Mr. Don Bell (North Vancouver, Lib.)
|| 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.
He said: 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.
Mr. Laurie Hawn (Parliamentary Secretary to the Minister of National Defence, CPC):
Mr. Speaker, I would like to thank the hon. member for North Vancouver for raising this important issue and also to make members of this House aware that the government is interested in finding common ground on this important issue.
The subject the hon. member has raised is quite serious for many people, those diagnosed with these rare diseases, their families and loved ones, and Canadians across Canada who sympathize with their suffering.
Rare disorders affect people of all ages, races and ethnic backgrounds. Some disorders are genetic, for example, Tay-Sachs, Huntingdon's, or Sickle Cell disease. Some disorders are acquired, such as Legionnaires' disease. Other rare disorders can cause general health conditions, such as heart disease and cancer. Most have no known treatment.
A rare, or orphan, disease is one that affects fewer than 1 in 2,000 people. However, there are more than 6,000 rare disorders that, taken together, affect approximately 3 million Canadians. One in ten individuals in Canada has been diagnosed with a rare disorder. Many Canadians are affected, but very few with any one particular disorder. This is a serious issue for this government.
We recognize that Canadians who suffer from rare diseases have health needs that frequently are not met, particularly, in terms of access to needed treatments. Few therapies exist to treat these diseases.
The rapid advance in medical technologies and therapies has given many Canadians new hope, but developing and providing these medical techniques, such as genetic therapies, requires enormous resources. Potential treatments have often been considered too difficult and expensive to develop because of a very small patient population.
It is often difficult to conduct clinical trials and to demonstrate outcomes because the number of people suffering the disease is too small to allow the standard scientific techniques, like random sampling, control groups and so on. Nonetheless, we cannot ignore the suffering of Canadians with rare diseases.
Recently, new drugs for rare disease have become available in Canada, but evidence on whether they work is often weak and the costs of these drugs are beyond the budgets of most Canadians. Thus, important public policy regarding these drugs is essential.
This government has not been idle on this issue. My colleagues can attest to the fact that we have already taken many steps towards improving pharmaceutical management in this country, most notably in relation to drugs used to treat rare diseases.
We are improving the way we regulate drugs as part of the food and consumer safety action plan. This includes a life-cycle approach which goes beyond a simple decision on market access and also monitors drugs once they enter the market.
Along with our provincial and territorial partners, we have been working on improving the common drug review process.
We continue to work with the provinces and territories to improve drug management, including for rare diseases, as part of our collaboration under the national pharmaceutical strategy, a component of the 2004 health care accord.
As mentioned before, the government and the hon. member for North Vancouver have been working behind the scenes to find common ground so that this motion can pass this House. We have focused our discussions on several aspects of the motion and I am happy to outline these areas for my colleagues.
First, and foremost, the government believes the motion ought to take into account the roles played by the provinces and territories with this issue.
Our provincial and territorial counterparts are primarily responsible for deciding the extent of drug coverage for Canadians. Provincial and territorial governments determine who qualifies for public coverage within their jurisdictions, what drugs qualify for reimbursement, and what portion of the costs will be covered.
They also negotiate with drug manufacturers on the prices for the drugs they choose to reimburse, including prices for rare disease drugs. They regulate prescribing and dispensing of these drugs within their jurisdictions. This is an important element of this issue, given the key roles doctors play in determining appropriate prescribing and developing clinical practice guidelines.
As such, the government believes that we must include a reference in this motion to our provincial and territorial colleagues; a fact that the hon. member for North Vancouver has listened to and demonstrated a willingness to accept.
The government also believes that Motion M-426 should note the important role of the common drug review process—a productive collaboration between federal, provincial and territorial governments that assists them in their decisions about drug coverage.
The common drug review process evaluates the therapeutic benefits of drugs as well as their cost-effectiveness in comparison with existing therapies. It also provides recommendations such as if and under what circumstances drugs should be covered under government drug plans.
