Blog
RSS FeedPosted by Michael Wonder on 05 Mar 2023
Our progress in the funding of medicines for rare disorders
3 March 2023 - PHARMAC will hear from people affected by rare disorders in its upcoming Rare Disorders Advisory Committee meeting ...
Posted by Michael Wonder on 02 Mar 2023
Asfotase alfa for the treatment of certain patients with paediatric-onset hypophosphatasia
1 March 2023 - This evaluation reviews the evidence for asfotase alfa for the treatment of patients with paediatric‑onset hypophosphatasia (HST6), ...
Posted by Michael Wonder on 27 Feb 2023
PHARMAC shares the agenda items for the upcoming Rare Disorders Committee meeting
27 February 2023 - PHARMAC is creating visibility of what medicines are being considered that could impact the rare disorders ...
Posted by Michael Wonder on 06 Nov 2022
Kiwis with rare disorders want faster progress on 'life or death' health strategy
6 November 2022 - For Samantha Lenik, being diagnosed with Pompe disease after seven years of symptoms was a “double-edged ...
Posted by Michael Wonder on 09 Sep 2022
Rare cancer sufferers fight to get drugs on PBS
9 September 2022 - People with rare cancers are being forced to sell their homes, raid their superannuation and fundraise ...
Posted by Michael Wonder on 04 Sep 2022
Australia among world's best in treating debilitating and often fatal muscle disorder
4 September - Australia has become one of the first countries in the world to fund a new, next-generation treatment ...
Posted by Michael Wonder on 18 Jul 2022
Velmanase alfa for the treatment of alfa mannosidosis
15 July 2022 - The Department of Health and Social Care has asked NICE to produce guidance on using velmanase alfa ...
Posted by Michael Wonder on 06 Jul 2022
Setmelanotide for the treatment of patients with obesity caused by LEPR or POMC deficiency
6 July 2022 - NICE has issued fvidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC ...
Posted by Michael Wonder on 01 Jun 2022
Cancer, rare disorder advocates call for more radical change after damning PHARMAC review
2 June 2022 - Radical action is needed urgently to tackle the delays patients face in getting the latest drugs for ...
Posted by Michael Wonder on 05 May 2022
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
5 May 2022 - NICE has published evidence based recommendations on the use of selumetinib (Koselugo) for the treatment of children ...
Posted by Michael Wonder on 21 Apr 2022
Ontario family forced to fight for life-saving drug highlights need for national rare disease strategy
21 April 2022 - Beth Vanstone doesn’t want anyone else to have to fight for access to life saving medications. ...
Posted by Michael Wonder on 31 Mar 2022
Statement from Rare Disorders NZ re Minister Little's comments on PHARMAC funding
31 March 2022 - Rare Disorders NZ is extremely frustrated to hear the Minister of Health this week dismiss the call ...
Posted by Michael Wonder on 01 Mar 2022
ABPI response to England Rare Diseases Action Plan
28 February 2022 - England's Rare Disease Action plan is published today with details of how to improve diagnosis, care and ...
Posted by Michael Wonder on 22 Feb 2022
NICE recommends Albireo’s Bylvay (odevixibat) for all PFIC types
22 February 2022 - Positive NICE review completed in less than six months post MHRA approval. ...
Posted by Michael Wonder on 13 Jan 2022
Orphan drugs: privilege of "fictitious" additional benefit not justified
12 January 2022 - Drugs for orphan diseases should also go through a regular benefit assessment procedure when they enter the ...