Thank you. Thanks to all my fellow speakers who have come today.
My name is Doug Coyle. I'm a professor at the school of epidemiology and public health at the University of Ottawa. I'm a health economist and have worked in this research area for the past 28 years. I have no conflicts of interest to report regarding this matter.
Previously, I've been a member of the Ontario Ministry of Health and Long-Term Care's committee to evaluate drugs and the drugs for rare diseases working group, as well as the Canadian expert drug advisory committee of the Canadian Agency for Drugs and Technology in Health, or CADTH. In all of these committees, I've helped make recommendations on the funding of new pharmaceuticals.
Thank you for giving me the opportunity to present my views today. My research and my teaching is driven by my passionate belief in the necessity of a publicly funded health care system. This is based on both the fundamental belief that equality in access to health care should be a right, and that the nature of health care as a commodity is such that provision through a market-based system is inefficient. The latter should really never be underestimated.
Despite my strong beliefs in the need for a publicly funded health care system, I feel my presentation today may be contrary to other speakers.
I've been asked today to present my views regarding barriers to access to treatment and drugs for Canadians affected by rare diseases and disorders.
Our health care system is under increasing pressures. New, more costly interventions have become available, and the clamour for their adoption is great. It is important that we have a process to assist decisions related to which technology should be funded, based on the values and wishes of Canadians. Given this, I believe that fair, equitable and transparent processes for making the complex and difficult decisions with respect to reimbursement for health care interventions are a necessity to ensure the sustainability of our health care system.
Although the processes currently in place are a start, they do not sufficiently address the challenges facing our health care system today. I suggest the fundamental principle by which we need to develop such processes is a need for fairness, as difficult decisions on what should and should not be covered need to be made through a process that recognizes the need to treat people equally—that is, a health benefit gain for any Canadian should be considered of equal value. Thus, the aim of our health care system would be to increase the overall health of the Canadian population.
However, given our limited budget for health care, not all new technologies will increase the overall health of the population. Despite claims, most if not all new technologies do not save money in the long term. Thus, we need to assess whether prices given for new technologies are justified, given their potential benefits.
We can assess how efficient the Canadian system currently is at producing improvements in population health. Current estimates suggest that we spend about $30,000 to $50,000 for every additional year of healthy life gained. That could be the benchmark to assess whether a new technology is worthwhile. Given our constrained budget for health, in deciding to fund technologies that cannot produce benefits at that price, the necessity is that we are therefore reducing the potential health of the Canadian population. By this, we are implicitly deciding that the health benefits to one group of Canadians are of greater value than similar benefits accrued by others.
Numerous surveys have been conducted both in Canada and worldwide that have explored whether the general public is willing to accept reduced levels of health across the whole population for improvements for specific populations—that is, are these populations for which they believe the health benefits are gained of greater value than other populations? The results of the surveys have repeatedly shown that the public does not value rarity as a sufficient reason to give greater weight to the health benefits accrued. Thus, to reflect societal values, technologies relating to rare diseases should be evaluated using the same criteria as technologies affecting more common conditions.
I'd like to present again the case of Soliris to this committee. Soliris is a drug for the treatment of a rare disease called paroxysmal nocturnal hemoglobinuria, or thankfully, PNH. It is a rare blood disorder. Soliris is effective. It reduces the instance of thromboembolism, the main cause of mortality in this disease, and it reduces the need for blood transfusions, a major management cost of the disease.
However, Soliris literally costs $500,000 per patient per year. An independent analysis is that funding Soliris could only increase the overall health of the Canadian population if a price reduction of 98.5% were achieved. Although Alexion pharmaceuticals has been required to reduce the price of Soliris, it is unlikely to have been to this extent.
We are often told that funding for rare disease will have little impact on health care budgets and there is a reason for being more lax in funding decisions. However, the funding of Soliris at the original listed price would cost more than $100 million per year if all those eligible received treatment. These resources could provide many other health care services to Canadians that would produce much greater health benefits.
