WHAT IS THE CANADA’S DRUG AGENCY REIMBURSEMENT RECOMMENDATION FOR CRYSVITA?: Canada’s Drug Agency recommends that Crysvita be reimbursed by public drug plans for the treatment of X-linked hypophosphatemia (XLH) in adult patients if certain conditions are met. The previous recommendation for Crysvita when initiated in pediatric patients who are at least 1 year of age and in whom epiphyseal closure has not yet occurred, continues to apply to those patients. WHICH PATIENTS ARE ELIGIBLE FOR COVERAGE? Crysvita should only be covered to treat patients aged 18 years or older who have a diagnosis of XLH supported by classic clinical features of adult XLH and a confirmed gene variant, and who have not previously received it. Patients should also have a specific threshold for kidney function or reduced kidney function if it is confirmed not to be due to nephrocalcinosis, bone pain that is caused by XLH or osteomalacia, and have not had a sufficient response to conventional therapy (therapy with active vitamin D and oral phosphate). If a gene variant is not confirmed, XLH diagnosis can be confirmed with a serum intact fibroblast growth factor 23 (FGF23) level by a Kainos assay. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Crysvita should only be reimbursed if it is prescribed by a physician who works in a comprehensive team of health care providers experienced in the diagnosis and management of XLH and if the cost of Crysvita is reduced. Reimbursement may be renewed on an annual basis for patients who do not meet any of the discontinuation criteria, which are the development of hyperparathyroidism, nephrocalcinosis, fasting hypophosphatemia, or fractures or pseudofractures based on X-ray. WHY DID CANADA’S DRUG AGENCY MAKE THIS RECOMMENDATION? • Evidence from a clinical trial showed that Crysvita normalized phosphorus levels in a majority of patients, showed potential for healing fractures and pseudofractures, and reduced pain and stiffness scores. • XLH is a rare disease that causes notable mortality and morbidity in patients. Crysvita has the potential to address several unmet needs of patients, such as reducing pain interference and stiffness as well as improving fracture healing. • Based on the Canada’s Drug Agency assessment of the health economic evidence, Crysvita does not represent good value to the health care system at the public list price. Therefore, a price reduction is required. • Based on public list prices, Crysvita is estimated to cost the public drug plans approximately $287,000,000 over the next 3 years. However, the actual budget impact is uncertain.
WHAT IS X-LINKED HYPOPHOSPHATEMIA? XLH is a rare genetic disorder that causes patients to produce too much FGF23, which makes them unable to retain phosphate. Adults with XLH can have fractures, pseudofractures, arthritis and connective tissue issues, as well as pain, stiffness, fatigue. These have considerable impact on patients’ mobility and health-related quality of life (HRQoL). The prevalence of XLH in Canada is unknown, but it is estimated to affect 1.57 per 100,000 adults in the UK. UNMET NEEDS IN X-LINKED HYPOPHOSPHATEMIA: There is an unmet need for effective therapies that are accessible, affordable, easier to take, boost energy and muscle function, reduce pain, improve HRQoL, and have fewer side effects in those patients whom conventional therapy did not achieve an adequate response. HOW MUCH DOES CRYSVITA COST? Treatment with Crysvita is expected to cost approximately $410,860 to $528,248 per patient annually (weight-based dosing).
