After years of lobbying by patient groups, Health Canada is preparing to change the way drugs are approved to accommodate rare diseases and the “orphan drugs” needed to treat them (Montreal Gazette).
Later in October, Health Canada will launch consultations on broad drug-regulation reforms and, for the first time, how orphan drugs should be treated differently and incorporated into the regulatory framework will be part of those conversations. The department hasn’t committed to implementing a formal orphan drug policy but the changes that will eventually be made as part of wider modernizations to Health Canada rules are considered major progress and a departure from previous positions.
There are thousands of rare disorders that affect only small groups of people, but altogether, an estimated one in 12 Canadians has a rare condition and many people are undiagnosed.
Unlike other countries, Canada doesn’t have a standard definition of rare diseases, let alone a national plan to deal with them. The United States has had an orphan drug policy since 1983 and the European Union has also implemented changes to their regulations to accommodate orphan drugs.
Canada needs to follow their lead, say groups such as CORD and BIOTECanada, because the absence of a policy means patients and researchers here are at a disadvantage.
The potential revisions could first include defining a rare disease as a condition that affects one in fewer than 2,000 people, and beyond that, may change the way drugs for rare diseases are developed, approved and brought to the market.
If the American and European models are followed, there will be more incentives for drug companies to pour money into research and development — a challenge considering the customer base for orphan drugs is so small. Those incentives could include more tax credits and funding grants for research, extending patent protection, and reducing drug-approval application fees or dropping them altogether.
Clinical-trial protocols might also be modified to accommodate the challenges associated with testing drugs for rare diseases, said David Lee, head of the Health Canada branch that is in charge of updating the regulations.
Lee said Health Canada is carefully studying how other countries handle orphan drugs with an aim to see what could be implemented here.
After the consultation period, which will last several months, draft regulations will be proposed for overhauling the drug-regulation system.