By Noah Lewellen. Full text here.
Decisions in the Supreme Court and, more recently, the Ninth Circuit have cast doubt on the role of statistical significance in drug development. In United States v. Harkonen, the defendant Harkonen was convicted of fraud for advertising successful testing of a drug when, in fact, the tests had not revealed statistical significance prior to any post
–hoc data manipulation. The government’s position, that statistical significance was key for a showing of causation, seemed directly opposite to the reasoning of the Supreme Court in Matrixx Initiatives, Inc. v. Siricusano. In that case, the Court found that Matrixx was liable to its investors for failing to disclose reported adverse effects of its marketed drug, even though the adverse effects had not been causatively tied to the drug via statistical significance. The opinions’ ostensible disagreement led to the question of whether or not statistical significance is a requirement to show causation in the context of prescription drugs.
Clarification of the issue requires a contextual examination of the cases and an analysis of the FDA’s public policy goals behind its use of statistical significance in drug safety and efficacy. This Note posits that Harkonen and Matrixx are not truly in conflict with one another because both comport with the public policy goal of consumer safety. Although the FDA has not issued official guidance on the treatment of statistical significance as a general concept, it has adopted an identical public policy. Drug developers must meet a stringent threshold of proving causation through statistical significance to ensure the efficacy of a drug, but such stringency is aimed, ultimately, at protecting the health and safety of consumers. This policy is not served by requiring statistically significant showings regarding adverse effects of drugs. In the emerging field of personalized medicine, wherein sample sizes are necessarily small and studies may lack the power to generate enough data to ensure statistical significance, the FDA has adopted a balancing approach, weighing statistical significance against other factors.
The theme tying these approaches together is the policy of consumer safety. As such, this Note argues that courts should expand on this policy and should hold presumptions against both the efficacy and safety of a drug when determining the disclosure obligations of drug developers.