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Capitol Checkup: US FDA's experimental drugs push; ACA ruling's political box


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Capitol Checkup: US FDA's experimental drugs push; ACA ruling's political box

In another sign the U.S. Food and Drug Administration is putting more effort into directing critically ill patients who want to try experimental medicines to the agency's expanded-access program and away from using unproven therapies outside of regulators' control, Commissioner Scott Gottlieb disclosed plans for a new compassionate-use pilot program.

Some medical ethics experts, however, questioned whether the FDA's new effort could undermine the clinical trials process and raise patients' expectations about the safety and effectiveness of early-stage drugs.

Congress passed a law in May that lets Americans seek investigational treatments from biopharmaceutical companies without going through the FDA's expanded-access process — the Right to Try Act.

Gottlieb initially opposed the legislation, sponsored by Sen. Ron Johnson, R-Wis., and publicly criticized it during an October 2017 hearing.

Johnson and his Right to Try Act supporters had argued the FDA's expanded-access program — also known as compassionate use — was not working sufficiently, despite Gottlieb testifying last year that the agency annually grants 99% of the requests it receives.

Critics of the new law said it would expose desperately ill patients to "snake oil" salesmen peddling false hopes.

After Gottlieb's boss, President Donald Trump, threw his full weight behind Johnson's bill — telling the Republican-controlled Congress to pass it — the commissioner backed away from his opposition and said he felt "comfortable" with the bill. After the Right to Try Act was passed, Gottlieb said his agency stood ready to implement it, though Johnson made it clear the new law was intended to "diminish the FDA's power over people's lives, not increase it."

Last month, the FDA unveiled a new website about the Right to Try Act, though it provides little information and directs viewers to the agency's expanded-access program site.

Now, the FDA plans to launch a pilot program in 2019 under what it is calling its "Project Facilitate," in which the agency will assist patients more directly in accessing unapproved drugs, such as filling out their expanded-access program paperwork — activities beyond the compassionate-use services regulators already provide.

The pilot program will involve access to experimental cancer therapies, Gottlieb told the trade publication BioCentury in an article that published over the weekend.

The commissioner said he wanted to "tip the scale in favor of expanded access."

On Twitter, Gottlieb said the FDA's "goal is to facilitate and streamline the patient experience with our expanded access program; to create concierge-like service for providers and patients who are trying to get access [to] drugs."

"We want to make the entire process more efficient and robust," the commissioner wrote in a Dec. 15 tweet.

The FDA, however, acknowledged it is unclear whether its Project Facilitate telephone staff could provide advice to patients about the availability of clinical trials or other drugs — approved or not yet cleared through the regulatory process.

Gottlieb said the FDA has the funds available to run the pilot and does not need Congress' approval for it, though he said the agency may need to seek additional resources from Capitol Hill to expand Project Facilitate.

While drugmakers will continue to have the final say on who uses their products, if they decline a patient, the companies will "have to give the reason for denying access," Richard Pazdur, director of FDA's Oncology Center of Excellence, told BioCentury.

It is unclear, however, whether the FDA has the authority to force drugmakers to provide that information to patients or if the companies' reasons for not permitting use of their experimental medicines would be made public.

A spokeswoman for the FDA was unable say why Gottlieb had never mentioned Project Facilitate before now — in testimony or at public forums — given the report said Pazdur initially proposed it in early 2018 or if he was trying to steer patients away from using the Right to Try Act.

She told S&P Global Market Intelligence the FDA would provide greater detail on the development of the initiative "in the near future."

A spokesman for Johnson was unable to obtain a response from the senator on if he had any concerns about the FDA's intent for the pilot.

Holly Fernandez Lynch, an assistant professor in medical ethics and health policy at the University of Pennsylvania, said the FDA's pilot is a "great effort to render Right to Try even less desirable," but she questioned whether the agency should be encouraging access to experimental drugs where the safety and efficacy is unknown.

Such a trend could change patients' expectations and willingness to enroll in clinical trials, "because it supports the notion that investigational drugs work and [patients] should want them," Fernandez Lynch wrote on Twitter.

