Trump Again Urges Right-to-Try Passage
President Donald Trump again has urged Congress to pass “right-to-try” legislation to allow patients with terminal conditions to have access to unapproved experimental treatments. Speaking at a 2/1 Republican retreat in West Virginia, Trump echoed remarks he made during his State of the Union address 1/30 (see story). Legislation passed the Senate last year and is currently being considered by the House Energy and Commerce Committee.
“I have known people like this,” Trump said. “They travel all over the world to try and find a cure but it will be years before they come on to the market.” He also said “right-to-try can help drug companies learn “pretty quickly how effective (a drug) is and if it works.”
Trump told the Republican lawmakers that FDA commissioner Scott Gottlieb is leading the right-to-try effort for the administration. However, during a 10/2017 House subcommittee hearing, one would not get an impression that Gottlieb was a strong proponent of the right-to-try legislation. His testimony focused on changes that the agency was making to ease patient access to experimental therapies under its expanded access program.
The hearing offered Gottlieb an opportunity to comment on the pending legislation. He said the agency recommends narrowing the eligibility from patients who face a “life-threatening disease or condition” to “terminal illness.” Many chronic conditions (e.g. diabetes) are life-threatening, but medical and behavioral interventions make them manageable without considering unapproved therapies. In addition, the term used in one bill for “terminal illness” would benefit from a clear definition. “We recommend defining it as ‘a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months,’” he said.
Additionally, Gottlieb said that over the last few years the agency has received over 1,000 applications annually for expanded access requests, noting that it authorizes 99% of these. “Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days,” he said. However, there is some “reluctance among companies to provide investigational drugs for expanded access,” he said in a related blog post. “This may have been due, in part, to uncertainty about how data for adverse events that occur during treatment under expanded access are viewed by FDA. Companies have voiced concerns that any apparent negative effects might jeopardize the product’s development.”
In addition, FDA senior management has historically been leery of “right-to-try” legislation out of a concern that it could interfere with recruitment of subjects in, and the scientific integrity of, blinded clinical trials.