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Osteoarthritis: Structural Endpoints for the Development of Drugs

DraftCenter for Biologics Evaluation and Research Center for Devices and Radiological Health Center for Drug Evaluation and Research08/23/2018

Description

The purpose of his guidance is to assist sponsors who are developing drugs, devices, or 20 biological products (medical products) to treat the underlying pathophysiology and structural progression of osteoarthritis (OA).2 21

Key Topics

Terms and concepts identified from this document

Scope & Applicability

Product Classes

3
Biological Products

Requires analytical comparability per ICH Q5E

Devices

Clinical investigations of medical devices

Drugs

Clinical investigations of drugs, including human drugs and biological products

Stakeholders

2
Stakeholders

Entities involved in the development of bispecific antibodies

Sponsors

Assist sponsors in the nonclinical evaluation

Regulatory Context

Regulatory Activities

2
Traditional Approval

Pathway based on clinical endpoints or validated surrogate endpoints

Accelerated Approval

Pathway for drugs and biologics for serious conditions; Regulatory pathway supported by surrogate or intermediate endpoints; Regulatory pathway based on surrogate or intermediate endpoints; Pathway for products based on surrogate or intermediate clinical endpoints

Attributes

3
Structural Progression

Common measures used to predict treatment effects

Structural Endpoints

Focus of the guidance for drug development

Clinically Meaningful Benefit

Evidence required to include software information in the PI

Technical Details

Processes

2
Statistical Analysis

Conducted to review submissions and verify quality of evidence; Programs used to perform the statistical analysis.

Clinical Trial Design

General issue not specifically addressed in this guidance but referenced to ICH.

Clinical Concepts

4
Patient Function

Direct measures of how patients function and feel

Joint Replacement

A clinical outcome of interest that structural endpoints aim to delay or avoid.

Patient-Reported Outcome Measures

Measures used to assess pain and function in OA approvals to date.

Osteoarthritis

manifestations of obesity beyond excess adiposity

Standards & References

Specifications

1
Structural Endpoints

The primary focus of the guidance for assessing OA disease progression.

ICH References (2)

ICH E10

Choice of Control Group in Clinical Trials.

ICH E9

Statistical Principles for Clinical Trials; Discourages deterministic procedures due to high risk of bias; Notes that the use of Bayesian methods in clinical trials may be considered.

Related MFDS Guidelines

Korean regulatory guidelines covering similar topics

See Also (7)

Osteoarthritis: Structural Endpoints for the Development of Drugs | Guideline Explorer | BioRegHub