Mary Butler (Waterford, Fianna Fail) | Oireachtas source

I thank the Minister of State for her comments and for acknowledging that today is International Day of Persons with Disabilities. I thank the Deputies who contributed on this discussion, in particular the Bill's sponsor, Deputy Pádraig O'Sullivan. I commend the goal of the Bill, which seeks to provide faster access for people to new medicines for the treatment of rare diseases.

I was particularly struck by Deputy Gould's comments. He was amazed that a backbencher could introduce a health Bill. Does that mean that Deputy Gould is not allowed to introduce health Bills? I compliment the two of his colleagues who are present - I am working with Deputy Colm Burke on a Bill on standards for home care and I am happy to work with Deputy Pádraig O'Sullivan on this Bill on orphan drugs. In government, we welcome all opinions. We all have individual minds, thoughts, strengths and ideas. I encourage anyone who wants to introduce a Bill. Members should not feel like they cannot. It is important that we hear from everyone across the House. That goes for all colleagues.

I have outlined some reforms of the existing assessment process carried out to date by the HSE and the National Centre for Pharmacoeconomics. I wish to take the opportunity to acknowledge their work in that regard. These reforms are ensuring that many expensive orphan drugs are getting reimbursed. I also wish to recognise the contribution made in recent years by the Joint Committee on Health. As Deputy Colm Burke mentioned, the committee has been considering this issue for the ten years he has been a member of it. It has held a number of productive discussions on the subject of rare diseases. Since the committee published its report in 2018, there has been substantial engagement between the Department of Health, the HSE and the National Centre for Pharmacoeconomics on each of the recommendations. In the previous Dáil, we all campaigned for the inclusion of Spinraza in the scheme on behalf of many kids who were moving into adulthood. We are all aware of how that drug has made such a difference to those families. It was an emotive issue at the time. There was only a small number of children involved, but it was great to see provision being made for the drug and those children being able to get on with their lives.

The substantial engagement I mentioned has resulted in a number of positive changes in the assessment process for orphan medicines. This includes the setting up of the rare diseases technology review committee, which facilitates patient and clinician input and gives these expensive medicines a fighting chance of getting through the system and becoming available to patients.

Irrespective of the costs involved, we must not forget the human element of this debate. We saw that with Spinraza and the effect it had on the children and their families. People with rare diseases are among our most vulnerable and they are often faced with a large level of unmet clinical need. Due to small patient populations, the development of new medicines for the treatment of rare diseases has historically been hampered by reduced commercial incentives. However, the pipeline of new medicines for rare diseases has become stronger in recent years thanks in part to the European Parliament's adoption of measures to encourage innovation in this area.

Today's debate offers an opportunity to progress our conversation around rare diseases. We must continue to question and improve our systems with a view to providing faster access to new innovations. Recent investment in research of treatments in this area is encouraging. However, more could be done to support access to treatments for rare diseases. The tabling of this Bill represents an opportunity to look comprehensively at the current system and consider how issues such as transparency and communication can be improved. I understand that the Minister has asked his officials to do that. In the circumstances, he does not propose to oppose the Bill.

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