It’s ironic that at a time when several high-profile marketed drugs have been given a black box warning or withdrawn from the market that the Abigail Alliance has gathered steam.
The Abigail Alliance is a patient advocacy group that is suing the U.S. Food and Drug Administration for the patients’ “right” to take an experimental drug after it has been through only one phase of drug development. Two members of the three-judge panel of the U.S. Court of Appeals for the D.C. Circuit found that patients with a life-threatening and otherwise untreatable disease have a constitutional right to seek experimental treatments, even if researchers have not determined efficacy. In addition, the court said that the government should be removed from the equation unless it has a “compelling interest.”
If by some strange series of events, the Abigail Alliance actually won its suit against the FDA, the repercussions would be immediately and unquestionably anti-consumer. It seems the public wants to have it both ways—drugs that are safe for all and at lightning speed. That is impossible.
Drugs approved by the FDA for the marketplace must complete all three phases of clinical research, and are often tested on as many as tens of thousands of people for some indications. But, safety is a small numbers game. Perhaps one in 10,000 people will suffer a serious adverse event after a drug is released to the market. Pharma are more attuned to this fact than ever, which is why adaptive clinical trial design is so appealing. Such adaptive trials would allow pharma companies to look at outcomes while a clinical trial is ongoing and be able to react faster. Implementing adaptive clinical trial design would allow biopharmaceutical companies to stop a clinical trial early if they know it will fail. It would also allow them to identify the subset of the population that has a severe side effect and exclude them to avoid further adverse events, instead of stopping a clinical trial altogether.
Yet, the Abigail Alliance is asserting the alleged right of American consumers to drugs that have only been tested for safety on fewer than 100 people and have very little to no evidence of efficacy. To further muddy the waters, these would be called “tier 1” drugs. It defies logic that critically ill patients would prefer to take a barely tested drug outside of a supervised medical experiment and contribute absolutely nothing to advance science in that therapeutic area. A recent Boston Globe op-ed highlighted the fact that since only 5% of cancer patients participate in clinical trials, it takes three to five years for a therapy to demonstrate effectiveness. That could be cut to one year if the percentage of these patients were doubled, according to a former National Cancer Institute head. Doubling the participation of patients with life-threatening diseases in clinical trials is what the Abigail Alliance should concentrate their efforts on, which would bring drugs sufficiently tested for safety and efficacy to market faster.
Drug companies cannot afford to stop learning about their drugs, especially after only one phase of development that studies 20 to 100 people. It would be impossible to recruit patients for phase II and III clinical trials where efficacy and further safety testing occur, if patients already had access to the study drug. If the Abigail Alliance wins its suit, it will wreak havoc on the drug development process and how it is regulated. Safety and efficacy standards will suffer, sending us back to the pre-FDA days of nostrums and snake oil.