President, Innovative Medicines Canada
Canada’s innovative pharmaceutical industry calls for a more collaborative, patient-focused approach to tackling rare diseases.
The social isolation and other disruptions created by the COVID-19 pandemic have added to the burden on those living with rare diseases, and also on the people caring for them. And while they face many of the same challenges that patients in other countries do, Canadian patients face added complications. Now is the time to develop a comprehensive rare diseases plan, and to provide the hope and relief that patients are asking for.
Unlike other countries, Canada doesn’t have a common definition of rare diseases, nor do we have adequate information about the different types of rare diseases or the size of the populations affected. This creates challenges in the delivery of health care. Better data would also help us more efficiently manage our health systems and would provide valuable insights for treatment and drug development.
Delivering effective change
The government has reiterated its commitment to a national strategy for drugs for rare diseases in the fall economic update and the ministerial mandate letters released in mid-January. The innovative pharmaceutical industry is ready to work with governments at all levels, patients, and other critical stakeholders to co-develop a plan to ensure that patients have better coverage for treatments, and that medicines that can make the difference between life and death — or contribute to better quality of life for patients — are delivered efficiently.
Access without barriers
In 2018, Innovative Medicines Canada (IMC) convened a group of Canadian industry representatives to develop policy options to address patient access to drugs for rare diseases. This work is a catalyst for industry collaboration with the federal and provincial governments, as well as patients and other stakeholders.
Our industry is ready to partner with all stakeholders to ensure that Canadians get the medicines they need, when they need them, while ensuring the sustainability of our health care systems
One of the key issues is, of course, affordability. To address this, we first need to understand the scope of the rare disease landscape in Canada. It starts with a common definition of rare disease and defining criteria for product eligibility. We can use this common understanding to build a rare disease patient registry and clinical network — something our industry is ready to invest in. This would benefit patients, governments, and the research community.
A true partnership among all interested parties, with common goals, is key to ensuring that patients have access to the drugs they need not just to survive, but to live longer, healthier lives. That access cannot be dependent on income, age, or postal code. Together, we can build a framework to support innovative agreements that measure the fair value and contribution of medicines for patients and payers through evidence-based reviews. This would produce fair, competitive prices and budget predictability for public drug plans and private insurers.
A commitment to collaboration
There’s an impediment, however. The pending changes to the Patented Medicine Prices Review Board will likely prevent or delay the launch of new medicines in Canada. Fortunately, there’s still time to choose a better path forward. IMC has proposed an alternate approach to the government, which includes investments in a manufacturing accelerator, as well as commitments to collaborating on a strategy for rare diseases and implementing measures to guarantee the un- or under-insured access to the medicines they need.
Fortunately, there’s clear political will to address the long-standing issue of drugs for rare diseases. Our industry is ready to partner with all stakeholders to ensure that Canadians get the medicines they need, when they need them, while ensuring the sustainability of our health care systems.