Toby Maher
Director of Interstitial Lung Disease Keck School of Medicine of the University of Southern California
Toby Maher, MBBS, MSc, PhD, is Professor of Clinical Medicine and Director of Interstitial Lung Disease at the Keck School of Medicine of USC. He has over 20 years of experience specializing in pulmonary fibrosis and orphan interstitial lung diseases, combining frontline clinical expertise with translational research leadership. Previously, he led the ILD unit at the Royal Brompton Hospital and headed the Fibrosis Research Group at Imperial College London, where he remains Professor of Interstitial Lung Disease. His research focuses on biomarker discovery, early-phase clinical trials, and the preclinical validation of novel therapeutic targets in fibrosing lung disease. Dr Maher trained in Respiratory Medicine at Royal Brompton Hospital, holds an MSc from Imperial College London, and completed a Wellcome Trust-funded PhD at University College London.
Seminars
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?
As pulmonary fibrosis trials continue to expand globally, sponsors must navigate an increasingly complex operational landscape. Differences in regulatory expectations, site capabilities, patient access, and trial infrastructure across regions can significantly influence study timelines, enrolment success, and data quality.
Key Discussion Points Include:
- Navigating regional regulatory and ethics requirements, including how differing approval timelines, IRB/ethics committee expectations, and evolving regulations in emerging markets can impact global trial execution
- Strategies for identifying and activating high-performing sites, engaging investigators with varying levels of ILD expertise, and preventing under-enrolment or inactive sites across geographically diverse trial networks
- Managing the operational complexities of multinational trials, including drug distribution, cold chain logistics, imaging standardization, biomarker collection, and cross-border data integrity while maintaining compliance with GCP and regional privacy regulations
- Lessons from recent global IPF studies on designing scalable protocols, anticipating operational bottlenecks, and integrating hybrid or decentralized approaches to maintain efficiency without compromising data quality
The past year has been transformative for IPF, with new therapies, emerging mechanisms, and evolving patient management strategies reshaping the landscape. This panel will provide a strategic, forward-looking overview of how the field has progressed and what the next 12-18 months may hold. Panellists will discuss the expanding pathophysiology of IPF, emerging classifications of therapies, and implications for patient care, clinical development strategy, and communication with stakeholders.
Key Discussion Points:
- How newly approved therapies and emerging candidates are expected to influence IPF treatment and clinical and research priorities in the next 12–18 months.
- Exploring the practical impact of emerging terminology, anti-fibrotic, anti-inflammatory, de-differentiation, fibrolysis, and how these distinctions may shape patient and clinician preferences
- Approaches for ordering, timing, and combining therapies in a multi-drug era, balancing efficacy, safety, and operational feasibility.
- Identifying opportunities, challenges, and areas for collaboration to ensure trials and programs remain aligned with evolving clinical practice
- Breaking down recent Phase II/III trial outcomes and approvals through a clinician’s lens, examining how efficacy signals, safety profiles, and real-world tolerability translate into meaningful benefit for patients in routine practice
- Understanding what these results reveal about mechanisms that are (and aren’t) working, and how emerging data are reshaping expectations around endpoints, disease modification, and what constitutes clinically meaningful improvement in IPF
- Looking ahead to the next generation of trials, exploring how recent successes and failures should inform study design, patient selection, combination strategies, and the evolving role of standard-of-care in future development programs
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
