Emily’s Entourage (EE) is pleased to announce that its first-ever, global survey on the health and perspectives of people with cystic fibrosis (CF) that do not benefit from approved mutation-targeted therapies was published this month in the peer-reviewed medical journal, Pediatric Pulmonology.
The organization will host an informational webinar featuring a presentation from EE Scientific Advisor Board member and co-author of the article, Dr. Jennifer Taylor-Cousar, to share survey findings and address questions from the community. The webinar will be held on Wednesday, Apr. 6 at 8 pm ET.
The purpose of this survey was to collect information to better understand the current health status, impact of CF, unmet needs, and clinical research interest of those in the final 10% of the CF community that do not benefit from existing mutation-targeted therapies (CFTR modulators) due to ineligible mutations, lack of access, or side effects.
“We hope these results can help direct and accelerate drug development and clinical trial design to meet the needs of the community and ensure that the voice of the CF community remains front and center in every step of the drug development and approval process,” said EE Co-Founder, Emily Kramer-Golinkoff.
Over 430 individuals from 5 continents participated in the 38-question study, which was conducted between June and July 2021, and disseminated via social media and email to individuals and families with CF as well as advocacy groups in and outside of the US.
HIGHLIGHTS FROM SURVEY RESULTS
- Approximately 26% of respondents had mild lung disease, 50.3% had moderate disease and 11.1% had severe disease
- The percentage of respondents who self-identified as a BIPOC individual who were ineligible for modulators (72.5%) was higher than those who self-identified as White (65.2%)
Impact of Disease and Burden of Therapy
- 13.8% of individuals with CF spent five or more nights in the hospital on an annual basis for a CF-related concern
- More than 86.1% of those surveyed reported that CF has a moderate to significant impact on their or their family member’s life with 93.5% of caregivers reporting that CF had a moderate to significant impact on daily life. Among caregivers, mental (66.7%) and physical (40.7%) health impacts were most highly reported.
- Among people with CF, the aspects of life most impacted by CF were mental health (45.5%), plans for the future (45.2%) and ability to spend time with friends/participating in social activities (39.7%)
Potential Clinical Research Participation
- 76.7% expressed interest in participating in future clinical trials
- Of those that had not or would not participate in clinical trials, the most common reason was lack of qualification (37.0%)
- The most important trial features were access to drug after the trial ends (92.5%) and addition of more clinical trial locations (78.7%)
Perceptions Regarding Access to CFTR Modulators for Some but not All People with CF
- When asked about how it feels to see others in the CF community benefit from modulators when they/their family member were unable to benefit, most felt happy and excited for those who were benefitting, but other recurring themes included “bitter sweet,” “envious,” “sad,” “frustrating,” “disappointing,” “angry,” “worried,” and “left behind.”
Please note that the article is open access to ensure everyone in the CF community can learn and benefit from the results, including, importantly, individuals with CF and their families.
EE will host an informational webinar featuring a presentation from Dr. Jennifer Taylor-Cousar on key survey findings followed by a Q&A session to address any questions from the community.
The webinar will be held on Wednesday, Apr. 6 at 8 pm ET and is free and open to all. Please note that registration in advance is required.
Thank you to all of the survey contributors and article authors, including Dr. Jennifer Taylor-Cousar and the individuals with CF and family members that generously participated in the survey.
If you or your family member has one or two copies of a CF nonsense mutation, you are eligible to join EE’s CF Nonsense Mutation Patient Registry and be informed about relevant research, clinical advances, and drug trials based on the information you provide. Learn more and join the patient registry.