Scientific publications
Strategies to design clinical studies to identify predictive biomarkers in cancer research. Scientific Publication
Jose Luis Perez-Gracia 1 , Miguel F Sanmamed 2 , Ana Bosch 3 , Ana Patiño-Garcia 4 , Kurt A Schalper 5 , Victor Segura 6 , Joaquim Bellmunt 7 , Josep Tabernero 8 , Christopher J Sweeney 7 , Toni K Choueiri 7 , Miguel Martín 9 , Juan Pablo Fusco 10 , Maria Esperanza Rodriguez-Ruiz 11 , Alfonso Calvo 12 , Celia Prior 13 , Luis Paz-Ares 14 , Ruben Pio 15 , Enrique Gonzalez-Billalabeitia 16 , Alvaro Gonzalez Hernandez 17 , David Páez 18 , Jose María Piulats 19 , Alfonso Gurpide 20 , Mapi Andueza 10 , Guillermo de Velasco 7 , Roberto Pazo 21 , Enrique Grande 22 , Pilar Nicolas 23 , Francisco Abad-Santos 24 , Jesus Garcia-Donas 25 , Daniel Castellano 14 , María J Pajares 26 , Cristina Suarez 8 , Ramon Colomer 27 , Luis M Montuenga 26 , Ignacio Melero 11
Abstract
The discovery of reliable biomarkers to predict efficacy and toxicity of anticancer drugs remains one of the key challenges in cancer research. Despite its relevance, no efficient study designs to identify promising candidate biomarkers have been established. This has led to the proliferation of a myriad of exploratory studies using dissimilar strategies, most of which fail to identify any promising targets and are seldom validated.
The lack of a proper methodology also determines that many anti-cancer drugs are developed below their potential, due to failure to identify predictive biomarkers. While some drugs will be systematically administered to many patients who will not benefit from them, leading to unnecessary toxicities and costs, others will never reach registration due to our inability to identify the specific patient population in which they are active.
Despite these drawbacks, a limited number of outstanding predictive biomarkers have been successfully identified and validated, and have changed the standard practice of oncology.
In this manuscript, a multidisciplinary panel reviews how those key biomarkers were identified and, based on those experiences, proposes a methodological framework-the DESIGN guidelines-to standardize the clinical design of biomarker identification studies and to develop future research in this pivotal field.
CITATION Cancer Treat Rev. 2017 Feb;53:79-97. doi: 10.1016/j.ctrv.2016.12.005. Epub 2016 Dec 30.
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