References
1. Kraljevic S, Stambrook PJ, Pavelic K. Accelerating drug discovery. EMBO Rep. 2004;5(9):837-42.
2. Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32(1):40-51.
3. DiMasi JA, Grabowski HG, Hansen RW. Innovation in the pharmaceutical industry: New estimates of R&D costs. J Health Econ. 2016;47:20-33.
4. MedicineNet. Drug Approvals - From Invention to Market … A 12-Year Trip 1999, July 14 [Available from: https://www.medicinenet.com/script/main/art.asp?articlekey=9877.
5. Goss CH, Mayer-Hamblett N, Kronmal RA, Ramsey BW. The cystic fibrosis therapeutics development network (CF TDN): a paradigm of a clinical trials network for genetic and orphan diseases. Adv Drug Deliv Rev. 2002;54(11):1505-28.
6. Fuchs H, Borowitz D, Christiansen D, Morris E, Nash M, Ramsey B, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med. 1994;331(10):637-42.
7. Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, et al. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019;381(19):1809-19.
8. Chmiel JF, Konstan MW. Anti-inflammatory medications for cystic fibrosis lung disease: selecting the most appropriate agent. Treat Respir Med. 2005;4(4):255-73.
9. Chmiel JF, Konstan MW. Inflammation and anti-inflammatory therapies for cystic fibrosis. Clin Chest Med. 2007;28(2):331-46.
10. Chmiel JF, Konstan MW, Elborn JS. Antibiotic and anti-inflammatory therapies for cystic fibrosis. Cold Spring Harb Perspect Med. 2013;3(10):a009779.
11. Konstan MW, Doring G, Heltshe SL, Lands LC, Hilliard KA, Koker P, et al. A randomized double blind, placebo controlled phase 2 trial of BIIL 284 BS (an LTB4 receptor antagonist) for the treatment of lung disease in children and adults with cystic fibrosis. J Cyst Fibros. 2014;13(2):148-55.
12. Doring G, Bragonzi A, Paroni M, Akturk FF, Cigana C, Schmidt A, et al. BIIL 284 reduces neutrophil numbers but increases P. aeruginosa bacteremia and inflammation in mouse lungs. J Cyst Fibros. 2014;13(2):156-63.
13. Torphy TJ, Allen J, Cantin AM, Konstan MW, Accurso FJ, Joseloff E, et al. Considerations for the Conduct of Clinical Trials with Antiinflammatory Agents in Cystic Fibrosis. A Cystic Fibrosis Foundation Workshop Report. Ann Am Thorac Soc. 2015;12(9):1398-406.
14. National Institutes of Health. Definitions 2015, April 7 [Available from: https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/fda-bioresearch-monitoring-information/definitions.
15. Cook N, Hansen AR, Siu LL, Abdul Razak AR. Early phase clinical trials to identify optimal dosing and safety. Mol Oncol. 2015;9(5):997-1007.
16. Goss CH, Kaneko Y, Khuu L, Anderson GD, Ravishankar S, Aitken ML, et al. Gallium disrupts bacterial iron metabolism and has therapeutic effects in mice and humans with lung infections. Sci Transl Med. 2018;10(460).
17. Takebe T, Imai R, Ono S. The Current Status of Drug Discovery and Development as Originated in United States Academia: The Influence of Industrial and Academic Collaboration on Drug Discovery and Development. Clin Transl Sci. 2018;11(6):597-606.
18. McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, et al. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). The Lancet Respiratory Medicine. 2014;2(11):902-10.
19. Busse WW, Morgan WJ, Gergen PJ, Mitchell HE, Gern JE, Liu AH, et al. Randomized trial of omalizumab (anti-IgE) for asthma in inner-city children. N Engl J Med. 2011;364(11):1005-15.
20. NIH Definitions Working Group. Biomarkers and surrogate endpoints in clinical research: definitions and conceptual model. . In: Downing G, editor. Biomarkers and Surrogate Endpoints: Clinical Research and Applications Amsterdam: Elsevier; 2000. p. 1-9.
21. Muhlebach MS, Clancy JP, Heltshe SL, Ziady A, Kelley T, Accurso F, et al. Biomarkers for cystic fibrosis drug development. J Cyst Fibros. 2016;15(6):714-23.
22. Mogayzel PJ, Jr., Naureckas ET, Robinson KA, Mueller G, Hadjiliadis D, Hoag JB, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187(7):680-9.
23. Chmiel JF, Konstan MW, Accurso FJ, Lymp J, Mayer-Hamblett N, VanDevanter DR, et al. Use of ibuprofen to assess inflammatory biomarkers in induced sputum: Implications for clinical trials in cystic fibrosis. J Cyst Fibros. 2015;14(6):720-6.
24. Fleming TR, Powers JH. Biomarkers and surrogate endpoints in clinical trials. Statistics in medicine. 2012;31(25):2973-84.
25. Mayer-Hamblett N, Rosenfeld M, Emerson J, Goss C, Aitken M. Developing cystic fibrosis lung transplant referral criteria using predictors of 2-year mortality. Am J Respir Crit Care Med. 2002;166(12 Pt 1):1550-5.
