April 7, 2023

international pricing

Why a Brazilian ad comm voted against CF drug Trikafta

A committee advising the Brazilian government said it shouldn't pay for Trikafta, a cystic fibrosis drug made by Vertex Pharmaceuticals. Although Trikafta is a potent medicine that treats about 90% of people with the rare genetic disease, Vertex is finding it difficult to sell the drug in low- and middle-income countries because of its high price tag. In Brazil, Vertex hopes to charge $126,000 per patient per year, which is too steep for the country, patient advocates say.

"It is deeply disappointing, if unsurprising, that Vertex's exorbitant price demands for Trikafta have been ruled as unaffordable by the Brazilian health authorities," one patient advocate said. "It is not acceptable that [the lives of cystic fibrosis patients in Brazil] should continue to be held ransom by Vertex's monopoly on this medicine."

car-t expanded

Newly identified risks from brain cancer immunotherapy

It's becoming increasingly possible to bring cellular immunotherapies like CAR-T to cancers of the brain. But the inflammation and other toxicities inherent to the CAR-T process can be even riskier when treating brain tumors — since untreated swelling can damage vital structures and cause serious neurological symptoms.

Stanford researchers tested a CAR-T therapy for diffuse midline glioma in children three years ago. "These are nearly universally fatal cancers," investigator Michelle Monje told STAT. "When we started the trial, we saw responses right away, more than we'd seen in any other context. What was amazing as a neurologist and oncologist was to see that kids got better." However, they published a study in Nature Medicine this week showing the toxicities unique to using immunotherapy to treat nervous system tumors.

The researchers call the new syndrome tumor inflammation-associated neurotoxicity, or TIAN, and they've created a grading scale to categorize severity. "It's kind of an electrical problem, if you will. Neural circuits are being temporarily compromised due to active inflammation," Monje said. "The other kind of TIAN is related to swelling."

AI

Can ChatGPT help unravel NASH?

NASH, or nonalcoholic fatty liver disease, has researchers stumped. Although it is extremely common and causes cirrhosis and loss of liver function, there are no approved treatments for the drug. Researchers don't even know what causes NASH. So they're turning to artificial intelligence to help probe for answers, using tools like ChatGPT. The idea is to perhaps integrate microscopic observations, from gene expression to clinical features, to identify patterns a human doctor might miss.

"And that's not something we can wrap our heads around conceptually, except that we will test these paradigms to see if they actually are more predictive," said hematologist Scott Friedman, who is leading the effort.