Advice for the advisory committees

Presented by The Campaign for Sustainable Rx Pricing

Driving The Day

TIME FOR AN UPGRADE — One of the only public windows into the FDA’s medical product approval process needs to be modernized to match the novel and complex drugs they’re reviewing, Katherine reports.

The FDA’s advisory committees are tasked with providing the agency with outside scientific expertise on drugs or other medical products. They’ve been a part of the agency’s regulatory decisions for more than 50 years. In addition to objective views, the committees provide a layer of transparency into the regulatory process — key for maintaining public trust in the FDA’s authority.

“For a public product as important as Covid vaccines, we need to maximize public trust in the process to ensure that people,” Aaron Kesselheim, a professor of medicine at Harvard Medical School and a former advisory committee member, told Katherine.

But in recent years, the FDA has steadily decreased the number of advisory committee meetings it holds. Between 2010 and 2021, the number of FDA drug approvals after an advisory committee meeting plummeted from 55 percent to 6 percent.

Some fear that a dearth of public meetings could hamper the review process, particularly as the agency has therapeutics, including the first-ever gene-editing treatments, on its plate and will review the new fall Covid vaccine soon.

Additionally, there’s no mechanism or mandate for the FDA to explain its rationale when it disagrees with its advisers’ recommendations. Kesselheim and two other members of the 10-person neurological drug advisory committee resigned in protest in 2021 after the FDA granted Biogen’s Aduhelm accelerated approval after the committee recommended against it. A fourth member later resigned in 2022.

Namandjé Bumpus, the FDA’s chief scientist, told Katherine the agency values the expert discourse — even more than any recommendations they make. She added that the agency is “currently thinking through the way we can optimize our advisory committee set-up.”

IT’S FRIDAY. WELCOME TO PRESCRIPTION PULSE. Your hosts are always seeking new obscure FDA trivia.

Send news, factoids and tips to David Lim ([email protected] or @davidalim) or Katherine Ellen Foley ([email protected] or @katherineefoley).

TODAY ON OUR PULSE CHECK PODCAST, host Alice Miranda Ollstein talks with Megan Messerly about the efforts being made by Planned Parenthood and partner organizations to put abortion on Florida’s ballot in 2024 and what that could mean for the six-week ban Republican Gov. Ron DeSantis recently signed into law and what the potential political implications are for the state.

Around the Agencies

FIRST IN RxP: ALZHEIMER’S GROUP KEEPS PUSHING — The Alzheimer’s Association, a patient advocacy group, sent a letter Thursday to the leaders of HHS and CMS asking Medicare to broaden its coverage of newly approved Alzheimer’s therapies, Katherine reports.

“CMS has been inconsistent in its methodology, and is sustaining a policy that cannot be justified on scientific or clinical grounds,” Joanne Pike, the Alzheimer’s Association’s president and chief executive, wrote to HHS Secretary Xavier Becerra and CMS Administrator Chiquita Brooks-LaSure in the letter shared with Katherine.

The letter comes in light of widely hailed new topline data from Lilly’s Alzheimer’s candidate, which the association hopes will help persuade the Biden administration to change its mind.

The group asked CMS in December to cover new monoclonal antibodies that target amyloid, including Aduhelm and Lequembi, for all beneficiaries. In February, CMS denied that request; coverage is currently restricted to those in a randomized trial even though no trials are enrolling patients.

Even FDA Commissioner Robert Califf appeared optimistic at a Wednesday Kitalys Institute webinar about donamemab’s prospects, making the case that there must be post-market evidence generation to help determine who should be treated with the drug.

“We’ll have now a mountain of data about reduction in the rate of decline of cognitive function,” Califf said while cautioning the FDA needs to ensure the actual data is consistent with Lilly’s press release.

Industry Intel

MODERNA STICKS TO VACCINE PRICE — Moderna anticipates that its updated Covid-19 vaccine will have a list price in the range of $110 to $130 a shot, the company said Thursday.

The price update comes more than a month after lawmakers on the Senate HELP Committee pressed Moderna CEO Stéphane Bancel to justify a $130 price point given the $12 billion the government spent to speed the company’s clinical trials and purchase its vaccine.

“In the commercial market, we will be providing differentiated discounts across different payer types from government agencies through to commercial players as well,” Arpa Garay, Moderna’s chief commercial officer, said on the company’s first-quarter earnings call.

The FDA doesn’t have firm recommendations for updated vaccines: Its independent expert panel will meet June 15 to discuss the best options for the fall-winter vaccine campaign.

FDA GREENLIGHTS FIRST RSV VACCINE — GSK’s respiratory syncytial virus vaccine for adults 60 and older received FDA approval Wednesday, making it the first RSV vaccine globally to get regulatory clearance, Katherine reports.

The vaccine, called Arexvy, was more than 80 percent effective in preventing moderate cases of RSV in a Phase III clinical trial and more than 90 percent effective at preventing severe disease.

RSV disproportionately sickens the youngest children and the oldest adults and kills between 6,000 and 13,000 adults over 65 annually, according to the CDC. GSK anticipates it will be able to roll out its vaccine ahead of the next RSV season, suggesting late fall or winter months. It is continuing to study participants for two more seasons to assess the vaccine’s durability.

Coronavirus

FDA: DON’T USE CERTAIN AT-HOME COVID TESTS — The FDA warned the public on Thursday not to use certain lots of SD Biosensor’s Pilot Covid-19 At-Home Tests — distributed by Roche Diagnostics — because of “significant concerns of bacterial contamination” in the test’s liquid solution.

“Approximately 500,000 tests were distributed to CVS Health, as well as about 16,000 tests to Amazon,” the FDA safety communication states. None were distributed through the government’s free at-home test program or other federal testing programs.

Tobacco

CA ‘MENTHOL-LIKE’ PRODUCTS ORDERED OFF SHELVES — Rob Bonta, the California attorney general, informed cigarette manufacturers ITG Brands LLC and R.J. Reynolds that their new “California compliant” cigarettes violate the state’s ban on flavored tobacco products, POLITICO’s Rachel Bluth reports.

The products, which appeared in stores shortly after the state banned most flavored tobacco products, including menthol cigarettes, contain a synthetic chemical, WS3, that has a similar cooling effect to menthol. Companies argued that WS3 was not a flavor and didn’t skirt the ban.

The state disagreed, noting the products’ packaging and promotional materials. Companies have until June 23 to remove them from shelves.

In Congress

FEDS: DO MORE TO PREPARE FOR PANDEMICS — The U.S. must do more to prepare for future pandemics and biological threats, top federal health officials told the Senate HELP Committee on Thursday.

“The increased frequency of outbreaks mean that we should not be asking if we will face another serious public health threat, but when,” CDC Director Rochelle Walensky said.

Lawmakers are weighing whether to reauthorize the Pandemic and All-Hazards Preparedness Act, which expires on Sept. 30.

SENATE FINANCE PLANS PHARMA TAX HEARING — The Senate Finance Committee will hold a hearing next week on the impact U.S. international tax policy has on the pharmaceutical industry, Chair Ron Wyden (D-Ore.) said Thursday.

Wyden said the hearing will focus on the effects of the Tax Cuts and Jobs Act of 2017, which Democrats widely criticize, and what he characterizes as the drug industry’s “tax avoidance schemes.”

Document Drawer

On Wednesday, the FDA issued draft guidance on decentralized clinical trials for drugs, biologics and medical devices.

The FDA and the Duke-Margolis Center for Health Policy will hold a virtual public workshop on the development of rare disease endpoints in clinical trials and the agency’s pilot on the topic on June 7-8.