(This story was updated with comment from the Pharmaceutical Research and Manufacturers of America at 5 p.m. ET on July 16, 2015.)
The "right to try” campaign that would allow terminally ill patients access to experimental, unapproved drugs is going national. Three House Republicans have introduced a bill that would ban federal regulators from taking action against those seeking access to such therapies.
The Right to Try Act of 2015 would prohibit the federal government from stopping or penalizing “the production, manufacture, distribution, prescribing, dispensing, possession, or use of” an experimental drug or medical device intended to treat a terminally ill patient.
The bill, introduced last week by Republican Reps. Matt Salmon and Paul Gosar, both of Arizona, and Marlin Stuzman, of Indiana, would apply only to the 22 states that have so far passed right-to-try laws. In those states, such laws have passed with overwhelming bipartisan support.
“In many cases, the right to try could mean the difference between life and death to otherwise terminal individuals; allowing them to take advantage of innovative, new options in hopes of saving or at least improving their quality of life,” Salmon said in a statement regarding the bill.
Under state right-to-try laws, a drug or device maker could provide therapies that have passed the Food and Drug Administration's Phase One clinical trials for approval, which according to the agency's website are conducted to determine potentially toxic effects and include less than 100 healthy subjects.
The state campaign to pass right-to-try laws has been led by the Goldwater Institute, a Phoenix-based, not-for-profit conservative research organization that drafted the state legislation.
“This common-sense law—a law protecting the terminally ill's right to work with their doctors to try to save their own lives—is literally sweeping the nation,” said Christina Sandefur, vice president for policy for the Goldwater Institute in an e-mail statement regarding the federal bill. “We will continue to work with federal policymakers to make it easier for terminally ill Americans to access promising investigational medications and remain focused on our goal of passing this law in all 50 states.”
Drugmakers and anyone involved in providing and administrating the treatments are shielded from lawsuits and disciplinary actions by state regulators under right-to-try laws. In many states, officials found to have blocked access to an eligible patient can be found in violation of a misdemeanor offense punishable by up to six months in jail.
Though the federal bill is intended to eliminate any potential conflicts between state laws and current federal regulations over the administering of investigational drugs, questions still remain as to the practicality of the laws in getting dying patients access to potentially lifesaving treatments.
Right-to-try laws do not mandate pharmaceutical companies to provide unapproved drugs and devices. Additionally, insurers are not required to cover either the costs for those treatments or any potential costs for healthcare services that may result from any adverse effects that occur from taking an unapproved treatment.
When asked about right-to-try laws, the Pharmaceutical Research and Manufacturers of America, the leading organization that represents the drug industry, expressed “serious concerns” back in November regarding any approach to make investigational medicines available that they said sought to “bypass the oversight of the Food and Drug Administration.”
In an updated response sent by e-mail on Thursday, PhRMA Vice President of Science and Regulatory Advocacy Sascha Haverfield acknowledged use of an experimental drug for a patient with a serious or life-threatening condition may be an option when they cannot participate in a clinical trial, but stressed that it should be done within the framework of the FDA's expanded-access program.
“Legislation at the state level, however well intentioned, is unlikely to add any meaningful new approaches that can optimize the federal expanded access process overseen by FDA,” Haverfield wrote. “All stakeholders—patients, physicians, biopharmaceutical companies, academia, and FDA—must come together to identify ways to improve the existing federal expanded access process and modernize the clinical trial, drug development and FDA review processes.”
Opponents of right-to-try laws point out that the FDA already has in place a process by which it provides terminally ill patients expanded access to investigational therapies on a case-by-case basis through its “compassionate” use program, which grants approval in about 99% of all cases.
But the law's supporters have argued that the FDA's compassionate use process is burdensome and lengthy. In February, the FDA issued draft guidance that introduced a new simpler, less time-consuming application form.
In a blog post on the agency's site, Dr. Peter Lurie, FDA associate commissioner for public health strategy and analysis wrote that the new application form would cut the time needed to complete an application down from the current 100 hours to just 45 minutes.
Others may contend that such laws, as well as the accompanying federal bill under consideration, are an attempt to bypass the FDA's authority. The agency has been criticized by some who say it has not taken steps to expedite and make less expensive the review process for new drugs and medical devices to come to market.
Such arguments have been behind the move for the comprehensive 21st Century Cures Act, which supporters say will speed the development of more breakthrough medications. That bill recently passed the House by a large majority and bipartisan support, but it is not clear at this point whether the Right to Try Act will be as successful.
