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Volunteer Advocate
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ORKAMBI™: WHERE ARE WE NOW?

Early this summer, people with cystic fibrosis were disappointed to learn they are going to have to wait even longer for access to a new CF drug, ORKAMBI™ (lumacaftor/ivacaftor). A final recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) to the public drug plans has been delayed until at least the fall of 2016.

WHAT IS ORKAMBI?

ORKAMBI™ is a combination therapy that targets the underlying cause of CF for people with two copies of the F508del mutation in the CFTR gene. Evidence from clinical trials indicates that ORKAMBI™ can improve lung function and reduce the number of pulmonary exacerbations that may result in hospitalization. In Canada, this therapy may benefit up to 1,550 people over the age of 12 living with CF, giving them an improved quality of life and a longer life.

THE JOURNEY SO FAR

January 2016

ORKAMBI™ received a Notice of Compliance from Health Canada. With this approval, ORKAMBI™ became available through some private insurance companies. However, in order to get ORKABMI™ covered by the public drug plans, there are more hurdles to cross.

February 2016

Vertex, the manufacturer of ORKAMBI™, submitted the drug to CADTH and, in Quebec, to the Institut National D’excellence en Santé et en Services Sociaux (INESSS). The role of CADTH and INESSS are to assess new drugs based on clinical evidence and make recommendations to the provincial and territorial drug plans on whether or not to cover the drugs. The decision from INESSS will be issued in October 2016 and will apply to Quebec.

Next Steps

In the rest of Canada, patients were hoping CADTH would issue a final positive recommendation in June or July. However, after reviewing the interim recommendation by CADTH, Vertex requested a reconsideration of the clinical evidence by CADTH. At this time, the funding decision has been delayed and CADTH will reconsider the clinical evidence at an upcoming meeting of the Canadian Expert Drug Committee (CDEC). Once the final recommendation is made by CADTH, all provincial and territorial health ministries will review ORKAMBI™ to determine whether or not the drug will be reimbursed by publically funded drug plans.  If there is a positive recommendation from CADTH, the price of the drug will be negotiated by the pan-Canadian Pharmaceutical Alliance.

Today

Cystic Fibrosis Canada is calling on the CDEC to carefully consider the criteria for the clinical use of ORKAMBI™ that was developed by CF Canada’s Physician Panel on lumacaftor/ivacaftor. It is the strong opinion of CF Canada that decisions regarding access to ORKAMBI™ should be made by CF experts, namely CF physicians. More specifically, it is CF Canada’s position that ORKAMBI™ should be recommended based on the criteria established by the expert panel.

CF Canada is sensitive to the reality that ORKAMBI™ is a very expensive drug at $249,000/year. CF Canada feels that the drug should be made available to everyone who can potentially benefit from it, which will mean that the negotiations at the pCPA will be very difficult since the price will have to be negotiated to a level at which would allow such access.

“The bottom line is that ORKAMBI™ needs to be negotiated so that everyone whose life can be improved by it, have access to it,” stresses Norma Beauchamp, CF Canada President and CEO. “That means the negotiations between government and Vertex are going to be difficult – but people living with CF are counting on them to be successful.”

“My daughter was fortunate enough to participate in a clinical trial for ORKAMBI™. We have seen a dramatic improvement in her lung function and a reduction in her infections and illnesses – which is hugely important for people with CF,” said Ron Anderson, the father of a 15-year-old girl living with CF. “We feel ORKAMBI™ has arrested the progression of the disease. We were hopeful that we could plan for the future in a way that we previously couldn’t, this news is certainly a setback.”

For advocacy resources and updates on ORKAMBI™, Health Canada, CADTH and how medications are funded, please continue following our updates on the blog.

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