Overview

Canadian Beth Vanstone advocates for CF patients, and the rare community. She and husband Glen have two daughters, Jessie and Madi (who has CF). Madi was diagnosed with CF at 8 months old. Over the years the Vanstone family has raised upwards of $100,000 for Cystic Fibrosis Canada. In 2011 Madi’s health was declining. A new CF modulator was being marketed. Madi participated in the trial and the results were outstanding. Due to an outdated system in Canada, Madi would not have access to this drug that could save her life. The drug was approved in Canada, and went through the Health Assessment process, but was in limbo with the pCPA an organization designed to negotiate these drugs. There was no action being taken, there was no urgency whatsoever to have the negotiations completed to have it added to provincial formularies. For two years, Beth and her daughter advocated to have the drug negotiated and added to the provincial formulary. The Mom and daughter team did countless TV and newspaper interviews. They met with the members of the provincial parliament, the health minister and the premiere of Ontario. Eventually, in June 2014 the drug was added to the formulary and in time, 118 people across Canada gained access to this life-giving drug. There were many lessons learned over those two years, the biggest being our Canadian System has forgotten about Rare Disease patients. As of 2024 Canada has no Rare disease strategy, and no orphan drug designation. Beth keeps going. She is currently working with the University of Toronto – Pharmacology Department as a rare disease partner liaison. Hear more of Beth’s incredible story.