As the House is aware, the Standing Committee on Health undertook an examination of the common drug review and released a final report this past December. In that report, it recognized the importance of the role played by the common drug review and it made recommendations to improve it. One of those recommendations was the creation of a public advisory board for the common drug review.
The recommendation is quite similar to an element of this motion, which suggests that we consider establishing “a multi-stakeholder advisory body, including treaters and patients, to recommend treatment access for life-threatening or serious rare disorders”.
This government believes that the common drug review should be included in any work on rare disease issues.
In our response to the committee's report, the government indicated its interest in pursuing discussions with participating provinces and territories on opportunities to appropriately involve the public in the common drug review process. Appropriate public involvement can lead to better decisions, as well as confidence in the fairness of the decision making process.
Adapting the common drug review approach to assessing drugs for rare diseases was also highlighted.
One of the more challenging aspects to the motion surrounds a reference to defining a “rare” disorder. While experts have focused their study on defining this issue, to date there is no common definition of a rare disease.
Determining what diseases count as rare and, therefore, who will benefit from any changes that governments might collectively or individually implement, is a vital step and not one to be taken lightly.
If governments adopt special approaches for rare diseases, how then do we deal with the almost rare?
For example, if diseases affecting fewer than 500 Canadians are considered rare and treated differently, what happens to the people suffering from diseases that affect slightly more than 500 Canadians?
The government feels that we need processes that can adapt to the needs of all Canadians and all diseases, and we thank the welcoming nature with which the sponsor of Motion No. 426 has received these comments.
We recognize that these diseases have few options for treatment and that available drug therapies are often extremely costly. However, in the absence of solid analysis of other ways in which we could address these challenges, we are not convinced that this motion's proposed fund is the best way to deal with this difficult issue.
The government recognizes the difficulties faced by Canadians suffering from rare diseases and acknowledges the spirit in which the motion was put forth. However, the hon. member's motion does not take into account the necessity of working with the provinces and territories and the government is also concerned that some elements of this motion are premature.
These are serious issues that need to be addressed but we must do so in way that is prudent and respects the roles of those involved.
I can identify personally in a very small way with those who suffer from rare diseases. When our son was born in Germany 34 years ago, he was initially diagnosed as having PKU disease. Although PKU disease is not that rare, it does involve severe lifelong dietary restrictions to prevent irreversible brain damage. The prospect for our son was scary but in the end the diagnosis, thankfully, proved to be inaccurate.
The motivation and intent of the motion are entirely honourable and worthy of very serious consideration. I know the hon. member for North Vancouver has a strong personal investment in this issue and that he is committed to doing the right thing. I and all members of this House applaud him for that.
We look forward to working with him and other colleagues to arrive at a positive course of action that will ease the load on families dealing with the impact of rare diseases, while still respecting the requirement for collaboration and cooperation with all levels of responsibility and authority in the delivery of effective and affordable health care to all Canadians.
Ms. Christiane Gagnon (Québec, BQ):
Mr. Speaker, I rise today to speak to Motion No. M-426, which calls on the government to respond to the challenges faced by patients with rare diseases and disorders. I thank the member for North Vancouver for making us aware of this important issue today. The lives and quality of life of many young people—most of these patients are young people—are at stake today in this debate.
This is a very important motion that sheds light on a regulatory problem that limits access to drugs to treat rare diseases, either because they are not allowed on the market or because they are not covered by provincial drug insurance plans.
Before I address the issue that is at the heart of the motion we are debating today, I would like to say that the Bloc Québécois cannot vote for this motion as is, not because of the principle behind it, but for a very specific reason that I will explain. The Bloc Québécois is in favour of the motion, but it requires an amendment, which we proposed to the sponsor of the motion, concerning how this motion applies to Quebec.
Under the 2004 10-year plan to strengthen health care, Quebec is not subject to the common drug review or the national pharmaceutical strategy. The 10-year plan to strengthen health care recognized Quebec's right to withdraw unconditionally, with full financial compensation, from any national health initiative.
