The Alpha-1 Foundation (A1F) Therapeutic Development Network (TDN) is a Foundation-owned, disease-specific research network designed to support efficient, ethical, and patient-centered Alpha-1 drug development.
This page is intended for pharmaceutical and biotechnology companies, as well as clinical research organizations (CROs), seeking to understand how the TDN can support Alpha-1 programs across the development lifecycle — from early feasibility through post-marketing research.
What the TDN Is
The TDN is a purpose-built clinical research infrastructure governed by the Alpha-1 Foundation.
- Engaged patient community
- Access to experts in Alpha-1 Antitrypsin basic and clinical science
- Clinical trial design advisory and operational feasibility review
- Routinely updated registry of patients with data focused on study eligibility and willingness criteria
- Participating sites with clinical trial capabilities
- Brings disease-specific expertise upstream into development planning
- Connects experienced Alpha-1 clinical sites, investigators, and patients
- Supports feasibility, site readiness, and participant access
- Operates under Foundation governance with strong ethical and data oversight
The TDN exists to reduce avoidable risk and inefficiency in Alpha-1 programs—while keeping patients and sites at the center of research.
To avoid duplication and confusion, it is important to clarify what the TDN does not do.
The TDN does not:
- Act as a CRO or replace CRO execution
- Replace clinical trial sites or institutional relationships
- Provide clinical care or medical advice
Instead, the TDN complements sponsors, CROs, and sites by improving inputs and alignment before and during trial execution.
Early Development & Feasibility
- Inform inclusion and exclusion criteria using real-world Alpha-1 data
- Assess feasibility and participant availability
- Provide Alpha-1-specific insights prior to protocol finalization
Site Identification & Readiness
- Identify experienced, research-ready, protocol-specific Alpha-1 centers
- Support site preparedness and alignment
- Reduce late-stage site performance variability
Recruitment & Execution Support
- Participant pre-screening and referral coordination
- Centralized operational support where appropriate
- Virtual site capabilities to expand access and equity
Observational & Post-Trial Research
- Natural history and disease progression studies
- Longitudinal follow-up and real-world evidence (RWE) generation
- Phase IV post-marketing observational studies
- Post-approval safety and effectiveness monitoring
- Label expansion and outcomes research support
The TDN maintains a highly curated, HIPAA-compliant dataset of individuals with Alpha-1 who have expressed interest in research participation.
Key features include:
- Structured clinical and demographic data relevant to Alpha-1 trials
- Regular data refresh to maintain relevance
- Alignment with common and anticipated eligibility criteria
- Secure, consented access governed by Foundation policies
Advanced analytics are used to support feasibility assessment, scenario modeling, and network planning—always with human oversight and ethical governance.
The TDN infrastructure also supports post-marketing safety surveillance, real-world effectiveness analyses, and longitudinal outcome tracking in approved Alpha-1 therapies.
The TDN is owned and governed by the Alpha-1 Foundation, with medical, operational, and ethical oversight.
Key governance principles include:
- Clear separation between the Alpha-1 Research Registry (patient-initiated) and the TDN (clinician-initiated)
- Standardized consent and privacy protections
- HIPAA-compliant systems and workflows
- A1F stewardship to ensure patient trust and long-term sustainability
This structure enables research agility while maintaining the trust of the Alpha-1 community.
By engaging with the TDN, industry partners may benefit from:
- Earlier, data-informed feasibility assessments
- Better-aligned inclusion and exclusion criteria
- More predictable site performance
- Improved confidence in enrollment assumptions
- Reduced late-stage protocol amendments
- Scalable infrastructure for post-marketing safety surveillance, label expansion studies, and long-term real-world evidence generation
The TDN integrates with existing CRO models and institutional relationships without disruption.
Industry partners at any stage in the drug development process who are interested in exploring collaboration with the Alpha-1 Foundation Therapeutic Development Network are encouraged to contact the TDN team at TDN@alpha1.org
The Therapeutic Development Network reflects the Alpha-1 Foundation’s commitment to advancing Alpha-1 therapies through ethical, patient-centered, and sustainable research partnerships.
