Blog
RSS FeedPosted by Michael Wonder on 25 Feb 2017
Without rare-disease policy, patients in Canada face steep costs for drugs
24 February 2017 - Alex Chiabai has been living with the effects of Duchenne muscular dystrophy, a genetic disease that weakens ...
Posted by Michael Wonder on 15 Feb 2017
NICE rejects Alexion’s rare disease therapy Kanuma
15 February 2017 - NICE has published a final evaluation determination rejecting the use of Alexion's Kanuma to treat infants, ...
Posted by Michael Wonder on 13 Feb 2017
Grassley launches inquiry into orphan drugs after KHN investigation
10 February 2017 - Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, has opened an inquiry into potential ...
Posted by Michael Wonder on 09 Feb 2017
FDA approves drug to treat Duchenne muscular dystrophy
9 February 2017 - The U.S. FDA today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 ...
Posted by Michael Wonder on 07 Feb 2017
Assessing the value of drugs for rare conditions: Institute for Clinical and Economic Review to host orphan drug assessment and pricing summit
7 February 2017 - Discussion will be anchored by an ICER Briefing Paper evaluating the evidence on the comparative clinical effectiveness ...
Posted by Michael Wonder on 07 Feb 2017
Aussie kids given immediate access to life-changing cystic fibrosis drug
7 February 2017 - From 7 February, children aged two to five will have immediate and free access to Kalydeco (ivacaftor) ...
Posted by Michael Wonder on 06 Feb 2017
Cystic fibrosis sufferers to get lifesaving medication for free after government intervention
6 February 2017 - A life changing drug — previously unaffordable for Australia’s littlest cystic fibrosis sufferers costing $300,000 a ...
Posted by Michael Wonder on 05 Feb 2017
Riverina mother to tackle government over PBS' refusal to list cystic fibrosis drug
5 February 2017 - A government decision to keep a revolutionary drug out of the hands of desperate parents has ...
Posted by Michael Wonder on 04 Feb 2017
Drug game-changer for thousands suffering from cystic fibrosis headed for the PBS
5 February 2017 - Australia's youngest cystic fibrosis sufferers will this week get a massive reprieve when the government lists ...
Posted by Michael Wonder on 26 Jan 2017
Biogen sees gradual Spinraza launch, eyes mid-year EU approval
26 January 2017 - Biogen Inc said on Thursday that the first U.S. patients have received its new drug for ...
Posted by Michael Wonder on 24 Jan 2017
Affordable orphan drugs: a role for not-for-profit organisations
23 January 2017 - The success of the regulation on orphan medicinal products in the European Union is evidenced by the ...
Posted by Michael Wonder on 24 Jan 2017
Novelion Therapeutics announces acceptance of marketing authorisation application for metreleptin by European Medicines Agency
23 January 2017 - Novelion Therapeutics today announced that the EMA has accepted for review the marketing authorisation application for metreleptin ...
Posted by Michael Wonder on 22 Jan 2017
Cystic fibrosis sufferer Scarlet Fox 1 million dollars away from access to life-changing drug Kalydeco
21 January 2017 - As a two-year-old living with cystic fibrosis, Scarlet Fox is $1 million away from access to ...
Posted by Michael Wonder on 17 Jan 2017
Drug makers manipulate orphan drug rules to create prized monopolies
17 January 2017 - More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical ...
Posted by Michael Wonder on 03 Jan 2017
Amicus Therapeutics set for share of Fabry market after NICE approval
3 January 2017 - England and Wales’ cost effectiveness watchdog NICE has given a final recommendation to a new treatment ...