I would like to reiterate certain principles set out in the 10-year plan to strengthen health care. The plan clearly states that nothing shall be construed as derogating from Quebec's jurisdiction over health.
We would have liked the Conservatives and the Liberals to agree to this amendment this morning so that we could go ahead with this motion today. Quebec has already set up a committee to look at this issue. This committee has begun looking at another way to deal with rare diseases and verify the efficacy and safety of drugs, taking into account their cost. This is the role of the common drug review. Quebec has its own drug review program, known as the Conseil du médicament du Québec, and is therefore not subject to the federal program. We need no lessons from the federal government, because Quebec is completely independent in this regard.
As such, any national strategy or policy concerning rare disorders and governing access to drugs to treat those disorders cannot apply to Quebec.
The only recommendation in this motion that can apply in Quebec is (g), which is about the drug approval process and clinical trials, issues that clearly fall under Health Canada's jurisdiction. We may be able to build consensus on this motion based on the proposal in (g).
That being said, as everyone knows, in the spring of 2007, the federal Standing Committee on Health conducted an in-depth study of the CDR, the common drug review. The committee then submitted a report containing five recommendations. The Bloc drafted a dissenting opinion to reiterate its preference that the national strategies for rare disorders not apply to Quebec. Instead, we asked the federal government to establish a specific approach to the evaluation of drugs for the treatment of rare disorders.
Last week, we received the government's response to the report. Even the government was quite vague with respect to recommendation 5, which called for a real policy on rare disorders. We are very disappointed in the government's response.
Here is what the government said in its response to the committee's report on the common drug review.
The government supports the “idea of exploring options”. It also recognizes that there might be “merit in exploring approaches to assessing these drugs as one of the first steps”, and in the end, it said it was interested in “pursuing discussions”.
That gives us some idea as to whether the government will support this motion or not. The response is pretty lukewarm and bears little resemblance to the proposals in the member for North Vancouver's motion. It is highly unlikely that the government will support this motion. I would be very surprised and also very pleased if the government decided to move more quickly and make the rare disorders policy more proactive and more considerate of the circumstances surrounding rare disorders.
As I said earlier, the Conseil du médicament du Québec has already begun working on a draft of the evaluation criteria for drugs to treat rare disorders. There must be specific criteria because rare disorders are not like other, more common disorders.
We know that in the United Kingdom, for example, 1 in 50,000 inhabitants has a rare disease, while here it is 1.1 in 10,000 inhabitants. That is why, in the approach set out in the motion—and this is what Quebec focuses its research on—we have to take into account the fact that clinical trials have been conducted in other countries in addition to those done in Canada and Quebec. Then we could have a better sample from a larger population base, instead of basing research only on the population of Canada. In fact, as far as certain diseases in certain countries are concerned and other diseases in Canada and Quebec, we could have better sampling and a much broader assessment of the safety and effectiveness of the drugs. That information could be used in approving the cost of drugs.
That is where the CDR or common drug review comes in, since it could recommend that a drug be covered. Quebec is not subject to a national strategy because it has its own drug review program.
Often the reason provided for not covering the payment of a drug treatment for a rare disease is that it is too costly and the patient base suffering from the disease is too small. The cost is astronomical.
In my opinion, we have to look at the situation from a human perspective. When we can offer not only a better quality of life to people with rare diseases, but also greater longevity, this certainly touches us emotionally. By agreeing to pay for a drug, regardless the cost, we are offering patients a better quality of life.
I know the hon. member presented this motion because people very close to him have suffered from a very rare disease and access to drugs was not possible.
I know that the hon. member is doing a good job raising awareness within his party and that is why I sincerely hope the Bloc Québécois amendment will be accepted in order to exempt Quebec from the strategy we are debating this morning. Nonetheless, we could support a national strategy for all of Canada, except Quebec, just to show some openness.
