That, in the opinion of the House, the government should respond specifically to the challenges faced by Canadians with rare diseases and disorders, and the initiative put forward by the Canadian Organization for Rare Disorders by: (a) establishing the definition for serious rare disorders as those with a prevalence of less than 1 in 2000 Canadians; (b) examining the feasibility of a national “Chance for Life Fund” equivalent to 2% of the total annual public drug expenditure to be designated for therapies for rare disorders; (c) considering the establishment of a multi-stakeholder advisory body, including treaters and patients, to recommend treatment access for life-threatening or serious rare disorders, based on scientific standards and social values; (d) considering the establishment of centres of reference for specific rare disorders, comprised of national and international experts, who will develop criteria for treating patients based on scientific evidence and patient impact and provide on-going surveillance into the real-world safety and effectiveness of these treatments on individual and group basis; (e) considering options to provide incentives through orphan drug regulation and policy, to assure Canadian organizations and researchers are motivated to conduct research and development into treatments for rare and neglected disorders; (f) supporting internationally accepted standards for conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations; (g) considering ensuring that Health Canada’s progressive licensing framework provide appropriate support to the design of clinical trials for very small patient populations and appropriate review of evidence submitted from these trials; and (h) reporting the progress accomplished to the House within six months.
Mr. Speaker, I am pleased to rise today to begin debate on my private member's motion, Motion No. 426, which addresses the issue of rare disorders. If passed, Motion No. 426 will begin the process of bringing Canada into line with numerous other developed nations that have already implemented effective policies offering extensive support and treatment for citizens with severe and life threatening rare disorders.
More specifically, Motion No. 426 calls on the government, through Health Canada, to consider the policy proposals advanced by the Canadian Organization for Rare Disorders. I consider this motion a starting point and CORD's Chance for Life Fund an ideal framework in which to examine many of the challenges faced by Canadians living with rare disorders.
Based on experience and evidence, I hope to prove that proposals within Motion No. 426, if implemented in Canada, would be as effective here as they have been in other nations. I also believe that doing so would reflect not only the principles of universality as outlined in the Canada Health Act but also the compassionate approach that most Canadians feel is at the core of our public health care system.
I have been greatly encouraged by the support I have received from the other parties on this issue. As rare disorders affect thousands of Canadian families from all walks of life and backgrounds, so too has my family dealt personally with this issue, and I have been impressed by the spirit of cooperation and non-partisanship that has been constant in my discussions with my colleagues as I have moved this issue forward and developed the motion we are debating today.
There are some issues that are truly non-partisan, and this is one of them. In particular, I would like to thank the Parliamentary Secretary for Health, the member for Charleswood—St. James—Assiniboia, as well as the health critic from the NDP, the member for Winnipeg North, and the Bloc health critic, the member for the riding of Québec.
I have also worked closely with CORD as well as many of the organizations affiliated with it, such as the Pulmonary Hypertension Association of Canada and the Canadian MPS Society, and wish to thank their representatives for their continued support and diligent work in bringing this issue forward.
Before I continue, I will clarify that I will often refer to “rare disorders”. Other nations have adopted similar language to describe these conditions, such as “orphan diseases, disorders or rare diseases”, and I will refer to them all throughout my remarks, but for the purposes of today's debate, they mean the same.
February 29 of this year marked the first annual International Rare Disease Day. With 2008 being a leap year, “the rarest date on the calendar”, February 29, was the perfect date to launch what will be an annual occurrence.
In the simplest possible terms, Motion No. 426 addresses the issues faced by Canadians who suffer from a wide range of rare diseases that affect very few people. At present, Canada does not have a definition of, or an official policy on, rare disorders and, as such, our health care system does not differentiate between someone who has a very common disease and one that is extremely rare. Essentially, the prevalence of their occurrence among Canadians is not considered.
In order to develop a comprehensive Canadian policy on rare disorders, it is necessary to establish a Canadian definition of rare disorder or, more specifically, what prevalence among Canadians a disorder should have to be considered rare. Motion No. 426 calls for the definition of rare disorders as those with a prevalence of less than 1 in 2,000 Canadians, and I will deal with this further in a moment.
