August 1, 2019

New program expands options for prescribing investigational therapies

A new group called Access to Investigational Medicine is assisting clinicians who want to seek compassionate use of investigational therapies for their patients.


by Jessica Pasley

Twice in one year, Sean Donahue, MD, PhD, was able to help patients receive an investigational drug through the U.S. Food and Drug Administration (FDA) Expanded Access program.

In both cases, the vision of the patient vastly improved.

Without the help of a new group created by the Vanderbilt Institute for Clinical and Translational Research (VICTR) called Access to Investigational Medicine, or AIM, Donahue knows both of his patients would have gone blind.

“There is no way this would have happened without AIM,” said Donahue, Sam and Darthea Coleman Professor of Pediatric Ophthalmology at Monroe Carell Jr. Children’s Hospital at Vanderbilt. “They did a great job shepherding this process through the FDA, getting all of the necessary approvals and making it seamless on the clinical side.

Sean Donahue, MD, PhD

“These kinds of things take weeks and weeks. I can’t say it enough how thankful I am for this group because it allowed me to continue my clinical work, and two patients received a much-needed treatment.”

Still in its infancy, AIM is ramping up to assist more clinicians who want to seek compassionate use of investigational therapies for their patients.

The goal of the program is to help Vanderbilt Health clinicians seek expanded access to use investigational therapies for their patients, said AIM Project Manager Meghan Joly, PhD.

She said that when patients have exhausted all standard of care treatment options, physicians will often introduce investigational agents that may have potential, but not all patients qualify for the relevant clinical trials.

Meghan Joly, Ph

Under the FDA’s Expanded Access pathway, sometimes referred to as Compassionate Use, patients can gain access to investigational therapies outside of the traditional clinical trial parameters.

“There is such a great need for this kind of service,” said Joly. “We support physicians seeking expanded access to investigational medications for patients. The process can be time consuming and complicated, and our team was designed to help improve efficiency and ameliorate potential barriers to Expanded Access by guiding the physician, patient and drug company through the entire process from beginning to end.”

AIM is supported by VICTR, which is working to transform how ideas and research discoveries make their way from origin to patient care.

“Our team is comprised of experts spanning multiple areas including regulatory processes, informed consent, project management and contracting, which allows us to handle the intricate and complex process from start to finish.”

Donahue is the principal investigator at the Vanderbilt Eye Institute for a gene therapy trial for Leber Hereditary Optic Neuropathy (LHON), a condition that leads to sudden onset of vision loss.

LHON is mainly characterized by bilateral, painless subacute loss of central vision during young adult life. In most cases, symptoms begin with one eye first, followed a few weeks later by visual failure in the other eye.

Vanderbilt is the only medical center in the country currently allowed to enroll children (ages 15-18). The two-year clinical trial is aimed at young adults.

Both of Donahue’s patients were younger than the assigned age for the trial and therefore did not qualify for clinical trial enrollment.

Donahue doesn’t know if the treatment works but knows that vision improved on the therapy and he is thankful that AIM was able to get the compassionate use approved.

“The real value of the AIM program is to minimize the regulatory burden for clinicians such that the opportunity to use an investigational therapy is more desirable,” said Terri Edwards, director of Research Support Services.

For additional information about AIM go to: