We applaud the global multisectoral collaborations coming together to combat COVID-19: to treat it, to find a cure and to prevent future infections. We're especially heartened by the public-private partnerships, which are essential for getting to successful interventions as quickly as possible.
However, in the same way that COVID-19 has exposed the inadequacies in Canada’s health care system, the experiences of rare disease patients in this time of COVID put in stark relief the lack of a comprehensive integrated approach to rare diseases pre-COVID.
While each rare disease affects only a small number of individuals, there are more than 7,000 rare diseases, which, together, affect one in 12 or nearly three million Canadians. Many of these conditions are associated with lifelong, debilitating symptoms affecting not only the patients, but also those who care for them. For Canadians with rare conditions, as you've heard for cancer and for lung disease, the fight against the disease doesn't stop during a pandemic. Indeed, in many cases, as you've already heard, it has become even more challenging. We have received numerous calls, requests and emails from patients trying to manage with their disease during this time.
In early April, we sent out a survey to our patient community to learn the impact of this. We had about 300 responses in just a couple of days. We were frankly shocked by the extent to which the lack of access to health care and other services is affecting them, and the seriousness of the consequences.
I'll give you a snapshot. Half of the respondents said they had experienced difficulty receiving medical care, such as delayed or cancelled surgeries, no blood work, limited access to dialysis and no physiotherapy. One patient said, “The surgery that is not happening would resolve a problem, in the meantime I am not functional and am bedridden and in a lot of pain.”
Second, about two-thirds were concerned about seeking health care because of the fear of contracting COVID-19. One respondent said, “I've been told should I contract COVID-19 I would be low on the list for treatment due to shortage and [a philosophy of] 'survival of the fittest.'”
Third, half said they could not access rehabilitation or critical services, including personal support care: “House bound/extreme mobility issues/no one able to help because of fears & stay at home in effect.”
About 40% could not access their prescribed medications, not because of drug shortages, but because of logistical issues in health care or pharmacy services, or lack of response to special access requests.
In fact, in a separate survey to the pharmaceutical manufacturers, CORD did receive assurances that the Canadian drug supplies were not in jeopardy and that steps were being taken to meet future supply needs and even to arrange alternative treatment sites if patients were getting infused. Furthermore, we were assured that ongoing clinical trials would not be interrupted, with adjustments made for alternative treatment sites, monitoring and data collection, if necessary.
In summary, it may seem self-evident that we should not neglect patients with serious chronic conditions while we battle a new disease, however rampant it is. Unfortunately, our survey and the calls to our infoline paint a stark and disconcerting picture of limited, delayed or denied access to testing, medical services, surgeries, rehabilitation therapy, supportive care and medicines, all of which inevitably put patients' lives and well-being at risk.
The underlying problem we come back to is that Canada has never approved a rare disease strategy, despite having national strategies for cancer, diabetes, cardiovascular disease and mental illness. Indeed, Canada is the only developed country in the world without an official national rare disease strategy.
Five years ago, in the House of Commons, CORD launched Canada’s rare disease strategy, which we had put together and which was developed and endorsed by leaders from all sectors. The strategy outlined five goals: improving diagnosis; providing for expert care and centres of excellence; ensuring community support, including patient organizations; access to treatments; and support for research.
While the strategy has yet to be endorsed nationally, it has nevertheless served as an important framework for a variety of important initiatives, including steps toward the development of provincial plans by Ontario and Quebec. However, it is clear that we cannot adequately and effectively address rare diseases through stand-alone provincial strategies and other piecemeal initiatives any more than we could conquer COVID-19 if each province were working in isolation. Rare disease needs national commitment, resources and leadership, particularly in this era of COVID-19.
Of course, I would be remiss if I did not acknowledge that the rare disease community is very grateful to see in the 2019 federal budget a $1-billion funding commitment to develop a rare disease drug strategy. Ideally, this would dovetail with the supplemental process proposed by the expensive drugs for rare diseases working group of the provinces and territories. However, I have to reiterate that in order for a rare disease drug strategy to be effective, it has to be imbedded in a comprehensive rare disease strategy.
Moreover, I'm also compelled to point out that the greatest threat to the potential benefits of a national rare disease drug strategy is the Patented Medicine Prices Review Board's regulatory changes that are scheduled for implementation on July 1, 2020, despite the lack of broad stakeholder consultation. This will affect the rare disease community probably more than any other.
To put it in context, the PMPRB changes, in particular the application of the economic factors to set maximum drug prices, would put some chronic disease patients at much greater risk than COVID-19. Ironically, if there were a breakthrough drug therapy for COVID-19, it would be unlikely to meet the new PMPRB pricing restrictions.
We obviously don't believe the government would reject a life-saving drug for COVID-19 on the basis of the PMPRB economic factors. We simply do not understand why the government is seemingly allowing the PMPRB to deny access to life-saving and life-enhancing drugs for rare disease patients.
I will conclude with two key learnings from our survey results, what we have identified and what we would like to have from the federal government.
The first is that while you deal with the crisis, don't sacrifice those with other health care needs. Set up a parallel team to identify, prioritize, triage and resolve the needs of those with chronic and other health care conditions. This extends, obviously, beyond the rare disease community, but it really does impact those with rare diseases disproportionately.
The other big learning is that Canada needs an innovative pharmaceutical industry. Companies in Canada are stepping up to ensure adequate supplies of emergency drugs and medical devices, and are partnering with researchers to develop new tests, therapies and vaccines for COVID-19. At the same time, they are ensuring that Canadians with rare and common conditions have continued access to the drug tests and other technologies that are needed. Perhaps this is truly the opportunity to develop effective public-private partnerships.
The problems experienced by the rare disease patients and families were not directly caused by COVID-19. Sadly, it has taken a pandemic to bring the pervasive deficiencies and dysfunctions in our health care system to the surface. We urge governments to address those issues now. We in the patient community are ready and wan to work with you, along with our clinicians and our researchers.
I would just add parenthetically that CORD has not applied for any of the support funds because we are, quite frankly, a very lean organization. We know how to work with limited resources. We have instead hoped that the funds would go to our hundred-plus patient groups, many of which are small, many of which are very much volunteer-led, and rely on fundraising events that are obviously not happening now.
Thank you very much.