Blog
RSS FeedPosted 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 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 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 ...
Posted by Michael Wonder on 13 May 2023
Orphan drugs: early generation of evidence urgently needed
10 May 2023 - A team led by IQWiG authors, together with Rita Banzi from the Italian Mario Negri Institute, analysed ...
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 ...
Posted by Michael Wonder on 08 Nov 2021
Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...
Posted by Michael Wonder on 02 Dec 2020
IQWiG publishes 1,000th report
2 December 2020 - In the 16th year of its existence, IQWiG has published its 1,000th report: a dossier assessment ...
Posted by Michael Wonder on 23 Mar 2020
Determinants of orphan drug prices in Germany
21 March 2020 - Legislation introduced in 2011 in Germany has instituted an early benefit assessment of newly licensed pharmaceuticals with ...
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 09 Sep 2017
Can a new value framework help ease friction over orphan drug prices?
7 September 2017 - America’s healthcare debate has stalled in Congress, but constructive dialogue and innovation are thriving in specialised ...
Posted by Michael Wonder on 19 Dec 2016
Vertex announces German reimbursement agreement for Orkambi (lumacaftor with ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation
19 December 2016 - Vertex Pharmaceuticals today announced it has reached a pricing and reimbursement agreement for Orkambi (lumacaftor with ...