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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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, ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →