Legislation signed this week by President Trump could pave the way for mesothelioma patients to access promising experimental treatments—even years before they are approved by the FDA. But not everyone thinks it is a good idea.
The “Right to Try” legislation gives patients with terminal illnesses like malignant mesothelioma the right to receive new treatments they would not normally be able to get because they haven’t gone through the FDA’s three-phase testing requirement.
Controversy Over New Legislation
Although the legislation could make it easier for some patients to access potentially life-saving mesothelioma treatments, it is also not without controversy. Some argue it could even be dangerous.
Typically, the FDA requires that all proposed mesothelioma therapies go through three-phases of studies or trials. In the first phase, the drug is tested in the laboratory and on animal subjects. In the second phase, the proposed mesothelioma treatment is tested in a small group of human patients to establish its safety and to determine the appropriate dose.
If the drug is determined to be safe and effective in the small group, it may then become the focus of a larger double-blind mesothelioma clinical trial. Most drugs that are rejected, fail in this second phase because of dose-limiting side effects.
But the “Right to Try” legislation would give mesothelioma patients and others who have run out of treatment options the opportunity to try new therapies even if they have only cleared the phase 1 hurdle.
While the legislation gives patients permission to try unapproved drugs, it does not require pharmaceutical companies to make them available. Some drug companies are reportedly nervous that granting access to drugs too early could open them up for lawsuits.
“Right to Try” versus Off Label
Although “Right to Try” is said to simplify drug access by eliminating the application process, many mesothelioma patients are already using investigational treatments through the FDA’s off-label, compassionate use, and orphan drug provisions.
“Off label” gives a doctor the opportunity to use an FDA-approved drug for a condition for which it has not been specifically labeled. Currently, pemetrexed (Alimta) is the only drug that is approved specifically for the treatment of malignant mesothelioma. All other drugs used in the treatment of the asbestos cancer are technically being used “off label”.
Under “compassionate use” (also called “expanded access”), certain patients diagnosed with mesothelioma and other serious illnesses can get access to drugs that are in clinical trials, even if the patient does not fit the criteria for the clinical trial. To recommend such a drug for a mesothelioma patient, a doctor must have a good reason to believe that the drug may help and that it can be administered safely.
“Orphan drugs” are drugs designed to treat rare diseases (like malignant mesothelioma) that have gone through the three-phase testing process, but have not yet submitted a marketing application.
The orphan drug designation can make it easier and faster for these medicines to make it into the hands of mesothelioma patients who need them. The VEGF-inhibitor nintedanib (Ofev) is an example of a drug that has been granted orphan drug status for malignant pleural mesothelioma.
“[Right-to-try] is really a law for people who are very sick, who have exhausted all treatment options and who cannot enroll in a clinical trial,” Starlee Coleman, senior policy adviser at the Phoenix-based conservative public policy think tank, the Goldwater Institute, told CNN.
Thirty-eight states already have ‘right to try’ laws in place but the federal legislation makes the policy applicable nationally.
Howard, Jacqueline, “What you need to know about right-to-try legislation”, Mary 20, 2018, CNN website
Expanded Access, FDA Website
Wendelbo, Morten and Callaghan, Timothy, “What is “right to try” and will it help terminally ill patients?”, May 30, 2018, The Conversation, CBS News website