scholarly journals The Past, Present, and Future of Economic Evaluations of Precision Medicine at the Committee for Economic Analyses of the Canadian Cancer Trials Group

2021 ◽  
Vol 28 (5) ◽  
pp. 3649-3658
Author(s):  
Kelvin K. W. Chan ◽  
Matthew C. Cheung ◽  
Dean A. Regier ◽  
Annette Hay ◽  
Alexander V. Louie ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Precision Medicine ◽  
Clinical Trial Design ◽  
Economic Evaluations ◽  
Economic Evidence ◽  
New Paradigm ◽  
High Performing ◽  
Clinical Trial Evidence ◽  
Cancer Trials ◽  
High Level

Precision medicine in oncology poses unique challenges to the generation of clinical and economic evidence used for cost-effectiveness analyses that can inform health technology assessment. The conduct of randomized controlled trials for biomarker-specific therapies targeted towards small populations has limitations in regard to feasibility, timeliness, and cost. These limitations result in associated challenges for groups involved in the generation of economic evidence to inform treatment-related decision making, including the Committee of Economic Analysis (CEA) at the Canadian Cancer Trials Group (CCTG). We provide a high-level description and vision about the new paradigm of clinical trial design, generation of economic evidence, and novel approaches to economic evaluations necessary in the space of precision medicine in oncology in Canada. The CEA’s previous approach to precision medicine, including master protocol designs and single-arm studies, is reviewed. Methods and approaches currently under consideration by the CEA and national collaborators, such as the role of real-world and clinical trial evidence in enabling life-cycle assessment of therapies, are explored. Finally, future initiatives being planned in the space of precision medicine at CCTG, such as the incorporation of correlative studies to identify and test high-performing biomarkers in trials, are discussed.

scholarly journals ANALYZING DIFFERENCES IN THE COSTS OF TREATMENT ACROSS CENTERS WITHIN ECONOMIC EVALUATIONS

2001 ◽  
Vol 17 (2) ◽  
pp. 155-163 ◽  
Author(s):  
Douglas Coyle ◽  
Michael F. Drummond
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
New Technology ◽  
Clinical Trial Design ◽  
Experimental Treatment ◽  
Economic Evaluations ◽  
Hospital Practice ◽  
Health Technologies ◽  
The United Kingdom ◽  
Study Results

Objectives: Assessments of health technologies increasingly include economic evaluations conducted alongside clinical trials. One particular concern with economic evaluations conducted alongside clinical trials is the generalizability of results from one setting to another. Much of the focus relating to this topic has been on the generalizability of results between countries. However, the characteristics of clinical trial design require further consideration of the generalizability of cost data between centers within a single country, which could be important in decisions about adoption of the new technology.Methods: We used data from a multicenter clinical trial conducted in the United Kingdom to assess the degree of variation in costs between patients and between treatment centers and the determinants of the degree of such variation.Results: The variation between patients was statistically significant for both the experimental and conventional treatments. However, the degree of variation between centers was only statistically significant for the experimental treatment. Such variation appeared to be a result of hospital practice, such as payment mechanisms for staff and provision of hostel accommodation, rather than variations in physical resource use or substantive differences in cost structure.Conclusions: Multicenter economic evaluations are necessary for determining the variations in hospital practice and characteristics that can in turn determine the generalizability of study results to other settings. Such analyses can identify issues that may be important in adopting a new health technology. Analysis is required of similar large multicenter trials to confirm these conclusions.

scholarly journals Adapting clinical trial design to maintain meaningful outcomes during a multicenter asthma trial in the precision medicine era

2019 ◽  
Vol 77 ◽  
pp. 98-103 ◽  
Author(s):  
Christine A. Sorkness ◽  
Tonya S. King ◽  
Anne-Marie Dyer ◽  
Vernon M. Chinchilli ◽  
David T. Mauger ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Precision Medicine ◽  
Trial Design ◽  
Clinical Trial Design

scholarly journals Phase III Precision Medicine Clinical Trial Designs That Integrate Treatment and Biomarker Evaluation

