Good Day BIO: What was CMS thinking?

We recap Day 2 of the BIO CEO and Investor Conference with what a former CMS Administrator said about the controversial coverage determination and what you need to know about our new FDA Commissioner. (630 words, 3 minutes, 9 seconds)

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What we’re watching today: We have a full day of virtual sessions, including creating biotech startups through incubators (11 AM ET), Cures 2.0 (1 PM ET), gene therapy approval and commercialization (2 PM ET), and more.

What was CMS thinking?

Alzheimer’s is on the rise—yet a controversial decision by the Centers for Medicare and Medicaid Services (CMS) could limit access to breakthroughs for patients. This set the tone for Day 2 of the BIO CEO and Investor Conference. 

ICYMI: The FDA recently granted accelerated approval for the first disease-modifying treatment for Alzheimer’s—but CMS proposed to restrict coverage to patients participating in clinical trials, setting a dangerous precedent. 

“This is the first time this has ever happened for a drug for an on-label indication,” said former CMS Administrator and FDA Commissioner Dr. Mark McClellan, speaking yesterday with Dr. Michelle McMurry-Heath in New York. 

“I don’t see this being a fundamental shift in CMS’ approach to coverage decisions as much as a new challenge,” in terms of how we deal with accelerated approvals for broad populations early on in the disease process when the condition is not life-threatening, he said. 

CMS’ argument that the trial wasn’t diverse enough is “interesting logic” given that “we have decades of clinical trials that were not representative,” said Dr. Michelle. Watch the discussion:

This “adds another level of uncertainty” for investors, said Dennis Purcell of Aisling Capital in a session on the policy outlook. 

Alzheimer’s drug development is already challenging. “I don’t know if any singular entity is going to bring forward a blockbuster in Alzheimer’s disease,” said Bill Martin, Head of Neuroscience at Janssen Pharmaceutical Companies, in a discussion on neurodegenerative therapeutics. 

But we’ve made “enormous progress,” especially in terms of identifying targets, said Rachelle Doody, Roche’s Global Head of Neurodegeneration—all the more reason why we can’t stop it in its tracks now.

We have a commissioner!

The Senate confirmed Dr. Robert Califf as the Commissioner of the Food and Drug Administration (FDA)—finally! 

The news: Dr. Califf was confirmed yesterday 50-46, with six Republicans voting in favor and five Democrats voting against the nomination; the vote otherwise followed party lines. A cardiologist, he previously served as FDA Commissioner from 2016-2017.

“Dr. Califf is an excellent choice to lead the FDA at this critical juncture,” said BIO President and CEO Dr. Michelle McMurry-Heath. “He’s a proven leader whose on-the-job experience and deep understanding of the agency will allow him to hit the ground running. I have known Dr. Califf for many years. He’s effective, inclusive, and makes decisions by following the science.” 

His leadership will drive several health priorities: “He’s driven efforts to modernize clinical trials, including the use of real-world evidence, and he’s pushed for greater diversity in science and clinical trials,” she added. 

He will also contribute to the oversight of animal and plant biotech: “BIO members are developing innovations that stand to benefit people and the planet,” said Sarah Gallo, BIO’s VP for Agriculture and Environment. “But these breakthroughs are dependent on a clear, timely and science-based regulatory approval process that provides a viable path to market. BIO looks forward to working with Dr. Califf to ensure the United States remains a leader in innovation and embraces the solutions that technology provides.”

More News:

The New York Times: A woman is cured of H.I.V. using a novel treatment
“A woman of mixed race appears to be the third person ever to be cured of H.I.V., using a new transplant method involving umbilical cord blood that opens up the possibility of curing more people of diverse racial backgrounds than was previously possible, scientists announced on Tuesday.”

President Biden’s Wednesday: No major events scheduled a day after giving a speech on Russia yesterday. The White House COVID-19 Response Team will hold a press briefing at 11 AM ET. 

What’s Happening on Capitol Hill: A subcommittee of the House Education and Labor Committee will hold a hearing on “promoting a healthier, more supportive school environment.” Democrats are considering putting inflation high on their agenda, The New York Times reports.