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Full Pfizer Vaccine Approval Stirs Questions Over Off-Label Uses

Aug. 23, 2021, 9:19 PM

Full approval of Pfizer’s Covid-19 vaccine makes it easier for providers to prescribe off-label uses—such as boosters or shots for children under 12—but federal agreements may curb an uptick in that practice.

The Food and Drug Administration approved Pfizer’s biologics license application Monday for those 16 and older, a landmark decision that makes for the first time a Covid-19 vaccine available outside of an emergency use authorization. Access to products under an EUA requires that providers stick to the terms of that authorization. Once approved, prescribers generally have more flexibility to offer a medical product for a reason not listed on the label.

Two unique factors associated with Pfizer’s vaccine, however, pose a challenge to off-label uses: It’s not a commercially available product because the government is distributing the vaccines, and Pfizer’s two-dose shot is now simultaneously available under a full approval and an EUA. The vaccine is available under an EUA for children ages 12 to 15.

“If it were any other product, once it’s approved, it’s fair game to be off label,” Alison Bateman-House, a bioethicist at New York University whose specialties include access to investigational medicines, said.

Off-label prescriptions are a fairly common and legal practice accounting for about 20% of prescriptions, according to the Agency for Healthcare Research & Quality. Questions have arisen over whether providers will be able to lean on this practice, and whether that would spur a flurry of vaccinations for young children or an uptick in third doses ahead of the Biden administration’s plan to roll out boosters.

“It’s an important discussion to have and to get information out,” said Heather H. Pierce, senior director for science policy and regulatory counsel for the Association of American Medical Colleges.

Pierce said that as schools start up again she can imagine that parents with kids under 12 will be seeking off-label use, especially in areas with high cases and where school mask mandates have been prohibited. Covid-19 vaccines are currently not authorized for children under that age, but pediatric vaccine trials are underway with data on safety and immunogenicity—the ability of the vaccine to stimulate an immune response—expected for all children by the first quarter of 2022.

Because vaccinating children under 12 would require a different dose than what’s been vetted, “the risks to the provider, and to the provider’s license, as well as potentially to children, will probably cause many to hesitate,” Pierce said.

‘A Little Wrinkle’

Providers administering a Covid-19 vaccine must sign an agreement with the Centers for Disease Control and Prevention that they will use the vaccine according to the agency’s terms.

That agreement may make it tougher to go off label for pediatric populations than it would be for booster shots, Govind Persad, a bioethicist at the University of Denver who’s working on a paper about off-label uses for Covid-19 vaccines, said. Compliance with the agreement includes only administering the vaccines in the age group that’s allowed under an approval or an authorization, he said.

“That is a little wrinkle that’s present for the pediatric doses that wouldn’t be present for things like giving boosters,” Persad said. “The provider agreement with federally procured vaccines seems to put more restrictions on the pediatric dose. But I don’t know whether this language is intended to have this effect. I don’t know whether the CDC might sort of change or clarify this.”

Pfizer’s Covid-19 vaccine is unique in that there’s still an EUA for children ages 12 to 15, so there’s both an EUA and a full approval for the same product, Bateman-House noted.

It’s reasonable to conclude that off-label use more widely wouldn’t be appropriate in this situation because there’s both a full approval for people ages 16 and older and a limited use for those ages 12 to 15, she said. If a provider administered a vaccine off label to a small child, “I’m not sure that there’s precedent there for them to be held legally liable. But generally speaking, ethically, you shouldn’t do it. And I’m pretty sure they will have already agreed in that provider agreement not to do it.”

The government’s terms for vaccination through an EUA may not change if it has essentially purchased a company’s entire vaccine supply, Geoffrey Levitt, co-chair of the DLA Piper’s life sciences policy and regulatory group, said.

Technically now with a licensed vaccine, the drugmaker has more flexibility to market and distribute the product, he said. “But if I’ve already promised my supply to the federal government, then in practice, I don’t have any product left to sell through normal market channels,” Levitt, who’s a former chief regulatory counsel for Pifzer, said.

He added it’s unclear whether Pfizer’s pledge to give its doses to the U.S. government and other countries will affect its “practical ability to distribute through other channels, as opposed to their legal ability to do so.”

Discouraging Off-Label Use

The FDA, CDC, and the American Academy of Pediatrics are actively discouraging off-label use.

FDA Acting Commissioner Janet Woodcock said there still isn’t full safety data or information on what the right dose is for children.

“We need to get the information and data on uses in younger children. They are not just small adults,” she said at a press briefing Monday. “We are not recommending that children younger than age 12 be vaccinated with this vaccine. It would not be appropriate.”

CDC spokeswoman Kristen Nordlund also said uses outside of the government’s authorization and recommendations raise issues like liability protections, compliance with the CDC provider agreement, and reimbursement for administration costs.

“Providers should administer COVID-19 vaccines in accordance with all program requirements and recommendations of CDC, the Advisory Committee on Immunization Practices, and the FDA. This applies to both FDA authorized and FDA approved COVID-19 vaccines,” Nordlund said in an email."Use of these products for purposes outside of those that have been approved and authorized by FDA (often referred to as ‘off-label use’) is not recommended.”

Yvonne Maldonado, chair of the American Academy of Pediatrics’ Committee on Infectious Diseases, urged physicians to wait until the clinical trials are complete in children to give the vaccine to those under 12.

“I know parents are anxious to protect their children, but we want to make sure children have the full benefit of ongoing clinical trials,” she said in a statement. “We do not want individual physicians to be calculating doses and dosing schedules one-by-one for younger children based on the experience with the vaccine in older patients.”

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bloombergindustry.com

To contact the editor responsible for this story: Alexis Kramer at akramer@bloomberglaw.com

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