What Is the CADTH Reimbursement Recommendation for Oxlumo?: CADTH recommends that Oxlumo be reimbursed by public drug plans for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients if certain conditions are met.
Which Patients Are Eligible for Coverage?: Oxlumo should only be covered to treat those with a genetically confirmed diagnosis of PH1 and whose urine oxalate excretion is not normalized with standard of care therapy (including 3 to 6 months of vitamin B6).
What Are the Conditions for Reimbursement?: Oxlumo should only be reimbursed if its cost is reduced and it is initially prescribed by a nephrologist or metabolic diseases specialist experienced in the diagnosis and management of PH1.
Why Did CADTH Make This Recommendation?:
One clinical trial demonstrated that Oxlumo reduced 24-hour urinary oxalate levels in patients 6 years or older compared to placebo. Two clinical trials provided evidence of Oxlumo lowering urinary and plasma oxalate levels for patients who were aged younger than 6 years and for those with kidney disease with or without hemodialysis.
Oxlumo may meet some of the needs identified by patients, such as reducing oxalate formation, but evidence was lacking to suggest that Oxlumo prevents kidney stones or end-stage kidney disease (ESKD), improves health-related quality of life (HRQoL), or delays the need for organ transplant.
Based on CADTH’s assessment of the evidence, Oxlumo does not represent good value to the health care system at the public list price; a price reduction is required.
Based on public list prices, Oxlumo is estimated to cost the public drug plans approximately $122 million over the next 3 years. However, the actual budget impact is uncertain as the total number of patients with PH1 who are eligible for funding of Oxlumo is unknown.
What Is PH1?: PH1 is caused by a genetic error that allows oxalate to build up in the body and form crystals, such as kidney stones, that are difficult for the kidneys to remove from the body. The accumulating oxalate crystals cause permanent damage to tissues, specifically the kidneys, leading to loss of function. Though prevalence in Canada is unknown, it is between 1 and 3 per million people in Europe.
Unmet Needs in PH1: Patients with PH1 need effective treatments that prevent further kidney damage, decrease oxalate accumulation throughout the body, and prevent the need for dialysis or organ transplant.
How Much Does Oxlumo Cost?: Treatment with Oxlumo is expected to cost approximately $581,132 for pediatric patients and $1,743,495 for adults in the first year, and $387,421 for pediatric patients and $1,162,263 for adults in subsequent years.