that today. We got a lot of obfuscation but no real answers to the questions. To summarise, we should not be reinventing the wheel. The drugs have been shown to act well. The next question is whether they are worth the price and if we can get the price down. That is where we should focus.

Ms Geraldine Kelly

I would like to make a point on that. In the context of the NCPE seeking additional resources, there is talk about an increase of nine staff. Those nine people will do work that is already being done elsewhere so it does not make sense. We should put the funding somewhere else, into something that is going to help the patients that are suffering from muscular dystrophy or alpha-1. There is a total lack of transparency on where their money is being spent by both the NCPE and the HSE. It is not obvious to any of us where the money is going. I think that is critical in all of this. Transparency is not there and that must be looked at across this whole process.

Ms Clair Kelly

I will refer to Deputy Durkan's question about the quickest route. We heard much today about Europe and what we can do there. Ironically, 80% of children in Europe are receiving this treatment, so we are one of the last to facilitate it. I can give an example of some of the mechanisms and programmes that are out there around Europe. In England and Scotland, they have individual funding requests for treatment and these can be applied for while the assessment process is ongoing. In Italy, they have fast-track mechanisms and in France they have authorised temporary use in the case of a condition that is life threatening or where there is no therapeutic alternative. That is the case for Duchenne muscular dystrophy. In Sweden, county councils can individually reimburse a treatment cost, which is quite different from what we do here. In Germany, there is automatic reimbursement. There are many examples of the different ways and as Professor McElvaney has stated, we do not need to reinvent the wheel. These programmes are out there already and we need to look at what fits for us.

Ms Geraldine Kelly

A question was asked about quality of life. Being faced continuously with a deadline on a drug that is allowing a person to live - what we around the table appreciate as normal daily living - is heartbreaking for these patients. It affects their family lives and it affects them financially. There is the continuous threat of ending up in hospital with a chest infection or the possibility of a lung transplant. It is not something any of us want to face. The threat over the alpha-1 patients is obvious, as it is with other patients suffering from rare diseases who require orphan drugs to help them maintain their lives.

The Health Service Executive or the National Centre for Pharmacoeconomics, NCPE, are looking at the effectiveness of a drug and they are concentrating on the cost effectiveness of a drug than whether it is effective for patients. That has already been proven;12 countries in Europe have approved this and are readily handing it out to patients, and we have already proven that it works in Ireland for 21 patients. We have got 40 more and probably ten on top of that waiting. It is a given that this needs to change.

I just have observations really. I thank the witnesses for coming here today. As I said to the previous groups, it is very important to note that behind every funding application, there is a life and families, etc., connected to that life. The witnesses know this more than most people. I am very glad the delegations are here this morning to see what happens and perhaps it is something we should do in future. Rather than us telling them what happened, it was great they could witness it.

I thank the witnesses for the work they do in their individual areas. I know they are extremely committed and dedicated to the projects in which they are involved and helping patients who have these difficult medical conditions. For the record, I will correct a comment from earlier this morning. My figures were correct. My understanding is that of the 148 orphan drugs, 133 have been approved in Germany, 68 have been approved in England and only 53 are currently reimbursed in Ireland. The HSE indicated my figures were exaggerated but they are not. These are the actual figures from a report I got. There are 53 reimbursed in Ireland, compared with 116 in France, 133 in Germany, 84 in Italy and 75 in Spain. Those are the figures. The other matter is the timescale. My understanding of orphan medication is that from the date of the initial application to the final HSE decision, it is an average of 23.6 months. They are the figures I got from a report I sourced. In the period 2012 to 2017, some 15 of the 45 applications made have been successful.

Professor Gerry McElvaney

It is a really important point. From our studies we know that when a person gets emphysema and loses lung tissue, he or she never gets it back. We have done the studies over two years. If a person is waiting two years to see if a drug is to be approved, he or she will have irretrievably lost lung tissue.

The figure I have is 23.6 months.

We could round it up to two years.

Mr. Richard Lodge

It would be exactly the same with the children with muscular dystrophy and Translarna. It is about the time.

I would imagine it is the same for a range of conditions.

Mr. Richard Lodge

Once they lose the ability to walk, they no longer fit the criteria for the original drugs trial.

I have a question on the work being done and contacts at European level. I presume there are equivalent organisations supporting patients with the same medical conditions at a European level so has there been any contact with them about how they feel about joined-up thinking between countries to expedite the process? I go back to the German example. We are talking about being tied with smaller countries in trying to do deals with pharmaceutical companies. My view is we should be tied to bigger countries as we would be in a far better position to negotiate. That is the big concern I have. As I stated earlier, if there is a drug to help 20 people here, in a country the size of the UK or Germany, a far bigger number would be involved. The pharmaceutical companies cannot afford not to do a deal over there. Have the witnesses spoken with comparable organisations in other countries? There is also the medical connection and there is much contact between medical people involved in these areas. That happens in Ireland, the UK and Germany, for example. Has there been a sharing of information or working together to try to bring about the change being sought?

Professor Gerry McElvaney

Absolutely. We can look at it in two ways, both in the US and the EU. The United States is different from Europe but there the patient organisation has a major role in acting as an intermediary between drug companies and patients. The drug is distributed by the patient organisation, which ensures a good price for the patient. It is a really good example of how patient organisations can take control.

