An effort to get the US Senate to pass a so-called Right to Try bill failed Wednesday when Senate Minority Leader Harry Reid blocked the effort, because a hearing was never held to review the legislation and only two of the 42 sponsors was a Democrat.
“It hasn’t been through the committee process as we were promised given the outstanding issues dealing with this policy,” Reid, a Nevada Democrat, told the Senate after Senator Ron Johnson (R-Wis.) attempted to win unanimous consent from the Senate for his legislation. “There are major players who have not had an opportunity to say what’s wrong with the bill.” (You can watch the video here).
Johnson later sent Reid a letter to note that, in fact, two hearings were held to review the bill. “It is beyond disappointing that Sen. Reid would ignore the pleas of those with terminal illnesses to score a political point,” Johnson later said.
The move was a setback — perhaps one that may be temporary — for the Right to Try movement. A spokeswoman for the Senate Homeland Security and Governmental Affairs Committee, which Johnson chairs, told us that he “will continue to work to get this bill passed.” A similar bill, by the way, is pending in the House.
The Right to Try laws allow patients to leapfrog a drug-development process that takes years before new drugs become available. The effort reflects rising frustration with a US Food and Drug Administration program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they aren’t enrolled in a clinical trial.
Under federal law, if terminally ill patients are not eligible to obtain an experimental drug through a clinical trial, they can apply to the FDA for the expanded access program, which is also known as compassionate use. The agency actually approves the vast majority of these requests, which must also be approved by drug makers. But critics maintain the federal program is arbitrary and cumbersome.
So far, 32 states have passed a Right to Try law, with California joining the list just last week.
But whether the Right to Try movement can succeed is uncertain. As we have noted previously, there are numerous questions about the extent to which the laws — which have been drafted by the Goldwater Institute, a Libertarian think tank, and introduced in more than a dozen other states — can accomplish their goals.
In an essay published last year in the Annals of Internal Medicine called “Hope, Hype and Unintended Consequences,” researchers from New York University and Columbia University argued that the laws “will do nothing to significantly change patient access to experimental medicines.”
Among the issues cited: the FDA does not provide oversight of the laws. The agency does not require drug makers to make medicines available. Insurers are not required to pay for treatment, since most states failed to create funding to help patients cover medical expenses. And there are unanswered legal questions about whether state laws trump federal regulations.
In a bid to dispel such concerns, Johnson last week held a hearing of the Homeland Security and Governmental Affairs Committee, but ran into opposition from the FDA, which had already ticked off the Senator because FDA Commissioner Dr. Robert Califf backed out of testifying at the last minute, purportedly due to scheduling conflicts.
In his stead, Dr. Peter Lurie, an associate commissioner for public health strategy and analysis for the FDA, testified that the agency has authorized more than 99 percent — or 7,110 of 7,176 — requests from physicians for patient access to investigational new drugs, as experimental medicines are called, between fiscal years 2010 and 2015. And he added that the agency has revamped its applications to make the process less time-consuming.
Lurie argued, though, drug makers may have concerns about making products available prior to a regulatory review as a key hurdle to access. Companies may deny requests in order to meet strict criteria needed to win FDA approval for a medicine — an unexpected patient reaction, for instance, might jeopardize the chance of success. Sometimes, a company claims to lack sufficient supplies for a large number of requests.
“It appears that pharmaceutical companies turn down considerably more applications from physicians than does the agency,” Lurie told the committee. He also expressed concern that the FDA may somehow not be privy to side effects that emerge during treatment with drugs under these programs.
“While we welcome suggestions that might improve the expanded access process,” he said, “we would caution against any changes that would reduce FDA’s role in expanded access or that might undermine the crucial clinical trial development process.”