“Right to Try” Drug Measures Gain Traction Among States

Legislators in 10 states will introduce “Right to Try” bills in 2015 that give critically ill patients access to medications that have not been approved by the FDA. Kansas, Tennessee, Utah, and Wyoming have already filed bills or announced intentions to do so. These “Right to Try” laws have already been approved in five states: Arizona, Colorado, Louisiana, Missouri, and Michigan.

Although federal law and FDA regulations permit expanded access to investigational drugs for treatment use to individual patients, including in emergencies,  intermediate-size patient populations, and larger populations under a treatment protocol or treatment investigational new drug (IND) application, drug manufacturers, for various reasons (such as liability concerns or lack of supply) are not interested in supplying unapproved medications outside the supervision of the FDA. In a story by the New York Times, the FDA noted that it was not taking a position on these state’s laws and bills, encouraging patients to use the existing expanded access program.

These laws are drawing comparisons to the 2013 movie “Dallas Buyers Club,” in which an AIDS patient in the mid-1980s smuggles in unregulated pharmaceutical drugs from across the border. Advocates of these laws argue that the current FDA approval process for experimental treatments is too slow and cumbersome to get these treatments to critically ill patients. Families of critically ill patients are frustrated with the pace of drug approvals, especially as their loved ones suffer while clinical trials are ongoing. Conversely, critics of the “Right to Try” laws say these unproven treatments could hurt patients and the drug development process. The drugs are in early stages of testing or untested in humans and liability and safety issues are at the forefront of drug manufacturer’s concerns. As noted, drug manufacturers do not have to provide these treatments. Insurers are also not required to cover the drugs.

Access Through the FDA

Widespread access to an experimental drug may be provided under a treatment IND. Under the requirements for treatment INDs, an investigational drug that is intended to diagnose, monitor, or treat a serious or immediately life-threatening disease or condition may be supplied to patients if there is no comparable or satisfactory alternative drug or therapy for that stage of the disease in the intended patient population; the drug is the subject of completed or ongoing controlled clinical trials under an IND; and the sponsor is actively pursuing marketing approval with due diligence. Providing the drug to additional patients must not interfere with the enrollment of patients in any clinical trials that are still under way.

In the case of a serious disease, the drug must have sufficient evidence of safety and effectiveness for its intended use. To treat a life-threatening disease, where there is a likelihood of death within months or of premature death without treatment , there must be a reasonable basis from the whole of the available scientific evidence to conclude that the drug may be effective for its intended use and would not expose patients to an unreasonable and significant additional risk of injury. A licensed medical practitioner wishing to obtain an investigational drug for treatment use would first request the IND sponsor to submit a treatment protocol. If the sponsor refuses, the practitioner could submit a treatment IND to the FDA.

Exemption and Fast Track

The expanded access protocol is an exemption to current federal law requires that a drug be the subject of an approved marketing application before it is transported across state lines. Under the terms of the exemption, all human test subjects must be informed that the drug is being used for investigational purposes and the consent of the subjects or their representatives must be obtained, except when an investigator deems it not feasible or contrary to the best interest of the subject.

Besides requests from patients, a new drug’s sponsor can ask for the investigational drug to be designated as a “fast track product.” If the drug receives the designation (which is to be granted or denied within 60 days), actions to expedite the development and review of the drug application may be permitted. Review of a fast track application may begin before the application is complete, as long as the applicant pays applicable user fees and provides a schedule for submitting the remaining information. An application for a fast track product may be approved on the basis of its effect on a clinical endpoint or a surrogate endpoint that is reasonably likely to predict clinical benefit.

Bypassing the FDA

These new laws seek to bypass the FDA and permit access to unapproved drugs by curtailing or outright eliminating the FDA’s role. Under these state laws, typically once a physician and a patient determine that treatment is the right choice, and key to bypassing the FDA, that other options have been exhausted, the physician and patient are permitted to approach the drug manufacturer for permission.