November 3, 2016
Cystic Fibrosis Canada is disappointed with the negative recommendation issued on October 26th, 2016, from the Canadian Agency for Drugs and Technologies in Health (CADTH) - Canadian Drug Expert Committee (CDEC) regarding ORKAMBITM.
ORKAMBI™ is the first drug to treat the basic defect in the largest population of people with cystic fibrosis (CF), those with two copies of the delF508-CFTR mutation. There are approximately 1,500 patients in Canada aged 12 years and older who could benefit from ORKAMBI™.
Cystic fibrosis is a progressive disease. Drugs that target the basic defect have the ability to slow that progression, allowing patients to live longer, healthier lives. Cystic Fibrosis Canada believes that ORKAMBI™ should be accessible to those patients for whom this drug can change, even save, their lives.
Although we’ve come a long way in improving health outcomes for Canadians with CF, we have more to do. The median age at death in 2014 was 32 years of age.
Cystic Fibrosis Canada, in collaboration with CF physicians, who are the recognized experts in CF care, established a set of clinical criteria using measurements accepted internationally by regulatory agencies for clinical trials, to allow continuation on ORKAMBI™ of only those patients that demonstrated a clinical benefit. It would appear that CDEC did not believe that CF physicians would be able to accurately distinguish between patients who benefit from the drug from those who do not. Further, the report cites a lack of sufficient effectiveness of ORKAMBI™ to justify reimbursement. Their report (available in English-only) states the following:
"CDEC noted that the absence of established thresholds for clinically significant changes in the clinical parameters that are routinely measured in clinical practice (e.g., ppFEV1) and variability in the occurrence and timing of pulmonary exacerbations makes it challenging to define discontinuation criteria for LUM/IVA that could be operationalized in a consistent manner across the CADTH Common Drug Review (CDR)–participating drug plans.”
However, the report also recognizes that 25% of patients see at least a 5% increase in ppFEV1, and one in eight sees an improvement of at least 10% (a similar effectiveness to KALYDECO®). This means that they accept that the drug will benefit a select number of individuals, but don’t believe that the discontinuation of those who do not benefit will work in clinical practice. That is very different from saying that the drug isn’t effective. No drug benefits everyone equally.
Cystic Fibrosis Canada believes that the proper recommendation should have been to reimburse with conditions, where the conditions include determining a means of discontinuation to the satisfaction of the provinces/payors.
CALLING FOR A TIMELY APPROACH TO MAKE PRORKAMBITM ACCESSIBLE AND AFFORDABLE FOR CANADIANS WITH THE MOST COMMON CF GENE MUTATION
Health Canada has issued a Notice of Compliance for the lumacaftor/ivacaftor combination drug PrORKAMBITM for use in Canada. This gives Canadians with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation a chance to gain access to this breakthrough treatment. PrORKAMBITM could benefit around 1,550 Canadians living with CF.
"Like Kalydeco before it, PrORKAMBITM treats the underlying defect in cystic fibrosis, but for a much larger part of the CF population. Health Canada’s approval of PrORKAMBITM is the first step in getting this new medicine into the hands of the people who need it most. We congratulate Vertex on the approval of PrORKAMBITM by Health Canada and look forward to working with Vertex and the provincial drug plans to ensure access to PrORKAMBITM is made available to eligible CF patients across Canada,” says John Wallenburg, Cystic Fibrosis Canada’s Chief Scientific Officer.
PrORKAMBITM improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. It is the second drug approved by Health Canada that targets the underlying cause of CF. Ivacaftor (as a single therapy) was the first. For a CF patient with two copies of the F508del mutation access to PrORKAMBITM could lead to a healthier, longer life.
“Among my many other roles, I am a wife, a mother, and a healthcare professional. I am also a woman living and sometimes struggling with cystic fibrosis. Gaining access to PrORKAMBITM gives me hope that I will have the opportunity to grow old with my husband, watch my children mature into adults, and choose when I am ready to retire because I have had a full and satisfying career, and not because my illness demanded it,” says Megan Parker.
Vertex, the manufacturer of PrORKAMBITM has submitted the drug to the Common Drug Review (CDR), a process run by the Canadian Agency for Drugs and Technologies in Health (CADTH), Quebec has its own process. A team of independent experts will review PrORKAMBITM and compare how well the drug works to current CF treatments, and whether the drug provides value for money. Based on their findings, a recommendation on whether or not drug plans should cover the drug will be issued. After the CDR recommendation has been made, all provincial health ministries will review PrORKAMBI™ for reimbursement by publicly funded drug plans.
Cystic Fibrosis Canada is calling for a compassionate and timely approach from the drug manufacturer and drug plan administrators when it comes to making PrORKAMBITM accessible to the Canadians with CF that would benefit from this life-changing treatment.