Last May, Gov. John Hickenlooper (D) signed into law the “Right to Try Act” (Colorado House Bill 14–1281), making Colorado the first state to give terminally ill patients access to drugs that have not been approved for patient use by the U.S. Food and Drug Administration. A month later, Gov. Bobby Jindal (R) signed similar legislation in Louisiana. Another month later, Gov. Jay Nixon (D) signed a “Right to Try” bill in Missouri. In November, Arizona voters will consider a ballot initiative that would likewise give terminally ill patients access to non-FDA-approved investigational drugs. The Goldwater Institute, a libertarian-leaning nonprofit organization, has drafted model right-to-try legislation and purportedly has met with lawmakers in Florida, Massachusetts, Michigan, Oklahoma, Texas, and Utah who are interested in introducing such bills in their legislatures.

Federal access / For compassionate, humanitarian reasons alone, who would not support granting access to potentially life-saving drugs to hundreds of thousands of terminally ill patients? On the face of it, virtually no one. Yet, at the federal level, efforts to pass similar legislation have met with little success. Rep. Diane Watson (D‑Calif.) most recently introduced H.R. 4732 (“Compassionate Access Act of 2010”) in the 111th Congress, only to see it flounder in the House Subcommittee on Health.

There are some existing federal regulatory options available for the terminally ill who want to try potentially life-saving, non-FDA-approved drugs. The FDA administratively allows expanded or “compassionate” use (on a case-by-case basis) of an investigational drug (“one not having been approved by the FDA as safe and effective”) that has cleared Phase I trials (involving early human testing with small pools of test subjects, usually 20–80 volunteers). The drugs can only be used to treat a patient with a serious or immediately life-threatening disease or condition provided that the patient has no comparable or satisfactory alternative treatment option.

If the patient is to take advantage of this allowance, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. As noted by the FDA, not all physicians are willing to manage the use of an investigational drug for patients in their care, nor are companies required to make their drug available through expanded access, or to manufacture a drug for “expanded use.” Moreover, while some pharmaceutical companies provide the drug at no cost to the patient, others will charge costs associated with the drug’s manufacture, and there can be additional expenses associated with administration and monitoring of the drug by health care professionals.

In the years 2010–2012, the FDA received between 950 and 1,200 annual requests through the expanded access programs to use non-approved drugs. The agency granted nearly all of those requests.

State action / For supporters of right-to-try, when their federal political lobbying strategy met resistance, they moved to a state political strategy. So what does the passage of state-level right-to-try legislation change for terminally ill patients? According to the Goldwater Institute (Policy Report no. 266, February 11, 2014):

For patients suffering from terminal illnesses, the FDA is the arbiter of life and death. These patients, suffering from diseases ranging from ALS to Zellweger Syndrome, face little chance of recovery. … [I]nvestigational medicines provide a glimmer of hope. The FDA, however, often stands between the patients and the treatments that may alleviate their symptoms or provide a cure. To access these treatments, patients must either go through a lengthy FDA exemption process or wait for the treatments to receive FDA approval, which can take a decade or more and cost hundreds of millions of dollars. Sadly, over a half a million cancer patients and thousands of patients with other terminal illnesses die each year as the bureaucratic wheels at the FDA slowly turn.

Ostensibly, the purpose of right-to-try legislation is to eliminate the bureaucratic federal process that hinders time-sensitive access to potentially life-saving drugs for terminally ill patients. Yet this administrative barrier is not necessarily removed by the passage of state legislation. Under the Interstate Commerce Clause of the U.S. Constitution, the FDA would be able to strongly (and convincingly) argue that state governments were in violation of this fundamental clause, thus striking down the state statute’s authority. There is recent legal precedent for this argument. On August 7, 2007, in Abigail Alliance v. von Eschenbach, the U.S. Court of Appeals for the District of Columbia ruled in an 8–2 decision against the Abigail Alliance for Better Access to Developmental Drugs, reversing a 2006 Court of Appeals ruling and upholding a previous court decision that found no constitutional right to unapproved drugs by terminally ill patients. In early 2008, the U.S. Supreme Court declined to hear an appeal of the 2007 decision.

The Pharmaceutical Research and Manufacturers of America, the industry’s trade group, has not been supportive of state efforts to usurp federal regulation of drug development protocols, arguing that the group has “serious concerns with any approach to provide patient access to investigational medicines that seeks to bypass the oversight of the [FDA] and clinical trial process, which is not in the best interests of patients and public health.” Under the FDA’s “expanded use” program, pharmaceutical companies have expressed concern that the agency would order additional testing of a drug candidate if unusual side effects are observed outside of trials. There is also industry concern about patients suing companies when such experimental drugs are proven unsafe. The variation in state laws may not adequately shield companies from tortuous action, even if companies require patients to waive their rights to sue. In spite of these regulatory concerns, some pharmaceutical companies have been willing to provide non-FDA-approved drugs outside of trials to patients under expanded access protocols.

Limits of state action / With state-level right-to-try legislation eliminating the need for the FDA approval process, will this encourage pharmaceutical companies to expand access to their experimental drugs? Not likely. When the “floodgates” open with no FDA “gatekeeper” available to manage patient requests, this will create an untenable ethical and operational dilemma for many research-based pharmaceutical and biotechnology companies.

State right-to-try legislation is a public policy attempt to help people who are desperately in need of help. Unfortunately, the legislation does not require that pharmaceutical manufacturers provide their investigational drugs to terminally ill patients, nor are physicians required to manage such an investigational drug protocol for their patients. Moreover, health care insurers (private or public) are not required to pay for the costs of such investigational drug applications for their terminally ill patients. While the FDA has not publicly addressed the issue of enactment of state right-to-try legislation, the recent wage of state actions could provide the stimulus for an FDA legal challenge at the federal level.

So, effectively, little has changed for terminally ill patients. What the state legislative efforts may accomplish is to reenergize this issue with the American public. Ultimately, the goal of “Right to Try” supporters is to secure the passage of similar legislation at the federal level and influence the pharmaceutical industry, the FDA, physicians, and health care insurers to cooperatively develop a workable, timely solution for their terminally ill family members and friends. It is unclear whether the state efforts will achieve that goal, regardless of noble intentions.