Saving lives with measles vaccinations, newborn screenings

Administration offers promising views on CGT during FDA roundtable

Top officials urged reduced regulatory barriers and alignment across the Health and Human Services Department (HHS) to spur access to Cell and Gene Therapies (CGT) during a Food and Drug Administration (FDA) roundtable on June 5.

FDA Commissioner Marty Makary, M.D. led a roundtable discussion and panel that included HHS Secretary Robert F. Kennedy, Jr., FDA Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad, M.D., National Institutes of Health Director Jay Bhattacharya, M.D., and Centers for Medicare & Medicaid Services Administrator Mehmet Oz, M.D.

HHS officialsheard from cell and gene experts and advocates on how to enable more efficient development and manufacturing of CGT, as well as barriers to access for patients. Speakers also praised progress in CGT under the leadership of Nicole Verdun, Super Office Director, CBER. Proposals from the roundtable participants included:

• Applying flexible evidence standards, especially for rare diseases.
• Continuing to expand the use of Accelerated Approval for cell and gene therapies, while also ensuring access.
• Ensuring U.S. CGT leadership in the face of Chinese competition.
• Exploring incentives and methods to bring clinical trials back to the US.

Value-based treatments: Kennedy urged payment overhauls enabling one-off CGT treatments that cure diseases instead of managing them, highlighting the success of the Centers for Medicare and Medicaid Innovation (CMMI) model for sickle cell gene therapy—and the potential to expand the model to other such treatments.

What they're saying: “We want to make sure that we get rid of the regulatory impediments,” Kennedy said. “We’re going to make this country the hub of biotechnology innovation.”

Watch the roundtable.

Survey finds lag in measles vaccination amid outbreak

Measles vaccination rates are below the 95% minimum needed to guarantee herd immunity in many U.S. counties, creating vulnerability to a preventable disease, a study finds.

The data: County-level data in 33 states shows measles, mumps, and rubella (MMR) vaccination rates decreased from 93.92% in the 2017-2018 school year to 91.26% in 2023-2024, says a study published June 2 in JAMA. Of 2,066 counties, 78% had declines in vaccination rates.

Why it matters: “The U.S. childhood vaccination program is estimated to have prevented more than 24 million cases of vaccine-preventable diseases in 2019 alone,” the study says. “Nonetheless, there is evidence of a U.S. national-level decline in the childhood [MMR] vaccination rate between 2019 and 2024.”

Why it matters now: In the worst outbreak since 2019, the Centers for Disease Control and Prevention (CDC) reports 1,168 confirmed U.S. measles cases; 95% of patients are unvaccinated or have unknown vaccination status.

What BIO’s doing: BIO’s campaign—“Invest in America. Vaccinate.”— uses clear messaging and images from a recent, less healthy past to remind everyone what we lose if vaccination slows down.

BIO’s view: “Decades ago, diseases like polio and measles brought suffering and death with no defense. Today, immunization represents the right and the ability to defend and protect those we love,” says the campaign website. “But a growing group are letting preventable infectious diseases make a comeback.” 

Visit www.whywevaccinate.org to learn more.

Patient’s mother advocates for newborn screening program reform

Because newborn screening for treatable genetic disorders saves lives, officials must revive and reform a federal advisory body to expand the number of diseases screened, a patient advocate tells Bio.News.

Why it matters: Many debilitating or potentially deadly diseases have much better outcomes if they are detected and treated early. Elisa Seeger’s son Aidan would not have died from adrenoleukodystrophy if he’d had the available screening.

Slow progress: Many states relied on the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) and its Recommended Uniform Screening Panel (RUSP) to add newly treatable genetic conditions to their screening program. But the process of adding new conditions to the RUSP was slow and tedious, and ACHDNC only added nine new conditions to the RUSP in its 15 years of existence.

ACHDNC was disbandedin April 2025, by the Department of Health and Human Services (HHS).

What’s needed: Seeger’s Newborn Screening Alliance advocates for a more effective body to replace ACHDNC and efficiently add to the current list of 38 diseases on the RUSP.

What they’re saying: “My son Aidan’s medical bills due to his late diagnosis of ALD exceeded $4,000,000. The cost to add ALD to the newborn screen panel in New York (with approximately 250,000 babies born annually) was approximately $500,000,” Seeger says. “We can clearly see the significance of saving thousands of lives as well as millions of dollars.”

Read more on Bio.News.

What Else to Read This Week

FDA launches AI tool to assist in application reviews. “Elsa” is a generative AI tool designed to increase the efficiency of the Food and Drug Administration’s scientific reviewers, investigators, and other staff. The FDA said the new tool would allow for faster, more efficient handling of the tasks involved in reviewing new drug applications.

FDA kicks off listening session series. The sessions, part of a listening tour led by Food and Drug Administration (FDA) Commissioner Marty Makary, M.D., provide industry executives with the opportunity to share feedback on opportunities to strengthen and modernize the US regulatory framework for medicines. FDA officials reported that approximately 70 industry leaders participated in the first session in Silver Spring, MD on June 5. Additional stops are planned throughout the summer in San Francisco, San Diego, Boston, New York and Atlanta. Read more here.

BIO President & CEO John F. Crowley joined former UK Prime Minister David Cameron to discuss rare disease drug development. Crowley and Cameron are members of the board of the Advisory Council for the Oxford-Harrington Rare Disease Centre, a transatlantic partnership between the University of Oxford, UK, and Harrington Discovery Institute at University Hospitals in Cleveland aimed at driving breakthrough treatments for rare diseases. The May 22 panel at the 2025 Harrington Scientific Symposium (pictured below) focused on progress in rare disease drug discovery and development.

We’ll see you at the BIO International Convention in Boston! From June 16-19, watch your inbox DAILY for updates from the world’s largest gathering of the biotechnology industry! Along with the remarkable partnering and networking opportunities, this year’s event will feature big names including FDA Commissioner Marty Makary, M.D., and Governors Maura Healey (MA), Josh Shapiro (PA), and Tim Walz (MN). Learn more at convention.bio.org.   

Beltway Report

In a June 5 House Science, Space and Technology Joint Research & Technology and Energy Subcommittee hearing on “Strategic Priorities in Biotechnology,” Research & Technology Subcommittee Chair Jay Obernolte (R-CA) highlighted the importance of public-private partnerships to advance biotechnology and cited the National Security Commission on Emerging Biotechnology report, which notes risks of China replacing America’s biotech leadership. “In just five years, the market value of Chinese biotechnology firms has increased 100-fold and is now valued at $300 billion,” he said. “The Golden Age of Innovation for biotechnology is here, and it is essential that the U.S. be at the forefront.”

On Tuesday, June 10, the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies will review the 2026 budget request for the National Institutes of Health (NIH), with testimony from NIH Director Jay Bhattacharya, M.D. The Senate will continue consideration of the One Big Beautiful Bill Act.