In 2019, the federal government announced a $1-billion investment over two years, with up to $500 million per year ongoing, to support Canada's first-ever national strategy for rare diseases beginning in 2022-2023. For the one in 12 Canadians who suffer from a rare disease, the news gave them hope for better health outcomes and cutting-edge treatment options. But as we near the end of the first year of that funding commitment, Canada has not yet implemented a comprehensive rare disease strategy that could positively impact the lives of rare disease patients and their loved ones.Canada remains the only G7 country without a national approach to rare diseases. To bring us in line with our peers, a comprehensive Canadian strategy should include a standard definition for what constitutes a 'rare disease,' an accelerated review pathway, real-world data, and rare disease centres of excellence.Most rare disease patients in Canada and elsewhere are children. As our scientific and medical understanding continues to grow – including through many world-class Canadian experts and institutions – so too does the possibility of better health outcomes for rare disease patients. This is a powerful illustration of the fundamental purpose of the innovative pharmaceutical industry: to move the best available research through clinical trials to result in useful therapies that respond to and help address unmet or under-treated medical needs in society.Unfortunately, only 60 per cent of treatments for rare diseases make it to Canada, and compared to the U.S. and Europe, it takes up to six years longer for those treatments to be approved due to Canada's fragmented process. There are also persistent gaps and challenges across the country in diagnosing, monitoring, and treating patients in an efficient and effective way. Canada doesn't currently have a streamlined review, approval, and access pathway for rare disease drugs that prioritizes timely access to the latest innovations. This lack of a dedicated pathway for rare diseases means Canadian patients – some only a few days old following newborn screening results – are left waiting too long for treatments that could change their lives.Health Canada must also ensure that other government agencies and policies support better access to drugs for rare diseases. For example, the lack of predictability and clarity in the recently proposed changes to the Patented Medicine Prices Review Board's (PMPRB) guidelines will discourage businesses from launching new medicines in Canada. If the guidelines remain unchanged, they will reduce patient access to new treatments and directly work against an effective pan-Canadian strategy for drugs for rare diseases.While Health Canada has consulted key stakeholders, patients, and their advocates as it develops a national strategy for rare diseases, action on the ground is needed. It's time for the federal government to work with provinces to put those significant dollars to work and make a difference for Canadians with rare diseases. There is a clear opportunity for the provinces to demonstrate leadership and prioritize rare diseases in their respective regions through provincial rare disease strategies, leveraging federal funds once available.A national strategy for drugs for rare diseases remains an important but uncompleted part of Health Canada's current mandate. The funding is available, and the federal government has an opportunity to lead the way with a rare disease strategy that will have a meaningful impact on patients and their families. But this challenge can only be met if the federal government charts a collaborative path that safeguards access to life-saving innovations by stopping the implementation of the proposed PMPRB guidelines.Pamela Fralick is President of Innovative Medicines Canada, the industry association representing innovative pharmaceutical companies.  Prior to joining IMC, Ms. Fralick was President and CEO of the Canadian Cancer Society. Previously, she served as president and CEO of the Canadian Healthcare Association, the Canadian Physiotherapy Association, the Collegium of Work and Learning and the Canadian Centre on Substance Abuse. She has also held senior public service positions with Health Canada and the Department of National Defense.