Federal Right To Try Legislation – Is It Any Better?

We've generally been skeptical of state "Right To Try" statutes, for several reasons. First, to the extent that they try to circumvent the FDCA, they're likely to be preempted. Second, drugmakers aren't likely to distribute experimental drugs due to liability concerns, and these statutes don't go far enough in removing that threat. Third, such statutes interfere with drug development because they involve the inclusion of people who don't meet study criteria - often because they are too sick - which is likely to increase the number of adverse results, all of which must be reported to FDA.

Apparently the number of states with Right To Try statutes is up to 37 - but we have yet to learn of a single individual that has successfully utilized any of them. For the reasons we've stated before, we're not surprised.

But earlier this month, the United States Senate just passed (apparently by voice ) a reworked version of the "Trickett Wendler Right to Try Act" (S. 4788) that we discussed in our prior post. A federal act would, of course, obviate the preemption problem, or at least replace it with issues of reconciling two federal statutes - which would be a somewhat less daunting a challenge.

Because of that senate action, we're taking another look at the proposed legislation, a link to which is here. First, it applies to anyone "with a life-threatening disease or condition" "who has exhausted approved treatment options and is unable to participate in a clinical trial." §561B(a)(1)(A-B). Eligibility must be "certified" by a treating physician who "will not be compensated directly by the manufacturer for so certifying." Id. §561B(a)(1)(B).

The "investigational drugs" that the legislation would make available must have completed a preliminary "Phase 1 clinical trial" and must be under active "investigation in a clinical trial that is intended to form the primary basis of a claim of effectiveness in support of approval or licensure." Id. §561B(a)(1)(C). Essentially S.4789 seeks to open up to terminal patients access to any unapproved drugs under current investigation, provided they have passed the relatively basic threshold Phase I determinations that they aren't acutely toxic to humans and might have some plausible benefit. Phase I investigations:

are designed to determine the metabolism and pharmacologic actions of the drug in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. During...