James Lawless (Kildare North, Fianna Fail)
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463. To ask the Minister for Health if a drug investigation or approval is in place for RTA 408 which may be an effective treatment for Friedreich's ataxia (details supplied); and if he will make a statement on the matter. [30501/19]

Simon Harris (Wicklow, Fine Gael)
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No medicine containing RTA 408 is authorised anywhere in the EU. RTA 408 (also known as omaveloxolone) is an investigational medicinal product currently undergoing clinical trials. The publicly accessible European clinical trials register lists two ongoing trials registered on the European Clinical Trials Database (EudraCT). Both are Phase II clinical trials, one of which is specifically investigating the safety, efficacy, and pharmacodynamics of RTA 408 in the treatment of Friedreich’s Ataxia.

Phase II trials test the efficacy of a drug and aim to find out:

- if the new treatment works well enough to be tested in a larger phase III trial

- which diseases the treatment works for

- more about side effects and how to manage them

- more about the best dose to use.

If phase II clinical trials prove successful, trials can advance to phase III.

A medicinal product cannot be placed on the market in Ireland unless it has a marketing authorisation granted by the Health Products Regulatory Authority or a centralised marketing authorisation issued by the European Commission. Following a successful application by the pharmaceutical company RTA 408 it has been designated as an orphan drug by the Commission. Therefore, a marketing authorisation application can only be submitted to the European Medicines Agency on behalf of the Commission.

For a medicinal product to be granted a marketing authorisation an applicant company must submit a dossier of all the trials and studies undertaken on the drug substance and the final pharmaceutical product, in order to provide evidence that the medicine adheres to clear and predefined standards of quality safety and efficacy relevant to its proposed therapeutic use. This dossier normally includes the results of phase III clinical trials.

The HSE has statutory responsibility for medicine pricing and reimbursement decisions, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies the criteria for decisions on the reimbursement of medicines.

In line with the 2013 Act and the national framework agreed with industry, if a company would like a medicine to be reimbursed by the HSE, the company must submit an application to the HSE to have the new medicine added to the reimbursement list.

Reimbursement is for licensed indications which have been granted a marketing authorisation.