Use of Patient-Reported Outcomes in Phase III Cancer Treatment Trials: Lessons Learned and Future Directions

2007 ◽  
Vol 25 (32) ◽  
pp. 5063-5069 ◽  
Author(s):  
Patricia A. Ganz ◽  
Carolyn C. Gotay
Keyword(s):  
Patient Reported Outcomes ◽  
Lessons Learned ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Future Directions ◽  
Missed Opportunities ◽  
Treatment Trials ◽  
Design Choice ◽  
Patient Reported ◽  
Phase Iii Trials

Purpose This article examines the challenges, opportunities, and successes that have occurred in the incorporation of patient-reported outcomes (PROs) in phase III cancer clinical trials. Methods An informal survey of the leadership of US cooperative group PRO investigators identified diverse trials in which PROs had been measured. Exemplary trials were selected for lessons learned and for examination of successful strategies. Results We review four challenging trials in depth, illustrating some of the difficulties in integrating PROs within treatment trials, including issues related to missing data, lack of procedures for monitoring patients and ensuring collection of PRO data, and missed opportunities in publication of treatment and PRO outcomes together. Four examples of successful trials are highlighted. Conclusion As a result of this review, the authors make specific recommendations related to the use of PROs in phase III trials, focusing on issues related to design, choice of PRO instrument and frequency of administration, analysis, and publication strategies.

Patient-Reported Outcomes in Phase II Cancer Clinical Trials: Lessons Learned and Future Directions

2007 ◽  
Vol 25 (32) ◽  
pp. 5058-5062 ◽  
Author(s):  
Lynne I. Wagner ◽  
Lari Wenzel ◽  
Edward Shaw ◽  
David Cella
Keyword(s):  
Clinical Trials ◽  
Phase Ii ◽  
Symptom Management ◽  
Patient Reported Outcomes ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
Phase Ii Trials ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
The Impact

With increasing limits on the resources available to conduct cancer clinical trials, the inclusion of patient-reported outcomes (PROs) in treatment and symptom management trials must be prioritized. Although it has been suggested on occasion that phase III trials should take precedence over phase II trials, we argue that there is a clear and important role for PRO assessment in phase II trials going forward. To illustrate the value realized from including PROs in phase II trials, we provide case examples from cancer treatment and supportive care. The benefits of including PROs in symptom management intervention research are exemplified using phase II trials targeting cognitive impairment. The inclusion of PROs in phase II cancer clinical trials adds important information about the impact of treatment in health-related quality of life, and advances the science of PRO measurement. These contributions significantly enhance the design of phase III trials, ultimately leading to the efficient utilization of clinical trial resources.

A comparison of patient-reported outcomes (PROs) in National Cancer Institute-sponsored cancer treatment trials conducted during two time periods: 1999–2003 and 2003–2008

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 6618-6618
Author(s):  
A. M. O'Mara ◽  
A. M. Denicoff ◽  
B. Reeve ◽  
R. Burns ◽  
T. Trimble
Keyword(s):  
Quality Of Life ◽  
Patient Reported Outcomes ◽  
Phase Iii ◽  
Disease Treatment ◽  
Treatment Trials ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
Secondary Endpoints ◽  
Time Periods

6618 Background: Over the past decade, the U.S. National Cancer Institute (NCI) support for incorporating Patient-Reported Outcomes (PROs) into disease treatment clinical trials has grown considerably, both in the number of trials, as well as the types of research questions being addressed and the measures used to answer the questions. This presentation will compare the breadth of NCI supported Phase III treatment trials in breast, colo-rectal, lung, and prostate cancers that include PRO secondary endpoints activated during two time periods: 1999–2003 and 2003–2008. Both time periods will be compared to identify the number and type of PRO measures; in addition the targeted PRO domain will be examined by cancer site. Methods: For this study, PRO was broadly defined to include any self-report questionnaire that gathered data on the potential impact that disease treatment might have on patient functioning, quality of life, or symptoms. Using the terms, 'protocol title, lead disease, phase III, quality of life, and instrument name,’ a search was conducted of the NCI database containing breast, colo-rectal, lung, and prostate Phase III treatment trials activated during the two time periods. If a scale was used more than once or if different versions of the scale were used, i.e., FACT-G and FACT-BR, it was only counted once. Results: Between 1999 and 2003, 42 phase III trials in the four major cancers were activated, with 15 trials having one or more PROs as secondary endpoints. Between 2003 and 2008, 50 phase III trials in the four major cancers were activated, with 28 trials having one or more PROs as secondary endpoints. Although the data reveal a 20% increase in the number of phase III trials in the 4 diseases between the two time periods, the number of trials with PRO endpoints has doubled. Conclusions: This significant increase in the use of PROs in phase III trials gives investigators additional data to fully analyze the impact of new treatments. These PRO data will provide researchers, clinicians, and future patients with a more in-depth understanding of the potential for new cancer therapies and technologies to extend both the quantity and quality of life. No significant financial relationships to disclose.