In Quebec, I met with one of the members of the committee, and he was saying that this issue has to be addressed differently and there should perhaps be more progressive reviews that take into account different factors. Then we might be able to approve the drugs for rare diseases.
Ms. Judy Wasylycia-Leis (Winnipeg North, NDP):
Mr. Speaker, I am pleased to participate in this very important debate on a motion submitted by my colleague from North Vancouver.
This motion represents an important discussion for the House of Commons and it is relevant for many Canadians. Although we are talking about rare diseases and treatment for such rare diseases, when we add up the numbers, in fact we are talking about a significant proportion of the Canadian population. Well over 10% of Canadians today are affected in one way or another by this issue and by the absence of adequate treatment and drug coverage for those who are affected by this very serious issue.
I want to thank the member for North Vancouver for bringing this issue to this House. It was inspired by his personal experience. His story of his grandchildren is tragic and devastating. We take courage in his ability to take some hope from these dire circumstances to change the world for others, to make this world a better place so that the kind of experience he and his family has had to endure will not have to be repeated by so many over and over again.
All of us bring our personal experiences to this House and they intersect here in the political world. It is here at that intersection where issues are most poignant and where they become relevant in a very deep, personal way for action.
I too bring to this House a personal experience on the issue of rare disorders. I have talked many times in this House about my son, Nicholas, who is now 23 and suffers from a very rare disorder. In fact there are only 11 boys in the world with his particular genetic disability or disorder. It is called band heterotopia, or double cortex syndrome. It causes uncontrollable seizures, profound intellectual disability, and of course many behavioural issues.
We became aware of this when Nick turned three. He started having seizures and he was diagnosed as simply having epilepsy. We were told not to worry, that everything would be okay. As the years passed and the drug trials continued and the tests persisted, we soon became aware that we were dealing with something very difficult. To this day we still struggle with drug therapies to bring his seizures under control. We keep hoping and praying that there will be a discovery that will help his particular situation.
In our case, we have been blessed with a wonderful medical system in Manitoba, with good access to neurologists. Obviously we have tried every drug in the world that exists. We have also used the exceptional drug program to access drugs that were not approved. We continue to be blessed by a system that is sensitive, insofar as it goes to the particular situation facing Nick.
However, we always hope that there will be the resources to find a cure, to find a solution, to find some remedy to help him, just as my colleague from North Vancouver hopes that something can be done to prevent the tragedy he has had to go through with the death of his grandchildren.
My other colleagues bring similar stories to this House. My colleague from Acadie—Bathurst is fighting for a family in his constituency. It is the case of a two year old girl in Paquetville, New Brunswick, suffering from xeroderma pigmentosum, a condition in which she cannot be exposed to sunlight. Cancer development is triggered by sun exposure and she has already recently suffered some new developments pertaining to cancer.
She lives in the dark all the time and can only go out at night after sundown. She requires special windows. She would need a special dome to protect her outside to play normally, and of course this is a very costly method of protection. Her parents have had to leave work to care for her. The family continues to struggle to ensure that this little girl has a future.
My colleague from Windsor—Tecumseh mentioned to me today that Szymon Cajmer, the little boy who is mentioned in the Globe and Mail story, is in his constituency of Windsor--Tecumseh. When this little boy was six years old, he was denied entry into a clinical trial to test the only drug to treat his rare disease because his lungs worked too well. Now at age 11 he suffers from severe hearing loss and easily loses his breath. The drug he did not qualify to receive years ago, idursulfase, has since been approved by Health Canada to treat his disease, Hunter syndrome, but now Szymon cannot obtain the enzyme replacement therapy for a different reason. It is funded in British Columbia and Alberta but not in Ontario.
With this range of personal stories we have a better understanding of the problems facing many Canadians. We need a strategy to help Canadians who are faced with rare disorders.
