News: Voucher program notches win as clock ticks

Friday, September 20, 2024

Voucher program notches win as clock ticks

Sep 20, 2024

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Driving the Day

Scientist filling tubes with blue liquid

The FDA's priority review voucher program, which helps accelerate the development of treatments for rare pediatric diseases, will lapse on Sept. 30 unless Congress acts. | Shutterstock

VOUCHING FOR RARE DISEASE DRUGS — The FDA’s priority review voucher program for rare pediatric diseases is set to lapse on Sept. 30 without congressional intervention. And while advocates and analysts expect lawmakers to reauthorize the program, the timing is unclear as House leaders struggle to pass a stopgap spending measure to avert a government shutdown on the same day.

Your host took a deep dive into the program to get a vibe check on its prospects.

Committee movement: The House Energy and Commerce Committee unanimously approved a bill Wednesday reauthorizing the rare pediatric disease program for five years, POLITICO’s Ben Leonard reports. Priority review vouchers are awarded to companies developing drugs for diseases with unmet needs. They can later cash in the vouchers to speed up the agency review process for another drug — or sell the voucher to another company.

The panel amended the legislation — which the health subcommittee had advanced in May on a party-line vote — to require a Government Accountability Office report within five years evaluating the indications of approved drugs for which the vouchers were awarded.

The committee’s top Democrat, Rep. Frank Pallone of New Jersey, has questioned how much the 12-year-old program has incentivized pharma companies to develop treatments for rare pediatric conditions. He signaled some reservations Wednesday, but he ultimately supported the bill.

Small drugmakers often plan their budgets expecting that the vouchers would be available, according to Kurt Karst, a pharmaceutical attorney at Hyman, Phelps & McNamara.

“Obviously, not reauthorizing is going to throw those plans into a little bit of disarray,” Karst said. “I would anticipate it gets reauthorized. Once something is in the law, it is difficult to actually get it out.”

The riskiness of rare disease drug development makes the prospect of earning a voucher that can be sold for millions attractive to small firms that need the funding, said Karin Hoelzer, senior director of policy and regulatory affairs at the National Organization for Rare Disorders.

A report NORD released in July found that the FDA had awarded 53 priority review vouchers across 39 rare childhood diseases, three of which had any agency-approved drugs before the voucher program’s debut.

Since the last GAO review of program data ended in 2019, the number of vouchers granted has more than doubled, Hoelzer said, adding that the growth tracks with the typical decadelong time frame to develop a rare disease drug win approval.

The group hopes for at least a temporary reauthorization to make its way into a continuing resolution this month, Hoelzer said.

“I’m realistic that Sept. 30 is not far away,” she added.

IT’S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. Fun fact: Neither of your morning hosts was born yet when Hatch-Waxman passed.

Reach out with tips to David Lim (dlim@politico.com or @davidalim) and Lauren Gardner (lgardner@politico.com or @Gardner_LM).

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Nearly two-thirds of Express Scripts by Evernorth customers spend less than $100 a year out-of-pocket for all their medications. Yes, despite the median annual price of new drugs to market soaring to an average of $300K in 2023. Yes, despite the median of all drug prices going up by 5% last year. That's not a middleman. That's an advocate. Express Scripts is fighting to make medications more affordable. Learn more here.

In Congress

Rep. Henry Waxman speaking at a microphone in Congress

Former Rep. Henry Waxman, pictured here in a 2009 photo, celebrated the 40th anniversary of the Hatch-Waxman Act on Thursday. | Susan Walsh/AP

HATCH-WAXMAN 40 YEARS LATER — Days ahead of the 40th anniversary of landmark legislation that established the modern generic drug industry, former Rep. Henry Waxman (D-Calif.) and a litany of former Hill staffers, government officials and academics gathered Thursday at the Brookings Institute to reflect on the law’s impact.

“The prices of drugs were quite high, and it just seemed to me that we would be better off if we had some competition of generic drugs,” Waxman, now 85, said.

Before the law’s passage, generic manufacturers had to undergo clinical trials to repeat safety and efficacy research instead of demonstrating bioequivalence to a brand drug.