ACA ruling's political fallout fears

Republican lawmakers showed little enthusiasm over a Texas district judge's late-night Dec. 14 ruling that the Affordable Care Act was unconstitutional — a court victory the party had long sought, but one its members are not embracing.

The House flipped from Republicans' control in the November midterm elections largely on the Democrats' consistent message that their rivals had tried — and failed — a number of times on Capitol Hill to dismantle the ACA and invalidate its protections for Americans with pre-existing medical conditions.

If upheld, the ruling in the Texas lawsuit, brought by 20 Republican state attorneys general, would do just that.

Polls, however, have shown the ACA's pre-existing conditions protections are popular with Americans.

Judge Reed O'Connor's decision also would take away the healthcare coverage that millions of Americans obtained under the ACA's Medicaid expansion, and seniors and people with disabilities enrolled in the federal government's Medicare Part D drug program would pay more for their prescription medicines.

Parents would not be able to keep their adult children up to age 26 on their healthcare insurance policies and insurers could again put lifetime caps on coverage.

In addition, the U.S. Food and Drug Administration could no longer approve lower-cost versions of biologic therapies, or biosimilars. And Health and Human Services Secretary Alex Azar would need to forget about some of the initiatives his agency planned to undertake to lower drug prices, given the authority it sought to use — specifically through the Center for Medicare and Medicaid Innovation — was granted by the ACA.

"It puts a lot of our Republicans in a box," Senate Minority Leader Chuck Schumer, D-N.Y., said during a Dec. 16 appearance on NBC's Meet the Press.

The judge, appointed by former President George W. Bush, and the 20 attorneys general "didn't do the Republican Party any favor," Democratic Senate Whip Dick Durbin of Illinois added on ABC's This Week.

On the same program, Republican Sen. Susan Collins of Maine called the judge's ruling "far too sweeping," and predicted a higher court would overturn it on appeal. She made similar remarks the same day on CNN's State of the Union.

Republican Sen. Roy Blunt of Missouri declined to say whether he agreed with Trump's assertion the ruling was "great news for America." Because there is no injunction, the judge's decision "has no immediate impact" and would likely spend years in the appeals process, Blunt said on NBC's Meet the Press. He added, though, that he expected healthcare to be "used as a political issue way beyond the ramifications of one district judge."

Rather than declaring victory, Rep. Greg Walden, R-Ore., chairman of the House Energy and Commerce Committee — a post he will lose in January when Democrats take control — called on his Democratic colleagues to work with Republicans on reforming healthcare.

Most legal experts — even several conservatives — said they expected the ruling to be overturned.

"I would be quite surprised if this opinion survives the inevitable appeal to the U.S. Court of Appeals for the Fifth Circuit, and even more surprised if this result garners the support of more than two justices on the Supreme Court, if the case even gets that far," Jonathan Adler, director of the Center for Business Law and Regulation at Case Western Reserve University, wrote in a Dec. 14 article in libertarian magazine Reason.

Senators take aim at price gouging

Meanwhile, four Democratic senators last week introduced legislation that would give HHS the authority to take actions beyond naming and shaming against drugmakers that price gouge.

The CURE High Drug Prices Act, sponsored by Democratic Sens. Richard Blumenthal of Connecticut, Kamala Harris of California, Jeff Merkley of Oregon and Amy Klobuchar of Minnesota, would require biopharmaceutical companies to submit justification to HHS for any price increases 10% or more from the preceding year, 20% over three years and 30% over five years and if the agency determines those hikes are unreasonable, it could require the company to reimburse consumers and payers, including government-run healthcare programs.

HHS also would be given the power to force the drugmakers to reduce their prices back down to their original level up to one year before the increases and to pay a civil penalty up to three times the excessive amount the manufacturer received as a result of any price gouging.

In addition, HHS could also refer the matter to the Department of Justice for enforcement.

"Giving HHS the authority to turn back exploitive price increases will help reduce delayed treatment and the extreme financial burden American families have had to shoulder due to big pharma's greed," Blumenthal said in a Dec. 13 statement.