K.K. Jain, Textbook of Personalized Medicine, DOI 10.1007/978-1-4939-2553-7_22, 665
© Springer Science+Business Media New York 2015
Chapter 22
Regulatory Aspects of Personalized Medicine
Introduction
The regulatory agencies have not laid down any specifi c guidelines for the personalized
medicines. Most of the discussion relevant to this topic is covered under the over-
lapping components of personalized medicine: pharmacogenetics, pharmacoge-
nomics, molecular diagnostics, and companion diagnostics. Accuracy, sensitivity
and reproducibility are required for any diagnostic procedure that is to be used for
predictive drug testing. It is desirable that companies developing genetic test meth-
ods should be certifi ed for their capabilities for detecting a genotype variant or a
SNP from any given patient sample. Only after confi rmation of the identity of the
polymorphism, should the company be allowed to proceed to the next step of analy-
sis, which involves proteomics or analysis of protein expression of the genotype
variant. Pharmacogenomic testing may be used in clinical trials of a drug, in reeval-
uation of a failed drug candidate or for evaluation of patient responsiveness to a
marketed drug. The quality of such testing is not yet adequately covered by the
regulatory agencies. Regulatory agencies will need to apply new approaches
towards the review and approval of molecular diagnostic tests that use new tech-
nologies as well as drugs that work in concert with companion diagnostics, often
using complex multianalyte test formats. The information revealed by pharmacoge-
nomic testing during drug development and that based on study of marketed drugs
might reveal potential hazards that need to be included in the labeling, which cur-
rently includes only known hazards. Labeling should disclose not only risk infor-
mation on the extrapolation of in vitro pharmacogenomic testing and in vivo drug
responsiveness but also the recommended dose based on stratifi ed patient groups
according to genotype/phenotype profi les.