Good morning, Mr. Chair. Thank you very much for the invitation to speak with you today. Just by way of disclosure, I'm the principal investigator in a CIHR team grant evaluating policy and reimbursement decisions around rare disease. I'm also a member of the B.C. Ministry of Health advisory committee for expensive drugs for rare diseases.
I'm going to talk to you today about my perspective and my experience specifically around orphan drugs and rare diseases.
In my discussions with provincial payers, particularly in B.C. as that's where my experience is, they have raised four key areas of concern, particularly around evidence, price, access, and communication and transparency of decisions in general, and specifically as these relate to reimbursement decisions for drugs for rare diseases.
We are in the midst of a paradigm shift and really a disruption in drug development, where we're moving from the blockbuster model and biologics to more niche products, targeted products around orphan drugs and rare diseases. This disruption has resulted in exorbitant costs of many of these drugs for rare diseases, in the order of millions of dollars per year per patient, for lifelong treatment. Now, with others in the pipeline, we're seeing prices in the order of $2 million to $3 million potentially, and the cost just seems to be continually pushed higher.
With new technology and incentives, the number of drugs in the pipeline with potential orphan indications continues to increase, which is a good thing, offering new treatments for our patients, but which will obviously bring further pressures on our system. Given the costs, it's obviously not feasible for any Canadian to be expected to pay out of pocket for these drugs if they are not insured benefits. Therefore, I think we also need a bold paradigm shift or disruption in our policy and reimbursement decision-making.
Now in Canada we know we have the common drug review, and more recently, the pan-Canadian pharmaceutical alliance. The common drug review was implemented with the specific objective of providing evidence of value, efficiency, and consistency of evidence evaluation across the provinces. The pan-Canadian pharmaceutical alliance was initiated with the objective of having a strategy for collective negotiation on drug prices by the provinces following CDR review. The theory is that this would result in greater cross-country equity. However, such is not always the case. Even in cases where pricing and product listing agreements haven't been reached with pharmaceutical manufacturers by the pCPA, some provinces have chosen to fund some drugs, resulting in inequity of access.
In a recent study of 2,600 Canadians, we asked Canadians what they felt were the most important considerations related to funding of new drug therapies. The top five considerations were the effect of the drug on quality of life, the effect on length of life, the safety of the drug, the ability of the drug to really work, and the severity of the disease that it's meant to treat. Those were the top five related specifically to the drug and the disease.
The next most important factor, however, was equity of access for minority populations, and in a more recent study, we found that equity across provinces was also very important. So despite the common drug review and the pCPA, we know Canadians do not have equal access to all treatments. However, for common diseases or even cancers, there are generally alternative therapies such that most patients are not necessarily left completely untreated, notwithstanding some of the comments we just heard this morning about the ability to pay and about the ability to get coverage. This is specifically speaking about equity.
For many rare diseases, however, there is generally only one therapy. Thus with different coverage decisions across provinces, there is truly differential access to treatment. I'm not suggesting that all treatments should be available to all patients across all provinces, whether they be for a rare disease or a common disease, but that the development of a national pharmacare program would prevent this from happening. All patients in Canada would either get access or no patients would get access, based on a transparent, consistent, evidence-based decision-making process, thus, evidence-based equity of access across all provinces.
We already have evidence of such a program having worked for a rare disease, specifically Fabry disease, with the Canadian Fabry disease initiative or CFDI. This initiative was conceived due to the inability of Nova Scotia to pay, given the high prevalence of Fabry disease in that province.
This is an initiative that, as I understand it, was initially funded as a partnership among provinces, the federal government, and the pharmaceutical industry, with two primary components: drug procurement for the entire country and data evaluation, data collection, and the development of a patient registry. As a result, we have an example of equal access. Only eligible patients receive treatment, based on guidelines and excellent data on the treatment effects and the natural history of Fabry disease, which contributes significantly to reducing the trepidation and uncertainty around treatment coverage decisions.
This initiative, I believe, could act as a model for other rare diseases as part of a national pharmaceutical strategy, given that this model provides a data collection platform to inform research, evaluation, and decision-making and supports evidence-based decision-making. It supported procurement and pricing negotiations. Also, in this environment, I think it could provide an opportunity for notice of compliance with conditions or reimbursement with evidence development, which we know lots of people are talking about but which we really haven't seen implemented in any situation yet.
This also speaks to what is included in the proposed Canadian orphan drug framework, specifically a post-marketing authorization plan, which I feel is imperative, given the limited evidence on the efficacy of these drugs when being reviewed for market authorization. Although I have been skeptical about how this could occur in the current regulatory and reimbursement environment, given the separation between federal and provincial responsibilities, a national strategy with an active post-marketing authorization pharmacovigilance plan would provide a framework that supports the growth of the evidence base. Economies of scale of a single system could be implemented across multiple rare diseases, resulting in system efficiencies. It could also provide a framework or a platform for international collaboration, and of course a national platform for price negotiation.
In closing, a national strategy for reimbursement, which could be the start of a national pharmacare strategy, would support equity of access or non-access, depending upon the evidence; system efficiencies on multiple levels; and potential earlier access to therapy for patients, given in an environment to support notice of compliance with conditions or reimbursement with evidence generation. It would support evidence-based, consistent reimbursement policy and decision-making, spending only where spending is warranted, and improved transparency and communication around systems and reimbursement decisions.
I would like to thank you, once again, for inviting me, and I hope I have provided you with some insightful comments for your deliberations.