Terminally-ill patients in Colorado, Louisiana, Missouri and Michigan have had access to experimental drugs not yet approved for use by the Food and Drug Administration since May, and patients in Arizona could join them if voters approve a ballot initiative Tuesday.
The laws are intended to give health providers and drug companies protection from liability if they provide a trial drug to someone whose illness is diagnosed as imminently fatal. The drug only needs to have completed the first phase of testing, which shows it is not toxic when given to humans at a particular dose. That level of testing, however, is not intended to prove safety or show effectiveness against a disease.
Proponents claim the laws are needed because the FDA’s existing “expanded access’’ system that allows critically-ill patients who are not part of clinical trials to get test drugs is too complex and too slow for someone with little time to live.
But it’s unclear just how effective the state codes will be in speeding along treatments. Although supporters say they’re aware of patients in the process of gaining access to drugs, none could cite anyone who had actually started treatment due to one of the laws.
``We don’t know anyone who has taken advantage of it yet,’’ said Victor Riches, vice president of external affairs for the free-market-oriented Goldwater Institute, a Phoenix think tank and advocacy group named for the late Sen. Barry Goldwater.
“I did talk to a doctor from Louisiana recently who seems to be getting close to being able to get a drug for a patient. I suspect as more people become aware of these laws, more will try to take advantage of them.”
The organization, along with some patient advocates, has been leading the effort to get “right-to-try’’ approved, including donating $70,000 to Your Right To Try, an Arizona committee set up to support approval of the referendum. Riches said the institute will work for passage of legislation in other states next year, including Florida and Texas.
No polling has been reported since the “right-to-try” initiative was added to the Arizona ballot, but a survey done last winter by the research firm Wilson, Perkins, Allen found that 79 percent of voters supported the potential ballot measure.
Regulators and experts in the drug industry note that the laws don’t guarantee or require firms to give terminally ill patients access to a trial drug, nor do they address some key concerns about sharing.
The FDA says it has no position on any state’s right-to-try bill, but says it is “strongly supportive of appropriate use of expanded access protocols and stands ready to work with companies that are interested in providing access to experimental drugs.”
The agency also notes that even if it permits use of an experimental therapy, it’s up to the company sponsoring a clinical trial to decide whether or not to provide the therapy to patients.
Riches said doctors seeking to go through the FDA’s current expanded use program for a patient face paperwork that takes more than 100 hours, and a review by an institutional oversight board that can take weeks or months to complete. “It can be 4 or 5 months to get the medicine, and for the terminally ill, that’s not going to work.’’
Dr. David Gorski, a surgical oncologist and editor/contributor to the blog Science Base Medicine, has been sharply critical of the laws, writing: “The entire justification for ‘right to try’ laws also seems to rest on a misperception that there are ‘miracle drugs’ out there that we will have to wait years for because the FDA is too slow to approve them.”
“Indeed, making essentially untested drugs more widely available, as these ‘right to try’ laws propose to do, will be far more likely to harm the individual patient than to help him,’’ he added.
Drug and therapy companies invest an average of 10 years and $1 billion to bring a new drug to market, industry officials say. “The whole reason they go through this extensive process to FDA approval is that it’s the best way to help the largest population of patients who need the drug,” said John Murphy, assistant general counsel for the Pharmaceutical Manufacturers Association.
Murphy said that while the association has no formal position on the state bills, it has attempted to educate lawmakers and others about the existing extended use program and how it acts as an extension of drug trials for individual patients. Having a two-track system for access to experimental drugs could make it harder in some cases to get enough patients to enroll in clinical trials.
“There should be concerns about any company that’s willing to bypass the FDA approval process,’’ Murphy said.
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