Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.
E9(R1) Statistical principles for clinical trials: addendum: estimates and sensitivity analysis in clinical trials.
International standards for the analysis of quality of life and patient-reported outcomes in randomized controlled cancer trials: recommendations from the SISAQOL consortium.
Lancet Oncol. 2020; 21: e83-e96
United States Food and Drug Administration review of statistical analysis of patient-reported outcomes in lung cancer clinical trials approved between January 2008 and December 2017.
Lancet Oncol. 2019; 20: e582-e589
Statistical analysis of patient-reported outcome data in randomized controlled trials of locally advanced and metastatic breast cancer: a systematic review.
Lancet Oncol. 2018; 19: e459-e469
Patient-reported outcomes as a component of the primary endpoint in a double-blind, placebo-controlled trial in advanced pancreatic cancer.
J Manage pain symptoms. 2009; 37: 135-143
A systematic review of reports on health-related quality of life in phase III ovarian cancer clinical trials: room for improvement.
Oncologist. 2018; 23: 203-213
Oncology Drug Advisory Committee Brief BLA 125477/S34 ramucirumab.
Review of patient-reported outcomes in multiple myeloma registration trials: highlighting areas for improvement.
J blood cancer. 2021; 11: 148
Time to deterioration of health-related quality of life score as a longitudinal analysis modality for health-related quality of life studies in oncology: do we need RECIST for quality of life to achieve standardization? .
Qual Life Res. 2015 ; 24: 5-18
Nivolumab versus standard monotherapy of investigator’s choice in relapsed or metastatic head and neck squamous cell carcinoma (CheckMate 141): health-related quality of life results from a randomized phase 3 trial.
Lancet Oncol. 2017; 18: 1104-1115
To assess time to progression of pain in multiple myeloma.
Blood. 2018; 132 2299
Time to deterioration in randomized cancer clinical trials for patient-reported outcome data: a systematic review.
Qual Life Res. 2020; 29: 867-878
E9 Statistical principles for clinical trials.
Estimands in clinical trials – broadening the perspective.
StatMed. 2017; 36: 5-19
Aligning Estimators with Estimators in Clinical Trials: Putting ICH E9(R1) Guidelines into Practice.
Ther Innov Regul Sci. 2020; 54: 353-364
A taxonomy of estimates for regulatory clinical trials with interruptions.
StatMed. 2016; 35: 2865-2875
Defining treatment effects: a regulatory perspective.
Clinical tests. 2019; 16: 345-349
Choice of estimands in clinical trials: implementation of ICH E9(R1).
Ther Innov Regul Sci. 2020; 54: 324-341
Demystifying the estimation framework: a case study using patient-reported outcomes in oncology.
Lancet Oncol. 2020; 21: e488-e494
Public Orientation Workshop on Patient-Oriented Drug Development: Integrating Clinical Outcome Assessments into Endpoints for Regulatory Decision-Making.
Contribution of early endpoints of clinical benefit to decreased lung cancer mortality rates.
JAMA Oncol. 2021; 7: 829-830
The effect of advances in lung cancer treatment on population mortality.
N Engl J Med. 2020; 383: 640-649
Key lung cancer statistics.
What bothers lung cancer patients the most? A prospective electronic longitudinal study of patient-reported outcomes in advanced non-small cell lung cancer.
Cancer Care Support. 2015 ; 23: 3455-3463
Prevalence of symptoms in patients with lung and colorectal cancer.
J Manage pain symptoms. 2015 ; 49: 192-202
114th US Congress. 21st Century Remedies Act.
FDA’s series of patient-centered drug development guidance to improve the integration of the patient’s voice into medical product development and regulatory decision-making.
Qualification Program for Clinical Outcome Assessment (COA).
Qualification Process for Drug Development Tools: Guidance for Industry and FDA Staff.
Key Patient-Reported Outcomes in Cancer Clinical Trials: Guidance for Industry – Draft Guidance.
Roydhouse J, Kluetz P, King-Kallimanis B. Time to endpoint deterioration in oncology clinical trials. Advance the science of endpoints; September 20-21, 2018.
New response endpoints in solid tumours: revised RECIST guideline (version 1.1).
Eur J Cancer. 2009; 45: 228-247
Patient-Reported Outcomes After Discontinuation of Treatment: Commercial Clinical Trial Data on Four Types of Cancer.
Value health. 2021; 24: 1302-1307
Issues with the use of progression-free survival when evaluating oncology products.
J Clin Oncol. 2009; 27: 2874-2880
Practical and statistical issues related to missing data for patient-reported longitudinal outcomes.
Statistical methods Res. med. 2014 ; 23: 440-459
Prevention and treatment of missing data in clinical trials.
National Academies Press,
Quantification of treatment effect for time-to-event parameters: estimates, analysis strategies and beyond.
PharmStat. 2019; 18: 145-165
Assessment of the impact of COVID-19 on the purpose of clinical trials and analysis of clinical trials in oncology – application of the Estimand framework.
Stat Biopharm Res. 2020; 12: 427-437
Estimand framework: are we asking the right questions? A case study in the setting of a solid tumor.
PharmStat. 2021; 20: 324-334
Clinical trial endpoints for approving cancer drugs and biologics: guidance for industry.
Quality of life: evaluation, analysis and interpretation of results reported by patients.
John Wiley and sons,
New York, NY2013
The role of statistics in regulatory decision-making.
Ther Innov Regul Sci. 2014 ; 48: 10-19