orphan diseases

orphan diseases

  • Germany approves award winning Cystic Fibrosis drug ‘Orkambi’

    Philip Watt CEO of Cystic Fibrosis Ireland welcomes today’s news (19 December 2016) that a pricing and reimbursement agreement for the ground breaking Cystic Fibrosis drug Orkambi has been reached with the German Statutory Health Insurances (GKV-SV).

    According to a press release issued by Vertex today, ‘the German Federal Joint Committee (G-BA) recognized the “considerable additional benefit” of Orkambi for people with CF who have two copies of the F508del mutation’.

    Orkambi has been available to eligible patients in Germany under their approval system (different to Ireland) that allows a drug to be made available on a one year trial basis once it has been approved by the European Medicines Authority (EMA). The EMA approved Orkambi in November 2015. Under the German system, authorities have a 12 month opportunity to assess the effectiveness and proposed pricing of a drug.

    Orkambi is now available to eligible people with CF in Germany, the United States and Austria. France has a similar system to Germany and is due to consider Orkambi in March 2017. CFI further notes that Orkambi has recently received the ‘Drug Discovery of the Year Award’ award from the British Pharmacological Society and the French ‘Prix Galien’ award for the most promising rare disease medicine in 2016.

    Cystic Fibrosis Ireland recently welcomed the news that the HSE and Vertex have entered into renewed negotiations on the potential of making Orkambi available in Ireland. CFI has urged that these negotiations are carried out in private and not over the airwaves and a fair agreement is reached as soon as possible. Philip Watt stated ‘both sides should make compromises in order to reach a fair agreement, including a considerable reduction in price from the manufacturer, Vertex and a greater acknowledgment by the HSE of the very high costs and risks involved in developing a drug for rarer diseases such as CF’.

    Philip Watt further stated: ‘CFI calls for an overhaul of the system of assessing drugs for orphan diseases (rare diseases) in Ireland, including consideration of adopting the French or German drug approval systems and/or adopting a similar approach to the way cancer drugs are assessed and approved in Ireland.’