	Introduction

The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try (RTT) Act of 2017 became federal law on May 30, 2018. Over the preceding five years, 40 states had enacted related legislation. The goal was to allow individuals with imminently life-threatening diseases or conditions to seek access to investigational drugs without the step of procuring permission from the Food and Drug Administration (FDA). Another goal—held by the Goldwater Institute, which led the initiative toward state bills, and some of the legislative proponents—was focused more on the process: to eliminate government's role in an individual's choice. 115-176 ). FDA-Related Issues

			Difficult Process to Request FDA Permission

Have FDA's procedures discouraged patients and their physicians from seeking treatment INDs? 114-255 ) and the FDA Reauthorization Act of 2017 (Section 610, P.L. It also required GAO to report to Congress on individual access to investigational drugs through FDA's expanded access program. These provisions
define an eligible patient as one who (1) has been diagnosed with a life-threatening disease or condition, (2) has exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (as certified by a physician who meets specified criteria), and (3) has given written informed consent regarding the drug to the treating physician; define an eligible investigational drug as an investigational drug (1) for which a Phase 1 clinical trial has been completed, (2) that FDA has not approved or licensed for sale in the United States for any use, (3) that is the subject of a new drug application pending FDA decision or is the subject of an active investigational new drug application being studied for safety and effectiveness in a clinical trial, and (4) for which the manufacturer has not discontinued active development or production and which the FDA has not placed on clinical hold; and exempt use under this section from parts of the FFDCA sections regarding misbranding, certain labeling and directions for use, drug approval, and investigational new drugs regulations;
The new FFDCA Section 561B has provisions that had not been necessary when access had been granted under FDA auspices. It states that, related to use of a drug under the new FFDCA Section 561B,
"no liability in a cause of action shall lie against ... a sponsor or manufacturer; or ... a prescriber, dispenser, or other individual entity ... unless the relevant conduct constitutes reckless or willful misconduct, gross negligence, or an intentional tort under any applicable State law"; and no liability, also, for a "determination not to provide access to an eligible investigational drug." This report discusses several provisions in the RTT Act that Congress could consider as it oversees the law's implementation. Those are the patients who would get access through the RTT pathway. The RTT Act, however, includes an exception. The no-liability provision in the RTT Act seems to remove that obstacle. It could answer three questions at the core of measuring its effect on FDA, drug manufacturers, and patients. First: Will more patients get investigational drugs? Third: How will this affect FDA? Writing in opposition to the bill, four former FDA commissioners warned that it would "create a dangerous precedent that would erode protections for vulnerable patients."
The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try (RTT) Act of 2017 became federal law on May 30, 2018. Over the preceding five years, 40 states had enacted related legislation. The goal was to allow individuals with imminently life-threatening diseases or conditions to seek access to investigational drugs without the step of procuring permission from the Food and Drug Administration (FDA). Another goal—held by the Goldwater Institute, which led the initiative toward state bills, and some of the legislative proponents—was focused more on the process: to eliminate government's role in an individual's choice.
The RTT Act (P.L. 115-176) offers eligible individuals and their physicians a pathway other than FDA's expanded access procedures to acquiring investigational drugs. It defines an eligible patient as one who (1) has been diagnosed with a life-threatening disease or condition, (2) has exhausted approved treatment options and is unable to participate in a clinical trial involving the eligible investigational drug (as certified by a physician who meets specified criteria), and (3) has given written informed consent regarding the drug to the treating physician.
It defines an eligible investigational drug as an investigational drug (1) for which a Phase 1 clinical trial has been completed, (2) that FDA has not approved or licensed for sale in the United States for any use, (3) that is the subject of a new drug application pending FDA decision or is the subject of an active investigational new drug application being studied for safety and effectiveness in a clinical trial, and (4) for which the manufacturer has not discontinued active development or production and which the FDA has not placed on clinical hold.
The RTT Act also has provisions that limit how the Secretary of Health and Human Services (through the FDA) can use data regarding clinical outcomes of patients who get these drugs through the RTT pathway; require drug sponsors (usually the manufacturers) to report annually to the Secretary on use of the pathway; and require the Secretary to post certain annual summaries. Finally, the RTT Act states that the sponsor or manufacturer has "no liability" for actions under the RTT provisions. The no-liability provision applies also to a prescriber, dispenser, or "other individual entity" unless there is "reckless or willful misconduct, gross negligence, or an intentional tort."
Before the RTT Act, observers discussed several obstacles to access to investigational drugs. These included some that were FDA-related: the difficult process to request FDA permission and the role of FDA as gatekeeper. Some related to why a manufacturer might decline to provide an investigational drug: limited available supply, liability, limited staff and facility resources, and concerns about use of outcomes data. The RTT directly eliminates some of these concerns, addresses some others, and leaves others alone.
Future Congresses could look at the RTT Act's effect on FDA, drug manufacturers, and terminally ill patients. Will more patients get investigational drugs? Congress could look at whether the law sufficiently removed obstacles to access. And how will the changes affect FDA? Four former FDA commissioners warned that the bill would "create a dangerous precedent that would erode protections for vulnerable patients." The first clue may come from how the current commissioner interprets FDA's role in the implementation of the new law.