Camilla Graham
Seminars
Fireside Chat: Designing the ‘Right’ PPF Trial – A Clinician & Developer Dialogue
- Identifying likely progressors by integrating clinical features, pulmonary function trends, imaging patterns, and recognizing the limitations of current PPF definitions
- Examining the current operational and scientific tension by exploring conflicts between ideal trial protocols and real-world patient heterogeneity, including practical recruitment, adherence, and site capability
- Balancing trial design with real-world practice, exploring conflicts, compromises, and the tension between idealized protocols and patient heterogeneity, including the artifact of a 52 week trial duration
- Collaborative perspective integrating clinician judgement, standardized imaging, and enrichment strategies to improve trial success as well as ability to generalize trial data to real-world patients
Designing the ‘Right’ PPF Trial: Clinical Reality vs Drug Development Needs
Drug development in interstitial lung diseases has traditionally focused on individual conditions such as IPF or connective tissue disease–associated ILD. However, increasing evidence suggests that many ILDs converge on shared fibrotic pathways. As PPF has emerged as a cross-disease construct to enable trial design, this raises a critical question: are we building therapies for diseases, or for fibrosis itself?
Key Discussion Points:
- What is best disease vs phenotype-driven development? Should therapies be developed for individual ILDs, or for fibrotic progression across diseases through constructs like PPF?
- How indication strategy influence trial design from patient stratification, endpoint selection, and the likelihood of detecting meaningful treatment effects
- Navigating the regulatory and commercial realities of broader indications vs disease-specific labels for approval, positioning, and reimbursement
- As shared fibrotic biology becomes clearer, should drug development move toward mechanism-based approaches rather than traditional disease categories?
Forced Vital Capacity remains the anchor endpoint for pulmonary fibrosis trials and the filed remains split on torn on its value. What’s more the field is evolving to capture a more complete picture of disease progression and therapeutic impact. This panel will dig into FVC and secondary endpoints, the emerging role of quantitative HRCT and other imaging biomarkers, the evidence required for regulatory acceptance, standardization across providers, and the integration of multi-modal endpoints into trial design, all to ensure you leave with a clear understanding of how to design trials that balance traditional FVC measurements with innovative endpoints, enabling robust efficacy assessment, regulatory alignment, and differentiation in an increasingly complex ILD landscape.
- The continued relevance of FVC as a surrogate for disease progression and mortality in IPF and PPF
- How secondary endpoints can complement FVC to reveal meaningful changes in symptoms, quality of life, and disease biology
- Advances in quantitative imaging, including AI-driven HRCT, and the pathway toward regulatory qualification
- Strategies for integrating multi-modal endpoints into trial design without overcomplicating study execution
- Practical considerations for trial sponsors: standardization, subgroup analysis, and managing background therapy
