Frank Feighan (Sligo-Leitrim, Fine Gael) | Oireachtas source

I thank Senator O'Loughlin for raising this important issue and for giving me the opportunity, on behalf of the Minister, Deputy Stephen Donnelly, to speak on the reimbursement of patisiran for hATTR amyloidosis. I fully understand that access to potentially beneficial drug treatments is an extremely important issue for people with this condition.

As the Senator is aware, the HSE has statutory responsibility for medicine pricing and reimbursement decisions under the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies certain criteria for decisions on the reimbursement of medicines which include unmet need, clinical evidence, the resources available to the HSE when making reimbursement decisions and budget impact. HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds, on the advice of the NCPE. It uses a decision framework to systematically assess whether a drug is cost effective as a health intervention.

The Minister is advised by the HSE that it received an application in December 2018 for the reimbursement of patisiran for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. In January 2019, a full health technology assessment, HTA, was commissioned by the HSE. In February 2020, following the HTA, the NCPE recommended that patisiran not be considered for reimbursement unless cost effectiveness could be improved relative to existing treatments.

In July 2020, the HTA report, the patient group submission received during the HTA process, and the outputs of commercial negotiations were reviewed by the HSE drugs group. The drugs group was unable to recommend in favour of patisiran at that time. The drugs group therefore sought additional patient and clinician engagement input via the rare diseases technology review committee, RDTRC, to assist the group in making its recommendation to the HSE executive management team.

In September 2020, the rare diseases technology review committee reviewed the clinical data previously submitted as part of the established pricing and reimbursement process and heard from consultants involved in the specialist management of patients with this illness. In November 2020, the committee convened again and continued its review of patisiran, which included patient input on the impact of this illness. Following these meetings, the committee finalised a statement summarising the clinician and patient engagement on patisiran, which was forwarded to the HSE drugs group for consideration.

The HSE has advised that patisiran currently sits as the third item for consideration on the January drugs group draft agenda. The statement from the rare diseases technology review committee will be considered by the drugs group along with the health technology assessment and the outcomes from commercial negotiations.

As the Senator will be aware, additional funding of €50 million was provided for new medicines in budget 2021. This funding will allow the HSE to approve medicines which have been recommended by the HSE drugs group on the basis of efficacy and value for money in line with the 2013 Health Act. The application for patisiran remains under consideration with the HSE and the reimbursement of this medicine will be considered in this context.