The Goldwater Institute, a think tank lobbying for the expanded access to experimental drugs, commonly known as the Right-To-Try Legislation, sued FDA to disclose the regulatory processes used to release an experimental Ebola drug to a few patients in the US during last year’s Ebola outbreak. In August 2014, FDA allowed use of Zmapp, an experimental drug being developed by the San Diego company Mapp Biopharmaceuticals, in 2 patients infected with Ebola virus. Both patients fully recovered from Ebola infection. It is widely accepted that the drug was made available under the compassionate use IND pathway but both the FDA and Mapp Pharmaceuticals have been secretive about the exact process used. FDA is bound by confidentiality rules since the compassionate use IND is a proprietary document that cannot be publicly disclosed by the Agency, and the pharma company obviously wants to closely guard its secret program from competition. However, in the process, patients who want access to other experimental drugs on a compassionate basis, and their supporters such as The Goldwater Institute, want to learn more about the regulatory precedence behind the compassionate IND review and approval process. It is widely believed that this suit will fail in the courts as asking FDA to illegally disclose proprietary documents could potentially open the floodgate to similar petitions for other drugs making the entire FDA confidential review process moot and subject to severe criticism. The law suit is erroneous in claiming that the FDA (by extension the Federal Govt.) is keeping it secret, while in fact it is the company which holds the key to the information.
Since early 2014, 22 states have passed the so-called “Right to Try” laws with similar bills at various stages of review at the other 28 states. These laws allow terminally ill patients access to experimental treatments more easily. The hope is that patients may be able to use potentially life-saving treatments before their approval by FDA that could take years. These state laws intend to further promote FDA’s compassionate use pathways to make it faster and easier for patients to obtain experimental therapies which they cannot get in a clinical trial setting. While the spirit of these laws is noble, they do create a severe challenge to the impartial regulatory review process and potentially forces companies to provide drug supplies to patients outside of ongoing clinical trials and could hinder the innovation process critical to make drugs available to everyone.
