Mr. Speaker, I am pleased to stand in the House today on behalf of my riding of Cambridge and North Dumfries and speak to this very important motion.
The government understands the seriousness of the issues faced by Canadians who suffer rare diseases. We have taken action on these issues for this vulnerable population of Canadians and will continue to do just that.
These disorders affect a very small number of people, which is the good news, sometimes fewer than 100 people in the entire country, making them difficult to diagnose and even harder to treat. The government recognizes that Canadians who suffer from rare diseases can face unique challenges, due in part to the limited information available to doctors, hence making it even more difficult to diagnose and leaving treatment options restricted.
Rare diseases means just that. They are rare. Very few people get them, but that truth makes it a very tricky thing to develop drugs for them. Appropriate testing is difficult and the potential market is very small. Research and development is often prohibitively expense for drug companies.
Perhaps it might be useful to briefly explain the system and how it works and the roles of the different participants.
The federal government is responsible for deciding what products can be sold on the Canadian market, based on sound and practical assessment of the drugs benefits and its risks. We first must do no harm. This is done through a review of the safety and efficacy data from clinical trials conducted on patients. In fact, therein lies one of the problems.
Traditional clinical trials use hundreds and in some cases thousands of patients in a study. Clearly this is not an option for analyzing treatment protocols for rare diseases. This means that by making regulatory decisions about drugs for rare diseases, it is just that much more difficult and that much more challenging.
However, after all that has been done, after all the trials and the research, the drug is approved for sale in Canada. Now each public drug plan, provincial, territorial and in some cases the federal drug plan, for example with first nations or veterans, must decide whether to pay for the drug treatment in their respective jurisdiction.
The high per patient treatment cost means that there will almost never be considered a cost effectiveness using traditional measures and traditional methodologies. It is challenging to know where to draw the line or whether we should even apply different standards if we were to do so, considering only the rarity of a condition.
Physicians must make difficult decisions on how to prescribe these drug therapies or even if they should prescribe them at all based on limited data. Patients must make the ultimate choice of whether to use them and, sadly, sometimes there are no other alternatives.
The good news is the government is taking steps forward on a system meant to improve the health of Canadians, including those with rare diseases. Our recent investment of $113 million in the food and consumer safety action plan is evidence of this progress. The action plan includes a comprehensive set of measures to improve the safety of products that we use, including prescriptions drugs for rare diseases.