Minimally invasive pharmacokinetic and pharmacodynamic technologies in hypothesis-testing clinical trials of innovative therapies

P Workman, EO Aboagye, YL Chung… - Journal of the …, 2006 - academic.oup.com
P Workman, EO Aboagye, YL Chung, JR Griffiths, R Hart, MO Leach, RJ Maxwell…
Journal of the National Cancer Institute, 2006academic.oup.com
Clinical trials of new cancer drugs should ideally include measurements of parameters such
as molecular target expression, pharmacokinetic (PK) behavior, and pharmacodynamic (PD)
endpoints that can be linked to measures of clinical effect. Appropriate PK/PD biomarkers
facilitate proof-of-concept demonstrations for target modulation; enhance the rational
selection of an optimal drug dose and schedule; aid decision-making, such as whether to
continue or close a drug development project; and may explain or predict clinical outcomes …
Abstract
Clinical trials of new cancer drugs should ideally include measurements of parameters such as molecular target expression, pharmacokinetic (PK) behavior, and pharmacodynamic (PD) endpoints that can be linked to measures of clinical effect. Appropriate PK/PD biomarkers facilitate proof-of-concept demonstrations for target modulation; enhance the rational selection of an optimal drug dose and schedule; aid decision-making, such as whether to continue or close a drug development project; and may explain or predict clinical outcomes. In addition, measurement of PK/PD biomarkers can minimize uncertainty associated with predicting drug safety and efficacy, reduce the high levels of drug attrition during development, accelerate drug approval, and decrease the overall costs of drug development. However, there are many challenges in the development and implementation of biomarkers that probably explain their disappointingly low implementation in phase I trials. The Pharmacodynamic/Pharmacokinetic Technologies Advisory committee of Cancer Research UK has found that submissions for phase I trials of new cancer drugs in the United Kingdom often lack detailed information about PK and/or PD endpoints, which leads to suboptimal information being obtained in those trials or to delays in starting the trials while PK/PD methods are developed and validated. Minimally invasive PK/PD technologies have logistic and ethical advantages over more invasive technologies. Here we review these technologies, emphasizing magnetic resonance spectroscopy and positron emission tomography, which provide detailed functional and metabolic information. Assays that measure effects of drugs on important biologic pathways and processes are likely to be more cost-effective than those that measure specific molecular targets. Development, validation, and implementation of minimally invasive PK/PD methods are encouraged.
Oxford University Press