Posted 21 August 2014
By Alexander Gaffney, RAC
The US Food and Drug Administration's (FDA) expanded access program, sometimes referred to as its "Compassionate Use" program, has frequently been in the news as of late, as dozens of patients have taken to social media in an attempt to pressure companies into allowing them access to clinical trials they believe might save their lives.
But while many companies have seemed reluctant to grant access to those trials, pharmaceutical giant Pfizer is taking a markedly different approach for a new expanded access trial for its breast cancer drug palbociclib.
Background: Expanded Access
Under normal circumstances, companies seeking FDA approval for a drug will begin a clinical trial, most often enrolling a group of patients most likely to benefit from the investigational product. Other patients might be unable to enroll in a trial, either because of exclusion criteria or because the study is for a markedly different indication (e.g. the trial is testing the drug in breast cancer, and a patient has colon cancer).
And so some patients, short either on time or available treatment options, petition a company to grant them access to a drug under FDA's compassionate use regulations. As we explain in great detail in this March 2014 article, there are four types of expanded access programs:
- Single Patient (Emergency Access): Used to grant access to a single patient who does not have time to obtain written permission from FDA
- Single Patient (Regular Access): Used to allow a single patient access to a trial
- Intermediate Size: Used for intermediate-sized patient populations
- Treatment: Used for large patient populations (i.e. widespread use)
Those programs can be initiated either as a new trial (with an investigational new drug application) or through the use of a protocol to amend an existing clinical trial.
But for various reasons, companies aren't always enthusiastic to grant expanded access requests. Use of the product outside of tightly controlled trials can be done incorrectly, cause harm to patients, raise troubling safety signals, jeopardize the supply of the product for existing trials and delay approval for the drug's intended use.
But for desperate patients, obtaining access to a drug product might very well represent their best—and sometimes their only—chance at survival. Over the last few years, dozens of patients have taken to social media in attempts to pressure companies into allowing compassionate use access to experimental drugs. In many of these cases, companies initially resisted before finally agreeing to provide access to the drug after public pressure on the company increased.
Pfizer: Come on in
And all that makes a recent announcement by Pfizer all the more unusual.
In a 21 August 2014 announcement, Pfizer announced that it was initiating a multi-center, open-label expanded access program that would allow its drug, palbociclib, to be used in patients with HR+ and HER2- advanced breast cancer.
"Through the program, palbociclib is being made available for use in combination with letrozole for post-menopausal women with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer for whom letrozole is considered appropriate therapy," Pfizer wrote.
Several factors might explain Pfizer's decision to expand the trial.
For one, the drug has already proven promising enough to warrant a "breakthrough product designation" label from FDA based on its clinical results. An application for approval ("new drug application") has also already been submitted to FDA, and two Phase III trials meant to support its approval have already concluded patient enrollment.
Those factors, combined with the size and experience of Pfizer, probably explain a lot about why the drug will now become available to new patients.
And for Pfizer, the prospect of obtaining data to support a new filing for approval probably doesn't hurt either.