Mr. John Hennessy:

I thank the committee for its invitation to attend again on the subject of orphan drugs. If I understood the Chairman's letter correctly, today's focus is on the process for evaluating orphan drugs and the potential reasons for delays in reimbursing approved drugs. The process for evaluating applications was covered at some length at the July committee meeting, so I will not repeat the full detail other than to re-emphasise that the criteria for assessment are those outlined in the legislation, namely, section 19(4) of the Health (Pricing and Supply of Medical Goods) Act 2013. I have the criteria with me and can go through them later if members wish.

The important point to bear in mind is that the legislation requires the HSE to have regard to the cost effectiveness and clinical benefits of the product being applied for and does not make specific provision or permit a different rule set to be applied in the case of orphan drugs. Nevertheless, particular features that relate to orphan conditions are considered during the assessment process and factors such as the clinical effectiveness of the product and the number of potential patients affected are clearly set out in the reports compiled. Professor Barry will allude further to this matter in his opening statement.

In the case of approved medicines, the reasons for delay can be many, but they are mainly logistical - putting in place the conditions that may be attached to an approval - or they are financial, for instance, in the case of products that confer clinical benefits and are deemed to be cost effect but budgetary provision for whose approval does not exist in the service plan. As members will recall from our previous meeting, a delay between approval and reimbursement occurred in 2017 in the case of products for the treatment of cystic fibrosis, cancer, heart failure and a number of other conditions.

Delay can also occur where the clinical benefits demonstrated by the trial studies are marginal but the prices charged are substantial. Everyone will be familiar with examples of products being referred back for further negotiations on price. Companies are encouraged to submit their initial applications based on the end price, but experience has shown that further price adjustments can, and do, become available after decisions are taken and notified.

I trust that this information is of assistance. My colleagues and I will try to answer whatever questions the committee may have or provide any further information that may be required.