Blog
RSS FeedPosted by Michael Wonder on 25 Mar 2025
Government of Canada signs bilateral agreement with Quebec for Drugs for Rare Diseases
21 March 2025 - In Canada, one in 12 people live with a rare disease, and for most people affected, the ...
Posted by Michael Wonder on 13 Mar 2025
First licensed treatment for ultra-rare immune disorder recommended
13 March 2025 - Leniolisib is the first ever treatment for activated phosphoinositide 3-kinase delta syndrome licensed for use in the ...
Posted by Michael Wonder on 03 Mar 2025
Canada’s Drug Agency publishes new guidance for rare disease registries in Canada
28 February 2025 - On Rare Disease Day — a global event dedicated to raising awareness for the millions of ...
Posted by Michael Wonder on 01 Feb 2025
High quality evidence for the treatment of very rare diseases: IQWiG is involved in the EU project RealiseD
29 January 2025 - Developing clinical studies for good evidence on very rare diseases in order to arrive at effective therapies ...
Posted by Michael Wonder on 28 Jan 2025
Drugs for rare diseases – Ontario agreement
24 January 2025 - Today, the Governments of Canada and the province of Ontario signed the National Strategy for Drugs ...
Posted by Michael Wonder on 06 Dec 2024
Jessica Lawrence’s meds cost her family $75k a year because they’re not listed on the PBS
6 November 2024 - Medications that are subsidised for cancer sufferers are critical to Jess Lawrence’s wellbeing, but because the 25-year-old ...
Posted by Michael Wonder on 06 Dec 2024
Government of Canada signs bilateral agreement with Alberta to improve access to drugs for rare diseases
5 December 2024 - Today, the Honourable Mark Holland, Canada's Minister of Health, and the Honourable Adriana LaGrange, Alberta's Minister ...
Posted by Michael Wonder on 05 Dec 2024
Results of health technology assessments of orphan drugs in Germany—lack of added benefit, evidence gaps, and persisting unmet medical needs
3 December 2024 - The number of orphan drug approvals has increased sharply in Europe. In Germany, all orphan drugs are ...
Posted by Michael Wonder on 24 Oct 2024
Canada’s Drug Agency announces recipients of funding to enhance rare disease registries
21 October 2024 - Canada’s Drug Agency is pleased to announce the successful applicants from our 2024 Request for Proposals (RFP) ...
Posted by Michael Wonder on 25 May 2024
Issues, challenges and opportunities for economic evaluations of orphan drugs in rare diseases: an umbrella review
25 May 2024 - There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. ...
Posted by Michael Wonder on 23 May 2024
Setmelanotide acetate for the treatment of patients with obesity and hyperphagia in Bardet-Biedl syndrome (final guidance)
22 May 2024 - NICE has published final evidence-based recommendations on the use of setmelanotide acetate (Imcivree) for treating obesity ...
Posted by Michael Wonder on 13 May 2024
PHARMAC shares the agenda items for the upcoming Rare Disorders Committee meeting (May 2024)
13 May 2024 - PHARMAC is sharing what medicine applications will be considered at the Rare Disorders Advisory Committee meeting ...
Posted by Michael Wonder on 29 Feb 2024
Collaborating to help improve access to drugs for rare diseases
29 February 2024 - On Rare Disease Day — a day when the international community raises awareness about rare conditions ...
Posted by Michael Wonder on 29 Feb 2024
Today is Rare Disease Day
29 February 2024 - PHARMAC understands that people living with rare disorders face many challenges, including access to health care ...
Posted by Michael Wonder on 18 Jan 2024
New orphan drug for a sub-type of acute myeloid leukaemia: G-BA sees significant additional benefit
18 January 2024 - Since July 2023, there has been one in Germany for patients who suffer from acute myeloid leukaemia ...