Legislation Seeks to Overhaul FDA's Compassionate Use Program

Regulatory NewsRegulatory News
| 09 December 2014 | By Alexander Gaffney, RAC

New legislation introduced in the House of Representatives would makes changes to the US Food and Drug Administration's (FDA) "expanded access"—sometimes called "compassionate use"—policies in the hopes of making it easier for terminally ill patients to access potentially life-saving drugs.


Under normal circumstances, companies seeking FDA approval for a new drug will first ask regulators for permission to begin a clinical trial using an application known as an investigational new drug application (IND). For most companies, this trial will seek to enroll a group of patients believed to be most likely to benefit from the investigational product.

Other patients might be unable to enroll in a trial, either because of exclusion criteria (e.g. too old or too many co-morbidities) 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).

However, some patients—typically terminally ill patients with no remaining treatment options—petition companies to allow them to access the investigational product using 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 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.

One patient in particular, Andrea Sloan, made headlines in late 2013 during her fight to obtain compassionate use access to BMN763—an experimental cancer drug made by BioMarin Pharmaceuticals. BioMarin refused Sloan access to the drug, and she died several months later.

New Compassionate Use Legislation

Now new legislation introduced by Rep. Michael McCaul wants to make it easier for patients to enroll in expanded access trials, if only for certain types of drugs.

McCaul's legislation, the Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act, would amend FDA's compassionate use policies to require any company whose drug is granted "breakthrough," "fast track," or "Qualified Infectious Disease Product" (QIDP) designation to make publicly available a corporate expanded access policy for compassionate use of the drug.

That policy would include, among other things, a point of contact at the company who can process requests, the procedures for making an expanded access request, the criteria for enrolling in an expanded access trial, and the amount of time the company expects to take to process a request.

The policy would need to be released to the public within 30 days of a sponsor receiving a designation from FDA.

Other Requirements

Companies would also be required to notify patients or their representative if their request for compassionate access has been denied within 5 days of the denial, along with an explanation for why the request was denied. The legislation does not define what constitutes a denial, however, and it remains unclear how this provision would be enforced in practice. A company might, for example, keep open a request for a lengthy amount of time instead of formally denying the request.

The legislation also calls for the formation of an "Expanded Access Task Force" that would be charged with evaluating "the access individual patients have to investigational drugs," as well as:

  • "ways to streamline and standardize the process for submitting requests"
  • challenges faced by children
  • ways biopharmaceutical companies could be incentivized to approve expanded access requests
  • the costs incurred by companies who grant access to expanded access programs
  • "how the Secretary of Health and Human Services interprets and takes into consideration adverse event data reported in the case of data from use under a request submitted under such sub section"

FDA would be also be tasked with finalizing a May 2013 guidance document within 180 days of the legislation's passage.


Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act


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