Do contemporary randomized controlled trials meet ESMO thresholds for meaningful clinical benefit?

Annals of Oncology

Volumn 28, Issue 1, 2017, Pages 157-162

  Del Paggio, J C ^a   Azariah, B ^b   Sullivan, R ^c   Hopman, W M ^a   James, F V ^b   Roshni, S ^b   Tannock, I F ^d   Booth, C M ^a,e  

^a QUEEN S UNIVERSITY   (Canada)

^b REGIONAL CANCER CENTRE   (India)

^c KING'S COLLEGE LONDON   (United Kingdom)

^d PRINCESS MARGARET HOSPITAL   (Canada)

^e QUEEN S UNIVERSITY   (Canada)

Author keywords

Benefit; ESMO MCBS; Meaningful; Value

Indexed keywords

ARTICLE; BREAST CANCER; CANCER GROWTH; COHORT ANALYSIS; COLORECTAL CANCER; DISEASE FREE SURVIVAL; EFFECT SIZE; EVENT FREE SURVIVAL; HUMAN; NON SMALL CELL LUNG CANCER; OVERALL SURVIVAL; PANCREAS CANCER; PRACTICE GUIDELINE; PROGRESSION FREE SURVIVAL; PROSPECTIVE STUDY; QUALITY CONTROL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECURRENCE FREE SURVIVAL; SAMPLE SIZE; SYSTEMATIC REVIEW; TREATMENT FAILURE; TREATMENT RESPONSE; EUROPE; MEDICAL SOCIETY; ONCOLOGY; STANDARDS;

EUROPE; HUMANS; MEDICAL ONCOLOGY; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SOCIETIES, MEDICAL;

EID: 85015742215     PISSN: 09237534     EISSN: 15698041     Source Type: Journal    
DOI: 10.1093/annonc/mdw538     Document Type: Article

References (15)

1. Light DW, Lexchin J. Why do cancer drugs get such an easy ride? BMJ 2015; 350: h2068.
2. Booth CM, Cescon DW, Wang L et al. Evolution of the randomized controlled trial in oncology over three decades. J Clin Oncol 2008; 26: 5458-5464.
3. Kay A, Higgins J, Day AG et al. Randomized controlled trials in the era of molecular oncology: methodology, biomarkers, and end points. Ann Oncol 2012; 23: 1646-1651.
4. Vera-Badillo FE, Shapiro R, Ocana A et al. Bias in reporting of end points of efficacy and toxicity in randomized, clinical trials for women with breast cancer. Ann Oncol 2013; 24: 1238-1244.
5. Cherny NI, Sullivan R, Dafni U et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol 2015; 26: 1547-1573.
6. Ridker PM, Torres J. Reported outcomes in major cardiovascular clinical trials funded by for-profit and not-for-profit organizations: 2000-2005. JAMA 2006; 295: 2270-2274.
7. Kiesewetter B, Raderer M, Steger GG et al. The European Society for Medical Oncology Magnitude of Clinical Benefit Scale in daily practice: a single institution, real-life experience at the Medical University of Vienna. ESMO open. 2016; 1(4): e000066.
8. Ellis LM, Bernstein DS, Voest EE et al. American Society of Clinical Oncology perspective: raising the bar for clinical trials by defining clinically meaningful outcomes. J Clin Oncol 2014; 32: 1277-1280.
9. Kumar H, Fojo T, Mailankody S. An appraisal of clinically meaningful outcomes guidelines for oncology clinical trials. JAMA Oncol 2016; 2(9): 1238-40.
10. Schnipper LE, Davidson NE, Wollins DS et al. American society of clinical oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol 2015; 33: 2563-2577.
11. Schnipper LE, Davidson NE, Wollins DS et al. Updating the american society of clinical oncology value framework: revisions and reflections in response to comments received. J Clin Oncol 2016; 2(9): 1238-40.
12. Saltz LB. Progress in cancer care: the hope, the hype, and the gap between reality and perception. J Clin Oncol 2008; 26: 5020-5021.
13. Hillner BE, Smith TJ. Efficacy does not necessarily translate to cost effectiveness: a case study in the challenges associated with 21st-century cancer drug pricing. J Clin Oncol 2009; 27: 2111-2113.
14. Katz R. FDA: evidentiary standards for drug development and approval. NeuroRx 2004; 1: 307-316.
15. Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA 2011; 305: 2320-2326.