Abigail Alliance v. Von Eschenbach: A closer look

Abigail Alliance v. Von Eschenbach: A closer look

In 2003, the Abigail Alliance for Better Access to Developmental Drugs sued the FDA to gain greater access to unapproved drugs. The Alliance was founded by Frank Burroughs, the father of Abigail Burroughs, a young woman who was diagnosed with squamous cell carcinoma of the head and neck. After conventional therapies failed, she campaigned hard for access to experimental drugs. However, she did not meet entry criteria for clinical trials of two (then) unapproved cancer therapies--ImClone's Erbitux and AstraZeneca's Iressa. Abigail eventually died while waiting for newer treatment. Erbitux was later approved for head and neck cancer [1] and Iressa [2] for lung cancer.

Currently, the FDA allows limited "compassionate use [3]" of experimental therapies. Patients who don't meet study entry criteria but have no other treatment options may be eligible receive unapproved drugs that have completed Phase II or III trials. But the Abigail Alliance wants patients to have access to therapies after Phase I trials are completed. In its suit, the Alliance argued that after a drug passes Phase I, they are sufficiently safe for expanded human use and should be available to terminally ill patients.

To dying patients, unapproved drugs must look like a treasure trove of possible cures and one can certainly understand their willingness to try any new therapy. But by their very nature, Phase I trials are small and are designed to expose as few people as possible to untested therapeutics to minimize risks. We often see promising drugs go up in flames [4] because of toxicity or efficacy concerns, and early-stage drugs (especially cancer therapies), which haven't survived the rigors of later testing, are more likely to be ineffective and even deadly. Only one in five drug candidates that enter clinical trials will receive FDA approval; most will be abandon because of safety or efficacy problems. Without Phase II and III clinical trials, there is a very real risk that a drug will do more harm than good.

But the Alliance argued that dying patients have a constitutional right to self-defense, which extends to choosing what risks they're willing to face to extend their lives. Should a terminal patient have the right to take a drug that may worsen their condition or even speed up their death? The court ruled that the constitutional right to self-defense doesn't include assuming any and all risk [5]. "Recognizing a right of self-defense cannot justify creating a constitutional right to assume any level of risk without regard to the scientific and medical judgment expressed through the clinical testing process," ruled the majority in the decision [6].

Aside from safety and efficacy, there are a number of other reasons why expanded access is problematic:

• Investigational drugs are extremely expensive to produce and supplies are generally limited.
• Finding a physician willing to administer an inadequately tested drug would be difficult at best given the potential liabilities.
• Expanded access would erode the FDA's authority. There would be loss of control over the actual access to prescription drugs, and untested therapies could be provided to patients outside of clinical trials which the FDA has not approved.
• Perhaps most importantly, if patients can get new drugs before clinical trials are completed, it would seriously undermine the entire clinical trial process because patients would have no reason to enroll in trials. If the drug is available outside of a clinical trial, the rigorous scientific controls over the data normally collected during the conduct of a study would be lost. This would slow the approval process to the detriment of the general public.

The FDA is under pressure from critics who say it allows unsafe drugs on the market [7]. But patient advocates castigate the agency for taking too long [8] to get new therapies to the public. Both arguments are valid, and it's essential that the FDA find the delicate balance between the two. The Abigail Alliance plans to appeal the case to the Supreme Court. - Maureen Martino [9]