Rose Conway Walsh (Sinn Fein) | Oireachtas source

I again wish to raise the matter of drugs that are used to treat rare diseases. There are two main issues here and the first is the lack of transparency within the current system.There is no way of finding out what cost-benefit analysis model is used for these drugs and what negative or positive externalities are used to make an assessment on whether the drugs will be approved. This process must become more transparent. The protectionism in the system stopping this from coming to the fore must be tackled. The only person who can tackle it is the Minister. I know it is the HSE's statutory obligation but the Minister is allowing it to have that protectionism and lack of transparency. Where we have a lack of transparency we have a lack of trust. The Cathaoirleach knows the two main drugs I have been following for months are Translarna and Spinraza. Time is of the essence in these situations for children to have the optimum benefit from the drugs in the case of Duchenne muscular dystrophy. Translarna is effective only between certain ages. Children admitted to intensive care units in hospital do not have the time to wait for Spinraza. They do not have the time for the obscure processes to work their way through the system.

At European level, the Benelux countries have approved them but we need to see the benefit of being part of the EU and we need to know when this will become apparent to the people who are most impacted by the lack of transparency and decision-making. If a medicine is approved by the European Medicines Agency in terms of it being effective and safe surely we do not have to make the same judgment again. We need to stop reinventing the wheel in these situations. We also need to have greater clinician involvement. This country has some of the most fantastic clinicians and we need to take their advice and guidance on board with regard to the assessment of these drugs. We also need to have patient advocacy. It is hugely important in terms of patient input to the decision-making process that the patients are not chosen by the agency. They need to be from a range of backgrounds and put forward in ways other than being selected by the HSE. I would like a debate in the House on the issue.