Canadian Cystic Fibrosis Patients See Access to Orkambi Delayed for Undetermined Time

Canadian patients with cystic fibrosis (CF) will have to wait for access to Orkambi (lumacaftor/ivacaftor), whose submission will be reconsidered at a future meeting by the Canadian Drug Expert Committee (CDEC) of the Canadian Agency for Drugs and Technologies in Health (CADTH).

CADTH is the nonprofit Canadian organization that makes recommendations for a drug’s optimal use and decides whether a drug is covered by the public drug plans.

“People with CF are going to have to wait even longer to get access to a medicine that has the potential to improve the quality and length of their lives,” Norma Beauchamp, president and CEO of Cystic Fibrosis Canada, said in a press release.

Orkambi was approved last year by the U.S. FDA for cystic fibrosis patients ages 12 and older who have two copies of the F508del mutation in the CFTR gene. Two double-blind, placebo-controlled studies have evaluated the safety and efficacy of Orkambi in 1,108 CF patients with the F508del mutation. The studies demonstrated the drug effectively improves lung function and reduces the number of pulmonary exacerbations, which can lead to accelerated lung disease and increased hospitalization rates.

In June, the CDEC shared an embargoed recommendation for Orkambi with Vertex, the drug manufacturer, and the participating drug plans. After reviewing the material, Vertex requested a reconsideration, claiming the embargoed recommendation is not supported by the submitted clinical evidence of Orkambi. CADTH agreed to reconsider based on Vertex’s request.

Members of Cystic Fibrosis Canada are disappointed that people with CF won’t have access to the drug right away.

“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 cystic fibrosis. “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.”

Cystic Fibrosis Canada has made a submission to CADTH’s CDEC requesting that Orkambi’s reconsideration takes place in the next CDEC meeting, in September 2016. CF Canada has also requested that CDEC considers the recommendations that were developed by a CF Physician Panel of CF Canada for the clinical used of Orkambi. The recommendations have been endorsed by 26 directors of Canadian CF clinics.

“Getting a new medicine to the patients who require it is a long and complicated process,” said Megan Parker, chair of Cystic Fibrosis Canada’s adult CF advisory committee. “It is imperative that the various bodies involved in the process, including Vertex, be driven by the evidence and patient need, to ensure access to Orkambi in a manner that serves the needs of patients, as well as protects the long-term sustainability of our public drug plans.”