by: Durhane Wong-rieger and christine beyaert
ORPhAN DRUGS
caring for canada’s most vulnerable citizens through a national plan for
rare disorders
Every year, hundreds of Canadians with severe or life-threatening disorders do not get access to the medicines they need. What do these patients have in common? They all have rare disorders. It’s almost unbelievable but true. With a public health system that is the envy of much of the world, Canada sadly lags behind almost all other developed nations, including the U.S., the EU, Japan and Australia, in our care and treatment for rare disorders. We’ve made progress over the past few years but it’s time for Canada to stop making excuses and bring in a comprehensive plan that will give Canadians with rare disorders the same treatment as those with common ones.
A rare disorder is defined as a disorder that affects less than one in 2,000 Canadians. Many are severe, progressive, and/or life threatening conditions, with high mortality rates and devastating impacts on patients, families and society. Although individually rare, collectively, they affect one in 12 persons or 2.8 million Canadians. There are no effective treatments for many of these disorders. However, for many others, if infants or children are diagnosed and treated early enough, they can avoid physical disability or cognitive delays and live nearly normal lives. Rare disorders present unique public health challenges. Few physicians are familiar with diagnosing and treating these conditions, which means most patients are undiagnosed, misdiagnosed, or delayed in diagnosis, so they do not get timely access to treatments even when therapies exist. There are relatively few therapeutic options - between 7,000 and 8,000 rare disorders have been identified, but there are only 400 ef12 BIOTECHNOLOGY FOCUS February/March 2014
fective drug treatments. This situation is expected to change dramatically with hundreds of new therapies in development and coming to market over the next decade. But without change in our rare disease environment, many of these therapies will not be accessible for Canadian patients. Currently, Canadians tend not to be included in clinical trials and companies are reluctant to bring their drugs to Canada. Approximately half of the rare disease drugs available to patients in the U.S. or Europe have been approved in Canada and only half of these are funded through public drug plans. Currently, Canada’s regulatory and reimbursement systems present significant challenges to researchers and developers of drugs for rare diseases, also known as orphan drugs. On the federal regulatory side, the review process is about to change. In October 2012, Health Canada announced their intention to implement an Orphan Drug Regulatory Framework, a review process for orphan drugs that is similar to those of the U.S. and the European Union. Health Canada has indicated that this framework will include tools to improve the quality of knowledge used by provincial and territorial decision-makers and healthcare professionals, provide the opportunity for patient input, encourage transparency and sharing information, and support international collaboration. By harmonizing Canada’s orphan drug regulatory process with those of the U.S. and the EU, companies will be able to set up clinical trials and to apply
