A new bill signed this month by President Trump may extend the options of terminally ill patients who want to use experimental types of drugs to treat their conditions. Traditionally, the FDA had various criteria governing the use of drugs that had not fully gone through the FDA approval process. The newly adopted law eliminates some of those requirements by essentially allowing an end run around some FDA protocols.
The new bill gives some patients the right to try new pharmaceutical products that have gone through initial round of clinical trials, but have not traveled the entire way through the FDA approval process. Reporting in the Washington Examiner clarifies that the first of three clinical trials looks at whether or not a new drug is safe to use, but does not evaluate its efficacy. With this in mind, the idea behind the bill seems to be that with the first clinical trial completed, patients can be reasonably sure that the FDA has pronounced the drug safe to use – but they can’t be sure that it will have any intended effect on their condition. Remaining concerns by critics of the bill center around whether patients are getting “false hope” in terms of treatment.
Existing FDA Processes
It’s important to note that the FDA already has some avenues in place for patients who want to try drugs that are not fully FDA approved.
The FDA website explains two of these processes, a protocol for investigational drug programs, and an expanded access program, which is also called compassionate use.
Compassionate use allows the use of investigational medical products outside of the clinical trial environment, but it does come with significant eligibility requirements.
First, the FDA specifies that the patient must have a licensed physician who is willing to use the product for treatment. The physician has to make sure there’s no other alternative treatment that’s comparable, and that the benefits of the treatment outweigh the risks.
The FDA also requires that any of these investigative uses not interfere with the product’s actual clinical approval.
The new bill, on the other hand, would give some patients the ability to go around the FDA approval process, so that some of these eligibility criteria may not be applicable. However, the law does not require drug makers to provide investigative drugs to patients – that’s something that will often get hashed out with insurance companies and other involved parties.
Protecting the Drug Makers
Although the majority of U.S. states already have their own “right-to-try” drug laws, the federal law takes the more open-ended approach to the next level. As such, proponents of the bill point out that with this new rule in place, there are provisions where the FDA can exclude from consideration severe side effects and deaths that happen outside of the clinical trial environment. In other words, in some senses, the patient is taking the investigative drugs at their own risk.
“Libertarian quackery or lifesaving hope?” asks the Big Think, reporting on how proponents of the bill suggest that dying patients don’t have time to comply with FDA approval rules.
Others like Frank Pallone, Jr. (New Jersey) ranking member of the House energy and commerce committee argues that existing FDA oversight helps protect patients. “(Eliminating FDA oversight) would provide fly-by-night physicians and clinics the opportunity to peddle false hope and ineffective drugs to desperate patients,” Pallone said according to Chicago Tribune coverage.
Concerns About Changes
The article also shows that hundreds of doctors have questioned right-to-try legislation on the basis that the FDA already has the compassionate use alternative on the books. The FDA Commissioner, Scott Gottlieb, has also expressed concerns about instituting a right-to-try bill on a federal level.
“This legislation sells vulnerable patients and families false hope at the expense of weakening the FDA’s critical role in making sure that all Americans can have confidence in the safety and effectiveness of our medical products,” reads a draft letter sent by physicians to the administration. “Patients with terminal conditions who access unapproved therapies outside of clinical trials may be at risk of hastened death or reduced quality of the life that they have left, and deserve protections similar to patients taking part in clinical trials.”
At its bottom, some of the debate essentially appears to be revolving around the role of the FDA – whether or not the FDA is an effective regulator, and whether its regulations are needed everywhere in the marketplace. However, as an incremental step in expanding patient freedoms, many would argue that it makes sense to empower the terminally ill over other classes of patients who may not need experimental treatments to the same extent.
As for the White House, Trump has been vocal about his support of the bill, along with Vice President Mike Pence, who put a similar bill into law in the state of Indiana.
Weighing in, FDA Commissioner, Dr. Gottlieb, indicated he feels that the FDA will still be involved in vetting requests to try experimental drugs, regardless of the new law.
“The compassionate use program will sit alongside ‘Right To Try,’ ” Gottlieb said at the annual meeting of the American Society of Clinical Oncology in Chicago, according to the Wall Street Journal. “Nothing’s going to change about our current approach.”
In some ways, debate over the bill has become a bellwether for talking about how regulations work in the U.S. Some officials have called for decreasing the power of the FDA, while others feel that’s a bad idea. The drug law will likely spur more back and forth about the role of the FDA and other federal regulators, in an environment where public policy is often under the microscope of public opinion.