June 18, 2018
Confusion expected in wake of 'Right to Try' bill's signing
Last month, Donald Trump signed the Right to Try bill ostensibly meant to allow terminally ill patients the opportunity to take experimental medications not yet approved by the FDA. Though on its face the bill could potentially give hope to those who thought they were out of options, many stakeholders, including doctors and pharma companies, think the new legislation will succeed only in sewing unnecessary confusion.
That's partially because similar legislation already exists in various forms by slightly different names. According to Martina Rozumberkova, the Director for Compliance, Forensics and Intelligence at the risk consulting firm Control Risks, comparable laws already exist at the state level across 38 states, with each state, of course, in control of its own regulations. In addition to that, the FDA already has a separate pathway, called expanded access, that expedites experimental drug treatments for the terminally ill.
While it's too early to say what changes pharma companies will have to make in order to comply with the new legislation, Rozumberkova told MM&M that at the very least, reports will have to be filed to the FDA whenever a patient receives an unapproved drug, in addition to any adverse effects it may cause.
“From my perspective, as someone who is interested in pharmaceutical compliance, there may be some additional reporting or requirements from the companies that will be providing the drugs based on this law,” she said. “What they will be exactly, it's hard to say at this point.”
Alison Bateman-House, an assistant professor in the Division of Medical Ethics at New York University Langone Medical Center, said that the first thing drugmakers will need to do is figure out the new law's implications. In general, the law will create a new mechanism for patients to use investigational drugs outside of clinical trials. But how exactly that will manifest in reality won't be know for several more months.
“There's a process by which a law is turned into rules,” she told MM&M. “And that needs to happen before we know exactly what the law's implications are.”
Bateman-House points out that the 21st Century Cures Act, which was signed into law at the end of 2016, requires companies to make their experimental drug policies available to the public, typically through their website. Now, pharma companies have to figure out where and how Right to Try fits into that.
“People are trying to figure out, does that mean I need to change my website to specifically mention ‘here's how we handle expanded access, here's how we handle right to try,' or do you not need to have that? Can you just have a more vague statement of ‘here's how we handle requests for access?' she said. “People are just trying to figure out what the actual legal implications are.”
One thing that both experts agreed pharma companies won't have to adjust, given the new legislation, is advertising. “Companies are not allowed to market investigational products, and this is solely of a path of access for investigational products,” said Bateman-House.
Rozumberkova agreed, saying that since the law applies specifically to drugs that are not on the market, there is no basis for advertising them. “They're just at the first stage of the testing, so they have a long way to go,” she said. “The patients are just getting them earlier.”
In terms of whether Right to Try will actually benefit anyone, Bateman-House is skeptical, particularly because it's entirely up to drugmakers whether they want to make their products available on an experimental basis. And if they hadn't done it under expanded access, it seems unlikely they will suddenly become interested in doing it under the new law.
“I really don't think that this is going to have much of an impact on patient access to drugs,” she concluded. “I think this is just going to be more of a headache for companies.”