“The Orphan Drug Act and Hatch-Waxman ... none of us appreciated how much of an impact they would have,” said Steven Grossman, who worked for late Sen. Orrin Hatch (R-Utah) as FDA counsel during the law’s passage.

Generic drugs now make up about 90 percent of the U.S. market but account for about 20 percent of drug spending.

“It was totally transformative,” pharmaceutical attorney Kurt Karst said. “There was essentially no generic industry before Hatch-Waxman.”

Markus Meier, who recently retired from the Federal Trade Commission’s health care division, said the Hatch-Waxman Act “largely achieved the goals it set out to do” despite the pharmaceutical industry’s patent manipulations and abuses that gamed the system over the years.

Antitrust enforcement, he added, “is, at best, a very second-best solution to legislation and regulatory fixes” because “it’s slow, it’s uncertain and it’s limited.” But Meier maintained that antitrust efforts to encourage competition are still important despite their limitations given the current political environment and the impact of recent Supreme Court rulings.

FDA MIDDLE EAST OFFICE — The priority review voucher legislation that advanced this week contained an unrelated FDA nugget: The legislation would establish an agency office in one of the so-called Abraham Accord countries — Israel, the United Arab Emirates, Bahrain and Morocco.

The FDA hasn’t staffed an office in the Middle East since it shut its Jordan outpost in 2013, and talk of establishing one in Israel has percolated for years. Supporters say having a presence in the region could help the U.S. “friend shore” manufacturing for essential medicines like penicillin that have been in shortage in recent years.

The economies in countries like Israel and the UAE would “get a real adrenaline kick, and we [would] get essential medicines from friendlier sources,” said Peter Pitts, a former FDA associate commissioner who’s advocated for the concept for the U.S. Israel Education Association.

By setting up shop on the ground, he added, regulators in those nations could better learn the process and philosophy by which the FDA considers drugs and devices, setting them up for greater success.

Pitts noted the provision’s addition to the House voucher bill could help its prospects, given high expectations that lawmakers will ultimately re-up the unrelated program. Still, its inclusion doesn’t guarantee that congressional leaders will endorse it alongside a program reauthorization.

Medical Research

CRENSHAW, CRAIG PRESS ON THIRD-PARTY REVIEWS — A pair of bipartisan lawmakers on the House Energy and Commerce Committee wants the FDA’s new top medical device regulator to explain what can be done to boost the use of the third-party review program for less risky medical devices.

Reps. Dan Crenshaw (R-Texas) and Angie Craig (D-Minn.) noted that the agency believes many in vitro clinical diagnostics that need to be reviewed under the FDA’s final rule for laboratory-developed tests will use the third-party review program.

The 510(k) third-party review program allows outside firms accredited by the FDA to conduct reviews of certain medical devices — but the FDA makes the final decision based on the review and recommendation.

“Our hope is that this will free up agency staff time to review more complex, innovative, high-risk devices,” Crenshaw and Craig wrote Thursday to Michelle Tarver, acting director of the FDA’s Center for Devices and Radiological Health.

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Pharma Worldwide

MPOX SHOT FOR TEENS — The European Commission endorsed Bavarian Nordic’s mpox and smallpox vaccine for adolescents ages 12 to 17, POLITICO’s Rory O’Neill reports.

It’s the first mpox shot approved on the continent for anyone under 18, a decision that comes a week after the World Health Organization approved it for adults.

Pharma Moves

Brent Eberle will join CivicaScript, a nonprofit generic drug manufacturer and seller, as its new president. He currently is chief pharmacy officer at Navitus Health Solutions.

Document Drawer

The FDA announced the fee Thursday for using a priority review voucher for fiscal 2025: approximately $2.5 million.

The FDA issued draft guidance Friday containing recommendations for manufacturers studying the biocompatibility of medical devices.

The FDA issued a direct final rule Friday outlining its regulatory hearing approach for tobacco-product manufacturing practice requirements.

WHAT WE'RE READING

Rep. Andy Harris (R-Md.) — the senior House FDA appropriator — is the new leader of the House Freedom Caucus, POLITICO’s Sarah Ferris, Daniella Diaz and Jordain Carney report.

Granules, a generic drugmaker in India, was hit with a rough 483 inspection report from the FDA that said the company was removing torn documents from a facility, STAT’s Ed Silverman reports.

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