Potential for therapeutic benefit among cystic fibrosis populations excluded from clinical trials or labeling of marketed therapies

Annals of the American Thoracic Society

Volumn 13, Issue 11, 2016, Pages 1890-1893

  VanDevanter, Donald R ^a   Heltshe, Sonya L ^b   LiPuma, John J ^c  

^a CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

Cystic fibrosis; Excluded subjects; Off label; Risk and benefit

Indexed keywords

ANTIBIOTIC AGENT; DORNASE ALFA; TOBRAMYCIN; AMINOPHENOL DERIVATIVE; IVACAFTOR; QUINOLONE DERIVATIVE;

CLINICAL STUDY; CLINICAL TRIAL (TOPIC); COHORT ANALYSIS; CYSTIC FIBROSIS; DRUG EFFICACY; DRUG SAFETY; FORCED EXPIRATORY VOLUME; HEALTH CARE ACCESS; HEALTH CARE COST; HUMAN; LUNG FUNCTION; NEPHROTOXICITY; OFF LABEL DRUG USE; PATIENT CARE; PATIENT SELECTION; PHYSICIAN; PROGNOSIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RISK BENEFIT ANALYSIS; SOCIAL STATUS; TREATMENT OUTCOME; RISK ASSESSMENT;

AMINOPHENOLS; CLINICAL TRIALS AS TOPIC; CYSTIC FIBROSIS; HEALTH CARE COSTS; HUMANS; OFF-LABEL USE; PATIENT SELECTION; QUINOLONES; RISK ASSESSMENT;

EID: 84995452405     PISSN: 23256621     EISSN: None     Source Type: Journal    
DOI: 10.1513/AnnalsATS.201606-462PS     Document Type: Review

References (31)

