National Right To Try Legislation Passes Congress

On May 22, 2018, the United States House of Representatives passed 250-169 the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Mathew Bellina Right to Try Act of 2017 (S. 204), intended to expand access by terminal patients to investigational drugs.1 Passage of this bill, first approved by the Senate on August 3, 2017, is the culmination of a national campaign, led by the Goldwater Institute, a libertarian public policy think tank, that has galvanized 40 states to pass their own right to try (RTT) laws. While clearly an important political and policy statement, the extent to which this legislation will have a practical impact on expanded access remains to be seen. This Advisory summarizes the existing framework for expanded access to experimental treatments, key aspects of the RTT legislation, and potential implications for stakeholders.

Expanded Access Under Current FDA Rules

State and federal RTT legislation purports to provide terminally ill patients with an alternative to obtaining investigational drugs through FDA's expanded access program.2 FDA's current regulations and guidance permit administration of an investigational drug to eligible individual patients3 under a single-patient, compassionate use investigational new drug application (IND) outside of the context of broader clinical trials. Eligible patients are limited to certain criteria, including that they have a serious or immediately life-threatening disease or condition.4

Under current regulations, the first step in obtaining an investigational drug for expanded access use is for the patient's treating physician to determine that the patient meets the eligibility criteria for expanded access, including that the "probable risk" to the patient from the investigational drug "is not greater than the probable risk from the disease or condition."5 Next, the physician must ask the drug manufacturer to provide the investigational drug for expanded access use.6 In order for the physician to proceed, the company developing the product in question must agree to provide the investigational drug and, typically, a limited right of reference to their IND. However, companies are under no legal obligation to support such INDs through provision of the drug or otherwise, and there are many reasons why companies may decline such access, including potential liability, cost, safety or other medical concerns about the particular proposed use, and other factors. If the company agrees, and the patient's physician is assuming responsibility for operating as both the sponsor and the investigator for the expanded access use,7 the physician must then submit the IND to FDA, which may include use of forms that FDA has recently streamlined to expedite expanded access requests.8 To grant access to an investigational treatment, FDA must find that there is no comparable or satisfactory alternative therapy, the patient cannot obtain the drug under another IND or protocol, the benefit to the patient justifies the potential risks of the treatment and those potential risks are not unreasonable, and providing the investigational drug will not interfere with clinical investigations that could support approval of the use or otherwise compromise development of the use.9 FDA authorizes more than 99% of the single-patient IND requests that it receives.10 If access is granted, then the physician must also obtain the patient's written informed consent, and obtain, at a minimum, concurrence by the institutional review board (IRB) chairperson or designated member prior to starting treatment.11

Path to RTT Legislation at the State and Federal Levels

As noted, to date, 40 states have passed RTT legislation, and RTT bills have been introduced in 9 additional states.12 Such legislation has largely been based on a model RTT law proposed by the Goldwater Institute, which purported to create a patient right to an alternative pathway bypassing FDA-expanded access requirements. However, because the Goldwater model law and subsequently enacted state RTT laws were widely thought to be at least impliedly preempted by the Federal Food, Drug, and Cosmetic Act (the FDCA), implementing a federal RTT law was viewed as instrumental to preserving an alternative, expedited pathway to investigational drug access for terminal patients. The FDCA, among other things, prohibits the introduction or distribution of an unapproved drug into interstate commerce.13 It also sets forth the framework that provides access to and authorizes distribution of investigational drugs.14 Therefore, state laws that circumvented FDA's authority by permitting distribution of an unapproved investigational drug from a manufacturer to a physician would directly conflict with the FDCA, and could be preempted due to the impossibility of complying with state and federal law.

At the federal level, RTT legislation faced considerable pushback by a variety of stakeholders as well as lawmakers in both chambers. Senator Ron Johnson (R-WI) led the push for federal RTT legislation, having...