We blogged a couple of years ago about the beginnings of what has become a wave of state “Right to Try laws” – laws that purport to give terminal patients with no other medical options the right to seek investigational drugs for their conditions from manufacturers who have yet to obtain full FDA approval.  Mostly, these laws are motivated by the unwieldy nature of the FDA’s “compassionate use” regulations, which are directed at the same problems.  Given that these laws originated with a “states’ rights group” called the Goldwater Institute, we strongly suspect that a second motive of gratuitously poking the FDA in the eye was also at work.

Whatever their provenance, we were deeply skeptical of their practicality. Three large obstacles loomed.  Number one:

For one thing, there’s the FDA. States can pass all the laws they want, but unless the FDA gives its okay to programs more expansive than its compassionate use (“expanded access”) program, nothing’s going to happen.

Number two:

The reason, as is the case for so many things these days, is the threat of liability. . . . You won’t induce a manufacturer to participate in a voluntary program by painting a target on its back.

Number three:

There’s no upside. These statutes are for use by very ill people, and if (as is unfortunately likely) the statutory participant died, then there’s an adverse event that must be reported to the FDA.  Companies investigate drugs in the hope of obtaining approval.  Adverse events definitely don’t help get approval.

Indeed, despite a couple dozen Right to Try statutes enacted over the last few years, we are unaware of even a single instance in which anybody successfully obtained treatment with an investigational drug under any of these state laws.


Continue Reading Developments in Compassionate Use and Right To Try Laws