September 2019 Edition Vol.11, Issue 8

FDA Eases Access to Unapproved Cancer Drugs

By Christina Bennett, MS

Patients with cancer are treated with drugs already approved by the U.S. Food and Drug Administration (FDA) or with an investigational agent in a clinical trial. But there are some patients who may need access to investigational drugs outside the realm of a clinical trial. Expanded Access to these investigational drugs, also known as “compassionate use,” is a pathway regulated by the FDA through which patients can access unapproved drugs outside of the clinical trial setting when no comparable or satisfactory treatment options are left.1

“When a patient has metastatic breast cancer and they’re running out of options, that’s their only hope,” said Wael Harb, MD, Chief of Medical Management, Verdi Oncology, Inc. Patients cannot wait six months until the drug gets approved—it might be too late, he noted.Howe

Expanded Access helps not only patients outside of clinical trials but also those for whom the trial has ended when they need to continue on the drug. For example, Dr. Harb had a patient with lymphoma who was treated with an investigational drug in a clinical trial—and her disease was responding—but the drug company stopped development of the drug. Dr. Harb requested the drug through Expanded Access, and the patient continued to receive the drug.

However, challenges with attaining Expanded Access may impede physicians from initiating a request in the first place.2 Specifically, the process can be burdensome and time-consuming. The current FDA application form alone takes an estimated 45 minutes to fill out, and physicians are often required to write a clinical protocol, similar to what is required for a clinical trial but shorter.3

In addition, the physician has to contact the drug manufacturer to make sure they will supply the drug and then secure approval from an Institutional Review Board (IRB) as well as obtain informed consent from the patient. If there is no IRB at a physician’s institution, the physician may need to go to an independent IRB, which incurs financial costs.2 Once the FDA, drug sponsor, and IRB agree, the patient can receive the drug through Expanded Access.

The process itself may be too difficult for physicians to navigate alone, especially for community oncologists with limited resources and support.

“I found [the process] very challenging,” said Apostolia Tsimberidou, MD, PhD, Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. Whenever Dr. Tsimberidou attempted to request drugs from a pharmaceutical company through Expanded Access, the process was arduous and complicated. She had to “spend a significant amount of time and energy” trying to contact the pharmaceutical company applying for the study drug, without success.

The time from initial request to final receipt of the drug takes several weeks, potentially impacting patient care. Chul Kim, MD, MPH, an assistant professor at Georgetown University, said the total process took him about five to six weeks to get approval. “Thankfully the patient didn’t progress fast during that time,” he said. However, for Dr. Harb’s patient, the process took eight weeks and treatment was interrupted as a result.

With the existing Expanded Access programs, the potential for unequal access among physicians is also present. According to Richard Pazdur, MD, Director of the Oncology Center of Excellence at the FDA, most of the Expanded Access requests the FDA receives are from university programs or from investigators who have had experience with the requested drug.


Project Facilitate

To improve on the application process, the FDA launched an oncology-specific pilot program on June 3, 2019, dubbed Project Facilitate.4 The program introduces a single point of contact at the FDA who can help guide physicians through the application process. The staff will also have contacts for the specific pharmaceutical companies so the physicians know who to call. In addition, there will be IRB resource options available.

Although Dr. Harb has not tried the new program, he said it sounds like it might help with logistical issues. He recalls having to make several calls and send several emails to just find the right person at the FDA who could help.

Dr. Kim agreed that having a single point of contact is beneficial, especially for community oncologists who don’t have the same resources as academic oncologists. Dr. Tsimberidou also expressed optimism, saying the program will make the process simpler and more efficient.

Unearthing Valuable Data

For the existing programs, the FDA has been limited in their data-gathering abilities, leaving several unknowns about the process. At the present, they don’t know how many patients are requesting single patient Expanded Access.

Dr. Pazdur explained that many times the treating physician may go to the drug sponsor and the FDA is not aware if the request is denied. The agency also doesn’t know the reasons for denying requests, the amount of time it takes for a patient to receive treatment once the request is approved, or patient outcomes, such as benefits and adverse events associated with the designated therapy. On the other hand, the FDA does track submissions to the agency and has the data to show that the agency approves nearly all Expanded Requests, with more than 99% of submitted requests approved between 2014 and 2018.5

Project Facilitate will collect metrics on this unknown information, and although the program is still in its infancy, some data have emerged. As of July 11, 2019—approximately five weeks into the program, the FDA has approved 61 Expanded Access requests.

The amount of requests is 20% higher than the same time interval last year, said Gideon Blumenthal, MD, Deputy Director of the Office of Hematology and Oncology Products (OHOP) at the FDA. He said they have also received many calls on what Project Facilitate does, the Expanded Access submission process, and being thanked by providers for offering this program.

Dr. Blumenthal assured that the data collected through Project Facilitate are not anticipated to require additional work on the physician’s behalf. According to current regulations, the treating physician is required to submit annual and safety reports. Project Facilitate will walk the provider through these processes and may be able to help the provider fill out these forms.

“We are hoping this streamlined process will decrease the regulatory burden on the treating physician and their team,” he said.

Dr. Blumenthal said that the time spent to compile the FDA submission is straightforward. As for the IRB submission and getting an approval from industry to gain access to the drug, the FDA does not have any regulatory oversight on those processes. He noted that the FDA is working with the Reagan-Udall Foundation and other key stakeholders to streamline those processes.

Dr. Harb said the data collected will be valuable, especially for understanding what the barriers are to Expanded Access so those can be addressed. Another area where the information can be useful is the number of requests for certain drugs, which may lead to the FDA working with the drug company to develop a protocol for more Expanded Access or even a clinical trial.

“Knowing where the demand is,” Dr. Kim said, “can be helpful.”

Here are the key steps for physicians to follow and submit Single Patient Expanded Access requests to pharmaceutical companies as well as the requirements for FDA and IRB review.


Source: Infographic adapted from the June 18, 2018 original ( courtesy of Janssen Pharmaceutical Companies of Johnson & Johnson.

Providers can call for assistance through Project Facilitate at 240-402-0004 or email at Call centers hours are 8 a.m. to 4:30 p.m., Eastern Daylight Time, Monday through Friday.


  1. Expanded Access: Information for Patients. U.S. Food and Drug Administration. Accessed August 21, 2019.
  2. Fountzilas E, Said R, and Tsimberidoua AM. Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits. Expert Opin Investig Drugs. 2018;27(2):155-162.
  3. Jarow JP, Lurie P, Ikenberry SC, and Lemery S, et al. Overview of FDA’s Expanded Access Program for Investigational Drugs. Ther Innov Regul Sci. 2017;51(2):177-179.
  4. FDA announces Project Facilitate to assist physicians seeking access to unapproved therapies for patients with cancer. U.S. Food and Drug Administration. Accessed August 21, 2019.
  5. Expanded Access (compassionate use) submission data. U.S. Food and Drug Administration. Accessed August 26, 2019.

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