Thus the decision to fund Soliris in certain provinces, even with the reduced price, should not be viewed as fair. Given the constrained resources facing our health care system, funding Soliris has led to many thousands of Canadians not receiving the health care that would have given them benefit. The overall health of the Canadian population has therefore declined as a result of such funding decisions.
There are many other examples of drugs for rare disease that are funded and for which the decision to fund will have resulted in a reduction in population health. More recently you may have heard of the coverage of the issues relating to the funding of the drugs Orkambi and Kalydeco for cystic fibrosis. There has been much media coverage relating to the inconsistency of coverage of these products across Canada. Analysis of the long-term benefits of these products by CADTH suggests that for their funding to result in an increase in the health of the Canadian population, the price of the product would have to be reduced by at least 90%. Not funding Kalydeco or Orkambi has been a responsible decision by the provincial ministries, in that funding these products would deny other Canadians the health care they require and that health care would provide greater benefits to the Canadian population as a whole.
An argument we typically hear is that Canada should be rewarding manufacturers of innovative medications. We should, however, support innovation by ensuring that funding is given to those technologies that represent value for money—that is, that they provide greater increases in the health of the Canadian population. It is hard to argue that a product that if funded will lead to reductions in the overall health of the Canadian population is truly innovating.
With the discussions in Canadian media coverage of issues regarding funding of drugs for rare disease, little focus is placed on the exceptionally high acquisition cost of these drugs and the role of manufacturers in creating these difficult situations, as evidenced by Dr. Midgley. Treatment with Orkambi costs $300,000 per patient per year. An interesting article recently explored the difference in the media coverage within the U.S. and Canada with respect to Orkambi. In Canada, the focus is typically on individual patients' fights for coverage, while in the U.S., the focus is on financial aspects relating to the product, the high level of sales and the financial performance of the manufacturer of Vertex pharmaceuticals.
Niche manufacturers of drugs for rare diseases are clearly not hurting. This is increasingly becoming the focus of the larger pharmaceutical companies, too. This has led to increasing questions about the pricing of such products and recent suggestions that having the Patented Medicine Prices Review Board bring considerations of impact on overall health of the Canadian population into price setting should be encouraged.
I strongly believe that before committing to fund products with such high annual costs, greater work is required to assess the reasonableness of the prices charged. A further point I would like to raise is the need for a more comprehensive approach to the funding of all health care interventions, not just those for which there is increased funding pressure due to the activities of a commercial sponsor.
When holding discussions within the rare disease space, much of the focus is on pharmaceuticals. This is frequently the primary focus of patient advocacy groups. However, the current focus within the health care system on pharmaceuticals leads to funding decisions that typically favour such technologies over alternative health care interventions, which may provide more benefit but where profit is not a driving factor for those advocating for the coverage. We need to consider all the technologies that are out there. Many existing technologies are underfunded, yet have evidence to support their effectiveness and cost-effectiveness. Many of these do not have commercial sponsors.
Given the changing demographics of our country and the increased long-term need for home care and long-term care, the continued focus on pharmaceutical coverage is in many ways missing the major problem facing our health and social care system today. Care through hospices, home care services and nursing homes suffers from a lack of commercial interest in promoting them, and they are often overlooked by those groups advocating for health care. There is a lack of funding for research to highlight their benefits and there is limited lobbying because of the lack of a commercial sponsor.
To summarize, I would like to reiterate the points that follow.
For a publicly funded health care system to be sustainable, we must have decision-makers who are willing to make the difficult decisions not to fund specific new technologies. By failing to make such decisions in a consistent and fair manner, decision-makers are leading to the reduction in the health of the overall population of Canadians, and, I am afraid—to be direct—just not doing their job.
Fairness should be the key principle in choosing which technologies to fund, and funding technologies that deny the availability of other technologies that provide more benefit is unfair and not consistent with societal values.
Today's focus on rare disease tends to lead to a refined focus on the coverage of pharmaceuticals. However, to reach our objectives of increasing the health of the Canadian population, it's essential that sensible and rational decisions be made on a consistent basis with respect to the funding of all health care interventions, not just those with commercial interests promoting them.
Thank you very much for your time.