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Safety Guidelines

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S10

Last Updated: March 3, 2025

PHOTOSAFETY EVALUATION OF PHARMACEUTICALS 1.      INTRODUCTION 1.1.   Objectives of the Guideline The purpose of this document is to recommend international standards for photosafety assessment, and to harmonise such assessments supporting human clinical trials and marketing authorizations for pharmaceuticals.   It includes factors for initiation of and triggers for additional photosafety assessment and should be read in conjunction with ICH M3(R2), Section 14 on Photosafety Testing (Ref. 1).   This guideline should reduce the   likelihood   that    substantial   differences    in   recommendations    for   photosafety assessment will exist among regions. This guideline is divided into several sections.    Section 2 discusses factors to consider in any evaluation of photosafety.    Section 3 describes existing...

S11

Last Updated: March 3, 2025

1.         INTRODUCTION 1.1        Objectives of the Guideline The purpose  of this  document  is to recommend  international  standards  for,  and  promote harmonisation  of,  the  nonclinical   safety  assessments  to   support   the  development   of pharmaceuticals intended for paediatric use. Harmonisation of the guidance for nonclinical safety  studies  will   define  the  current  recommendations   and  reduce  the  likelihood  that substantial differences will exist among regions. It should facilitate the timely conduct of paediatric  clinical  trials  and  reduce  the  use  of  animals   in   accordance  with  the   3R (replace/reduce/refine) principles....

S12

Last Updated: March 3, 2025

1.   INTRODUCTION 1.1. Objectives of the ICH S12 Guideline The objective of this guideline is to provide harmonised recommendations for the conduct of nonclinical biodistribution (BD) studies in the development of gene therapy (GT) products. This  document  provides  recommendations   for  the   overall   design  of  nonclinical   BD assessments. Considerations for interpretation and application of the BD data to support a nonclinical development programme and the design of clinical trials are also provided. The recommendations in this guideline endeavour to facilitate the development of GT products while avoiding unnecessary use of animals, in accordance with the 3Rs (reduce/refine/replace) principles. 1.2. Background An understanding of the BD profile of...

S9

Last Updated: March 3, 2025

NONCLINICAL EVALUATION FOR ANTICANCER PHARMACEUTICALS 1.       INTRODUCTION 1.1     Objectives of the Guideline The purpose of this guidance is to provide information to assist in the design of an appropriate   program   of  nonclinical   studies   for   the   development   of  anticancer pharmaceuticals. The guidance provides recommendations for nonclinical evaluations to  support  the  development  of anticancer  pharmaceuticals  in  clinical trials  for the treatment of patients with advanced disease and limited therapeutic options. This  guideline  aims  to  facilitate  and   accelerate  the   development  of  anticancer pharmaceuticals  and...

S8

Last Updated: March 3, 2025

IMMUNOTOXICITY STUDIES FOR HUMAN PHARMACEUTICALS 1.         INTRODUCTION 1.1      Objectives of the Guideline The  objectives  of this  guideline  are  to  provide  (1)  recommendations  on  nonclinical testing   approaches   to   identify   compounds   which   have   the    potential   to   be immunotoxic, and (2) guidance on a weight-of-evidence decision making approach for immunotoxicity testing. Immunotoxicity is, for the purpose of this guideline, defined as  unintended  immunosuppression  or  enhancement.  Drug-induced  hypersensitivity and autoimmunity are excluded. 1.2      Background Evaluation  of potential  adverse  effects  of human  pharmaceuticals  on  the  immune system  should  be  incorporated  into  standard  drug  development.  Toxicity  to  the immune system encompasses a variety of adverse effects. These include suppression or enhancement of...

S7B

Last Updated: March 3, 2025

THE NON–CLINICAL EVALUATION OF THE POTENTIAL FOR DELAYED VENTRICULAR REPOLARIZATION (QT INTERVAL PROLONGATION) BY HUMAN PHARMACEUTICALS 1.        INTRODUCTION The assessment of the effects of pharmaceuticals on ventricular repolarization and proarrhythmic risk is the subject of active investigation.  When additional data (non- clinical and clinical) are accumulated in the future, they will be evaluated and this guideline might be revised. 1.1      Objective of the Guideline This guideline describes a non-clinical testing strategy for assessing the potential of a test   substance   to    delay   ventricular    repolarization.      This    guideline   includes information concerning non-clinical assays and...

S7A

Last Updated: March 3, 2025

SAFETY PHARMACOLOGY STUDIES FOR HUMAN PHARMACEUTICALS 1.        INTRODUCTION 1.1      Objectives of the Guideline This  guideline  was   developed  to  help  protect  clinical  trial  participants   and  patients receiving  marketed  products  from  potential  adverse  effects  of  pharmaceuticals,  while avoiding unnecessary use of animals and other resources. This  guideline  provides  a  definition,  general principles  and recommendations for  safety pharmacology studies. 1.2      Background...

