Blog
RSS FeedPosted by Michael Wonder on 16 Feb 2020
After a new version of a decades-old drug gets orphan status, the price suddenly skyrockets
12 February 2020 - For years, hospitals and clinics have used an injectable medicine called dehydrated alcohol to treat such ...
Posted by Michael Wonder on 11 Feb 2020
SMC approves first two ‘ultra-orphan’ pathway meds
11 February 2020 - The Scottish Medicines Consortium has released its February guidance, advising six new medicines to NHS Scotland. ...
Posted by Michael Wonder on 11 Feb 2020
SMC - February 2020 decisions
10 February 2020 - The Scottish Medicines Consortium, which advises on newly licensed medicines for use by NHSScotland, has today ...
Posted by Michael Wonder on 30 Jan 2020
ProQR receives rare paediatric disease designation from FDA for QR-421a
30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...
Posted by Michael Wonder on 21 Jan 2020
Mum says 'miracle' drug saved ill baby Kaan Hunter's life
21 January 2020 - A baby who was given only four months to live after being diagnosed with an extremely ...
Posted by Michael Wonder on 19 Jan 2020
FDA harnesses technology and collaboration to support rare disease product development
17 January 2020 - Announcing an orphan drug technology modernisation effort and the 2020 FDA Rare Disease Day meeting. ...
Posted by Michael Wonder on 16 Jan 2020
Asfotase alfa in hypophosphatasia in children and adolescents: survival advantage for small children
15 January 2020 - Weak evidence does not allow a specific statement to be made about the extent of the added ...
Posted by Michael Wonder on 02 Jan 2020
Does an orphan drug policy make a difference in access? A comparison of Canada and Australia.
2 January 2020 - Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, ...
Posted by Michael Wonder on 11 Dec 2019
Vertex announces European Commission approval for Kalydeco (ivacaftor) in infants with cystic fibrosis ages 6 months to less than 12 months with certain mutations in the CFTR gene
10 December 2019 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...
Posted by Michael Wonder on 27 Nov 2019
Our drug policy often prioritises eliminating rare disease. One researcher asks: Is that the right goal?
26 November 2019 - Dr. Peter Bach wants to turn the entire philosophical underpinnings of America’s health care system upside ...
Posted by Michael Wonder on 18 Nov 2019
Funding orphan medicinal products beyond price: sustaining an ecosystem
18 November 2019 - Following a similar legislation in the USA, the EU passed a legislation in 1999 to incentivise ...
Posted by Michael Wonder on 12 Nov 2019
Rare Disease Fund now covers Pompe disease, a rare inherited neuromuscular disorder
3 November 2019 - The Rare Disease Fund now covers Singaporeans with Pompe disease - a rare inherited neuromuscular disorder ...
Posted by Michael Wonder on 11 Nov 2019
FDA admits it goofed when granting orphan status to an opioid addiction treatment
8 November 2019 - In an unusual move, the FDA has acknowledged a mistake and revoked orphan drug status for ...
Posted by Michael Wonder on 01 Nov 2019
Vertex announces EMA marketing authorisation application validation for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination treatment in cystic fibrosis
31 October 2019 - Application supported by positive results from two global Phase 3 studies in people with cystic fibrosis ages ...
Posted by Michael Wonder on 29 Oct 2019
Battle for life-saving drugs now harder as PHARMAC absorbs $5m rare disorders fund
29 October 2019 - A $5 million fund set up by PHARMAC in 2014 to address rare disorders no longer ...