Non-Clinical Contribution to Clinical Trials during Lead Optimization Phase.

Lázara Martínez Muñoz

doi:10.3390/bs8010017

Abstract

This manuscript comments on guidelines related to requirements for clinical trials for new drugs and the importance of considering regulatory criteria in the planning phase, in order to enhance the utility of data generated in basic research. Suggestions are made for optimizing regulatory management to improve the likelihood of acceptance of pre-clinical data prior to Clinical Phase I trials (early clinical trials).

Highlights

The growing incidence of neurodegenerative diseases makes it imperative for scientists conducting research in this field to design projects in ways that minimize delay for patients in need of access to new technologies or medicines
There is concern about the limited understanding many investigators have of the importance of correctly integrating regulatory approaches and criteria into their preliminary work; such understanding allows planning for an effective research program in new drug development that improves the chances of positive regulatory review in order to support progress to clinical trials
The most-desired result may be the publication of data; that data may be relevant for supporting early clinical trials if the information is produced following appropriate criteria for quality assurance and meets regulatory requirements

Summary

Introduction

The growing incidence of neurodegenerative diseases makes it imperative for scientists conducting research in this field to design projects in ways that minimize delay for patients in need of access to new technologies or medicines. This guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products (R1) of 2017 [8] with few differences makes similar suggestions for safety studies for clinical trials prior to Clinical Phase I. Highlighted in these regulations are an extended set of parameters to be evaluated, as well as the consideration that an exaggerated pharmacological effect may be a cause of toxic effect, which should be included as a measure of the margin of safety. Endpoints should include—at a minimum—body weights, clinical signs, clinical chemistries, hematology, and histopathology

Selection of Doses for Exploratory Clinical Trials

Challenges Involved in Translating Preclinical Data to Clinical Set-Up

Concluding Remarks