Terminally ill patients in California will be able to try potentially life-saving medication before it passes FDA final review thanks to a new piece of legislation inspired by the movie "Dallas Buyers Club."
The bill, dubbed the "Right to Try" law, makes California the 32nd state to allow patients with terminal illnesses to try drugs that have passed the FDA's Phase 1, but haven't been fully approved.
Phase 1 is the first stage of drug testing in human patients. Drugmakers earn approval to conduct clinical trials on people after presenting the results of successful trials on animals, according to the FDA. After presenting the data -- and a plan for human trials -- the FDA determines whether drug companies can go forward with additional testing.
Patients can try experimental treatments only after exhausting all other options, according to the libertarian think tank Goldwater Institute, and the patients' treatments with Phase 1 drugs cannot be included as data in ongoing clinical trials.
California Gov. Jerry Brown signed the law on Sept. 27 after he vetoed a similar law last year, Reason notes. At the time, Brown said the state should take a wait-and-see approach with the FDA's own “compassionate use” program, which similarly allows terminally ill patients to try experimental drugs after exhausting other treatment options.
Under compassionate use, formally dubbed Expanded Access by the FDA, a patient must have the approval and full participation of his or her doctor, and the sponsor -- the company seeking FDA approval for a drug -- must submit a treatment plan for the patient.
The FDA also requires medical analysis that says "the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition."
Neither the Right to Try nor compassionate use programs require drugmakers to demonstrate efficacy, only that they prove that they won't do additional damage to patients in therapeutic doses.
Programs like Expanded Access and Right to Try laws are also called Dallas Buyers Club bills, after the 2013 movie starring Matthew McConaughey as a Texas AIDS patient who defies the FDA in the mid-1980s by smuggling experimental, unapproved HIV and AIDS medication into the country.
In a guest column for the Washington Post, 32-year-old Matthew Bellina says Right to Try laws are an improvement on the FDA's Expanded Access program because they stipulate that the FDA won't shut down or delay clinical trials if an experimental treatment goes wrong.
Because derailing clinical trials can deal a significant blow to drug companies that have poured millions of dollars into research and development, drug companies are less likely to sponsor terminally ill patients without guarantees that the FDA won't retaliate for failed treatment.
Bellina, a military veteran and father who has terminal ALS -- amyotrophic lateral sclerosis, also called Lou Gehrig's disease -- testified before the Senate on a federal version of Right to Try.
"The state laws allow terminal patients like me to work directly with their doctors and drug manufacturers to access promising treatments now being safely used in clinical trials," Bellina wrote. "The federal law under consideration would prevent the Federal Drug Administration or any other federal agency from blocking or interfering with the implementation of these state-passed laws."
Sen. Ron Johnson, a Wisconsin Republican, said a federal version of the legislation is necessary because it isn't easy for terminally ill patients to apply for a compassionate use exemption. Only 1,268 patients were permitted to participate in the program last year, according to FDA statistics.
"Without the protections of federal Right to Try legislation, tens of thousands of patients never even apply," Johnson said.