S6(R1)

Last Updated: March 3, 2025

PART I: PRECLINICAL SAFETY EVALUATION OF BIOTECHNOLOGY-DERIVED PHARMACEUTICALS ICH Harmonised Tripartite Guideline Having reached Step 4 of the ICH Process at the ICH Steering Committee meeting on 16 July 1997, this Guideline is recommended for adoption to the three regulatory parties to ICH 1.      INTRODUCTION 1.1     Background Biotechnology-derived pharmaceuticals (biopharmaceuticals) were initially developed in  the  early  1980s.  The  first  marketing  authorisations  were  granted  later  in  the decade.  Several...

S5(R3)

Last Updated: March 3, 2025

DETECTION OF REPRODUCTIVE AND DEVELOPMENTAL TOXICITY FOR HUMAN PHARMACEUTICALS S5(R3) ICH Consensus Guideline TABLE OF CONTENTS TABLE OF CONTENTS LIST OF ABBREVIATIONS  6 1.     INTRODUCTION & GENERAL PRINCIPLES   7 1.1.     AIM OF STUDIES ...

S4

Last Updated: March 3, 2025

DURATION OF CHRONIC TOXICITY TESTING IN ANIMALS (RODENT AND NON RODENT TOXICITY TESTING) ICH Harmonised Tripartite Guideline Having reached Step 4 of the ICH Process at the ICH Steering Committee meeting on 2 September 1998, this guideline is recommended for adoption to the three...

S3B

Last Updated: March 3, 2025

PHARMACOKINETICS: GUIDANCE FOR REPEATED DOSE TISSUE DISTRIBUTION STUDIES ICH Harmonised Tripartite Guideline Having reached Step 4 of the ICH Process at the ICH Steering Committee meeting on 27 October 1994, this guideline is recommended for adoption to the three regulatory parties to ICH Introduction A   comprehensive   knowledge   of   the    absorption,...

S3A

Last Updated: March 3, 2025

NOTE FOR GUIDANCE ON TOXICOKINETICS: THE ASSESSMENT OF SYSTEMIC EXPOSURE IN TOXICITY STUDIES 1.    Introduction This   Note   for   Guidance   concerns   toxicokinetics   only   with   respect   to   the development of pharmaceutical products intended for use in human subjects. In this  context, toxicokinetics is  defined  as the  generation  of pharmacokinetic data,  either  as  an  integral  component  in  the  conduct  of  non-clinical  toxicity studies or in specially designed supportive studies, in order to assess systemic exposure.  These data may be...

S2(R1)

Last Updated: March 3, 2025

GUIDANCE ON GENOTOXICITY TESTING AND DATA INTERPRETATION FOR PHARMACEUTICALS INTENDED FOR HUMAN USE 1.        INTRODUCTION 1.1      Objectives of the Guideline This guidance replaces and combines the ICH S2A and S2B Guidelines.    The purpose of the  revision  is  to  optimize  the  standard  genetic  toxicology  battery  for  prediction  of potential human risks, and to provide guidance on interpretation of results, with the ultimate goal of improving risk characterization for carcinogenic effects that have their basis...

S1C(R2)

Last Updated: March 3, 2025

DOSE SELECTION FOR CARCINOGENICITY STUDIES OF PHARMACEUTICALS 1.        INTRODUCTION Traditionally,  carcinogenicity  studies  for  chemical  agents  have  relied  upon  the maximally tolerated dose (MTD) as the standard method for high dose selection (Note 1). The MTD is generally chosen based on data derived from toxicity studies of 3 months’ duration. In the past, the criteria for high dose selection for carcinogenicity studies of human pharmaceuticals have not been uniform among international regulatory agencies. In Europe  and  Japan,  dose  selection  based  on  toxicity  endpoints  or  attaining  high multiples of the maximum recommended human daily dose (>100x on a mg/kg basis) has been accepted. However, in the United States, dose selection based on the MTD has traditionally been considered the only appropriate practice. All regions have used a maximum feasible dose as an appropriate endpoint. For  pharmaceuticals  with  low  rodent...

S1B(R1)

Last Updated: March 3, 2025

1.   OBJECTIVE This document provides guidance on approaches for evaluating the carcinogenic potential of pharmaceuticals. 2.   BACKGROUND Historically, the regulatory requirements for the assessment of the carcinogenic potential of pharmaceuticals in the three regions (E.U., Japan, U.S.) provided for the conduct of long-term carcinogenicity studies in two rodent species, usually the rat and the mouse.  Given the cost of these studies and their extensive use of animals, it is in keeping with the mission of ICH to examine whether this practice requiring long...

S1A

Last Updated: March 3, 2025

GUIDELINE ON THE NEED FOR CARCINOGENICITY STUDIES OF PHARMACEUTICALS 1. INTRODUCTION The objectives of carcinogenicity studies are to identify a tumorigenic potential in animals and to assess the relevant risk...