Clinical Research: Phase 1 - Phase 4

Fit-For-Purpose: A Strategic Approach to Biomarker Method Validation for Rare Disease

Ensuring the integrity of your surrogate endpoints

Rare disease studies present unique obstacles to drug developers, including small patient populations, slow disease progression, lack of prior clinical research, and uncertainty around validating biomarker measurements intended for use as surrogate endpoints. Our white paper describes a fit-for-purpose approach to validating bioanalytical methods in orphan drug trials. Among the topics we explore:

  • Establishing normal ranges for biomarkers that take into account variations resulting from gender, age, and many other factors
  • Three things key to defining criteria for assay acceptance, a prerequisite for biomarker method development and validation
  • Creating standard procedures for collecting, handling, and storing assays — essential for maintaining sample and/or biomarker integrity.