At a recent internal summit with colleagues from all over the world, I had the honor of meeting Emily Schaller, a patient advocate and the CEO of Rock CF, a Detroit-based non-profit aimed at improving quality of life for people with cystic fibrosis through exercise. In the early 1980s, Emily was diagnosed with cystic fibrosis (CF) at 18 months, when the life expectancy for people with the disease was about 18 years. Today, the life expectancy for people living with CF is 40.
Emily attributes her life – and not just quality of life, but actual survival – to clinical trials. The cystic fibrosis community, made up of about 30,000 people in the U.S., is incredibly involved in clinical trials because of the lack of successful treatments for CF and the large number of gene mutations that can affect response to those treatments that have been approved.
For those of us in the room at the summit, Emily brought to life our work’s purpose. We had colleagues from the clinical team for the first time seeing a patient who had been through one of their trials, and colleagues on the commercial side seeing the results of a brand they named and promoted. It was incredibly gratifying, but we knew it wasn’t the end of the story for people like Emily.
As Emily said in a 2013 Atlantic article, “I worry about things I really never thought I’d have to think about—like health insurance and having an income to support myself and my parents getting older.” For a patient population that previously hadn’t thought about life past the age of 18, and that is still short of a cure, there are still plenty of things to worry about. The most pressing on Emily’s mind regarding the work we do:
- Healthcare access – not only cost, but continued access. For people like Emily, it’s a matter of survival.
- Improved drug delivery – ensuring that companies continue to innovate and find better ways to help patients stay compliant.
- Clinical trial ease – cumbersome consent forms wear on trial participants. Companies need to find better ways to ease the administrative side for participants via digital tools or portals.
- Value – ensuring the public knows more than just the price of the drug. Emily noted her drug was absolutely expensive, but prior to taking it, she had been in the hospital three times every year at a cost that equaled the annual price of the drug. Factoring in her newfound ability to work, get off disability and improve quality of life overall, and the question of value disappears.
Our conversations with Emily brought to life just how real healthcare is for millions of people. It was a good reminder of how important our work is – and how important people like Emily, who are willing to participate in clinical trials, are to healthcare innovation.