In a European context, it is sad for us that the pivotal study that other European countries used to get the drug approved was done mainly in this country. We had the largest number of patients of any country in Europe on that trial. Our patients ended up not getting the drug but organisations in other countries used the trial to get drugs for their patients. We are perceived as being a very strong alpha-1 community in a European context. We have spoken with colleagues but the problem is 12 of them have already bolted the cage as they have the drug. That includes Greece and similar countries that we might see as being in a worse economic position than ourselves. They have the drug and we do not.

The Senator is absolutely right and we should make common cause with European neighbours. We would be doing so at a remove and as a poor relation for the moment. They have the drug and we do not. It is a major cause of concern for us.

Deputy Durkan and others indicated that it does seem we speak much about co-operation at a European level but on this issue there seems to be none. The witnesses were here when we questioned the HSE and I did so myself. We asked about European standards and they just do not seem to exist. It strikes me that it is an area on which we should put a heavy focus.

Professor Gerry McElvaney

I do not want to dwell too much on it. From a diagnostic perspective, Ireland is perceived as one of the European leaders and we teach other European countries how to diagnose the issue. We are working closely with European colleagues but our colleagues in the HSE do not seem to see this as an option. I would see it as a suitable option.

It should be the first port of call.

Ms Clair Kelly

I will go back to Deputy Brassill's question, which we did not answer. The NCPE process was probably one of the most transparent elements as it was published. There is a 90-day timeline, albeit with a stop-the-clock element as well.

They have that period within which they have to report back. The problem arises for us when the recommendation leaves the NCPE. There are then no timeframes and there is very little transparency. There is also no direct communication with the HSE. That is the stage at which we come up against most of the problems. In the case of the NCPE, we do not know what actual studies were reviewed. While the NICE in the United Kingdom has a list of every document it has received, the NCPE does not. There is no record of an entire study and it is the same with Respreeza. We do not know where the study went or if and when it was assessed.

As regards the children and clinical trials, we did not have a clinical trial site in Ireland for this treatment. The children have been at a double disadvantage in that they have not had an opportunity to take part in a clinical trial and, when trials do come through, they have to waiting longer. It is 22 months since the beginning of the NCPE process and three years since the EMA approved the drug. That is a long time in anybody's life but particularly so in the case of a five year-old child.

Mr. Richard Lodge

Let me pick up on another question. We were asked what drugs we were aware of coming through the system. Ms Kelly has just touched on one of the problems in treating rare diseases, namely, that Ireland is small and that as a result the numbers are very small. Until recently we did not have a clinical trial site, although we do now. Our very real concern is that unless we can change the current system to make it easier for the drug companies to operate trials in Ireland and have their drugs passed and licensed and because we are dealing with such small numbers of orphan drugs and rare diseases, we may get to a stage at which a drug company may simply not bother with Ireland or may not be inclined to invest in research here to the same level. Many of the drugs trials about which we are talking simply do not happen Ireland. There may be a raft of reasons behind this.

The other point the Vice Chairman made was about the cost of caring for patients. A study in 2014 put the cost of caring 24/7 for a non-ambulatory patient at around $130,000 per year. The question was how confident were we that they had taken all costs into account. We have never been told that Translarna failed on a cost basis. However, we have no idea what costs were included. We would like to think and have to assume all costs were included. However, because there are such gaps in the communication process, we can only assume that they were.

That issue is central to both cases. When we put a straight-up question to the HSE such as does it consider everything, it answers yes. I have met numerous advocacy groups, as we all have, and the point keeps being made that if it did actually look at the real savings involved, the conclusion could not be anything other than that the drug is cost effective. There is obviously some slippage and if we have done nothing else today, we have managed to isolate the fact that a lot of work needs to be done on the process of communication between patients, their advocates, the HSE and the NCPE. The impression was created - I will say no more than that - that there was massive communication, but there does not seem to have been that much. The committee will certainly take on board the idea that more needs to be done in increasing patient involvement and communication.

Professor Gerry McElvaney

I did not answer one question about companies discontinuing drugs. It does happen. When conducting Alpha-1 trials in Ireland, we insist on a patient continuing on the drug until a decision is made on reimbursement. That is the very least companies should do. The problem for us is that the issue of reimbursement drags on so long.

I would like to ask the professor, in particular, about the reason we have made so little progress in reducing the overall cost of drugs. If we were to reduce the overall cost - I am talking about straightforward medication - we would obviously have a lot more money available for the purchase of orphan drugs. However, we seem to be purchasing a lot more drugs per head of population. Does Professor McElvaney think we need to do some work on that issue to get a message out to the general public?

Professor Gerry McElvaney

I do not think so. There are ways to get around it. Purchasing for bigger numbers leads to decreased costs. We have to control who prescribes drugs. We have to make sure those who prescribe know what they are doing and that it is being kept within a well demarcated area. There also has to be a review of a drug further down the line to see if it does what has been said it does. If not, we need to be prepared to pull it. There are a few things we have to do. We have to stake a claim that, while we want such drugs, we want them to be effective and that we will take all measures necessary to show that they are effective.

On behalf of the committee, I thank Professor McElvaney, Mr. Lodge and Ms Kelly for coming before us and putting their case so eloquently. I am glad that they had the opportunity to listen to the HSE and the NCPE. It is helpful for those who are advocates who are welcome to attend public sessions of the committee at any time to see that when representatives of the HSE and NCPE come before us, we do attempt to hold them to account and ask the important questions.

The joint committee adjourned at 12.20 p.m. until 11 a.m. on Wednesday, 20 September 2017.