My colleague from North Vancouver has presented a comprehensive motion that does not prescribe exactly what the federal government ought to do, but recommends that the government consider various steps to ensure that a strategy is put in place. The first recommendation my colleague has made is that there be a strategy, that we define what rare disorders are, that we do what the United States has done at least, which is to give it a word, give it definition, give it meaning, and then develop a strategy to act on those issues.
I commend my colleague for that. I certainly support in general his motion. We can quibble with some of the aspects but that is for us to sort out as we move this along.
It is the responsibility of the federal government to take the wisdom of the experience of the member for North Vancouver and the personal experiences of so many in this House and build on them and come up with solutions. We do not want these rare disabilities and disorders to become a jurisdictional football, as is the case of the family living in Windsor—Tecumseh.
It is wrong that provinces with more wealth and ability to pay for expensive rare drugs can do so while others cannot. We need a national strategy.
The government could immediately recommit itself to a national pharmaceutical strategy which is sitting on a shelf somewhere gathering dust without action and without purpose. We need a minister of health and a Government of Canada that is prepared to get back to the drawing board and recognize that this country needs a national pharmaceutical strategy with a national formulary that would have a specific fund allocated for people suffering from rare diseases as a part of it. Otherwise, it will never be possible to address those particular conditions. They are too specific, too small, too narrow to be part of a common drug formulary. That is clear.
We are not suggesting that every province reinvent the wheel and come up with funds from scarce resources in order to cover these particular rare disabilities. We need a central clearinghouse. We need a place where those families with family members who suffer from rare disabilities, disorders and diseases can have some hope. These families are facing serious challenges in terms of drug coverage. Drugs may not be thoroughly clinically tested, may not be thoroughly without side effects, but might provide people facing these circumstances with some hope that they might be able to enhance the quality of their lives or even add to their lives. The government must recognize that and put a plan in place.
There is no excuse for the government not to recognize the importance of having a national drug coverage plan that would ensure that all Canadians would never have to make the difficult choice between paying for needed drugs or buying food for their family and paying the rent.
There needs to be a planned approach on all fronts. Serious discussions are needed with the brand name drug companies that are still spending very little of their profits on research and development which would help people with rare diseases and would be of significant benefit to Canadians in these circumstances.
We need to convince the drug companies that spending 90% of their budget on marketing and advertising does not make sense. They have to invest that money in helping Canadians, whether it is in terms of rare disorders and disabilities or whether it is in terms of a universal program that would help many across all jurisdictions in this country.
I commend the member for North Vancouver for bringing this motion to the House. I will support him in his efforts. We look forward to strategies and recommendations and courage from the federal government on this issue.
Mrs. Susan Kadis (Thornhill, Lib.):
Mr. Speaker, I am very pleased to rise today to address and fully support Motion No. 426, a motion to address the lack of a rare disease drug policy in Canada.
Health care is always among the top concerns of residents in my community of Thornhill and, of course, all Canadians. From access to treatment to quality of care, residents in my community and all across Canada want a health care system that is there when they need it and that is inclusive.
I am very proud to speak in support of the motion by my colleague from North Vancouver, which has been very much inspired from his own very difficult, sad family experience and has been used in such a positive fashion to help others in the same predicament. I am very proud to support all of his efforts. We were all very moved today to hear the story that he shared, as well as other members' personal experiences.
His motion would move Canada toward a system where Canadians with rare disorders would receive the same standard of care as patients with more common disorders. As a breast cancer survivor for 16 years, if I had not had the drugs available to me at that time, I cannot imagine what would have happened to me. The same is true for many others. I can well understand all those who need to be part of our system. When we consider that other countries are doing so, it is vital that we move together. I am happy to see there is some consensus.
As a member of the health committee and part of the common drug review, it was very clear from the organizations and advocacy groups that this issue has not been adequately addressed. The time has come to do so now.
