On May 30, President Trump signed into law the bipartisan “Right to Try Act” which offers people with advanced and terminal illnesses who have failed all available treatments to have access to experimental drugs that might help if the drugs have passed Phase 1 FDA trials. 40 states have enacted similar laws. The federal law expands access to those people living in the 10 states that haven’t passed such laws, but does not add much else that’s new.
The Act has been hailed by advocates as a giant step in support of patient choice, but it is not such a simple issue as "bureaucrats were preventing sick people from getting treatments that can help them”- as some would imply.
Let's start with the fact that the FDA already has an expanded access ("compassionate use”) policy that allows access to drugs that have passed phase 1 trials for terminally ill patients who have no other treatment choices left.
USA Today reported (March 31, 2018), that "of the nearly 5,800 requests the FDA received from 2012 through 2015, the agency approved 99 percent of them, according to the Government Accountability Office. The most common reasons requests were denied included incomplete applications, safety concerns, the availability of adequate alternative therapies and evidence that the drug was ineffective for the intended use, according to the GAO." These requests can be approved over the phone or in some cases in a few days. Unfortunately, this was not the case, for instance, 30 years ago when AZT, then experimental, wasn’t available to patients with HIV infections and AIDS.
The Act and the state laws that it mimics, while appearing to be a reasonable approach that allow dying patients access to drugs that offer a chance of benefit, raise several causes for concern - the first of which is that they open the door for quacks and fly-by-night clinics that abuse patients by offering false hope of inflated results and miracle cures.
Phase 1 (safety and dosage) studies are very early in the drug development process. According to the FDA website (www.fda.gov) Phase 1 studies enroll 20-100 participants, who may be healthy volunteers, except that if it’s a cancer treatment drug the participants will have that type of cancer. The FDA explains that “researchers adjust dosing schemes based on animal data to find out how much of a drug the body can tolerate and what its acute side effects are.” There is no useful data on efficacy at this stage. 30% of drugs fail to get past Phase 1. Phase 2 studies enroll “up to several hundred people with the condition" but the FDA warns "these studies aren't large enough to show whether the drug will be beneficial.” 33% make it to Phase 3 studies of "efficacy and monitoring for adverse reactions". These larger studies are designed "to demonstrate whether or not a product offers a treatment benefit to a specific population.” Only 25-30% of drugs that start the process are found to be safe and effective, pass Phase 3, and are released to the market. Large (several thousand participant) post-marketing Phase 4 studies continue to monitor for adverse drug reactions.
On January 22, 2018 the Los Angeles Times reported in Right-to-try laws are hazardous to your health…: “As…reported...in the Journal of the American Medical Association, 'most of the drugs that succeed in phase 1 trials turn out to be too unsafe or ineffective for clinical use.’"
Information about these drugs in early stages of development is not readily available to most physicians, who are under no obligation to prescribe an experimental drug even if they know about it. The law doesn't require a drug company to provide the drug and it doesn't limit the price, which could be exorbitant (likely more than the exorbitant prices of some drugs when they do reach the market). The cost is unlikely to be paid by insurance and certainly not by Medicare, which doesn't pay for experimental treatments that haven't been approved by the FDA. Moreover, insurers and Medicare won’t pay for treatment of complications of unapproved therapies, leaving patients exposed to unlimited costs if something goes wrong. And most of the state laws, which are still on the books, rescind eligibility for hospice if the patient is receiving active therapy, including these unapproved drugs.
There is always the risk, since the drug hasn't gone through the full testing process, that it will fail to work, increase pain and suffering, and/or shorten life. The FDA approval process is designed to detect these risks and protect the public by preventing dangerous and ineffective drugs from reaching the market. The pharmaceutical company may indeed drop development of the drug based on studies showing lack of effectiveness or harm and may never bring it to market, leaving those who used it under the Right to Try Act in the lurch, having paid dearly for a useless or dangerous treatment.
There is a political agenda at work here that must be acknowledged - an anti-regulatory philosophy that would weaken FDA oversight of drug development even though that system has generally worked well and has been modernized and streamlined in recent years, including the expanded access program mentioned above.
On a positive note, the law holds physicians and pharmaceutical companies harmless if there are adverse effects from prescribing or providing an unapproved drug and since the drugs is being used in the most advanced cases, negative outcomes won’t be used to delay or block FDA approval.
So while I can see that very sick people who have exhausted all other avenues of treatment are anxious to find something else that can help, I’m not so sure this approach is the silver bullet. That being said, if I were in this position with a terminal illness and all other treatment options had been tried and failed, I would probably try a Phase 1 experimental drug myself figuring that I had nothing to lose.