Journal article
CUSTOM (Molecular Profiling and Targeted Therapy for Advanced Non-Small Cell Lung Cancer, Small Cell Lung Cancer, and Thymic Malignancies trial): A radical redesign of clinical trial structure—Use of mass customization and product postponement
Journal of clinical oncology, v 31(15_suppl), pp 6524-6524
20 May 2013
Abstract
Abstract only 6524 Background: Traditionally, the structure of clinical trials has relied upon histology as the top eligibility criterion for enrollment. While more modern trial designs (i.e. BATTLE and I-SPY2) have implemented molecular selection for treatment assignment, histology has remained at the top of the clinical trial structure (1. Histology 2. Molecular selection 3. Treatment assignment). CUSTOM is the first completed prospective clinical trial designed by implementing the ideas of mass customization and product postponement into its structure. This report discusses CUSTOM’s innovative clinical trial design strategy. Methods: In CUSTOM, the task of differentiating a particular treatment’s efficacy by statistical analysis based on histology was postponed until the latest possible point in the trial’s structure (1. Molecular selection 2. Treatment assignment 3. Histology). An optimal two-stage phase II design was used to identify compounds with high clinical activity defined by a response rate of more than 40%. Patients were eligible for enrollment regardless of performance status, previous number of therapies or organ function. Results: CUSTOM was able to enroll 668 patients in only 18 months at 2 academic institutions and conduct a parallel and simultaneous evaluation of multiple targeted therapies (5) in molecularly selected patients in 3 cancer histological types. Its design allowed for the inclusion of rare cancers resulting in the largest prospective molecularly based clinical trial in patients with thymic malignancies and small cell lung cancer. Overall, its design resulted in a more rapid, cost-effective and efficient strategy for pharmaceutical development compared to traditional approaches. Conclusions: The traditional methodology used to design and execute clinical trials (i.e., histology-driven, single treatment vs. SOC) is dated, inefficient and expensive. The CUSTOM’s clinical trial design is one approach to increase the efficiency, reduce the cost, and increase the speed of the early phase pharmaceutical development process. Clinical trial information: NCT01306045.
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Details
- Title
- CUSTOM (Molecular Profiling and Targeted Therapy for Advanced Non-Small Cell Lung Cancer, Small Cell Lung Cancer, and Thymic Malignancies trial): A radical redesign of clinical trial structure—Use of mass customization and product postponement
- Creators
- Ariel Lopez-Chavez - Oregon Health & Science UniversityAnish Thomas - National Cancer InstituteArun Rajan - National Cancer InstituteSeth M. Steinberg - National Cancer InstituteDavid M. Dilts - Oregon Health & Science UniversityAustin Doyle - National Cancer InstituteGiuseppe Giaccone - National Cancer Institute
- Publication Details
- Journal of clinical oncology, v 31(15_suppl), pp 6524-6524
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- General Internal Medicine
- Other Identifier
- 991022060043104721