Dr. Lorraine Nolan:

I thank the Deputy for the question, which is obviously a pertinent one in the context of the role we have. He is absolutely right that these MCAP products are not medicines in the conventional sense in which he and I would understand that word. The extent of the data behind these products is limited and I have been clear in that regard. We are, however, satisfied. What is here is in line with the recommendations we made in 2017. This is a very limited form of access for those three specified conditions. The cascade and progression to treatment under MCAP are important. These patients must have exhausted all of the available options so that when they are granted access to MCAP, there is a clear unmet need for those patients. In that context, there are different considerations regarding benefit and risk. We are satisfied with the programme.

There is a little bit of overlap between the benefit and risk at the individual patient level and the control of the system. In line with the clinical guidance to which I have referred, every time a patient is put onto the MCAP programme, it must be initiated by a consultant. The enrolment process has been outlined. A treatment programme must then be put into the medical record of each patient. That sets out the background to the decision to introduce the patient onto the programme and includes the product and dosage that are going to be used. It is important that there is an agreement on what the expected outcome should be in terms of improvement. Actual measurements are put in place. There is a monitoring plan for the patient. Patients are monitored at regular intervals to ensure that those improvements are being realised.

The overriding principle is that anybody who starts on one of these products starts low and goes slow. The dosage starts at the lowest level and is worked upwards. If the measurements I have spoken about are not met, there is also a work-down. The treatment can be stopped. It is an individualised programme that is supervised at an appropriate medical level, which is part of the control system. I do not want to overlap with what Mr. O'Connor already said. These substances are controlled under the misuse of drugs legislation and there is an additional tracking system. For example, if a wholesaler is bringing in stocks that must be held for onward supply to patients, a separate licensing system tracks them. A reconciliation process is done on the amounts that come in, are used and are in stock. The HPRA has a role in monitoring that as well. There are many levels of control across the system to prevent leakage. From the benefit-risk point of view, it comes down to an individual patient decision, closely supervised by a doctor.