Unlocking Access: Building a National Strategy to Support Canadians with Rare Diseases

Access to healthcare is an often-used phrase. 

In some cases, it means a patient’s ability to receive primary care or a consultation from a specialist. In other instances, it may mean access to surgical care or critical pharmaceutical products, like vaccines or curative and life-extending treatments. A number of factors can affect one’s access to care. However, the barriers are particularly pronounced for Canadian patients living with rare diseases. 

One in 12 Canadians – about 3 million people – is living with a rare disease. Two-thirds are children. There is no perfect definition that encompasses the more than 6,800 rare diseases, which include cystic fibrosis, multiple sclerosis and hemophilia. But rare diseases are often chronically debilitating or life-threating. For many patients, access to care can be delayed or derailed by a variety of factors, including diagnostic delays, reimbursement challenges or the limited therapeutic options available. There are treatments for only about 10 percent of rare diseases, and only 60 percent of those treatments for rare diseases make it into Canada. Even when treatments are available, access to the product varies greatly depending, in part, on where a patient lives, how the treatment is paid for and what type of coverage the patient and their families have.  

Health Canada took a positive, much-needed step toward addressing this longstanding issue and the existing access disparities when it announced plans to develop a national strategy for drugs for rare diseases. With the first $500 million earmarked for this strategy in 2022, this year represents a monumental moment in time to accelerate this urgent effort and formally establish a single, national framework to ensure equitable and consistent access to therapies—no matter where patients live or what access to funding they have. As Health Canada builds out the initial framework in collaboration with stakeholders across the industry, it is critical they consider the factors throughout the entire patient journey and implement an innovative approval and coverage process, with payment ideally tied to long-term, real-world studies on effectiveness and safety. 

Support Across the Entire Patient Journey
Due to lack of disease understanding and awareness, it can take a patient, on average, seven years to receive an accurate rare disease diagnosis. But even after patients receive a diagnosis and are prescribed a medication, their treatment journey is complex – particularly when patients are quite ill and have little support to navigate this difficult process. Each patient journey is unique. A limited understanding of public and private reimbursement mechanisms and criteria, coupled with the complexity of paperwork and testing requirements, can delay treatment and cause increased anxiety and fear among patients and caregivers. Many therapies indicated for rare diseases have unique administration requirements that necessitate additional customized training and ongoing safety and clinical monitoring. Reimbursement processes can be daunting, and patients and families often have many questions about how to access specialized support services.

Those barriers can be compounded by affordability-related challenges. As of 2019, there were 93 drugs for rare diseases approved in Canada that cost more than $100,000 per patient per year, according to the Patented Medicines Prices Review Board. The costs of these drugs can also pose a challenge to the long-term sustainability of government and employer-sponsored drug plans. To control the impact of these drugs on health system budgets, Health Canada can incorporate specific options, such as pay-for-performance models or the sharing of costs and pooling risks. 

Patient support programs—funded by manufacturers—play a valuable role in helping many patients navigate the healthcare system and access their medications by providing services, such as financial assistance solutions and reimbursement support, that enable patients to start on therapies sooner. For example, clinic and field nurses deliver self-injection education and training, provide rigorous safety monitoring and administer injections and infusions at local private clinics or in patients’ homes. Through its patient support programs, Innomar Strategies helped more than 10,000 patients access rare disease drugs in 2020. The complex patient journey underscores the demand for end-to-end support. As such, it’s critical the national framework extends beyond just access to the medication, but also factors the services required to access and obtain treatment. 

Using Real-World Data to Emphasize Outcomes
One key component to optimize patient access to these therapies, both now and in the future, is establishing an effective process to collect and analyze real-world data and evidence. The data will provide clarity around the use and effectiveness of the treatments, which is critical not only to emphasize patient outcomes but to provide decision-makers with the information needed to support regulatory and reimbursement decisions. The use of real-world data and evidence is particularly important for these therapies, as they target diseases that affect small numbers of people. During the product development phase, clinical trials for Rare Disease therapies often involve fewer patients and are more likely to be non-randomized – factors that make it more difficult to generate the data and information payers seek. 

As part of its strategy, Health Canada can establish a national system to collect and analyze data. There is a significant opportunity to leverage current data sources and to create partnerships between integrated service providers and public payers to collect real-world evidence to support the overall strategy for using real-world data to inform decision-making. This data collection process, which can be initiated following product approval from Health Canada, can accelerate patient access to therapies and help regulators learn sooner how the drug performs in a real-world setting. Additionally, Health Canada can seek opportunities to join an international network to gain access and contribute to a larger pool of real-world data. By working with countries around the world, Canada can adopt best practices to improve patient access and leverage existing data sets to support decisions on products available in Canada. 

While the heightened focus and investment to build this strategy represents a monumental step forward, it is important to maintain this momentum and drive long-term change that positively impacts the lives of Canadians living with rare diseases. By Rare Disease Day in 2023, it’s critical for Health Canada – in collaboration with stakeholders across the industry – to take meaningful steps toward unveiling a national strategy that adequately supports the unique needs of patients and caregivers to ensure consistent and fair access to the medication and services they need.