Scientific publications

Strategies to design clinical studies to identify predictive biomarkers in cancer research

Feb 1, 2017 | Magazine: Cancer Treatment Review

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


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.

CITA DEL ARTÍCULO  Cancer Treat Rev. 2017 Feb;53:79-97.  doi: 10.1016/j.ctrv.2016.12.005. Epub 2016 Dec 30.