Since his days on the campaign trail, President Trump has not minced words about an aggressive agenda to cut regulations “at a level no one has ever seen before.” Paramount on his list of deregulatory targets is the Food and Drug Administration (FDA), an agency whose rules he has vowed to slash by 75-80 percent. So, it hardly comes as a surprise that the Trump Administration would support new legislation that interferes with the FDA’s ability to perform oversight. However, the Trickett Wendler Right to Try Act of 2017 (S.204), which the administration has recently endorsed, not only undermines the FDA, but opens the floodgates for a host of unintended consequences that ultimately place patients and consumers at risk.
Right-to-try laws have been enacted in 33 states, including Vice President Pence’s state of Indiana, where he championed the law as governor. These measures are designed to grant terminally ill patients the “right to try” an unapproved drug or medical device by allowing patients and healthcare providers to work directly with drug companies and circumvent the FDA altogether. Proponents allege that FDA red tape restricts access to experimental drugs and right-to-try laws help patients get “immediate” access to treatments they need. The reality is current state right-to try laws have done little to widen patients’ latitude to try unapproved drugs, and federal legislation would do little to strengthen state laws. What federal legislation WILL do is create a lax legal and regulatory environment for industry, while compromising patient safety.
The FDA’s expanded access program was put into place more than two decades ago for the specific purpose of granting compassionate use of experimental treatments to terminally ill patients for whom there is no alternative treatment and, for various reasons, are not able to participate in a clinical trial. To participate in this program, a patient’s physician must first reach out to a drug company to receive approval to use the treatment. The drug company is not required to provide the treatment, and the FDA cannot compel it to do so. However, if the company does indeed agree to provide it, the physician would then request approval from the FDA and—almost always—that approval is granted. Criticism that the FDA is somehow holding experimental treatments hostage from patients who desperately need them is baseless; in fact, the FDA has authorized more than 99 percent of the requests it has received for expanded access to investigational products between 2010-2015.
The expanded access program achieves the right balance between facilitating patient access to unapproved drugs and providing appropriate protections for patients, without undermining the clinical trials process. Though patients who are terminally ill are often more willing to try experimental treatments, the FDA’s role in protecting these patients from quack treatments, and unnecessary physical and financial risk is essential. Therefore, with every request FDA receives, the agency ensures that the potential benefit of the treatment justifies the potential risk, and the potential risks are not unreasonable in the context of the patient’s disease. The FDA also prohibits drug companies from charging patients more than the manufacturing costs for an unapproved drug, and in most cases, the company doesn’t charge the patient anything at all.
Right to try legislation addresses none of these issues. No risk analysis or evidence of a drug’s efficacy is required under the state or proposed federal laws, which makes patients significantly more vulnerable to horrible side effects or earlier death. Also, without the FDA’s cost regulation, companies might take the opportunity to profit at the expense of the patient. The legislation also does not address drug company participation. While the FDA cannot compel a company to provide a treatment, proposed state or federal right to try laws do not compel companies to do so either. Companies may be even less likely to provide an unapproved drug if demand increases, due to potentially burdensome costs or bad publicity due to poor health outcomes. Also, should a company provide an unapproved treatment to a patient, the proposed federal law includes a provision that would block the FDA from using information about unintended side effects against the company if/when the drug is submitted for approval.
Perhaps one of the direst consequences of S.204 is that it compromises the clinical trials process. If patients can simply request a drug from the company with no further oversight, they may be less likely to view clinical trials as a primary option, and enrollment could significantly decrease. Robust clinical trials are the most critical mechanism for determining whether a drug is safe and effective and minimal participation could slow or stop the process that ultimately brings new drugs to market for the public at large.
Since its founding more than 100 years ago, the National Consumers League has worked alongside regulatory agencies, such as the FDA, to ensure the goods and services consumers receive are safe and reliable. While we are all committed to ensuring new treatments and therapies are available to patients, we must not sacrifice safety in that pursuit. Right to try legislation is not only ineffective, but it also creates a dangerous environment with no checks and balances for patients and families who are already vulnerable. Rather than focusing on this harmful and ethically questionable legislation, the efforts of Congress would be better spent on finding real solutions to improve health care delivery and the drug development process, such as reducing the generic drug backlog and safeguarding the Affordable Care Act. The FDA’s importance in protecting public health cannot be understated, and we must be careful not to undermine the regulations that have made it the global gold standard.