Catherine Byrne (Dublin South Central, Fine Gael) | Oireachtas source

On behalf of the Minister, Deputy Simon Harris, I thank the Senators for raising the matter. Ataluren, also known by the brand name Translarna, is indicated for the treatment of Duchenne muscular dystrophy, DMD, a genetic disorder characterised by progressive muscle degeneration starting in early childhood. I understand that access to potentially beneficial drug treatments is an important issue for people with DMD. The Minister, Deputy Harris, appreciates that DMD sufferers and their families face enormous day-to-day challenges in dealing with this condition.

The Health (Pricing and Supply of Medical Goods) Act 2013 gives full statutory powers to the HSE to assess and decide on the reimbursement of medicines, taking account of a range of objective factors and expert opinion, as appropriate. The Act specifies criteria for reimbursement decisions. These include the clinical and cost effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE.

Ataluren is a high cost medicine manufactured by PTC Therapeutics for the treatment of a subgroup of patients with DMD, a rare disease. The medicine has conditional market authorisation for Europe from the European Medicines Agency, the condition being that the company carries out further clinical trials to determine the clinical efficacy of the drug. Applications for reimbursement were considered by the HSE drugs committee and leadership team in 2016 and 2017. The applications were considered carefully. However, the final decision was that the HSE was not in a position to fund the medicine on the basis of the current clinical evidence of effectiveness and the prices being charged by the pharmaceutical company. The HSE informed the applicant company of this decision, in keeping with the requirements of the 2013 Act. The applicant appealed the HSE's decision to the High Court, which is part of the statutory process under section 27 of the 2013 Act.

On 6 July 2018, the Department was informed by the HSE that a settlement of the High Court action had been reached. The terms of settlement stipulate that in the event that the manufacturer submits a new application to add ataluren to the reimbursement list, it would be reviewed by the HSE in a timely fashion having regard to the relevant statutory framework.

The Minister for Health is very much aware that this protracted process is upsetting to the patients and their families who are affected by this condition. As of yesterday, 11 July, the manufacturer has not submitted an application for reimbursement of Translarna. I call on the company to come to back to the table with a fair offer so that patients may access this treatment as soon as possible.