This article is the first in an 8-part series from the authors. The series, Waiting for new drugs for rare disorders in Canada, is an in-depth look at the disadvantages faced by Canadians with rare disorders in accessing needed, innovative drugs.
By Nigel Rawson and John Adams, July 12, 2023
Canadians living with rare disorders are seriously disadvantaged compared with sufferers in other countries because, so far, Canada has no national strategy for these disorders. Most developed countries have policies encouraging manufacturers to launch “orphan” drugs for rare disorders. This is the first in a series of articles about obstacles facing patients in Canada to access rare disorder drugs.
In Canada, we make a short-sighted virtue of raising barriers that delay or deny access to important innovative medicines, especially costly ones, for patients with unmet or poorly met health care needs. Canadians with rare disorders are particularly impacted. Patients’ experiences manifestly demonstrate evidence of the barriers they confront to gain much-needed access to new medicines.
The primary step in accessing any new medicine is for its developer to submit an application regarding the drug’s safety, effectiveness and manufacturing quality for regulatory authorization. Let’s look at a new medicine called Roctavian for severe hemophilia A as an example to see differences between review processes of the US Food and Drug Administration (FDA) and Health Canada.
Hemophilia A is a rare genetic bleeding disorder experienced by around one in 10,000 people. Sufferers lack the normal ability for blood to clot after an injury due to a deficiency of an essential clotting protein called Factor VIII. This inherited deficiency places them at risk for painful, potentially life-threatening bleeds from even modest injuries such as dental work.
The standard of care for severe hemophilia A is infusions of Factor VIII administered intravenously two to three times per week (that’s 100 to 150 infusions per year). Sufferers’ lives revolve around these infusions. Nevertheless, many continue to experience breakthrough bleeds resulting in progressive and debilitating joint damage.
Few new treatments for hemophilia A have been introduced for decades. However, the new science of human genome sequencing has resulted in novel therapies for many previously untreatable or poorly treated disorders – Roctavian is one. For several years, Roctavian has been undergoing trials in humans that have demonstrated its efficacy, safety and manufacturing quality. A single Roctavian infusion results in low levels of bleeding without need for Factor VIII infusions. That’s one infusion versus hundreds.
The FDA has at least four programs to encourage drug developers to bring new therapies to patients who need them and granted Roctavian:
- Orphan drug status. This is a program intended to advance the evaluation and development of drugs that demonstrate promise for treatment of rare disorders.
- Breakthrough Therapy Designation. A program to allow Americans early access to important new medicines.
- Priority review status. This means the FDA’s review performance target is four months shorter than its usual standard, although the review is no less stringent.
- Regenerative Medicine Advanced Therapy Designation. A recently-introduced program to facilitate the development and review of new treatments for unmet medical needs in patients with serious conditions.
Canada has no program or law to provide incentives for new orphan drugs and Health Canada has no similar programs for breakthrough or advanced regenerative therapies. Health Canada has a priority status review process but can only cope with a limited number of these reviews at a time and the priority reviews we do manage usually duplicate earlier reviews by American or European regulators.
Health Canada talks about being a world-class regulator. However, the lack of incentive programs and the limit on priority reviews tell a different story. Instead, Health Canada merely repeats the work of world-class American and European regulators.
The FDA approved Roctavian in June 2023. The European Union also gave Roctavian orphan drug status and conditionally authorized the medicine in June 2022. No submission for regulatory approval for Roctavian has been made to Health Canada so far. This could be due to the lack of a rare disorder strategy or other incentives, combined with the last six years of uncertainty around Ottawa’s plan to reduce the cost of new expensive therapies to a level that would be unsustainable for their developers. It could also be due to the multiple barriers erected by payers in Canada over the last 20 years that manufacturers must overcome to get new medicines to patients who need them, which we discuss in the next four articles in this series.
Canada’s place in global launches of new drugs is slipping. We have fallen out of the top tier. Wait times for patients to access new medicines are growing. Canadians with rare disorders desperately need incentives to encourage developers to launch their drugs here. Health Canada should work collaboratively with drug developers to rapidly move new medicines through its regulatory process – as the FDA does – or mutually recognize medicines approved in the United States or the European Union as being marketable in Canada.
Nigel Rawson is a Senior Fellow with the Macdonald-Laurier Institute and an Affiliate Scholar with the Canadian Health Policy Institute. John Adams is cofounder and CEO of Canadian PKU and Allied Disorders Inc. which has received sponsorship from the developer of Roctavian, a Senior Fellow with the Macdonald-Laurier Institute and volunteer board chair of Best Medicines Coalition.
The views expressed are the authors’ own and do not necessarily represent those of organizations with which they collaborate.
