Part 2 of this blog series questions whether expanded access meets a fundamental right to access investigational drugs.
Quick Answer: The current laws do not support a fundamental right to access investigational drugs.
According to the FD&C Act, “no person shall introduce or deliver for introduction into interstate commerce any new drug” unless it has been approved for marketing by the FDA. Approval requires that the new drug has been proven both safe and effective in humans based on at least 3 phases of “adequate and well-controlled” clinical studies [See 21 CFR 312.21 Phases of an investigation and 21 USC 355 Section (d)]. Drug sponsors are permitted to submit treatment INDs to provide Phase 3 or, less commonly, Phase 2 drugs to patients with “a serious or immediately life-threatening disease condition in patients for whom no comparable or satisfactory alternative drug or other therapy is available” [See 21 CFR 312.34]. However, the FDA reserves the right to deny access if there is “insufficient evidence of safety and effectiveness to support such use” or there is an “unreasonably and significant additional risk of illness or injury,” even if the patient’s illness is immediately life-threatening [See 21 CFR 312.34]. Furthermore, no one can compel the drug sponsor to provide investigational drugs to patients not enrolled in clinical studies, or make such a request on their behalf; participation in a treatment IND is voluntary. So the current laws do not support the existence of a fundamental right.
This argument is supported by the 2007 ruling in a recent case: Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach. The Abigail Alliance was formed by Frank Burroughs after his daughter, Abigail, died of head and neck cancer in 2001. She had been denied access to then-investigational treatments cetuximab (Erbitux) and gefitinib (Iressa), both of which went on to win FDA approval after her death. Convinced the denial of access (and possibly Abigail’s premature death) was caused by unnecessary regulation and bureaucracy, the Abigail Alliance sued the FDA in an attempt to change the existing regulations and permit terminally ill patients to access new drugs as soon as they have completed Phase 1 trials.
In its argument the Abigail Alliance claimed “the concepts of self-defense, necessity, and interference with rescue are broad enough to demonstrate the existence of the fundamental right they seek—a right for ‘persons in mortal peril’ to ‘try to save their own lives, even if the chosen means would otherwise be illegal or involve enormous risks.’” In support of its argument, the Alliance cited Washington v Glucksberg saying the FDA’s regulations violated the Constitutional concept of Due Process and the right to self-protection. But the US Court of Appeals for the District of Columbia dismissed the argument by saying, “The Alliance’s effort to focus on efficacy regulation ignores one simple fact: it is unlawful for the Alliance to procure experimental drugs not only because they have not been proven effective, but because they have not been proven safe.” The Court disagreed with the Abigail Alliance’s interpretation of Washington v Glucksberg stating, “A tradition of protection does not alone establish a fundamental right.”
In addition, the US Court of Appeals for the District of Columbia asserted, “There is no fundamental right ‘deeply rooted in this nation’s history and tradition’ of access to experimental drugs for the terminally ill.” The Abigail Alliance took its appeal to the US Supreme Court, but they were denied certiorari in 2008 and the District Court decision stood.
I feel it is also important to add here that a fundamental right should not, and could not, apply only to a segment of the population (terminally ill people). If the right is truly fundamental, all persons should have the same access to investigational drugs even if their illness is mild and non-life-threatening. Current laws and the Constitution include no such fundamental right, so this first criterium is not met.
Part 3 of this series discusses whether the benefits of taking investigational drugs outweighs the risks.
For more, contact the Kulkarni Law Firm.