scholarly journals Suggestions for improving the design of clinical trials in multiple sclerosis—results of a systematic analysis of completed phase III trials

2019 ◽  
Vol 10 (4) ◽  
pp. 425-436 ◽  
Author(s):  
Sinje Gehr ◽  
Thomas Kaiser ◽  
Reinhold Kreutz ◽  
Wolf-Dieter Ludwig ◽  
Friedemann Paul
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Progressive Multiple Sclerosis ◽  
Phase Iii ◽  
Primary Progressive Multiple Sclerosis ◽  
Systematic Overview ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
Secondary Endpoints

AbstractThis manuscript reviews the primary and secondary endpoints of pivotal phase III trials with immunomodulatory drugs in multiple sclerosis (MS). Considering the limitations of previous trial designs, we propose new standards for the planning of clinical trials, taking into account latest insights into MS pathophysiology and patient-relevant aspects. Using a systematic overview of published phase III (pivotal) trials performed as part of application for drug market approval, we evaluate the following characteristics: trial duration, number of trial participants, comparators, and endpoints (primary, secondary, magnetic resonance imaging outcome, and patient-reported outcomes). From a patient perspective, the primary and secondary endpoints of clinical trials are only partially relevant. High-quality trial data pertaining to efficacy and safety that stretch beyond the time frame of pivotal trials are almost non-existent. Understanding of long-term benefits and risks of disease-modifying MS therapy is largely lacking. Concrete proposals for the trial designs of relapsing (remitting) multiple sclerosis/clinically isolated syndrome, primary progressive multiple sclerosis, and secondary progressive multiple sclerosis (e.g., study duration, mechanism of action, and choice of endpoints) are presented based on the results of the systematic overview. Given the increasing number of available immunotherapies, the therapeutic strategy in MS has shifted from a mere “relapse-prevention” approach to a personalized provision of medical care as to the choice of the appropriate drugs and their sequential application over the course of the disease. This personalized provision takes patient preferences as well as disease-related factors into consideration such as objective clinical and radiographic findings but also very burdensome symptoms such as fatigue, depression, and cognitive impairment. Future trial designs in MS will have to assign higher relevance to these patient-reported outcomes and will also have to implement surrogate measures that can serve as predictive markers for individual treatment response to new and investigational immunotherapies. This is an indispensable prerequisite to maximize the benefit of individual patients when participating in clinical trials. Moreover, such appropriate trial designs and suitable enrolment criteria that correspond to the mode of action of the study drug will facilitate targeted prevention of adverse events, thus mitigating risks for individual study participants.

Patient-Reported Outcomes Assessment in Cancer Trials: Taking Stock, Moving Forward

2007 ◽  
Vol 25 (32) ◽  
pp. 5133-5140 ◽  
Author(s):  
Joseph Lipscomb ◽  
Bryce B. Reeve ◽  
Steven B. Clauser ◽  
Jeffrey S. Abrams ◽  
Deborah Watkins Bruner ◽  
...  
Keyword(s):  
Data Collection ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Lessons Learned ◽  
Phase Iii ◽  
Health Providers ◽  
Cancer Trial ◽  
Management Procedures ◽  
Patient Reported ◽  
Cancer Trials

To evaluate and improve the use of cancer trial end points that reflect the patient's own perspective, the National Cancer Institute organized an international conference, Patient-Reported Outcomes Assessment in Cancer Trials (PROACT), in 2006. The 13 preceding articles in this special issue of the Journal were commissioned in preparation for or in response to the PROACT conference, which was cosponsored by the American Cancer Society. Drawing from these articles and also commentary from the conference itself, this concluding report takes stock of what has been learned to date about the successes and challenges in patient-reported outcome (PRO) assessment in phase III, phase II, and symptom management trials in cancer and identifies ways to improve the scientific soundness, feasibility, and policy relevance of PROs in trials. Building on this synthesis of lessons learned, this article discusses specific administrative policies and management procedures to improve PRO data collection, analysis, and dissemination of findings; opportunities afforded by recent methodologic and technologic advances in PRO data collection and analysis to enhance the scientific soundness and cost efficiency of PRO use in trials; and the importance of better understanding the usefulness of PRO data to the full spectrum of cancer decision makers, including patients and families, health providers, public and private payers, regulatory agencies, and standards-setting organizations.

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