Downside? What downside?

At the Brooking-AEI Joint Center site, they have posted a brief by John E. Calfee, Daniel B. Klein, Sam Peltzman, Alex Tabarrok, and Benjamin Zycher, submitted to the DC Court of Appeals, in the case of Abigail Alliance for Better Access to Developmental Drugs, et al., v. Andrew C. Von Eschenbach, et al. The case involves FDA delays in approving new drugs. Here is the gist of their argument.

Preventing terminally-ill patients from pursuing potentially life-saving treatments until effectiveness has been thoroughly substantiated ignores the fact that for patients with no remaining treatment options, any significant, evidence-based chance of an effective outcome may be worth taking. The FDA’s motivation for its unfounded delay in releasing safe drugs stems from an institutional desire to avoid potentially negative public opinion. There are two types of errors that can be made in deciding whether to permit new drugs to be sold: erroneously identifying drugs as reasonably safe that are in fact unsafe, and refusing to permit the sale of drugs that are in fact reasonably safe. These errors are known as Type I errors and Type II errors, respectively. In arguing for reconsideration, the FDA suggested that releasing post-Phase 1-approved drugs to volunteers would risk exposing terminally-ill patients not only to inefficacious cancer treatments, but also to potentially unsafe side effects—a classic Type I error. But the FDA’s institutional incentives to exercise excessive caution in approving new drugs, even if rational and well-informed patients would want access to such
drugs, is not always consistent with patient welfare. Empirical evidence strongly suggests that the FDA historically has been over-concerned with avoiding Type I errors while underestimating the damaging public health consequences of the Type II errors that affect patients such as the members of the Abigail Alliance.