By Mario Moretto
Maine could become one of a growing number of states to allow terminally ill patients the right to try experimental drugs before they're approved by federal regulators for public use.
A bill by state Rep. Thomas Longstaff, D-Waterville, would make Maine the latest state to pass such a "right-to-try" law. The move would make available for terminal patients any drug that had passed the first phase of the federal Food and Drug Administration's three-step drug approval process.
"The government shouldn't say, 'Well, it's not finally approved, so too bad. You're going to die,'" Longstaff, a former hospital chaplain who worked with some terminal patients, said in a recent interview.
Last year, five states -- Arizona, Colorado, Louisiana, Michigan and Missouri -- passed versions of the plan, known by advocates as the "right-to-choose" law. Maine is one of 26 states pursuing such a law this year, according to the Goldwater Institute, a libertarian group that supports the reform effort.
The FDA currently has a process for expanded access to experimental drugs, or "compassionate use," which gets experimental medicine into the hands of patients, effectively allowing them to participate in clinical trials from a distance.
The FDA approves nearly every request for compassionate use, but critics have said the process is too complicated and slow. Longstaff's right-to-try bill cuts the FDA out of the process entirely, allowing terminally ill patients to go to drug companies directly if their doctor recommends it.
Access to drugs in the early stages of testing may give patients options where previously there were none, but it's hard to say whether those drugs would stand much chance of improving the lives of the terminally ill.
The first phase of the FDA's testing process involves few subjects and is designed only to test dosage and toxicity. The drug's safety and effectiveness in fighting the symptoms or conditions it targets isn't tested until Phase 2.
Longstaff's bill is sponsored by a bipartisan collection of House and Senate lawmakers -- though it notably does not carry the stamp of approval from any of the four medical doctors in the Legislature. Gordon Smith, executive vice president of the Maine Medical Association, said his group has taken no stance on the bill but is sympathetic to its aim.
"If you've been told that you have a terminal illness and that nothing more can be done, but a clinical trial is going on somewhere in the country with promising results, who amongst us would want to deny you a chance to try that?" he said.
Smith said he has questions about whether the federal government would approve of such a law, given the FDA has exclusive jurisdiction over the testing and public availability of medical drugs.
Even if the FDA agrees to let states do what they please, other questions remain. Under Longstaff's proposal, pharmaceutical companies would not be obligated to provide drugs to patients who requested them, and it seems many might be unlikely to do so.
The Pharmaceutical Research and Manufacturers of America, the leading industry group for drug manufacturers, said last year that right-to-try laws are well-meaning but sidestepping the FDA is "not in the best interest of patients and public health, and is unlikely to achieve our shared goal of bringing innovative, safe, and effective medicine to patients as quickly as possible."
Even if the drug manufacturers opt not to hand out preliminary drugs, that doesn't mean the proposed law would be a failure, state Sen. Eric Brakey, R-Auburn, said. Brakey had proposed a similar bill, but opted to sign on as a co-sponsor to Longstaff's instead.
The whole point, he said, isn't to guarantee anything to patients. It's to allow them to make their decisions, with their doctor and with the drug companies, as long as all three are willing partners.
Patients should "know the risks" of unproven drugs, he said, and an informed consent requirement in the bill is meant to show that patients enter any new drug treatment program with full understanding.
"There's no guaranteed outcome, but the whole point is the choice," Brakey said. "The bill doesn't force patients, hospitals or pharmaceutical companies to do anything. All it does is get government out of the way to let a terminally ill patient try something when they've run out of options."
Brakey, the Senate chairman of the Health and Human Services Committee, said Longstaff's bill had not yet been scheduled for its first public hearing.
(c)2015 the Bangor Daily News