Chair and members, we appreciate your invitation to be here today, and we thank the committee and staff for their work throughout the pandemic challenges.

I represent a coalition of 30 patient organizations, national and regional, working together through the Best Medicines Coalition to support patients and advocate for better access to life-saving medicines. I volunteer as the chair of the board of directors of BMC.

Our non-profit supports any actions by governments, public or private insurers and civil society that can deliver greater access to medicines for Canadian patients. We're a small but mighty organization made up of broad-based charities and grassroots groups, including the one I lead, Canadian PKU and Allied Disorders, which involves rare brain-threatening diseases. These groups involve patients and caregivers to improve care and are active in research, patient services, education and issue advocacy.

BMC member organizations include diverse conditions, including mental illness, arthritis, asthma, blindness, Parkinson's, psoriasis and cancers, including breast, kidney, lymphoma and ovarian, and their survivors.

I am here today as a patient advocate, because when our youngest child arrived, the miracle of universal newborn screening discovered his rare genetic condition, enabling immediate therapy. As an adult, he volunteered for a clinical trial for its first drug, and that drug transforms his brain and life. His mother, my wife, died in 2014 of two neurodegenerative diseases, ALS and FTD, which still do not have effective treatments. It is a big deal when we can move lethal diseases like ALS from untreatable to treatable, and it is a big deal when we can permanently fix genetic problems, such as my son's, in the new era of cell and gene therapies.

Let me address the important question of real, perceived or imagined conflicts of interest. I happily completed the committee's disclosure form and want to address how the Best Medicines Coalition funds and governs its activities. We do not sell a service. We do not have union or other dues. We don't have university tenure or other job security. We don't charge patients to advocate on their behalf.

If governments establish a means to fund our efforts to support patients in the public interest, we will apply for that funding. This is not without precedent, as it exists in the context of climate change advocacy, telecommunications regulation or legal challenges for human or charter rights. We ask HESA to recommend such public interest funding.

BMC accepts funding from the pharmaceutical industry for our patient support activities. We make no secret of this and are aware of possible perceptions. It is why this funding is received with an explicit agreement that funding does not influence our policies, advocacies or operations in any manner. All activities and interactions are pursued within a framework of integrity. BMC and its member organizations adhere to a code of conduct regarding funding, which is publicly available on our website. All initiatives are conceived and developed in and by the organization, aligned with its missions and goals, without influence from any funder on content, messaging or execution. I'd be pleased to answer questions on this, and I trust that we all must be transparent about all our conflicts, perceived or otherwise.

The matter at hand today is the government's approach on drug prices and the critical risk this poses to some Canadian patients. Our focus is ensuring that all Canadians have access to medicines needed when needed. Medical necessity is paramount for all patients. We support the need to make medicines more affordable for all patients, especially those who pay out of pocket, and we support the goal of actual prices in line with similar countries. We also believe regulations and guidelines must encourage, not deter, new medicines and vaccines plus those clinical trials sponsored by drug developers, which are important to patient volunteers in providing early access to promising new therapies, as was the case for my son.

There's a natural tension between how much insurers are willing to pay and patient access to medicine. Whether we like it or not, pharmaceuticals are a global market and Canada is competing globally to be an attractive market. This is clear in terms of medicines, vaccines and PPE.

If Canada regulates prices too low, the patients we represent will not gain access to life-altering or life-saving medications. If prices go too high, public insurance systems could become overwhelmed and unsustainable. This would be disastrous for patients and taxpayers alike. The challenge for Parliament, government and PMPRB is to balance those interests. It's not an easy task.

The government and PMPRB decided to move forward with their approach despite uncertainties on patient impacts. This is at the core of our concern. At this committee, PMPRB was unable to tell when certain conditions in the guidelines will come into effect. Consider that a global entity is deciding on the sequence of product launches and Canada's processes and pricing rules lack clarity for the foreseeable future, why would it be a surprise that a new medication or indication would not be launched in Canada?

This government is unwisely taking on significant risks and the consequences will be borne by patients. We are not willing to provide informed consent. As written, the PMPRB regulations and guidelines have not balanced price and access. Specifically, anticipated price reductions go beyond original intent, and initial evidence shows significant negative impact: a decline in new drug introductions and reductions in new clinical trials.

This government confirmed the legitimacy of these concerns and, in my view, lost the moral high ground when it exempted COVID vaccines and drugs from the PMPRB rules. Why exempt COVID and not other lethal diseases?

A frequent criticism is that few new drugs provide significant additional benefits to patients. COVID vaccines reveal this but also show that it's not helpful. We have one approved vaccine today. It is a breakthrough. Other vaccines may be approved, but the others will not likely offer a substantial clinical benefit over that first approved vaccine. It's clear that our health systems and individuals will benefit substantially from access to many approved vaccines, even if only the very first one is a breakthrough. That is how innovation works.

A key marker for patient communities is whether medicines will be available to Canadians both affordably and in a timely manner. Since the announcement of the new rules, applications for new drugs have declined compared to similar countries. This is troubling.

Health Canada and PMPRB want to lower drug prices to reflect what the public health system is willing to pay or able to pay. Nothing in these changes makes it certain that health systems will deliver patient access to medicines at these prices. There could be a double whammy for patients. Price regulations might mean new drugs will not come and, if they do finally come, the system still may not cover them.

We wish to express four recommendations for this committee to consider.

First, the Best Medicines Coalition supports step-by-step implementation to lower drug prices. PMPRB indicated some phasing. They did not articulate when and what would be phased. We ask for clarity and decisions based on best evidence. We urge this committee to recommend that the federal government, through cabinet, direct a stay of implementation of parts of the regulations and guidelines, deferring the novel economic factors to a second stage pending further study and consultation. The new basket of comparator countries should proceed to bring down list prices immediately, especially for those patients who pay out of pocket, which, I point out, represents 21% of all drug spending in Canada.

Second, BMC recommends that this committee call for greater policy-making disclosure, and we support any efforts of the committee to bring about that transparency. The government should disclose inputs provided as part of formal consultations and otherwise, including analysis of the nature and breadth of concerns and how concerns have been addressed or will be mitigated. Further, it is important that the government and PMPRB disclose correspondence and other inputs from provinces and territories.

Third, the Best Medicines Coalition asks that this committee recommend that the government develop and publish comprehensive evidence, including initial impacts on critical markers of policy success or failure; that is, key metrics of introductions of new medicines and initiation of clinical trials sponsored by drug developers. There is a need for disclosure regarding the government's and PMPRB's assumptions, which the regulations and guidelines rely upon. We urge this committee to request that the PMPRB case studies on specific drugs back in 2018, prepared to show the impact of possible proposals, be updated and published based on the final regulations and guidelines.

Fourth, the Best Medicines Coalition asks that this committee recommend that the government study and provide public interest funding so that the voices of patients and their needs and interests can be more adequately represented in public policy and regulatory matters, including PMPRB.

I would be pleased to answer any questions.

Thank you.

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