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 ...

Read more →

Today is Rare Disease Day

29 February 2024 - PHARMAC understands that people living with rare disorders face many challenges, including access to health care ...

Read more →

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 →

Sebelipase alfa for the treatment of patients with Wolman disease (final guidance)

10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if ...

Read more →

Velmanase alfa for the treatment of patients with alfa mannosidosis

13 December 2023 - NICE has published final evidence-based recommendations on the use of velmanase alfa (Lamzede) for treatment of ...

Read more →

PHARMAC calls for applications for medicines for rare disorders

13 November 2023 - PHARMAC is calling for applications for medicines to treat rare disorders.  ...

Read more →

Elexacaftor with tezacaftor and ivacaftor, tezacaftor with ivacaftor and lumacaftor with ivacaftor for the treatment of patients with cystic fibrosis

3 November 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...

Read more →

Olipudase alfa for the treatment of patients with acid sphingomyelinase deficiency (Niemann-Pick disease type B and AB)

26 October 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...

Read more →

Cipaglucosidase alfa and miglustat for the treatment of patients with late-onset Pompe disease

15 August 2023 - NICE has published evidence-based recommendations on the use of cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for ...

Read more →

PHARMAC’s Rare Disorders Advisory Committee makes new recommendations for funding

10 August 2023 - PHARMAC has published the record of the March Rare Disorders Advisory Committee meeting, sharing details on ...

Read more →

Setmelanotide acetate for the treatment of people with obesity and hyperphagia associated with Bardet-Biedl syndrome

3 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...

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 →

New life saving medicines

11 April 2023 - The Australian Government has added the two medicines, Kanuma (sebelipase alfa) and Cerdelga (eliglustat) to the Life ...

Read more →

Investments to support access to drugs for rare diseases

22 March 2023 - Today, the Government announced a total investment of up to $1.5 billion over three years in ...

Read more →

Government of Canada improves access to affordable and effective drugs for rare diseases

22 March 2023 - Today, the Honourable Jean-Yves Duclos, Minister of Health, announced measures in support of the first-ever National Strategy ...

Read more →