Following 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.
At this meeting, 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.
On 5 November, the Committee met again and received input from patients with each of the above conditions.
A statement summarising the clinician and patient engagement was finalised and forwarded to the HSE Drugs Group during the week ending 27 November.
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
The applications for Burosumab and Patisiran remain under consideration and are being assessed in line with the Health (Pricing and Supply of Medical Goods) Act 2013.