Donald R VanDevanter1, Nicole Mayer-Hamblett2, Michael Boyle3. 1. Case Western Reserve University School of Medicine, Cleveland, USA. Electronic address: drv15@case.edu. 2. University of Washington and Seattle Children's Hospital, Seattle, USA. 3. Cystic Fibrosis Foundation, Bethesda, USA; John Hopkins School of Medicine, Baltimore, USA.
Abstract
BACKGROUND: New CFTR modulators are in development that sponsors anticipate will be comparable or superior to approved modulators. Testing these agents for efficacy will require either placebo-controlled or active-comparator trials. METHODS: We surveyed US CF physicians and their patients eligible to receive approved modulators or their families for willingness to participate in placebo-controlled modulator trials of varying duration. RESULTS: Interest in placebo-controlled trials of short duration (2-4weeks) was greatest, with few respondents, particularly among patient respondents, willing to consider 6month studies. Patients/families with access to approved modulators were consistently less interested in placebo-controlled modulator trials of any duration. CONCLUSIONS: Sample size and interpretability advantages of placebo-controlled trials outweigh alternative active-comparator trials, but must consider physician and patient thresholds for forgoing treatment with approved modulators. Enrollment will be most feasible for short-duration trials and those conducted among populations without access to approved modulators.
BACKGROUND: New CFTR modulators are in development that sponsors anticipate will be comparable or superior to approved modulators. Testing these agents for efficacy will require either placebo-controlled or active-comparator trials. METHODS: We surveyed US CF physicians and their patients eligible to receive approved modulators or their families for willingness to participate in placebo-controlled modulator trials of varying duration. RESULTS: Interest in placebo-controlled trials of short duration (2-4weeks) was greatest, with few respondents, particularly among patient respondents, willing to consider 6month studies. Patients/families with access to approved modulators were consistently less interested in placebo-controlled modulator trials of any duration. CONCLUSIONS: Sample size and interpretability advantages of placebo-controlled trials outweigh alternative active-comparator trials, but must consider physician and patient thresholds for forgoing treatment with approved modulators. Enrollment will be most feasible for short-duration trials and those conducted among populations without access to approved modulators.
Authors: N Mayer-Hamblett; S van Koningsbruggen-Rietschel; D P Nichols; D R VanDevanter; J C Davies; T Lee; A G Durmowicz; F Ratjen; M W Konstan; K Pearson; S C Bell; J P Clancy; J L Taylor-Cousar; K De Boeck; S H Donaldson; D G Downey; P A Flume; P Drevinek; C H Goss; I Fajac; A S Magaret; B S Quon; S M Singleton; J M VanDalfsen; G Z Retsch-Bogart Journal: J Cyst Fibros Date: 2020-06-07 Impact factor: 5.482
Authors: Scott C Bell; Marcus A Mall; Hector Gutierrez; Milan Macek; Susan Madge; Jane C Davies; Pierre-Régis Burgel; Elizabeth Tullis; Claudio Castaños; Carlo Castellani; Catherine A Byrnes; Fiona Cathcart; Sanjay H Chotirmall; Rebecca Cosgriff; Irmgard Eichler; Isabelle Fajac; Christopher H Goss; Pavel Drevinek; Philip M Farrell; Anna M Gravelle; Trudy Havermans; Nicole Mayer-Hamblett; Nataliya Kashirskaya; Eitan Kerem; Joseph L Mathew; Edward F McKone; Lutz Naehrlich; Samya Z Nasr; Gabriela R Oates; Ciaran O'Neill; Ulrike Pypops; Karen S Raraigh; Steven M Rowe; Kevin W Southern; Sheila Sivam; Anne L Stephenson; Marco Zampoli; Felix Ratjen Journal: Lancet Respir Med Date: 2019-09-27 Impact factor: 30.700