It’s time to move forward. The recent approval by the U.S. Food and Drug Administration of the triple-combination treatment Trikafta (elexacaftor, tezacaftor, and ivacaftor) has sparked hope in the cystic fibrosis community.
While it’s not a cure, the therapy has shown to have positive health benefits in clinical trial participants. However, with these benefits come serious considerations.
The medication has potential benefits for an estimated 90 percent of CF patients ages 12 and older. Even if only a small percentage of them experience improvements in their health with Trikafta, the changes to the CF community could be significant. We must be prepared to adapt to these new developments.
I hope that the various cystic fibrosis foundations will create plans moving forward to address the following issues:
- Disability support: In clinical trials, Trikafta increased lung function by an average of 13.8 percent compared to treatment with a placebo in patients with a single CF mutation. For some, this improvement in health status could have negative financial consequences. For example, those who are receiving disability support may worry about losing their benefits. Others might be concerned about their medical insurance status. I have read posts by CF patients in online support groups expressing this as a serious concern. How does one get on Trikafta and reap the benefits without losing financial security?
- Body image: Trikafta patients have reported weight gain. As CFers, we understand that gaining weight is beneficial for most of us to help maintain lung function; however, societal pressures tell us that skinny is good. We need to develop mental health guidelines and employ professionals to guide patients through the process of weight gain and its effect on their body image.
- Work and education: Some people with CF don’t see the point of spending time on education because of the disease’s reduced life expectancy. But if Trikafta has the potential to improve those numbers, career advisers will be needed. Employment opportunities and status also should be addressed. I’m among many CF patients who have been unable to work in a traditional full-time job. Older adult patients may find returning to the workplace particularly challenging due to a spotty or nonexistent résumé. How does one begin to look for a job with no work experience? Disclosing your disability can be risky, and patients shouldn’t wade into these waters alone without expert guidance. Many patients will want to work, and some will be entering the workforce for the first time.
Of course, we need to consider other factors. But I believe that the three issues mentioned need immediate attention. Some people already have started taking Trikafta. The clock is ticking — it’s time to shape our futures.
Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to cystic fibrosis.