John Brassil (Kerry, Fianna Fail) | Oireachtas source

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?