Pursuing the expanded access process for treatment may be the best option for patients who have exhausted all available FDA-approved drugs and clinical trials.
For patients with cancer who have progressed through all treatment options available for their disease state, pursuing the expanded access process (EAP) for treatment may be the best option in such cases, explained Micha Koentz, PharmD, an investigational pharmacy supervisor at Cook Children's Medical Center in Fort Worth, Texas, during a presentation at the 2022 ATOPP Summit.
However, there remain several obstacles for pharmacists to overcome when pursuing this option for patients, including whether the drug is available and stocked at the hospital or pharmacy and whether the FDA approval covers that patient population and/or disease state. If the FDA approval does not cover that patient and/or disease state, such as in the case of a pediatric patient for a drug approved by the FDA for adult patients only, then insurance will likely not reimburse for that treatment, leading to financial toxicity for that patient and/or their caregivers.
“Overall, there are 3 main ways that a patient can get a drug,” Koentz said. “First and foremost, we are all most familiar with FDA-approved drugs. These drugs have safety and efficacy data out there, they have labeling, they're often reimbursed by insurance companies, and they have the broadest availability.”
Koentz explained that following exhausting all options from the FDA-approved drug category, the next option to help patients get access to a drug under investigation for a certain disease state is by enrolling them in a clinical trial.
“Institutions may have a clinical trial that is open, so enrolling your patient for this can be a great aid,” Koentz said. “This avenue will help provide safety and efficacy data, and clinical trials are very important because it helps us to get those drugs FDA-approved and available for patients through insurance reimbursement.”
Once a pharmacist has exhausted all treatment options through clinical trials, they may then need to pursue getting the patient access to a drug through the EAP, Koentz explained.
“I never knew this route existed in pharmacy school or even residency. I became familiar with it over the past couple of years through some passion projects of mine,” Koentz said. “So when all other options have been exhausted, and you have a patient with cancer, and they've gone through the standards of care options, and there are no more clinical trials available; well, maybe they will be eligible for a drug that's not yet available. You can call a drug company and ask if the [patient] can have access to their drug.”
Koentz noted that under the FDA, the EAP is defined as the use of an investigational medical agent to treat a patient with a serious disease or condition who does not have a comparable or satisfactory alternative therapy to treat their disease or condition. In this way, the primary intent of the EAP is to treat a patient, which is in contrast to the primary intent of a clinical trial, which is to gather safety and efficacy data in a systematic collection and learn from it.
“So, in a clinical trial, you want to learn. In expanded access, you want to treat,” Koentz said.
Koentz noted that there are also several different names used for an EAP in the field, the most common alternative being “compassionate use.” Koentz explained that she uses the terms “compassionate use” and “EAP” interchangeably for her purposes.
There are also multiple parties involved in the EAP, with the first party being the patient, Koentz explained. Following the patient is the physician, who can explore and assess the treatment options that are out there for an EAP.
Working with the physician is the pharmacist, who can help the physician oversee the patient's treatment. Additionally, the pharmacy can help work with the industry to file paperwork with the FDA and the institutional review board (IRB) for the EAP. The pharmacy is also responsible for patient care and reporting any adverse effects the patient experiences back to the drug company.
The next party involved in the EAP is the drug company, who must be willing to provide the patient with the drug for free. If the drug company does provide the drug to the patient, then a letter of authorization needs to be completed as well.
“So here's a quick overview of how you open up a single patient [investigational new drug application (IND)]. The physician or pharmacist will contact the industry or drug manufacturer. Then, you must have FDA and IRB approval,” Koentz said. “You can then order the medication and obtain informed consent, like you would on a clinical trial. From there, the treatment can begin.”
In 2018 and 2019, the FDA noticed that their single patient IND oncology requests increased by 20%, Koentz explained. Due to this high demand, the FDA’s Oncology Center of Excellence developed Project Facilitate, which is a program that creates a single point of contact to the FDA for single patient IND oncology requests. Project Facilitate has a telephone number available during normal business hours, but they also have a 24-hour call line as well.
“I’ve called [the 24-hour line] before and they do answer on the weekends and nights, and you can get a hold of them when it’s really needed if it's a true emergency,” Koentz said. “There is also a Project Facilitate email that can be used. This email will direct your request to the group of people that works there, and hopefully somebody will answer you quickly and help you out with what you need and will help you with the step-by-step process.”
Koentz M. A One Size Fits All Solution to Off-Label Procurement: Lessons Learned From Pediatrics That Anyone Can Use. San Diego, CA: 2022 ATOPP Summit; July 14, 2022.