Mr. Chair, thank you for the opportunity to present to the committee this morning.
I'd like to start by telling you about the Canadian Agency for Drugs and Technologies in Health, CADTH, and how our work enhances the accessibility, affordability and appropriate use of pharmaceuticals and other health technologies in Canada.
CADTH is an independent not-for-profit corporation that was established in 1989. Our primary goal and principal success criterion is impact—by providing better health, better patient experience and better value for Canadians. The members, or the owners, of CADTH are the federal, provincial and territorial deputy ministers of health who fund CADTH. This includes Health Canada and all provinces and territories except Quebec. We are governed by a board of directors that reports to the deputy ministers.
We refer to ourselves as an HTA, a health technology assessment agency, meaning we provide evidence-based assessments of the clinical and cost-effectiveness of drugs, diagnostics, and medical, dental and surgical devices, procedures and programs. In essence, we have two broad areas of work, our drug portfolio and our medical devices portfolio. We have a number of programs and products in place to support the management of medical devices in Canada. However, I will focus my comments today on our drug portfolio.
CADTH provides a range of services to support the effective management of pharmaceuticals in Canada, most notably the CADTH common drug review, CDR, and the CADTH pan-Canadian oncology drug review, pCODR. The CADTH common drug review program is a federal, provincial and territorial process that was established in 2004 to provide a common approach for reviewing the clinical and cost effectiveness of new drugs and existing drugs that may have new uses. We also receive input from patient groups as part of that review.
The common drug review supports coverage decisions for 18 of the 19 publicly funded drug plans in Canada, including the six plans managed by the Government of Canada for such specific populations as the military, veterans and Canada's indigenous peoples. Quebec has its own system for drug reviews through its HTA organization, INESSS, which is very similar to the approach CADTH uses and is increasingly becoming well aligned with CADTH through our excellent relations.
The pan-Canadian oncology drug review program was established by the provinces and territories in 2010, again with the exception of Quebec, and was transferred to CADTH on April 1, 2014. The federal government joined as a funding partner on April 1, 2016. Similar to CDR, the pan-Canadian oncology drug review provides a common process for the assessment of cancer drugs and makes reimbursement recommendations to Canada's federal, provincial and territorial public drug plans and cancer agencies to guide their cancer drug-funding decisions. The CADTH CDR and pCODR programs support funding decisions for individual drugs. We also conduct multi-drug reviews on classes of drugs under the auspices of our optimal use program.
One other service I would like to mention is our rapid response service, where we provide quick evidence reviews on the dauntingly large and complex medical literature. This service is extremely helpful in that it directly addresses the urgent needs for evidence that informs policy and practice. It's a widely used service, with more than 4,000 reports completed in the last decade by federal, provincial and territorial governments.
Early in 2018, CADTH adopted a new three-year strategic plan. The plan articulates a bold new direction for CADTH, and positions us as a key player in enhancing the accessibility, affordability and appropriate use of health technologies in Canada. Under the strategic plan, CADTH is building upon its success as a health technology assessment agency to become a health technology management, HTM, enterprise. We are implementing strategies to enable life-cycle health technology assessment, increased collaboration and engagement, and comprehensive implementation support. We are expanding our reach by embedding CADTH staff in jurisdictions across Canada and by being responsive and agile, catalyzing opportunities to align efforts across the drug review and approval system. It is CADTH's commitment to collaboration and integration that has brought us before the Standing Committee on Health today.
CADTH is pleased to be supporting a national consultation on a proposed supplemental process for highly specialized and complex drugs, including those used in the treatment of rare diseases. As this new initiative will help shape the future of Canada's drug review process, CADTH is planning to initiate an extensive consultation to ensure that Canadians have an opportunity to be able to provide input and to align our own processes with this new supplemental process.
It is estimated that there are more than 7,000 known rare and ultra-rare diseases. Of these, approximately 95% have no effective treatment options. This gap has become the focus of a considerable amount of attention by the pharmaceutical manufacturers for research and development, which can be associated with both high risk and high reward; by clinicians who are searching for effective options to improve quality of life, alleviate pain and suffering, and cure disease; and, more importantly, by patients and families who live with the often significant impact of these diseases.
In our experience, submissions from pharmaceutical manufacturers for drugs that treat these diseases are often associated with several challenging realities.
