Since last May, five states have passed Right to Try legislation. These new laws allow critically ill patients the right to try medications that have not yet been approved by the Food and Drug Administration (FDA). In Colorado, Michigan, Missouri, Louisiana, and Arizona, patients now can gain access to medications that are still in testing if all other medical options have been exhausted. Ten other states plan to introduce similar bills this year. Will these laws help terminally ill patients? Or will they weaken the drug development process and ultimately cause more harm than good?
Right to Try laws allow terminally ill patients who have exhausted all other options, including clinical trials, to obtain medications as long as the drugs have passed at least the first of three FDA investigation phases. But that’s where these laws stop. They do not require pharmaceutical companies to provide the drugs. Many companies may be unwilling to dispense unapproved medications for a number of reasons that include a lack of adequate supply and exposure to liability. Under these laws, health insurers are not required to cover the cost of the drugs, and they can deny coverage to patients who use investigational drugs.
The laws have been nicknamed the Dallas Buyer Club laws after the story portrayed in the movie about an AIDS patient who smuggled unapproved pharmaceutical drugs into the U.S. in the 1980s and distributed them to others who were also HIV-infected while fending off opposition from the FDA.
The Goldwater Institute, a libertarian group, is the political force behind efforts to pass Right to Try legislation. The institute’s claim is that too many terminally ill patients are blocked by bureaucratic red tape from accessing experimental drugs that could help them. Right to Try laws would cut through the red tape by circumventing the federal government entirely. Under Right to Try legislation, patients could obtain approval from a physician and then approach the appropriate drug company to access an investigational drug.
Opponents of these laws believe that they are intended to weaken the FDA’s authority and its drug development structure and fear that bypassing safety procedures established by the FDA could weaken the agency, thereby jeopardizing public health and setting the stage for a protracted legal battle over who has the authority to decide whether a drug is safe or not.
Right to Try laws don’t seem to have helped anyone gain access to any investigational drugs so far, largely because drug companies are not open to the idea of sidestepping the FDA and supplying unapproved medications. Several pharmaceutical companies have said they will continue to ask patients to go through the FDA despite the legislation.
In fact, the FDA does have a process in place for “compassionate use” exemptions to help terminally ill patients gain access to yet-to-be approved treatments. The FDA allows patients who have serious or life-threatening illnesses and who do not have an option for clinical trials to access investigational drugs when no comparable alternatives exist. Compassionate use—also known as the Expanded Access Program—was created in 1987 to respond to requests from AIDS patients who wanted to try treatments that were still under investigation. Since then, the FDA has processed requests and granted permission for about 1,000 people to use unapproved treatments each year. Even so, the FDA cannot force a drug company to provide the drugs. Some patients and their family members have attacked the FDA’s program, claiming that the agency’s process takes too long when every minute counts for a terminally ill patient. The FDA has not taken a position on Right to Try legislation but has stated its concern about any efforts to weaken the “congressionally mandated authority and agency mission to protect the public from therapies that are not safe and effective.”