Orladeyo recommended for reimbursement in Québec
The oral therapy is taken preventively by hereditary angioedema patients
The Institut national d’excellence en santé et services sociaux (INESSS) has recommended that BioCryst Pharmaceuticals’ Orladeyo (berotralstat) be reimbursed by public health plans for eligible adults and adolescents 12 and older with hereditary angioedema (HAE) in the Canadian province of Québec.
A similar recommendation was made earlier this year by the Canadian Agency for Drugs and Technologies in Health (CADTH).
Guidelines from INESSS and CADTH, among other local organizations, are considered by national and regional health authorities when deciding which therapies will be accessible to patients at little or no cost through public health plans.
“We are committed to bringing Orladeyo to as many Canadians living with HAE as possible,” Anand Janack, vice president and general manager of BioCryst Canada, said in a press release.
“This announcement from INESSS, which follows the positive recommendation from CADTH earlier this year, takes us one step closer to making our oral, once-daily therapy available to Canadians who are in need of a new option to help improve control of their HAE attacks,” Janack said.
Orladeyo is a daily oral therapy that works by suppressing kallikrein, an enzyme that regulates the production of a molecule called bradykinin that triggers HAE attacks.
It was approved in Canada in June 2022 for the routine prevention of HAE attacks in patients ages 12 and older. It is approved similarly in the U.S., European Union, U.K., and Japan.
Clinical benefits, cost are considerations
When endorsing a medication’s listing on public health plans, organizations like INESSS consider its clinical benefits for patients, as well as its cost.
INESSS’ analysis included a review of data from the Phase 3 APeX-2 trial (NCT03485911) that backed Orladeyo’s regulatory approvals. Data from that trial indicated that Orladeyo was tolerated well, reduced the number of monthly HAE attacks, as well as the need for standard treatment relative to placebo.
The recommendations noted that Orladeyo’s oral route of administration may be preferred by patients relative to other preventive therapies like Takhyzro (lanadelumab), which typically are given by injection.
Patient associations support reimbursement
INESSS also received communications from two patient associations — Hereditary Angioedema Quebec and Angioedema Canada — supporting Orladeyo’s reimbursement. These communications emphasized the potential quality-of-life gains for patients.
However, the medication is expensive, with an estimated cost of $23,800 for a month’s supply. Still, INESSS noted that listing the medication would provide an estimated $5 million in savings to the overall public healthcare system in the first three years.
Ultimately, INESSS suggested that Québec’s Minister of Health and Social Services reimburse the therapy for people with HAE types 1 and 2, ages 12 and older, who experience swelling attacks that interfere significantly with daily activities.
This guideline comes with two conditions — that its use is regulated, and that its manufacturer help to reduce the economic burden on the health system.
This also was reflected in CADTH guidelines, which suggested a price reduction given the fact that Orladeyo’s current public list price does not offer good value to the healthcare system.
“On behalf of people living with HAE and their caregivers, I appreciate this positive recommendation from INESSS, as it should help ensure this oral prophylactic [preventive] therapy is available to those who could benefit from it,” said Charles St-Pierre, president of Hereditary Angioedema Québec. “This recommendation is a step forward as it highlights the need for continued, and simple access for people living with HAE, and their caregivers.”
Orladeyo also has been added to Japan’s National Health Insurance System, making it available to eligible patients there at low, or no, cost.
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