One, the size of the target population is small, making it difficult to conduct a clinical trial. Regulators such as Health Canada and health technology assessment organizations like CADTH consider randomized clinical trials the gold standard. When less-than-robust trial data are available, there is a correspondingly high degree of uncertainty about the true magnitude of the clinical benefit, or the efficacy, and also the safety and cost-effectiveness of the therapy being reviewed.
Two, clinical trials for rare and other diseases often use surrogate end points rather than real outcomes, which further increases the uncertainty regarding the effects of the treatment on clinical outcomes that are of primary concern to patients, such as mortality and quality of life.
Three, for reasons that are not always fully understood—they perhaps relate, at least in part, to the high cost of research and development, small eligible population and length of time required to recoup the investment to bring the drug to market—the cost of these therapies is invariably extraordinarily high.
In short, drugs used to treat these highly specialized and often complex diseases are often plagued by limited evidence, lower confidence in the magnitude of clinical benefit, extremely high cost and highly uncertain cost-effectiveness. This reality makes it difficult for CADTH's expert review committees to recommend reimbursement and for public payers to offer reimbursement given the finite resources of the public health care system.
In the past several years, CADTH has engaged in numerous initiatives to clarify and enhance our pharmaceutical review processes to address these challenges. These include enhanced training of our review staff to enhance our assessment of treatments with limited evidence; updating the deliberative framework of the CADTH expert review committees to allow for leeway in making funding recommendations for treatments of rare diseases; allowing greater opportunities for manufacturers to submit alternative types of data, so-called real-world evidence; strengthening clinician and patient engagement in the review process; and expanding opportunities for early dialogue and engagement with manufacturers. Most importantly, CADTH recently established a new parallel review process, in collaboration with INESSS and Health Canada, that will allow reviews to be completed within the shortest possible timelines.
Recently CADTH announced an initiative to integrate panels of clinical specialists into our review process to better inform and assist the pan-Canadian pharmaceutical alliance and provincial drug plans to implement drug funding recommendations. These panels enhance CADTH's engagement with the clinical community and further reinforce regional representation for important pan-Canadian funding recommendations. We have partnered with our HTA colleagues at INESSS in Quebec in a pilot initiative to hold joint panels for a truly national approach to these challenging reviews.
Finally but importantly, CADTH is pleased to be able to support the expensive drugs for rare diseases, EDRD, working group in conducting a national consultation about a proposed supplemental process for highly specialized and complex drugs. The EDRD was established by the deputy ministers of health in 2014 to explore the management of rare diseases with evidence-based approaches. The EDRD working group is being co-led by three provinces—British Columbia, Alberta and Ontario. As they were unable to send a representative today to appear before the committee, they gave me approval to share some of the information with you on their behalf.
The proposed supplemental process includes provisions to address several of the challenges I have already spoken about. These include limited data regarding efficacy, safety and cost-effectiveness; high uncertainty in the magnitude of clinical benefit; and the high cost of these drug treatments. While the members of the EDRD working group are best positioned to explain the specifics of the proposed supplemental process, we can confirm that it includes options that may help public drug plans provide time-limited access to these therapies as additional clinical evidence is being gathered.
Consultations have been organized for the EDRD working group to hear directly from clinicians, ethicists, researchers, patient groups and the pharmaceutical industry during web-based sessions on November 5, 6, 8, 13 and 14 respectively. More than 180 individuals have registered to participate in one of these consultations. Following this, there will be an opportunity for interested stakeholders to provide a written submission.
Information from the consultation process will be considered by the EDRD working group to inform discussion at the federal, provincial and territorial level about potential enhancements or changes to the proposed supplemental process. Because CADTH is integrally involved in supporting the provinces in this work, we are able to consider how this new proposed supplemental process might impact CADTH's review process and to modify our own approach accordingly to align efforts.
Ultimately, CADTH exists to serve the needs of its customers by providing evidence-based funding recommendations to support jurisdictional decision-making. We will continue to be responsive to opportunities to enhance our process, and are pleased to continue to support the EDRD working group and our other partners to address access, appropriate use and affordability of drugs, including those for highly specialized and complex diseases.
Thank you, Mr. Chair, for allowing me to present today, and I welcome any questions that the committee members may have.