PMID- 37877021
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20240210
IS  - 2296-858X (Print)
IS  - 2296-858X (Electronic)
IS  - 2296-858X (Linking)
VI  - 10
DP  - 2023
TI  - Taking AIM at serious illness: implementing an access to investigational 
      medicines expanded access program.
PG  - 1287449
LID - 10.3389/fmed.2023.1287449 [doi]
LID - 1287449
AB  - When seriously ill patients have exhausted all treatment options available as 
      part of usual care, the use of investigational agents may be warranted. Food and 
      Drug Administration's (FDA) Expanded Access (EA) pathway provides a mechanism for 
      these patient's physicians to pursue use of an investigational agent outside of a 
      clinical trial when trial enrollment is not a feasible option. Though FDA has 
      recently implemented processes to significantly streamline the regulatory portion 
      of the process, the overall pathway has several time-consuming components 
      including communication with the pharmaceutical company and the associated 
      institutional requirements for EA use (contracting, Institutional Review Board 
      [IRB], pharmacy, billing). Here, we present our experience building 
      infrastructure at the Vanderbilt University Medical Center (VUMC) to support 
      physicians and patients in pursuing EA, called the Access to Investigational 
      Medicines (AIM) Platform, aligning the needs and responsibilities of 
      institutional stakeholders and streamlining to ensure efficiency and regulatory 
      compliance. Since its launch, the AIM team has experienced steady growth, 
      supporting 40 EA cases for drugs/biologics, including both single patient cases 
      and intermediate-size EA protocols in the emergent and non-emergent setting. As 
      the EA pathway is a complex process that requires expert facilitation, we propose 
      prioritizing EA support infrastructure at major academic medical centers as an 
      essential regulatory knowledge function.
CI  - Copyright (c) 2023 Joly, Edwards, Jerome, Mainor, Bernard and Pulley.
FAU - Joly, Meghan Morrison
AU  - Joly MM
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
FAU - Edwards, Terri L
AU  - Edwards TL
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
FAU - Jerome, Rebecca N
AU  - Jerome RN
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
FAU - Mainor, Alex
AU  - Mainor A
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
FAU - Bernard, Gordon R
AU  - Bernard GR
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
FAU - Pulley, Jill M
AU  - Pulley JM
AD  - Vanderbilt Institute for Clinical and Translational Research, Vanderbilt 
      University Medical Center, Nashville, TN, United States.
LA  - eng
GR  - UL1 TR000445/TR/NCATS NIH HHS/United States
PT  - Journal Article
DEP - 20231009
PL  - Switzerland
TA  - Front Med (Lausanne)
JT  - Frontiers in medicine
JID - 101648047
PMC - PMC10590908
OTO - NOTNLM
OT  - FDA expanded access
OT  - access to investigational medicines
OT  - compassionate use
OT  - investigational medicines
OT  - regulatory knowledge
OT  - single patient IND
COIS- The authors declare that the research was conducted in the absence of any 
      commercial or financial relationships that could be construed as a potential 
      conflict of interest.
EDAT- 2023/10/25 06:42
MHDA- 2023/10/25 06:43
PMCR- 2023/10/09
CRDT- 2023/10/25 04:03
PHST- 2023/09/01 00:00 [received]
PHST- 2023/09/15 00:00 [accepted]
PHST- 2023/10/25 06:43 [medline]
PHST- 2023/10/25 06:42 [pubmed]
PHST- 2023/10/25 04:03 [entrez]
PHST- 2023/10/09 00:00 [pmc-release]
AID - 10.3389/fmed.2023.1287449 [doi]
PST - epublish
SO  - Front Med (Lausanne). 2023 Oct 9;10:1287449. doi: 10.3389/fmed.2023.1287449. 
      eCollection 2023.