Key Considerations for Supporting IITs in EU/US

• IITs are increasingly used by early-stage companies to generate early / proof-of-concept data in a faster and more cost-effective way.
• IIT data can support fundraising but carry risks: poor data quality, inconsistent safety reporting, and unexpected findings.
• Also depends if the IIT is your primary source of data or if its an add on to a more advanced and larger program.

• Full IIT: Investigator holds IND in US/clinical trial application in EU, sponsor provides drug/funding. Least sponsor control, highest risk.
  • EU clinical trial application seems always owned by Investigator for IIT
  • US IIT can be its own IND or under Company IND (the latter seems preferred from a (safety) reporting perspective.
    • Note NIH does not seem to be very flexible with IIT. NIH contracting (CRADA) is mentioned to be very challenging.
• Hybrid IIT: Sponsor retains IND (US) or structured support (EU), while investigator leads scientifically. Sponsor involvement for protocol development, database build, safety reporting, and data outputs.
• Timing is critical: IITs should align with development plans and financing events, not be initiated too early.

• Data quality is the biggest challenge; its recommended sponsors be involved in
  • database built (e.g., REDCap can be compliant),
  • monthly data reviews/check-ins,
  • funding for resources e.g. staff for data entry
• All safety events must flow into Company’s safety database and DSUR. Pharmacovigilance outsourcing is feasible and relatively low cost.
• GDPR and data transfer agreements must be planned early.

Prioritize solid documentation and engagement through

• Contract/ clinical trial agreement (CTA): Define roles and responsibilities, involvement in study design, data collection, access to and review of data, safety reporting, publication rights, IP protection, confidentiality, drug storage and accountability, monitoring, biomarker testing, role during audit / inspection etc.
  • It was suggested to introduce milestone payments if Company is considering an independent grant payment.
• Charter: Internal IIT steering committee proposed to consolidate input across ClinOps, Quality, Safety, Regulatory, Biostats, and Clinical Supplies.
• SOPs: Governance process for IIT evaluation, approval, oversight, safety reporting, inspection readiness.
• Data Transfer Agreements: Required in EU and some US institutions.
• Real time check ins: A proactive collaboration and real time check ins are flagged to be very important.

• Strong site relationships are essential: monthly calls, on-site visits, trackers for performance.
• Providing sites with manuals, templates, and CRFs reduces deviations.
• Milestone-based payments can encourage timely enrollment and data entry.

• Risks include poor data quality and missed safety signals.
• Recommended: proactive sponsor visits, contingency planning for inspections.
• Predetermined milestones and oversight measures help manage liabilities.

• IITs are increasingly used for proof-of-concept in tight funding environments.
• Trade-offs discussed: IITs as side projects vs. central to development strategy.

• ClinOps should have a big role in IITs and this might impact workload depending on the project and collaboration with Investigator.
• Proposal to form an internal IIT steering committee for cross-functional review and oversight.
• Ensures decisions align with company development strategy and regulatory expectations.

• Publication rights must be carefully managed in agreements to avoid premature disclosure of material information.
• Clear delineation of sponsor vs. investigator responsibilities is critical to avoid regulatory or reputational issues.
• Hybrid IITs can balance independence with sponsor oversight but require strong governance and documentation.
• Label should include “for investigational use only” or language as required per local regulation.
• Navigate information related to Investigational Product and/or intellectual property as documented in the agreement