Drug funding for people under 18 with rare bone disorder
Wednesday, December 15, 2021 11:00 AM
Eligible people in B.C. living with X-linked hypophosphatemia (XLH), a rare genetic and chronically debilitating bone disorder, will have help as the provincial government is covering costs for burosumab, or Crysvita, the medication used to treat the disease.
Symptoms typically develop around the ages of one or two and include rickets, delayed walking, bowed legs, bone pain, impaired growth and severe dental issues. Adults with XLH can have symptoms such as bone and joint pain, fractures, severe dental issues, hearing loss and fatigue.
The annual cost of burosumab at list price ranges from $130,000 to $1.17 million, depending on the patient’s weight and age. B.C. participated in pan-Canadian Pharmaceutical Alliance negotiations with the manufacturer to finalize its funding decision.
The Ministry of Health will provide funding for burosumab through the BC Expensive Drugs for Rare Diseases (EDRD) process for eligible XLH patients. EDRDs are considered non-benefits, but some drugs and patients may be eligible for coverage on a case-by-case, last-resort basis. Patients will need to discuss their eligibility with their health-care provider.
Prior to the approval of burosumab in Canada, there had been no approved treatments for XLH that target the underlying cause of the disease.
Treatment usually starts at the time of diagnosis and continues until bones stop growing, and includes phosphate supplements combined with high-dose calcitriol. Growth hormones may also be required, as well as orthopedic surgery.
Clinical trials of burosumab in patients under the age of 18 showed reduced deformities in lower extremities, as well as significant improvements in stiffness and the severity of rickets.
There are approximately 15 people with XLH under the age of 18 in B.C.
Fri, Jan 21, 2022 at 9:00 am - Doug Smith posted on their blog: Snowshoeing the Bush Lake North Loop
Thu, Jan 20, 2022 at 10:01 am - David Suzuki posted on their blog: Is there a rational argument against climate action?
Wed, Jan 19, 2022 at 8:30 am - Doug Smith posted on their blog: Clapperton Hill Traverse Video – Kamloops Trails
Mon, Jan 17, 2022 at 9:12 am - Your DJ created a new Profile
Mon, Jan 17, 2022 at 9:00 am - Doug Smith posted on their blog: Red Hill Ramble – Kamloops Trails
Sat, Jan 15, 2022 at 9:00 am - Doug Smith posted on their blog: Georgina Point Heritage Park – Kamloops Trails
Thu, Jan 13, 2022 at 10:02 am - David Suzuki posted on their blog: Rest is good, but resolving global crises means not looking away
Thu, Jan 13, 2022 at 9:00 am - Doug Smith posted on their blog: Exploring the Hills in the Snow
Tue, Jan 11, 2022 at 8:30 am - Doug Smith posted on their blog: Red Rock Canyon Loop – Kamloops Trails
Mon Feb 28 The Offspring
Thu Mar 24 to Sat Apr 2 WCT: Clean
Thu Mar 31 to Sat Apr 9 WCT: Kim’s Convenience
Thu Apr 21 to Sat May 7 WCT: Hey Viola!
Thu May 12 to Sat May 28 WCT: The Wizard of Oz
Thu Jun 2 to Sat Jun 11 WCT: Echoes of the Homesick Heart