2019 ◽  
pp. 1-9 ◽  
Author(s):  
Mei-Yin C. Polley ◽  
Edward L. Korn ◽  
Boris Freidlin
Keyword(s):  
Clinical Trial ◽  
Drug Development ◽  
Precision Medicine ◽  
Randomized Clinical Trial ◽  
Cancer Genomics ◽  
Clinical Trial Design ◽  
Predictive Biomarkers ◽  
Phase Iii ◽  
Cancer Trial ◽  
Clinical Trial Designs

Recent advances in biotechnology and cancer genomics have afforded enormous opportunities for development of more effective anticancer therapies. A key thrust of this modern drug development paradigm is successful identification of predictive biomarkers that can distinguish patients who might be sensitive to new targeted therapies. To respond to this challenge, a number of phase III cancer trial designs integrating biomarker-based objectives have been proposed and implemented in oncology drug development. In this article, we provide an updated review of commonly used biomarker-based randomized clinical trial designs, with a particular focus on design efficiency. When the efficacy of a new therapy may be limited to a biomarker-defined subgroup, the choice of an appropriate randomized clinical trial design should be guided by the strength of the biomarker’s credentials. If compelling evidence indicates that a targeted therapy is beneficial only in a particular biomarker-defined subgroup, an enrichment design should be used. If there is strong evidence that the treatment is likely to be more beneficial in the biomarker-positive patients but a meaningful benefit is also possible in the biomarker-negative patients, then a properly powered biomarker-stratified design (eg, a subgroup-specific or Marker Sequential Test strategy) would provide the most rigorous determination of the sensitive populations. If the evidence supporting the predictive value of the biomarker is weak and the treatment is expected to work in the overall population, then a fallback design could be used. Careful selection of an appropriate phase III design strategy that integrates evaluation of a new anticancer therapy and its companion diagnostic is critical to the success of precision medicine in oncology.

scholarly journals Evolution of Clinical Trial Design in Early Drug Development: The Use of Expansion Cohorts (ECS) in Phase I Cancer Trials (PITS)

2012 ◽  
Vol 23 ◽  
pp. ix158
Author(s):  
A. Manji ◽  
I. Brana Garcia ◽  
E. Amir ◽  
I.F. Tannock ◽  
P. Bedard ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Drug Development ◽  
Phase I ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Cancer Trials ◽  
Early Drug

Integrating radiomics into clinical trial design

2019 ◽  
Vol 63 (4) ◽  
Author(s):  
Jessica J. Waninger ◽  
Michael D. Green ◽  
Catherine Cheze Le Rest ◽  
Benjamin Rosen ◽  
Issam El Naqa
Keyword(s):  
Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design

Avenues to Precision Oncology

2020 ◽  
pp. 36-45
Author(s):  
Alexander Meisel
Keyword(s):  
Precision Medicine ◽  
Trial Design ◽  
Ad Hoc ◽  
Molecular Targets ◽  
Clinical Trial Design ◽  
Predictive Biomarkers ◽  
Precision Oncology ◽  
Companion Diagnostics ◽  
Bona Fide ◽  
The One

Until recently, the clinical management of cancer heavily relied on anatomical and histopathological criteria, with ad hoc guidelines directing the therapeutic choices in specific indications. In the last years, the development and therapeutic implementation of novel anticancer therapies significantly improved the clinical outcome of cancer patients. Nonetheless, such cutting-edge approaches revealed the limitation of the one-size-fits-all paradigm. The newly discovered molecular targets can be exploited either as bona fide targets for subsequent drug development, or as tools to precision medicine, in the form of prognostic and/or predictive biomarkers. This article provides an overview of some of the most recent advances in precision medicine in oncology, with a focus on novel tissue-agnostic anticancer therapies. The definition and implementation of biomarkers and companion diagnostics in clinical trials and clinical practice are also discussed, as well as the changing landscape in clinical trial design.

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