FDA Guidances: COPD, Histopathology for Biomarkers
FDA BRIEF: Week on May 23, 2016
INDICATION: Whole spectrum or defined entities
TYPES OF DRUGS:
- Improving airflow obstruction .
- Providing symptom relief
- Modifying or preventing exacerbations
- Altering disease progression
- Modifying lung structure
DOSE SELECTION: To optimize benefit/risk
EFFICACY ASSESSMENT:
- Objective: Pulmonary function tests, Exercise capacity,
- Patient Reported Outcomes: Symptom scores, Activity scales, Health-related quality-of-life instruments
- Biomarkers, Surrogate Endpoints:: lung function test parameters, such as FEV1,
PRIMARY EFFICACY ENDPOINT:
- Improving airflow obstruction
- Providing symptom relief
- Modifying or preventing exacerbations
- Altering disease progression.
- Modifying lung structure.
DURATION: 3 mo. – several years, dependent on endpoint
DRUGS ADMINISTERED VIA INHALED ROUTE: Assessment of delivery systems on safety/efficacy
COMBINATION DRUGS: Compare product to each of its constituents to demonstrate combination product superior to each constituent
SCOPE: Biomarker qualification using histopathology as reference standard
BIOMARKER PERFORMANCE:
- Performance characteristics
- Sensitivity and Temporal Correlation
- Specificity
- Reversibility/Resolution
- Alternative Testing Models.
HISTOPATHOLOGIC METHODS:
- General Planning
- Slide examination
- Timing of Sample Collection
- Controls
- Fixation
- Number of Sections and Sampling Location (at Necropsy)
- Staining
- Special methodology
OTHER CONSIDERATIONS: Digital Pathology and Slide Sharing, Lexicons, Filtering
OTHER FACTORS: Diagnostic Drift, Chronological Bias