Disparities in Access to Medications

 Cystic fibrosis is at the forefront of precision medicine with a new class of drug called CFTR modulators. However, CFTR modulators are only approved for a limited number of CFTR variants leaving some people with CF without access. People who are Asian, Black, Indigenous or Latino are less likely to qualify for and be prescribed CFTR modulators.

Prior Findings

We have found that people with CF who are Asian, Black, or Latino are proportionally underrepresented in CF pharmacology clinical trials compared to the CF population. Over 80% of CF pharmacology clinical trials did not even report subjects’ race and ethnicity. We found that people with CF who are Asian, Black or Latino are less likely to qualify for CFTR modulators which are disease altering and greatly improve outcomes.

We previously conducted a N-of-1 clinical trial of ivacaftor versus placebo in patients with CF and clinical signs of residual CFTR function. Some CF patients with residual CFTR function have decreased sweat chloride concentration with ivacaftor. Increased chloride current in HNE cultures among subjects with decreased sweat chloride concentrations may predict clinical response to ivacaftor.

Dr. McGarry continues to advocate that people with CF all over the world should have access to CFTR modulator therapy that is affordable.

Key Publications

• In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies
• Left behind: The potential impact of CFTR modulators on racial and ethnic disparities in cystic fibrosis
• Minorities Are Underrepresented in Clinical Trials of Pharmaceutical Agents for Cystic Fibrosis
• In response to “who are the 10%? – Non eligibility of cystic fibrosis (CF) patients for highly effective modulator therapies”
• Triple Therapy for Cystic Fibrosis with a Phe508del CFTR Mutation
• Normalization of sweat chloride concentration and clinical improvement with ivacaftor in a patient with cystic fibrosis with mutation S549N