Posted 09 October 2014
By Alexander Gaffney, RAC
A libertarian-leaning think tank is pressuring the US Food and Drug Administration (FDA) to release information regarding the internal approval processes it used to permit two US citizens stricken with Ebola access to Mapp Biopharmaceuticals' ZMapp, an experimental drug.
In August 2014, the American public became fixated on an outbreak of Ebola in West Africa after two US missionaries, Dr. Kent Brantley and Nancy Writebol, became infected with the disease. In the effort to save their lives, they were flown back to the US and treated with Zmapp, a monoclonal antibody derived from mice.
But as Focus has explained in the past, the story behind how Brantley and Writebol actually obtained access to Zmapp has been something of a mystery.
For example, did the missionaries obtain access to the drug through FDA's "compassionate use" program, which is used to give small numbers of patients access to experimental drugs outside a clinical trial? Neither the company nor FDA has said whether this is the case.
Due to commercial confidentiality laws, FDA isn't allowed to confirm if a company has or has not filed an investigational new drug (IND) application, which is used to start a clinical trial. As such, the agency wouldn't confirm to Focus if Zmapp had been cleared through FDA's compassionate program. Mapp Biopharmaceutical's CEO, Larry Zeitlin, told The Washington Post that the company is "discussing with the FDA the right path to make the drug available to people as quickly and safely as possible."
However, it largely confirmed that if any drug was being used experimentally, it would need to go through FDA.
Here's FDA's statement on the matter: "Currently there are only experimental treatments for Ebola virus infection in the earliest stages of development. When a drug is not approved, the FDA can authorize access to potentially promising products through other mechanisms, such as through an emergency Investigational New Drug (IND) application. In order for an experimental treatment to be administered in the U.S., such a request must be submitted to and authorized by the FDA. The FDA cannot comment on the specifics of ongoing drug development programs and cannot reveal information that is not otherwise public concerning submissions covering such programs such as IND applications submissions. The FDA stands ready to work with companies and investigators treating these patients."
FDA confirmed to Focus that an emergency IND request can be authorized by the agency "within a very short period of time, depending on the urgency of the situation and the nature of the available information." There are currently no FDA-approved treatments for Ebola, FDA confirmed.
FDA also has other avenues through which it could provide access to an experimental therapy. For example, it can exercise its "Enforcement discretion"—basically which regulations it chooses to enforce and which ones it does not. It's possible that in the interest of expediting access to the drug, FDA simply chose to defer to public health officials overseeing the treatment of Writebol and Brantley.
If Compassionate Use Doesn't Work for Ebola…
But hypotheticals aren't of interest to The Goldwater Institute, a think tank heavily involved in pushing for nationwide reform of FDA's compassionate use system. The organization has already successfully lobbied for state-level compassionate use laws, and similar legislation has been introduced in the US House of Representatives.
The gist of the laws: Shield drug companies from legal liability, allowing them to more easily offer experimental therapies to desperate patients who might otherwise not have access to the treatment, or have to wait too long for the treatment to be useful.
The Institute filed a Freedom of Information Act (FOIA) request with FDA, seeking to find out if the agency had used the compassionate use process to grant Writebol and Brantley access to the drugs, as well as to "understand the internal decision-making process used by the FDA" during that time.
"The Goldwater Institute wants to know if the doctors were required to comply with the FDA’s standard 'compassionate use' process and procedures that other Americans seeking access to experimental medications must follow," the group wrote in a press statement. "The average compassionate use request takes 300 hours to complete," they contended, though it's unclear from where that data originates.
But the Institute's broader intent is clear: If FDA can't even use its compassionate use system for Ebola patients, why should other patients have to abide by it as well?
“If these doctors were allowed access to experimental medication without following the FDA’s normal process, we want to know why and how other sick or dying Americans can also get an exception,” said Jon Riches, an attorney at the Goldwater Institute, in their statement.
For now, though, the institute will—like the rest of us—have to wait for that information. FDA denied the organization's FOIA request, citing the same commercial confidentiality concerns that it had communicated to Focus in August 2014. Goldwater said it plans to appeal that denial.
Focus Explainer on Ebola Regulation
Focus Explainer on Compassionate Use