President Donald Trump last week insisted that his commissioner of the Food and Drug Administration, Scott Gottlieb, was leading the effort to allow critically ill patients who have exhausted all other options to have broader access to experimental drugs that have not yet gone through the U.S. approval process.
But Gottlieb has gone on the record as objecting to the so-called right-to-try legislation, which passed the Senate last summer and is currently pending in the House.
Trump used his Jan. 30 State of the Union address to publicly declare his support for the right-to-try legislation. His vice president, Mike Pence, also told lawmakers in a Jan. 18 tweet that it was time to get the bill out of the House and to the president's desk.
"I hope you folks can approve it and I hope you agree with it," Trump told Republicans Feb. 1 at their retreat at the Greenbrier resort in White Sulphur Springs, W.V. "It's so important. And Scott Gottlieb is heading it up."
But Gottlieb, whom Trump recently called a "star," told lawmakers at an Oct. 3, 2017, hearing that the right-to-try bill contained flawed criteria, which he said could expose more people unnecessarily to potentially dangerous side effects. He called for changes to the bill.
The FDA chief noted that his agency already has a long-running expanded access program, also known as compassionate use, in which regulators help terminally ill patients obtain and use investigational medicines outside of the clinical trial process.
Gottlieb's handling of the right-to-try legislation going forward will be a test of his loyalty to Trump, which is likely to be played out in the public eye.
More than three dozen states have passed their own versions of the right-to-try bill.
The pharmaceutical industry insists the FDA needs to remain a part of the process — something not required by the Senate bill that was sent to the House — and the medical community has also largely pushed back against the legislation.
In a Jan. 10 op-ed in the peer-reviewed medical journal, the New England Journal of Medicine, Steven Joffe and Holly Fernandez Lynch from the University of Pennsylvania asserted the motivation behind the proposed legislation threatened to weaken the FDA's ability to pursue its public health mission.
"Are we prepared to abandon the FDA's gatekeeping role in favor of unfettered patient autonomy and market forces, risking precisely the problems that prompted Congress to grant the FDA its present authority?" they asked. "The agency has made substantial progress in balancing the needs of desperate patients and the principle that all patients deserve evidence that the benefits of medical products justify their risks. We upset that balance, and diminish the FDA's public health mission, at our peril."
Cutting the FDA out as a gatekeeper "increases the risk of patients being harmed through the peddling of false hope and snake oil," said former biotech executive Michael Becker, a doctor and president and founder of MDB Communications LLC, who has been diagnosed with terminal cancer.
In a Feb. 1 op-ed in Stat News, Becker noted that under the right-to-try bill, a death caused by the use of an experimental drug could not be considered by the FDA in deciding whether to approve or reject the therapy.
"While this may help address pharmaceutical and biotechnology industry concerns that expanded access programs could jeopardize ongoing studies, it is simply unethical at its core," he wrote.
Becker also noted it was unclear who would pay for hospitalization or other care if something goes wrong with the experimental drug under the right-to-try use.
Cutting drug prices Azar's No. 1 job
Meanwhile, at the Republicans' retreat and the State of the Union, Trump reiterated his desire to reduce the price of prescription drugs — a promise he had made throughout his presidential campaign but has taken little action to achieve.
He charged his new secretary of Health and Human Services, Alex Azar, with getting the job done.
Prices will "come rocketing down," Trump pledged during Azar's Jan. 29 swearing-in ceremony.
Speaking from Indiana on Feb. 2, Azar said he and the president were "working on an aggressive agenda on lowering list prices and costs to the system from prescription drugs."
"I've only been on the job for several days and I have had many interactions already with the president of the United States on this issue," he added.
Azar said Trump told him lowering prices was "job number one."
The new health chief said he has been meeting with most senior members of the administration on a "game plan," which he said involved working on how to increase generic and branded drug competition to lower prices.
He said that he and other administration officials have been examining "how we end gaming of the patent system by branded pharmaceutical companies so that we get more low-cost generics out on the market" — actions Gottlieb has been working on at the FDA.
Azar said he and his team were also looking at ways to ensure the benefits from the discounts insurance companies receive from drug manufacturers — which are paid through middlemen entities known as pharmacy benefit managers — are passed on to patients so they can pay lower out-of-pocket costs at the pharmacy.
But "most importantly" he was searching for ways to create incentives aimed at getting drugmakers to lower rather than raise their prices, he added.
Azar, a former executive at Eli Lilly and Co., had complained during his Jan. 9 confirmation hearing that biopharmaceutical manufacturers do not lower their prices because the system does not provide incentives to do so.
Indiana opioid funds
Azar's Feb. 2 drug pricing remarks came during a news conference in Indiana, where he ventured outside of Washington for the first time in his new role to announce his agency had granted that state the second ever federal waiver to require able-bodied adults to work or engage in other community activities, such as skills training, education, job search, volunteering or caregiving, in exchange for receiving Medicaid benefits.
Kentucky was the first state granted the work requirement waiver, receiving it on Jan. 12.
As part of Indiana's waiver, the federal government is allowing the state to use up to $80 million each year for the next three years — or up to $240 million in total — to support efforts to combat the opioid epidemic.
A range of addiction treatment options — inpatient, outpatient and residential — will be available for low-income beneficiaries enrolled in either the state's traditional Medicaid program or its expanded Healthy Indiana Program, said Jennifer Walthall, secretary of Indiana's Family and Social Services Administration.
Walthall emphasized the $240 million was not a grant, but would come through the Medicaid reimbursement process and was based on an estimate of the number of beneficiaries anticipated to use the opioid addiction services.
"This is about giving them access to more hospitals and other facilities to help them recover, as well as expanding access to recovery opportunities in their communities," Walthall told reporters.
"It is not an overstatement to say this is not going to just change lives. This in fact is going to be able to save lives," Indiana Gov. Eric Holcomb said.
While Indiana may have additional opioid funding support, Trump has yet to ask Congress for new funds to fight the epidemic nationwide.