26. Konstan MW, Byard PJ, Hoppel CL, Davis PB. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med. 1995;332(13):848-54.
27. Sawicki GS, McKone EF, Pasta DJ, Millar SJ, Wagener JS, Johnson CA, et al. Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data. Am J Respir Crit Care Med. 2015;192(7):836-42.
28. VanDevanter DR, Konstan MW. Outcome measures for clinical trials assessing treatment of cystic fibrosis lung disease. Clin Investig (Lond). 2012;2(2):163-75.
29. Stanojevic S, Ratjen F. Physiologic endpoints for clinical studies for cystic fibrosis. J Cyst Fibros. 2016;15(4):416-23.
30. Konstan M, Morgan W, Butler S, Pasta D, Craib M, Silva S, et al. Risk factors for rate of decline in forced expiratory volume in one second in children and adolescents with cystic fibrosis. J Pediatr. 2007;151(2):134-9, 9.e1.
31. Ellaffi M, Vinsonneau C, Coste J, Hubert D, Burgel P, Dhainaut J, et al. One-year outcome after severe pulmonary exacerbation in adults with cystic fibrosis. Am J Respir Crit Care Med. 2005;171(2):158-64.
32. Britto M, Kotagal U, Hornung R, Atherton H, Tsevat J, Wilmott R. Impact of recent pulmonary exacerbations on quality of life in patients with cystic fibrosis. Chest. 2002;121(1):64-72.
33. Vandevanter DR, Yegin A, Morgan WJ, Millar SJ, Pasta DJ, Konstan MW. Design and powering of cystic fibrosis clinical trials using pulmonary exacerbation as an efficacy endpoint. J Cyst Fibros. 2011;10(6):453-9.
34. Goss C, Burns J. Exacerbations in cystic fibrosis. 1: Epidemiology and pathogenesis. Thorax. 2007;62(4):360-7.
35. Treggiari MM, Rosenfeld M, Mayer-Hamblett N, Retsch-Bogart G, Gibson RL, Williams J, et al. Early anti-pseudomonal acquisition in young patients with cystic fibrosis: rationale and design of the EPIC clinical trial and observational study’. Contemp Clin Trials. 2009;30(3):256-68.
36. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79.
37. Quittner AL, Buu A, Messer MA, Modi AC, Watrous M. Development and Validation of the Cystic Fibrosis Questionnaire in the United States: A Health-Related Quality-of-Life Measure for Cystic Fibrosis. Chest. 2005;128(4):2347-54.
38. Goss CH, Caldwell E, Gries KC, Leidy NK, Edwards T, Flume PA, et al. Validation of a novel patient-reported respiratory symptoms instrument in cystic fibrosis: CFRSD-CRISS. Pediatric Pulmonology. 2013;48(S36):A251.
39. Retsch-Bogart G, Quittner A, Gibson R, Oermann C, McCoy K, Montgomery A, et al. Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. Chest. 2009;135(5):1223-32.
40. Ramsey B, Boat T. Outcome measures for clinical trials in cystic fibrosis. Summary of a Cystic Fibrosis Foundation consensus conference. J Pediatr. 1994;124(2):177-92.
41. Stalvey MS, Pace J, Niknian M, Higgins MN, Tarn V, Davis J, et al. Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor. Pediatrics. 2017;139(2).
42. VanDevanter DR, Mayer-Hamblett N. Innovating cystic fibrosis clinical trial designs in an era of successful standard of care therapies. Current opinion in pulmonary medicine. 2017;23(6):530-5.
43. VanDevanter DR, Mayer-Hamblett N, Boyle M. Feasibility of placebo-controlled trial designs for new CFTR modulator evaluation. J Cyst Fibros. 2017;16(4):496-8.
44. Clancy JP, Cotton CU, Donaldson SH, Solomon GM, VanDevanter DR, Boyle MP, et al. CFTR modulator theratyping: Current status, gaps and future directions. J Cyst Fibros. 2019;18(1):22-34.
45. Durmowicz AG, Lim R, Rogers H, Rosebraugh CJ, Chowdhury BA. The U.S. Food and Drug Administration’s Experience with Ivacaftor in Cystic Fibrosis. Establishing Efficacy Using In Vitro Data in Lieu of a Clinical Trial. Ann Am Thorac Soc. 2018;15(1):1-2.
46. Rosenfeld M, Wainwright CE, Higgins M, Wang LT, McKee C, Campbell D, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. The Lancet Respiratory medicine. 2018;6(7):545-53.
47. Milla CE, Ratjen F, Marigowda G, Liu F, Waltz D, Rosenfeld M. Lumacaftor/Ivacaftor in Patients Aged 6-11 Years With Cystic Fibrosis Homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2016.
48. Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, et al. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. The Lancet Respiratory medicine. 2016;4(2):107-15.
49. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-72.
50. Burgel PR, Munck A, Durieu I, Chiron R, Mely L, Prevotat A, et al. Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. Am J Respir Crit Care Med. 2020;201(2):188-97.