The national council for priority setting in health care discussed a case on the introduction of a costly drug (ivakaftor, Kalydeco ®) for treating cystic fibrosis and the individual reimbursement scheme on April 7 2014. This is a summary of the paper the Norwegian Knowledge Centre for the Health Services wrote on the issue.
The case was introduced to the council by the clinical ethical committee in “Helse Bergen”, and presented by Ingrid Miljeteig (June 2013). The case links to issues like orphan drugs (drugs for rare conditions) and willingness-to-pay thresholds. It addresses several relevant priority setting challenges.
Cystic fibrosis (CF) is a congenital, inherited progressive disease that mainly affects the lungs and bowel function. CF is a rare disease, and less than 300 people have the disease in Norway. A new drug, ivakaftor, is developed for treatment of a very small subset CF patients. Efficacy and safety of ivakaftor have been studied in two randomized, double blind, placebo-controlled clinical trials in which patients were followed for 48 weeks. The results are good; including improved pulmonary function, but it is not known whether life is prolonged. The drug should be taken lifelong in addition to other treatments, and costs about 2.5 million NOK per patient per year. Swedish estimates of cost per quality adjusted life year (QALY) gained is between 3.5 million and 10.4 million SEK. In Norway, the relevant patients get the treatment funded through the individual reimbursement scheme. As of now (June 2014), no assessment of cost-efficiency is performed when applications for reimbursement under this scheme is assessed. However, this is currently under review by the ministry of health and care services.