The inspiration for Motion No. 426 is found in the Canadian Organization for Rare Disorders Chance for Life Fund, an action plan that CORD developed to address this issue and begin the process of establishing a made in Canada policy that will ensure patients with rare disorders have the exact same right and access to effective therapies, or the same “chance for life”, as all Canadians.
A policy for rare disorders is based on the basic principle of the equitable provision of health care that many Canadians assume is an unshakable element of Canada's universal health care system, but in the case of rare disorders, this principle of universality is currently missing.
Rare disorders include such conditions as cystic fibrosis, Huntington's disease, legionnaires' disease, muscular dystrophy, thalassemia, MPS, pulmonary hypertension, Fabry disease, Gaucher disease, ALS, commonly known as Lou Gehrig's disease, Waldenstrom's anemia, AIDS, and acromegaly, to name a few.
Rare disorders can be acquired or genetic. It is also important to note that several rare disorders fall within more common conditions. For example, while cancer is obviously not considered a rare disorder because of its prevalence, kidney cancer is. Many of these rare disorders we may have heard of, because self-help groups have done a good job of public awareness.
From the brief list of rare disorders that I just named, I am sure many Canadians can already identify a family member or friend who suffers from a rare disorder. My grandson, Dylan Bell, was diagnosed at the age of 3 with a rare disorder, pulmonary hypertension, and passed away the day after his 12th birthday. In my own riding, young Nicolas Harkins has MPS 1, an enzyme deficiency disease that is also life threatening. Today's Globe and Mail article on page 3 by Lisa Priest refers to 11 year old Szymon Cajmer, who has MPS 2, otherwise known as Hunter syndrome.
However, that list of disorders is minuscule when compared to the complete list of approximately 7,000 rare disorders that are currently identified. Using the prevalence standard or definition of rare as being 1 in 2,000, it is estimated that 1 in 12 Canadians has been diagnosed as having, or being a carrier of, a rare disorder. That is over two and a half million Canadians who today are affected by a rare disorder.
Clearly the impact of rare disorders is much greater than most realize. While only a small number of Canadians may have a specific rare disorder, as a group Canadians with rare disorders are far from rare. It is for this reason that CORD, which is the only organization of its kind in Canada, plays such a vital role. By collecting information on rare disorders and raising awareness, CORD is developing networks for Canadians with rare disorders, networks that allow them to speak effectively with one voice and, most importantly, to realize they are not alone. Simply put, there is strength in numbers.
Not only does Canada not have an official definition of prevalence of rare disorders, which is critical in order to move forward on this issue, but it lags far behind most countries in the developed world when it comes to rare disorder policy. For a country that considers its health care system to be a badge of honour, the envy of the world, as it is often referred to, I believe the manner in which we deal with this issue would surprise most Canadians.
The United States was the first country to enact rare disorder legislation 25 years ago in 1983 and was quickly followed by Australia, Japan, Singapore, Taiwan and South Korea in the 1990s. In 2000 the European Union established its own orphan drug legislation.
Official definitions or prevalence of rare disorders vary in each country. In the U.S., a prevalence of less than 200,000 persons in the total population, which is approximately 1 in 1,300, is the official definition. On the other end of the spectrum is Australia, where “rare” is a prevalence of 1 in 15,000. Other nations generally fall somewhere in between. Establishing a definition, or prevalence, is only the starting point, and it is through that definition that other policies and changes to our system can flow.
Access to medication for Canadians with rare disorders has been described as “trying to untangle a Gordian knot” and is the greatest challenge faced by those with rare disorders. Because Canada does not have a rare disorders policy, medications and treatments available to a person with cystic fibrosis, for example, in the United States, South Korea or France may not be available to a Canadian or, worse, are available but not necessarily covered under all provincial health plans, and therefore weakens the aspect of universality.
