Dáil Éireann Debate, Thursday - 5 November 2020
316. Deputy Louise O'Reilly asked the Minister for Health the outcome of the meeting of the rare diseases technology review committee in the week commencing 21 September 2020; the status of the assessment of the two medicines referred to the committee; and the specific dates which he expects the committee to meet in 2020 and 2021. [34429/20]
View answerFollowing a request from the HSE Drugs Group the HSE Rare Diseases Medicinal Products Technology Review Committee convened on 24 September to engage with patients and clinicians in the assessment of two drugs. These are:
1. Burosumab (Crysvita®) for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.
2. Patisiran (Onpattro®) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or 2 polyneuropathy.
The Committee reviewed the clinical data previously submitted as part of the established pricing and reimbursement process and also heard from Consultants involved in the specialist management of patients with these illnesses.
The Committee is scheduled to meet on 05 November and is arranging for patients with these conditions and/or their representatives to take part. The aim is to conclude discussion of these drugs and issue a formal response to the Drugs Group as soon as possible after this meeting.
The Rare Diseases Medicinal Products Technology Review Committee meets in response to requests for assessment of new products for rare diseases or expanded indications for existing products for rare diseases. The frequency of subsequent meetings will relate directly to the number of requests received.
