Research Study: Projecting the impact of delayed access to Trikafta

In August 2020, Cystic Fibrosis Canada shared news of a game-changing study published showing the dramatic life-saving benefits of the breakthrough cystic fibrosis modulator drug, Trikafta.

The study examines the potential health outcomes for the Canadian cystic fibrosis population by 2030 assessing three scenarios: getting access to the drug in 2021, getting access to the drug in 2025 and not getting access at all.

The study shows that early introduction of Trikafta in 2021 would result in life-changing health improvements by 2030 in comparison to no drug available, including:

• The estimated median age of survival for a child born with CF would increase by 9.2 years
• 60% fewer people living with severe lung disease due to cystic fibrosis
• 15% fewer deaths
• 18% increase in people with mild lung disease
• 19% fewer hospitalizations or home intravenous courses for chest infections
• A reduction in the number of transplants that are required for severe lung disease

About the study

The study was led by researcher Sanja Stanojevic, PhD, of Dalhousie University. Cystic fibrosis clinician Dr Anne Stephenson, of St Michael’s Hospital, was the senior author and clinician lead. Read the article published on August 24 in the peer-reviewed Journal of Cystic Fibrosis.

The multi-disciplinary research team from Dalhousie University, SickKids and St Michael’s Hospital has been investigating the changing CF population in Canada to help inform future resourcing of the population’s needs. The team initiated the study following the US FDA approval of Trikafta and the remarkable results of the phase III clinical trials for Trikafta. Their aim was to consider the impact Trikafta could have on the population in the future, and in the Canadian context, the impact of delayed access. The study reflects outcomes across the whole Canadian cystic fibrosis population and was informed using data from CF Canada’s Cystic Fibrosis Registry and observations from patients in phase III clinical trials of Trikafta.

This independent, peer-reviewed study was funded by CF Canada.

Sanja Stanojevic and Dr Anne Stephenson presented their study in the following recorded briefing: