A site where you can find recommended contractors for non-clinical testing｜effical " Summary of basic information to keep in mind before requesting a non-clinical study " Collection of Official Guidelines for Drug Development

Collection of Official Guidelines for Drug Development

A Bridge from Preclinical to Clinical Development: Insights from Official FDA, EMA, and PMDA Guidelines and Approval Cases

In pharmaceutical development, it is crucial to connect data obtained in non-clinical studies to clinical trials and ultimately to approval. The FDA, EMA, and PMDA, through ICH guidelines and their own directives, respectively, provide concepts regarding non-clinical safety, First-in-Human (FIH), Good Clinical Practice (GCP), trial design, and dose optimization. This page introduces official guidelines that are highly valuable for practical reference, as well as case studies of approvals that are useful for deepening understanding.

Basic Regulations and General Guidelines

ICH M3(R2) - Nonclinical Safety Studies

A core document outlining when and to what extent non-clinical safety studies should be conducted and how data can be bridged to clinical trials and regulatory submissions.

Official PDF: https://database.ich.org/sites/default/files/M3_R2__Guideline.pdf

ICH E6(R3) – Good Clinical Practice (GCP)

The latest GCP guidelines adopted on January 6, 2025, are international standards that integrate quality assurance for clinical trials, subject protection, and risk-based management.

Official PDF: https://database.ich.org/sites/default/files/ICH_E6(R3)_Step4_FinalGuideline_2025_0106.pdf

EMA "FIH/Guideline on Risk-Reducing Measures in First-in-Human Clinical Trials"

An important EMA document that shows how to integrate non-clinical data to design the first dose and dose escalation, effective February 1, 2018.

Official PDF: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-strategies-identify-and-mitigate-risks-first-human-and-early-clinical-trials-investigational-medicinal-products-revision-1_en.pdf

FDA "Cancer Clinical Trial Eligibility Criteria: Organ Function and Prior or Concurrent Malignant Tumors" Final Guidance (July 2020)

Guidance outlining how to handle criteria such as kidney, liver, and heart function, as well as previous or concurrent malignancies, to avoid unnecessarily narrowing patient selection for cancer trials.

Official PDF: https://www.fda.gov/media/123745/download

FDA "Eligibility Criteria for Cancer Clinical Trials: Laboratory Values" Draft Guidance (April 2024)

Guidance on the scientific basis of eligibility criteria using examination values and promoting re-evaluation. This is a draft stage, and comments are being sought.

Official PDF: https://www.fda.gov/media/178013/download

FDA "Project Optimus"

FDA's Tumor Dose Optimization Initiative. This initiative focuses on the appropriate dose exploration, aiming to determine the optimal dose before registration trials by utilizing preclinical and clinical data.

Official page: https://www.fda.gov/about-fda/oncology-center-excellence/project-optimus

EMA Non-clinical Development Guidelines List

Entry page to non-clinical development related guidelines at EMA. Useful as a guide for referring to individual non-clinical evaluation guidelines and related documents.

Official page: https://www.ema.europa.eu/en/human-regulatory-overview/research-development/scientific-guidelines/non-clinical-guidelines/non-clinical-development-guidelines

PMDA ICH Related Pages

The PMDA's ICH-related pages are useful as a reference for checking ICH documents in Japanese and can be used as a guide for confirming clinical-related guidelines, including the E series.

Official page: https://www.pmda.go.jp/int-activities/int-harmony/ich/0011.html

Approval examples for reference

Rezdiffra (resmetirom) – FDA (March 14, 2024)

The first drug approved for adults with non-cirrhotic NASH and moderate to advanced fibrosis.

FDA Press Release: https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease

Ofev (nintedanib) – EMA

Anti-fibrotic medication approved in Europe for idiopathic pulmonary fibrosis (IPF), systemic sclerosis-associated interstitial lung disease (SSc-ILD), and other progressive fibrotic ILDs.

EMA EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/ofev

Tecentriq (atezolizumab) + Avastin (bevacizumab) combination – FDA (HCC, May 29, 2020)

Approved by the FDA as a combination therapy for unresectable or metastatic hepatocellular carcinoma. Extended survival in the IMbrave150 trial.

FDA Approval Information: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-atezolizumab-plus-bevacizumab-unresectable-hepatocellular-carcinoma

3 Recommended Contract Research Organizations for Non-Clinical Studies
— by Target goal and Expertise

In drug discovery, the quality and efficiency of non-clinical studies have a direct impact on clinical success rates, development costs, and overall length of time required in R&D. In recent years, there has been more demand for clinically relevant data, globally accepted reliability, and accurate early-stage screening. Thus, it is more important than ever to select the right CRO (Contract Research Organization) for strategic approach.
In this article, we highlight three CROs with proven technical capabilities, expertise, and long standing track records. These are our TOP 3 choices based on their capabilities and the specific target goals of the researchers for their non-clinical studies.

Pharmacology (Efficacy) Studies
Replicate unknown pathological models and
Discovery to clinically oriented drug evaluation

SMC Laboratories, Inc.

Reference: SMC Laboratories official website (https://www.smccro-lab.com/jp/)

SMC Laboratories has established a disease models using patented mouse technologies. The company has established proprietary pathological models—particularly in liver disease and fibrosis—and continues to expand their approach across a wide range of models in cancer, inflammation, and metabolic diseases.
From exploratory research to clinically oriented efficacy evaluation, SMC offers customized study designs, dosing strategies, and evaluation analysis tailored to each project. Their collaborative approachallows researchers to discuss and refine study plans together with SMC’s expert scientists.
With flexible small-scale study options and strong technical support, SMC Laboratories is an ideal partner for start-ups, biotech ventures, and academic institutions alike.

Consult study design and disease model development
with SMC Laboratories

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Safety Studies
Comprehensive Safety Evaluation for FIH Applications

labcorp
(Labcorp Drug Development)

Reference: labcorp official website (https://jp.labcorp.com/)

labcorp provides a fully integrated GLP testing system aligned with international regulatory standards, including FDA, EMA, and PMDA requirements. All studies are conducted under ICH-compliant quality assurance, making it ready for data submission.
The company has extensive expertise in long-term toxicity studies such as Segment I–III reproductive and carcinogenicity studies, as well as 2-year chronic toxicity assessments.
labcorp’s comprehensive approach enables sponsors to efficiently outsource the entire preclinical package from toxicology, toxicokinetic (TK), and safety pharmacology study design to execution. This accelerates a path to First-in-Human (FIH) trials. For most of the global drug developers, this all-in-one service structure minimizes cost, risk, and expedite the time before advancing to clinical phase.

Consult labcorp
Discuss GLP/ICH-compliant study design for your FIH submission.

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Pharmacokinetic (PK/PD) Studies
High-Precision Bioanalysis for Clinically Predictive PK/PD Evaluation

PhoenixBio

Source: PhoenixBio Official Website (https://phoenixbio.co.jp/)

PhoenixBio offers pharmacokinetic and hepatic metabolism studies using their proprietary PXB-mouse®, a humanized-liver chimeric mouse model. This platform enables the acquisition of data with high clinical correlation in ADME, drug–to-drug interaction studies, bridging the gap between preclinical and clinical stages.
With advanced LC-MS/MS-based bioanalysis, PhoenixBio provides aseamless workflow from plasma concentration measurement and metabolite identification to quantitative validation.
The company offersan integrated evaluation analysiscovering pharmacokinetics, hepatotoxicity, and safety with flexibility to accommodate complex modalities such as oligonucleotide and middle-molecule therapeutics. For compounds where hepatic metabolism is a development bottleneck—or where quantitative, reproducible exposure data are critical—PhoenixBio delivers unmatched analytical precision and consistency.

Consult Phoenix Bio for PK/PD analysis with a clinical focus

Phoenix Bio.
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