1. Vertex Pharmaceuticals. Kalydeco prescribing information. 2015 [accessed 2016 June 14]. Available from: http://pi.vrtx.com/files/ uspi-ivacaftor.pdf
2. Vertex Pharmaceuticals. ORKAMBI prescribing information. 2016 [accessed 2016 June 14]. Available from: http://pi.vrtx.com/files/ uspi-lumacaftor-ivacaftor.pdf
3. Orenstein DM, O'Sullivan BP, Quinton PM. Cystic fibrosis: breakthrough drugs at break-the-bank prices. Glob Adv Health Med 2015;4:8-57.
4. Silverman E. Orkambi's slick unveiling puts insurers in a bind. Managed Care Magazine 2015 [accessed 2016 June 14]. Available from: http://www.managedcaremag.com/archives/2015/8/orkambis-slickunveiling- puts-insurers-bind
5. American Academy of Pediatrics, Committee on Drugs. Uses of drugs not described in the package insert (off-label uses). Pediatrics 2002; 110:181-183.
6. Dresser R, Frader J. Off-label prescribing: a call for heightened professional and government oversight. J Law Med Ethics 2009;37: 476-486, 396.
7. Center for Biologics Evaluation and Research (CBER), Center for Drug Evaluation and Research (CDER), Food and Drug Administration, U.S. Department of Health and Human Services. Guidance for industry: providing clinical evidence of effectiveness for human drug and biological products. 1998 May [accessed 2016 June 14]. Available from: http://www.fda.gov/downloads/Drugs/GuidanceCompliance% 20RegulatoryInformation/Guidances/UCM078749.pdf1 Providing1clinical1evidence1of1effectiveness1for1human1 and1bioclient=FDAgovsite=FDAgovlr=proxystylesheet= FDAgovoutput=xml-no-dtdie=UTF-8access=poe=UTF-8
8. Dodge JA, Lewis PA, Stanton M, Wilsher J. Cystic fibrosis mortality and survival in the UK: 1947-2003. Eur Respir J 2007;29:522-526.
9. Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry, 2014: annual data report to the center directors. Bethesda, MD: Cystic Fibrosis Foundation; 2015.
10. Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams- Warren J, Vasiljev-K M, Borowitz D, Bowman CM, Marshall BC, et al.; Cystic Fibrosis Inhaled Tobramycin Study Group. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. N Engl J Med 1999;340:23-30.
11. Retsch-Bogart GZ, Quittner AL, Gibson RL, Oermann CM, McCoy KS, Montgomery AB, Cooper PJ. Efficacy and safety of inhaled aztreonam lysine for airway Pseudomonas in cystic fibrosis. Chest 2009;135:1223-1232.
12. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, Griese M, McKone EF, Wainwright CE, Konstan MW, et al.; VX08- 770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011;365: 1663-1672.
13. Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, et al.; TRAFFIC Study Group; TRANSPORT Study Group. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015;373:220-231.
14. VanDevanter DR, Kahle JS, O'Sullivan AK, Sikirica S, Hodgkins PS. Cystic fibrosis in young children: a review of disease manifestation, progression, and response to early treatment. J Cyst Fibros 2016;15: 147-157.
15. Ramsey BW, Boat TF. Outcome measures for clinical trials in cystic fibrosis: summary of a Cystic Fibrosis Foundation consensus conference. J Pediatr 1994;124:177-192.
16. Fuchs HJ, Borowitz DS, Christiansen DH, Morris EM, Nash ML, Ramsey BW, Rosenstein BJ, Smith AL, Wohl ME; Pulmozyme Study Group. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med 1994;331:637-642.
17. VanDevanter D, Konstan MW. CF drug developers: victims of our own success. Respir Drug Deliv 2008;1:11-18.
18. State of California-Health and Human Services Agency, Department of Health Care Services. Lumacaftor/ivacaftor (Orkambi). N.L. 06-0915. 2015 Sept 4 [accessed 2016 Sept 10]. Available from: http://www.dhcs.ca.gov/services/ccs/Documents/ccsnl060915.pdf
19. Illinois Department of Healthcare and Family Services. Orkambi (lumacaftor-ivacaftor tablet): prior authorization criteria. 2015 September [accessed 2016 Sept 10]. Available from: http://www. illinois.gov/hfs/SiteCollectionDocuments/OrkambiCriteria.pdf
20. Division of Medical Assistance Programs, Oregon Health Authority. Oregon Medicaid Pharmaceutical Services prior authorization criteria. 2016 Jan 1 [accessed 2016 Sept 10]. Available from: https:// www.oregon.gov/oha/healthplan/tools/Oregon%20Medicaid%20PA %20Criteria, %20January%202016.pdf
21. Magellan Health Services. Clinical criteria, step therapy, and quantity limits for TennCare preferred drug list (PDL). 2016 July 20 [accessed 2016 Sept 10]. Available from: https://tenncare.magellanhealth.com/ static/docs/Preferred-Drug-List-and-Drug-Criteria/Criteria-PDL. pdf#nameddest=cystic-fibrosis-agents
22. Quittner AL, Schechter MS, Rasouliyan L, Haselkorn T, Pasta DJ, Wagener JS. Impact of socioeconomic status, race, and ethnicity on quality of life in patients with cystic fibrosis in the United States. Chest 2010;137:642-650.
23. Schechter MS, McColley SA, Silva S, Haselkorn T, Konstan MW, Wagener JS; Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis; North American Scientific Advisory Group for ESCF. Association of socioeconomic status with the use of chronic therapies and healthcare utilization in children with cystic fibrosis. J Pediatr 2009;155:634-9.e1-4.
24. VanDevanter DR, Pasta DJ, Konstan MW. Improvements in lung function and height among cohorts of 6-year-olds with cystic fibrosis from 1994 to 2012. J Pediatr 2014;165:1091-1097.e2.
25. Isles A, Maclusky I, Corey M, Gold R, Prober C, Fleming P, Levison H. Pseudomonas cepacia infection in cystic fibrosis: an emerging problem. J Pediatr 1984;104:206-210.
26. Muhdi K, Edenborough FP, Gumery L, O'Hickey S, Smith EG, Smith DL, Stableforth DE. Outcome for patients colonised with Burkholderia cepacia in a Birmingham adult cystic fibrosis clinic and the end of an epidemic. Thorax 1996;51:374-377.
27. Lipuma JJ. The changing microbial epidemiology in cystic fibrosis [review]. Clin Microbiol Rev 2010;23:299-323.
28. Kaminski R, Nazareth D. A successful uncomplicated CF pregnancy while remaining on ivacaftor. J Cyst Fibros 2016;15:133-134.
29. Goss CH, VanDevanter DR. CFTR modulators and pregnancy: our work has only just begun. J Cyst Fibros 2016;15:6-7.
30. Quan JM, Tiddens HA, Sy JP, McKenzie SG, Montgomery MD, Robinson PJ, Wohl ME, Konstan MW; Pulmozyme Early Intervention Trial Study Group. A two-year randomized, placebo-controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities. J Pediatr 2001;139:813-820.
31. Murphy TD, Anbar RD, Lester LA, Nasr SZ, Nickerson B, VanDevanter DR, Colin AA. Treatment with tobramycin solution for inhalation reduces hospitalizations in young CF subjects with mild lung disease. Pediatr Pulmonol 2004;38:314-320