If a ship was sinking, you would want the right to cling to anything that might save your life, even if not officially sanctioned by the government. If lifeboats were available but not Federally approved due to the possibility of leaking, should a person not be allowed access to that chance? Leftist government says no.
Opponents of the approach call it an ill-advised effort that circumvents federal law, undermines the drug development process and threatens to harm more people than it helps by providing access to medications that haven’t been proven safe and effective.
“The notion is based on the ‘Dallas Buyers Club’ — the idea that you have to get around the indifferent and cruel government to get access to drugs,” said Arthur Caplan, director of the bioethics division at New York University Langone Medical Center, referring to the Oscar-winning movie based on an AIDS patient who smuggled unapproved drugs into Texas during the 1980s.
The truth is that the system desperately needs reform.
For decades now the Food and Drug Administration has maintained an onerous and slow approval process that delays the debut of new drugs for fatal diseases, sometimes for years longer than the life span of the patients desperate to try them. Attorneys and scholars at the Goldwater Institute of Arizona have crafted legislation for the states that would allow terminally ill patients to try experimental drugs for cancer or degenerative neurological diseases earlier. These “Right to Try” bills are so scripted that they overcome the usual objection to delivery of such experimental drugs: safety. Under “Right to Try,” only drugs that have passed the crucial Phase 1 of FDA testing could be prescribed, thereby reducing the possibility of Thalidomide repeat. Second, only patients determined to have terminal cases would be eligible to purchase the drugs, making it harder to maintain that the drug will jeopardize their lives. Representatives in Colorado, Louisiana, and Missouri approved the “Right to Try” measure unanimously. Citizens of Arizona will vote on the effort to circumvent the FDA process this fall.
In cases of terminal illness, there is just no argument to ban access to potentially life saving medicine. This is not a case of the government protecting people from wasting time and money in unfounded “rhino horn” style scam cures. These are people trying to improve their terminal conditions with medicine that has been tested but continues to be tested for FDA approval, and in some cases approved in other countries.
Regulators and others in the pharmaceutical industry say sidestepping the later stages of testing exposes patients to unknown hazards. The first stage, usually involving only a few dozen subjects, is designed to learn whether a compound is safe for consumption. The laws would give patients medicines before rigorous second- and third-phase trials have established their safety and efficacy compared with placebos. Those later-stage studies help scientists determine proper dosages and understand how a drug affects sick patients who may be taking other medications. “These products have serious risks, and maybe even more concerning, we don’t even know what the risks are,” says Coleen Klasmeier, a former attorney for the FDA who represents drug companies at Sidley Austin. “You can’t just say, ‘Sure, take it. It might kill you, but vaya con Dios.’ ”
The new laws can’t compel pharmaceutical companies to make their newest drugs available before they’ve been vetted. It takes about 10 years and $1 billion to win FDA approval for a compound, and only 16 percent of treatments that begin clinical trials ultimately hit the market. Giving an experimental drug to terminally ill patients could tarnish the safety profile of a new drug out of the gate. “We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process,” Sascha Haverfield, vice president for scientific and regulatory affairs at the Pharmaceutical Research and Manufacturers of America, wrote in an e-mail.