Rare disorder policy, such as the orphan drug act in the United States, which, I would like to add, was the result of a bipartisan effort, has produced positive, tangible results; a key area of improvement being a dramatic increase in the number of innovative therapies in development due in part to economic and regulatory incentives included in rare disorder legislation. This has led directly to an exponential growth of the biotechnology industry and the development of more cutting edge technologies and treatments for those living with rare disorders.
Canada's policy void in this area has forced many Canadian biotech firms to offer free clinical trials to small groups of patients as the only way to give them access to life-saving therapies. In many cases these clinical trials have proven to be immensely successful. Unfortunately, such trials were limited to only a few individuals and participating in them often involved regular travel which can be too expensive or difficult for those with advanced conditions.
A Canadian rare disorder policy must view the biotechnology industry as a priority sector in a knowledge-driven economy that not only helps with the “brain gain”, but can also produce life-saving technologies for those suffering from rare disorders.
Motion No. 426 asks the government to consider the establishment of centres of reference for clusters of rare disorders, comprised of national and international experts, who would develop criteria for treating patients based on scientific evidence and patient impact, and provide ongoing surveillance into the real world safety and effectiveness of these treatments on an individual and group basis.
Motion No. 426 also asks the government to consider options to provide incentives through orphan drug regulation and policy to ensure Canadian organizations and researchers are motivated to conduct research and development into treatments for rare and neglected disorders.
There is a tremendous pool of talent in Canada in the health research fields that we must encourage through a made in Canada policy on rare disorders. In the other countries I have mentioned, orphan drug legislation has provided incentives for the development and marketing of medicines for rare disorders that otherwise would not have gone forward.
Motion No. 426 can begin an evaluation of how this process could develop in Canada. Recent data reveals how effective such changes have been in helping those in need. For example, CORD reports that during the 2003 review of the U.S. orphan drug policy, it was found that 1,100 products received orphan drug designation, 231 were marketed, thereby impacting over 11 million patients. During the 2005 review of the European Union policy, 260 products received orphan drug designation, and 22 received market authorization, thereby impacting over 1 million EU patients.
Clearly, Canada has work to do to catch up to our friends to the south and in Europe. Perhaps the greatest roadblock for those with rare disorders accessing drugs and treatments is the common drug review process.
As part of the Canadian Agency for Drugs and Technologies in Health, the common drug review performs systematic reviews of clinical evidence, pharmacoeconomic information, and with detailed recommendations by the Canadian Expert Drug Advisory Committee, CEDAC, it provides evidence-based recommendations to provincial drug plans on which drugs should be covered under provincial health plans. All provincial drug plans, with the exception of Quebec, participate in this process and use CDR decisions as a starting point when deciding on funding.
While there are some provincial programs that do offer assistance for specific rare disorders, AIDS being one example, these programs can be temporary and only create a patchwork system. While the CDR provides a streamlined drug review process in Canada, it only accepts specific evidence-based data in its decision making process.
More simply put, stringent evidence, a mandatory requirement for a drug even getting to the starting point in the CDR process, must have been produced from a study with a minimum number of patients. Without a specific minimum number of patients enrolled in a study or trial, evidence is simply not considered to be of high enough quality to advance in the CDR process let alone be considered for approval.
We can imagine the frustration of researchers and doctors who are unable to find enough patients to participate in a process that they believe could result in life-saving treatments becoming available to those who so desperately need them.
Motion No. 426 calls on the government to consider supporting internationally accepted standards for conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations and to consider ensuring that Health Canada's progressive licensing framework provides appropriate support to the design of clinical trials for very small patient populations and appropriate review of evidence submitted from these trials.
Some rare disorders are known to affect as few as 20 Canadians. One can only imagine the incredible challenge of not only attempting to identify enough cases for a clinical study, but the tremendous travel costs and other concerns such as work, family and health concerns of individuals who may be able to participate.
It is my hope that with the passage of Motion No. 426, we can begin the process to move Canada a little further down the road in developing a rare disorder policy and at the same time raise public awareness on this important issue.
As parliamentarians, I encourage all members to rise above partisanship and continue to work together to ensure that those Canadians with rare disorders are given a chance for life.