A telling example was brought to the committee's attention, and it was particularly disturbing. It revolved around Nexavar, a drug to treat kidney disorders. It was rejected by the CDR essentially because it was too effective. The interim data from clinical trials of Nexavar were producing stellar results and the U.S. Food and Drug Administration said that patients in the control arm of the study should be allowed to enter the treatment arm. It was the obvious ethical choice.
Many patients in the control arm of the study, obviously enthusiastically embraced the opportunity to receive this new, groundbreaking drug, but because most U.S. patients had left the control arm, there was not enough data left to satisfy CDR requirements. The placebo data was determined to be statistically insufficient and the drug was rejected on these grounds. Clearly, the placebo data would not be forthcoming and access to Nexavar, available to those suffering in the U.S. and Europe, was severely hampered in Canada.
There is not much motivation in these kinds of cases for Canadian biotech companies at this time to simply duplicate a clinical trial that has already proven to be successful elsewhere. That is one of the other dilemmas.
Because data from trials conducted in other countries is not accepted by the CDR process presently, patients are often forced to wait years for clinical trials to conclude in Canada while patients in other countries, as I have said, have already had access to the drugs being tested. In Canada, patients with rare disorders are forced to wait for no logical reason and to their detriment, except for the rigid requirements presently, in this case, in the CDR process.
Drugs that are often covered by private drug plans to treat conditions such as gigantism, Fabry disease, Fabrazyme, MPS, Gaucher disease and kidney cancer have been rejected by the CDR and are not accessible to those with public drug plans.
Who is being denied treatment the most often? The sad reality is that in many cases it is Canadian children who have rare childhood disorders. In fact, it is those children and their families who are being denied access to treatment. Even in cases where drugs for rare disorders have been approved through Health Canada's progressive licensing framework, often CDR will stand in isolation and essentially reject the conclusions of Health Canada and numerous international studies.
Motion No. 426 asks the government to consider the establishment, and rightly so, of centres of reference for specific rare disorders that would be comprised of national and international experts who would develop the criteria for treating patients based on scientific evidence and patient impact. They would provide ongoing surveillance into the real world safety and effectiveness of these treatments on individual and group bases so that we could consider supporting internationally accepted standards for the conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations.
By expanding the CDR and developing a separate process for the consideration of rare disorder drugs, we can even the playing field as so many other countries have already done, be it in the U.K., the U.S. The examples are everywhere. It is time for Canada to follow their lead and embrace the international framework model that has proven to be so successful.
Many countries, such as France and the U.K., and others in the EU have successful models of separate bodies that consider treatments for rare disorders. Canada must take a hard look at the cooperation that is happening in these countries as there is much it can learn from their experiences. The time has come, again with our collective resolve, to do so.
It was suggested to our committee that changing the CDR process to allow for the pooling of limited Canadian data rejected by the CDR with rare disorder patient groups outside Canada would likely set the stage for more approvals of treatments of rare disorders, which is what is necessary.
The testimony the health committee heard on this issue was compelling. When our final report on the issue was tabled, included in it was the recommendation, as referenced today by the government, that the government look at options. We are happy to see the government has accepted that recommendation favourably. Again, we will very much be looking to see this is implemented and moved forward in a very expeditious fashion. Often we hear there is interest by a government, but it does not always moved on it. In this case there is no other acceptable option but to move on it.
Clearly there is consensus, as I have mentioned, including the government and the health committee, that a new approach is required and that the one currently taken by the Canadian Agency for Drugs and Technologies in Health in approving drugs for rare disorders is not yet there.
When the government tabled its response, I was pleased it was received well. I very much look to see that this has everyone's total support and that we will see action in this area, which is so vital. It is predominantly affecting children and other Canadians.
I fully support this, and again commend my colleague for moving on and taking leadership forward. We want to see this happen not in a long period of time, but in the very near future so those suffering currently will feel they are part of our health system in every fashion and that they do not have to look elsewhere.
Mr. Gary Goodyear (Cambridge, CPC):
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.