Michael Harty (Clare, Independent) | Oireachtas source

I thank Deputy Brassil for introducing this Bill and applying to it his expertise in this area. It involves an amendment to the Health (Pricing and Supply of Medical Goods) Act 2013. Deputy Brassil is proposing to establish specific criteria that would be applicable to orphan medical products in regard to the HSE making decisions on reimbursement and prescription by doctors.

The health committee has had several meetings on orphan drugs, most recently last month. One of the recommendations of the committee was that there should be a review of the legislation. Recommendation No. 2 states: "The Committee recommends that a review of the 2013 Act be commenced by the Department of Health, to present a report to the Minister for Health within 6 months of the publication of this report, identifying potential legislative barriers to the reimbursement of orphan drugs and corresponding legislative amendments, as appropriate." It was identified by witnesses who appeared before the committee that there was a legislative barrier to the consideration of orphan drugs. It was stated by representatives from the HSE and the Department that the 2013 legislation limited the examination of orphan drugs. It was stated that since it did not specify specific criteria for orphan drugs, the examination of orphan drugs was limited. It was said that if there were to be change, there would have to be a change to the legislation. That was one of the recommendations of the committee.

When the representatives of the HSE and Department were before the committee last month, however, the representatives of the Department said the Minister was satisfied there is no legal barrier to the assessment of orphan drugs because there is sufficient scope for the HSE to have regard to the special circumstances that apply to them. The legislation was introduced in 2013. It is now almost six years later and the issue of orphan drugs is far more complex than the legislation envisaged. That is why Deputy Brassil's Bill is timely. It is introducing new, specific criteria to apply to orphan drugs. The Minister has determined and said to the committee that he sees no legal barriers but there are legal barriers because orphan drugs are different from regular drugs.

The committee made 15 recommendations. I will not go through them all. There was a recommendation that specific evaluation of orphan drugs take into account that the drugs are for rare and very rare diseases, with perhaps 5,000 and 10,000 people affected.

The numbers in Ireland are small and that is an issue for patients and their advocacy groups. There was a recommendation that because of the small numbers we should look internationally at the evaluation process for orphan drugs in Europe and tap into it and avail of the qualitative information available throughout Europe. As the numbers in Ireland are small, we have to have a collaborative approach throughout Europe and Ireland has started to engage with other European countries on health technology assessment and, perhaps, on negotiating a price from the pharmaceutical companies. Orphan drugs are confined by existing legislation and what Deputy Brassil has proposed is that the quality assessment threshold of 45,000 should not apply to orphan drugs because they are a unique niche-type of drug that apply to a very small number of people but have a huge effect on them if they are seen to be effective. The assessment process needs to be more transparent. The companies, patients and support groups feel they are left out of the assessment process. There is also a recommendation to appoint an independent expert to review the assessment process. I do not believe this has been achieved yet.

The proof of the pudding is that the number of orphan drugs available in Ireland is low compared to other European countries. This points to a deficiency in the assessment process. Ireland has approved only 22 orphan drugs whereas many European countries have approved two to three times this number. We must bring it back to the patient because there is a detachment between the assessment process and the patient. There are opportunities to have managed access programmes to negotiate a price only for those who respond to treatment, because not everybody responds to treatment, and to pay only for the responders.

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