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Getting Experimental Drugs to Patients Who Need Them

May 27, 2016
Baby in NICU with canula

When a patient’s traditional treatment options are exhausted, sometimes there is an experimental drug—not yet approved by the FDA—that could be beneficial.

Baystate's Human Research Protection Program may be able to help doctors get these treatments for their patients through the FDA's compassionate or emergency use process.

When You're Down to the Final Treatment Option

Last summer, a young man arrived at Baystate Medical Center’s ED in sickle cell crisis, a painful episode where the sickle-shaped red blood cells block blood vessels, preventing blood and oxygen from getting through. It can damage tissues and cause liver or kidney failure, even death.

He was admitted, but his health declined over the next few days. A blood transfusion—the only proven treatment—was recommended. But the patient declined due to his faith. As his condition continued to worsen and death seemed imminent without a transfusion, experimental treatments were explored.

Hemopure, which is based on bovine hemoglobin, holds potential for patients with life-threatening anemia for whom human blood is not an option. The family agreed to try it. But, it is not approved by the Food and Drug Administration.

Emergency Use May Be the Last Resort

In certain cases, the FDA allows experimental drugs to be accessed by patients with serious or life-threatening diseases who have no comparable therapeutic alternatives and are ineligible for clinical trials of the drug.

The manufacturer must agree to provide the drug to the patient, and the doctor must get an investigational new drug application approved by the FDA.

“When we get a request for emergency or compassionate use, it bumps everything else off our schedule,” says Jennifer Pacheco, Director of Baystate’s Human Research Protection Program. “We know there is a specific patient that needs help and we drop everything to do it. It’s not unusual for staff to stay late or put in weekend hours.”

The doctor of the young man with sickle cell anemia called the HRPP office, and Ann Marie Collins and Pacheco worked with the clinical care team, the pharmaceutical company, and the FDA to get an emergency use request approved in about 24 hours.

When Treatment is Needed Right Away—Even on Christmas

According to Pacheco, most emergency use requests are from doctors on clinical service. They are usually not researchers used to dealing with the FDA approval process. “So, we try to complete as much of the required paperwork for the doctor as we can.”

When HRPP knows a request is coming, they reach out to IRB chairs and vice chairs asking for help finding a reviewer for the application.

"We will page, phone, email, whatever is needed," says Pacheco. “We’ve had reviewers in clinic and if they have a 15 minute slot due to a patient cancellation they will review it. They drop everything they can to move this through, too.”

It takes a lot of coordination between the clinical team, the IRB, Baystate’s investigational pharmacy, the FDA, the pharmaceutical company—and sometimes some unexpected outside agencies.

“We’ve had product needing to be here on Christmas day. No shipping company delivers on Christmas, “ recounts Pacheco. “We had to coordinate troopers meeting at the state line to receive it from another state’s police officers so that child could get the drug when it was needed.”

Emergency NICU Nutritional Supplement Approval in Under 3 Hours

In March, the HRPP office received an email from Dr. Rachana Singh regarding an emergency use request for Omegaven for a 2 month old in the NICU. Omegaven is a nutritional supplement for NICU patients not able to take breast milk, and for whom tube feeding is not possible or it has had an adverse effect on their liver. It’s not approved in the U.S. and is available only from Germany.

The FDA agreed to allow an emergency use Single Patient Investigational New Drug application for this specific patient. The IRB staff worked with Dr. Singh, who was on service, to complete the required paperwork on her behalf,  secured the required IRB approval, then worked with the pharmacy to get the order ready to send to the manufacturer in Germany —in 2 hours and 48 minutes.

More Doctors Taking Advantage of this Service

According to Pacheco, she is seeing an increasing number of emergency use requests—6 over the past 18 months instead of the more usual 1 per year.

She thinks it is because word of mouth has spread that the HRPP has this service and can help doctors facilitate the compassionate and emergency use of experimental therapies for the benefit of their patients.

"We assess where they are in the process and piece together the puzzle to get them where they need to be."

 > More information about what the Human Research Protection Program does
 > Learn about clinical trials at Baystate