Thank you, Madam Chair.
On October 3, 2012, the Minister of Health announced the development of a modern framework for orphan drugs. These are drugs used to treat rare diseases. This opens the way for increased Canadian research and development of these drugs and for improving Canadian patient access to treatment.
It's a pleasure to appear before the House of Commons health committee to explain more about how this proposed regulatory framework would benefit Canadians affected by rare diseases.
There are dozens of well-known diseases in Canada. They're well known because they affect the lives of many people. There are charities, associations, and support groups for people suffering from those diseases.
But there are thousands of people suffering from other diseases that most of us have never heard of. That’s because they're so rare, they can affect fewer than 12 people in the country. At any given time, even internationally you can have very small numbers of patients.
While some of the rare diseases may affect only a handful of Canadians, in all, hundreds of thousands of Canadians are dealing with these conditions, and they need effective treatments. In Canada it's estimated by some that one out of 12 Canadians is affected by a rare disease.
The diseases are often linked to genetics, as Dr. Wong-Rieger suggested. They can have a very early onset. They can be diagnosed during childhood, often very young. And they're very difficult to study, to treat, and to understand how to regulate because of the small size of the population. It's scientifically difficult to tell how a therapy would work in that population and to pick up safety information.
Rare diseases can be serious chronic conditions—they last throughout the lifespan of the patient. They can be seriously debilitating or life-threatening. They often are life-threatening.
The drugs that demonstrate promise for treating these diseases are often referred to as “orphan drugs”. It's a term that has developed globally, and the United States started that.
Today when a patient with a rare disease needs access to an orphan drug, because we don't have rare-disease regulations currently, it's not available in Canada. The patient’s doctor, often a specialist in that disease, will obtain it through our special access program at Health Canada. But every time the specialist uses this option, he or she has to take time to write out a form to request in writing the allocation of the drug to the patient, and then the department can contact the manufacturer to release the drug to that patient. While this works—and it's what's operating right now—it is time-consuming, and each decision is made on a case-by-case basis. It's an unnecessary burden on the health care system.
Health Canada has also approved some orphan drugs as new drugs under division 8. That's our normal review provision for commonly marketed drugs. While this path has worked in the past as well, it's limited because it was not designed to address the unique challenges of rare diseases. It really doesn't pay attention to the data requirements, for example, that we would tailor in a new framework.
What is needed is a new regulatory framework designed to gather information used to treat small, vulnerable patient populations, specifically tailored to facilitate the development and approval of drugs meant to treat rare diseases, an orphan drug framework.
An orphan drug framework will level the playing field for Canadian rare-disease patients so they, too, can share in the benefits rare-disease patients in the U.S., and in many European countries with such frameworks in place, already have. In those countries and those jurisdictions, they have a lot of experience with rare diseases by now, and they've been very helpful in teaching us about that.
The orphan drug framework will allow Health Canada to approach the approval of these drugs in a flexible manner, recognizing that greater uncertainties may exist for orphan drugs, given the complexities of the diseases and again the small size of the patient population.
First, we're aligning with trusted international counterparts, the European Medicines Agency and the U.S. Food and Drug Administration. And I will say that they're very generous in their advice, opening up what has worked for them and not jurisdictionally. Both have had frameworks in place for orphan drugs for over a decade, and in the case of the States, more than two decades. International alignment of Canada’s regulations will allow our scientists, Canadian scientists, researchers, and regulators, the ability to pool increasingly limited resources to help us to better understand these complex diseases and their treatments.
Second, drawing on the idea of life-cycle management, the framework will also allow us, the regulator, to more closely follow the safety and effectiveness of these drugs once they're brought into the market. This will be done by ongoing post-market data collection relating to the drug’s safety and efficacy or effectiveness. This innovative approach complements the current pre-market focus with a more balanced, dynamic, and fluid set of regulatory interventions, and it will better serve the patients’ needs while maintaining a strong safety oversight.
We want to make sure the design of our approach is very patient-centred. The patient's voice needs to enter into that regulatory process, so we want to enable patients to have a voice throughout the decision-making.
Because the proposed regulations will align with international frameworks, which is very important, it will be more commercially feasible for pharmaceutical companies to develop and then bring their drugs to market in Canada. This is because the international alignment gives drug manufacturers a more predictable, operationally less burdensome path to follow. What they follow in other jurisdictions will be typically sufficient here as well.
A more predictable regulatory path, with clear research requirements and flexibilities to enable international collaboration, also creates space in which Canadian research and innovation can thrive, and we're trying to pay attention to this in designing the framework. The framework will also provide for greater transparency to improve the gathering and sharing of information among patients, health care professionals, researchers, payers, and international regulatory partners.
Improved transparency is expected to result in more informed, evidence-based decision-making. It also brings with it increased public confidence in evidence-based research and the safety of research participants. This is because the broad sharing of research data accelerates the research, fosters data integrity, and increases accountability.
Most of all, the new framework will benefit Canadian patients with rare diseases by improving access to new and existing drug therapies that might have been harder to get or not available at all without these new rules.
As part of our work to better understand the impact that the orphan drug framework will have on Canadians, Health Canada has also met with many Canadian researchers, clinicians, rare disease representatives, and patients. I've done this often in conjunction with CIHR and other valuable domestic colleagues.
In response to what we heard, Health Canada, with support from the Canadian Institutes of Health Research, has launched Orphanet. There's been a lot of effort around that. It's a very important online resource, a global resource that offers a directory of specialized information for people with rare diseases and health care service providers. It includes information about specialized clinics, medical laboratories, clinical trials, and registries. Together the new framework for orphan drugs and Orphanet will create a better environment to increase access to information for patients with rare diseases and the new treatments coming onto the market.
In closing, the proposed framework is in the final design stages. We will soon be broadly targeting public consultation. Comments and feedback would then be gathered during consultation and incorporated into a final version of the proposal.
Thank you for inviting me to appear today.