Patients with mesothelioma may soon find an easier path to experimental drugs and treatments after President Donald Trump signed the “Right to Try Act” into law last week.
The new legislation will give terminally ill patients a more direct and quicker line to drugs still awaiting approval from the U.S. Food and Drug Administration (FDA), bypassing the often-cumbersome application process.
The law also will protect doctors and pharmaceutical companies from the legal risks of allowing unapproved treatments.
The bill was passed by the Senate in August 2017 and cleared the House of Representatives late last month.
“I do like the idea behind this law, yes, if the title reflects what the actual law is,” Dr. Raja Flores, mesothelioma specialist and thoracic surgeon at Mount Sinai Hospital in New York City, told Asbestos.com. “I just need to read more about it in depth.”
Dr. Michele Carbone, a pathologist at the University of Hawaii Cancer Center and mesothelioma specialist, disagreed with the premise behind the new law.
“Terminally ill patients should not be used in place of experimental mice,” Carbone told Asbestos.com. “Mesothelioma patients are among those at risk of these new policies. The world is filled with stories of magic potions that never worked. I support a rigorous vetting of drugs by the FDA before distribution to the public.”
Patients Searching for Effective Treatments
Mesothelioma is a rare and aggressive cancer with no definitive cure.
It typically comes with a poor prognosis. Most patients live about one year after diagnosis. Fewer than 10 percent of patients survive five years or more.
Standard-of-care treatment can include a combination of surgery, radiation and chemotherapy.
The FDA has not approved any new treatments for mesothelioma since the chemotherapy drug pemetrexed (Alimta) in 2004, although several immunotherapy drugs are slowly working their way through the approval process.
There still is no FDA-approved second-line treatment for mesothelioma, often leaving doctors, patients and families feeling helpless and frustrated when first-line therapy fails.
The new law will allow patients and doctors to work directly with drug manufacturers without requiring FDA approval, taking the government agency out of the equation.
“The law puts these important personal decisions where they rightfully belong — into the hands of patients and their doctors,” Naomi Lopez Bauman, director of healthcare policy at the Goldwater Institute, which helped craft the legislation for Congress, told Asbestos.com. “It also provides hope for patients who have exhausted all other options.”
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Some Experimental Drugs Already Available
Some patients — including those with mesothelioma — already have access to experimental drugs seeking FDA approval through clinical trials or the “compassionate use” exception.
Clinical trials, though, are competitive and restrictive. Many patients fail to qualify under stringent eligibility requirements or proximity issues.
They may live too far away from a cancer center to participate. Patients also can be too sick, or not sick enough, to qualify for a clinical trial testing a new drug.
Some mesothelioma patients have qualified for experimental drugs under the FDA’s “compassionate use” provision, which was implemented in 2009. To qualify, the physician and each patient must complete an application, which is reviewed by the FDA before approval is granted.
According to the FDA, 99 percent of those applications are approved, although there have been only an estimated 1,500 applications submitted each year, covering a wide range of diseases and drugs.
“The FDA does approve almost all the applications, but it is time consuming for physicians, requiring patients to jump through bureaucratic hoops that don’t need to be there,” Lopez Bauman said. “It’s a hurdle the patient doesn’t need to face now.”
New Hope for Patients
Immunotherapy drugs nivolumab (Opdivo) and pembrolizumab (Keytruda) have been the two most popular, yet-to-be-approved medications obtained by mesothelioma patients, according to oncologists. Both have shown promise in extending survival times and have been approved for use with other types of cancer.
The “Right to Try Act” does require the drug to have passed through the first of three phases in the FDA approval process. Phase I includes testing for safety, side effects and proper dosage.
There had been significant opposition to the bill’s passage. Much like Carbone’s assessment, the American Cancer Society believes the law will give false hope to patients with unapproved drugs, while questioning the safety aspect.
Less Government Restrictions
Similar “Right to Try” laws, crafted by the Goldwater Institute, already have been implemented or passed in 40 states. The first was enacted in Colorado in 2014. The federal law, though, provides some uniformity.
FDA Commissioner Scott Gottlieb expressed his concerns before the law was passed, believing it was unnecessary legislation. But he also promised to support it, even though it reduced his agency’s authority.
“We stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients,” Gottlieb said in his statement.
Trump had been pushing for the bill’s passage since he spoke on the issue at his State of the Union address in January.
It was another step in his efforts to reduce federal government regulation.
“Patients with terminal conditions should have access to experimental treatments that could save their lives,” Trump said. “It’s time to give these wonderful Americans the ‘Right to Try.’”