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Journal of Cystic Fibrosis ; 21:S135, 2022.
Article in English | EMBASE | ID: covidwho-1996794


Objectives: Research conducted on patient experiences looks at older modulators;Orkambi and Kalydeco®. There is little research on patient experiences with the newer modulator Kaftrio®. There is also a gap in research regarding qualitative methodology and Kaftrio®. Qualitative approaches provide an insight into the understanding of how people with CF are adjusting to new CF treatment. This study asks the question "What are patients’ feelings and experiences of taking Kaftrio® for cystic fibrosis?" Methods: Ethical approval was gained through the University of Southampton. Nine people with CF (5 women, 4 men) were interviewed. The interview covered 5 areas: 1) expectations before starting Kaftrio®, 2) experiences of taking Kaftrio®, 3) the positives and negatives of Kaftrio®, 4) the day-to-day change to life, and 5) feedback to healthcare professionals and those eligible to take Kaftrio®. Interviews lasted from 23 minutes to 1 hour 10 minutes. Interpretative phenomenological analysis (IPA) was conducted on the data. IPA produces themes and reflects on how these themes are different between each participant, focusing on the individual linguistic and conceptual commentary from person to person. Results: Four themeswere prominent. Thesewere "Preparing for Kaftrio®", "The new normal", "Concerns about Kaftrio®" and "Kaftrio® and COVID- 19". The study found that Kaftrio® has a greater impact in psychosocial domains of life. Participants explained they were having to future plan, consider retirement, start work and family plan. These considerationswere unexpected for many participants. Conclusion: This study reveals the psychosocial impacts and the unexpected lifestyle changes when taking Kaftrio®. As less medical management is needed due to the improvement of symptoms, there is a shift to life management. These impacts are likely to continue in the modulator era of CF care. Future research is needed to ascertain if these impacts continue with prolonged use of Kaftrio®

Springer Series Pharm. Stat. ; - ((Chuang-Stein C., Kalamazoo, MI, United States):1-21, 2021.
Article in English | EMBASE | ID: covidwho-1410939


Developing a new drug is a high-risk and high-reward enterprise. The high risk is reflected by the low success rate of turning a new molecular entity (NME) into an approved drug even after the NME has successfully met the preclinical testing requirements. In this chapter, we offer a high-level summary of the clinical testing which is composed of four distinct phases under a traditional development plan. We discuss deviations from the traditional development plan and new regulatory approval pathways. In particular, we highlight the speed with which COVID-19 vaccines were developed and authorized for emergency use through extensive public–private partnerships and the adoption of a high-risk business model. We offer some examples of recent advances in clinical trial designs and conclude the chapter with a short summary of the salient points covered in the chapter.