Taking AIM at serious illness: implementing an access to investigational medicines expanded access program

Area of Science:

• Medical Science
• Regulatory Affairs
• Patient Access to Treatment

Background:

• Critically ill patients may exhaust conventional treatments.
• The Food and Drug Administration's (FDA) Expanded Access (EA) pathway allows use of investigational drugs when clinical trials are not feasible.
• EA involves complex logistical and regulatory hurdles, including pharmaceutical company communication and institutional requirements.

Purpose of the Study:

• To describe the development and implementation of the Access to Investigational Medicines (AIM) Platform at Vanderbilt University Medical Center (VUMC).
• To streamline the process for physicians and patients seeking investigational agents through the EA pathway.
• To ensure efficiency and regulatory compliance in managing EA cases.

Main Methods:

• Established the AIM Platform infrastructure at VUMC.
• Integrated needs and responsibilities of institutional stakeholders (e.g., contracting, IRB, pharmacy, billing).
• Facilitated EA cases for physicians and patients.

Main Results:

• The AIM Platform successfully supported 40 EA cases for drugs and biologics.
• Supported both single-patient and intermediate-size EA protocols.
• Managed emergent and non-emergent EA cases.

Conclusions:

• Implementing dedicated infrastructure, like the AIM Platform, is crucial for facilitating Expanded Access.
• Prioritizing EA support at academic medical centers enhances regulatory knowledge and patient care.
• Streamlined processes improve efficiency and compliance for investigational medicine access.