Policy

HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy.

According to President Trump, CMS Administrator Mehmet Oz is a “tough hombre” who can bring down drug costs “like a rock.”

Vocal skeptics of COVID-19 vaccinations gave mRNA a bad name and government funding for mRNA research is now being cut. On the flip side, at least one CEO said the pandemic also provided “elevated acceleration” for the field, which also holds promise in therapeutics for cancer and rare diseases.

The “One Big Beautiful Bill Act” includes negotiation exemptions for orphan drugs approved to treat more than one rare disease and has implications for PBMs. Also on Thursday, the White House released its MAHA report with a mission to “make our children healthy again.”

The advisory committee meeting comes days after the FDA unveiled a new risk-based approval framework for COVID-19 vaccines.

Canada’s health agency says it has been “taking all necessary action safeguard the drug supply and ensure Canadians have access to the prescription drugs they need.”

China continues to be a source of innovation as Pfizer strikes biggest pact yet; HHS provides more info on Trump’s Most Favored Nation executive order; FDA Commissioner Marty Makary and CBER director Vinay Prasad reveal new COVID-19 vaccine strategy following Novavax approval; ODAC underway after chaotic planning; more.

From a higher bar for regulatory clearance to pricing limitations, drug development is more expensive than ever. This has led firms to make tough pipeline decisions early in the development process. The result may be costly for all of us.

While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.

The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.