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The Readout Damian Garde & Meghana Keshavan

A bid to encourage ‘value-based’ drug pricing

Drug makers have long shied away value-based pricing arrangements — in which insurers pay for drugs only when they work. Their argument: Medicaid rules get in the way of these deals because of the way they force them to calculate the best price.

Now, as STAT's Nick Florko reports, the Trump administration has unveiled a proposal it thinks can encourage value-based pricing arrangements, anyway. The gist: Manufacturers would be able to calculate more than one “best-price.” 

The proposal might not leave everyone happy. Independent Medicaid experts have previously argued existing rules don't prevent value-based pricing arrangements and changes could allow drug makers to game the system.

Read more.

How long does gene therapy last?

BioMarin looks almost certain to win FDA approval for its hemophilia A gene therapy, but the latest data suggest its effects may wear off over time, clouding the question of just how valuable the one-time treatment might be.

Yesterday, BioMarin presented details on 13 patients followed for up to four years. The seven patients who got the high dose of gene therapy — the one under FDA review — saw a 95% reduction in bleeding episodes sustained over four years, and their need for doses of costly protein replacement therapy fell by about 96%.

However, their levels of Factor VIII, a protein necessary to prevent bleeds, has been on the decline. No one knows whether — or when — the benefits of the gene therapy will wear off. That probably won’t affect the FDA’s decision, expected in August, but it will weigh on the minds of patients and doctors once BioMarin’s treatment reaches the market.

Read more.

A little CAR-T keeps mice young

Scientists hoping to prolong human life have trained their attention on senescent cells, the building blocks of life that have ceased to live and instead contribute to disease of aging. And CAR-T, famous for its effects on cancer, could be an effective way to get rid of those dead cells and improve health.

As STAT’s Sharon Begley reports, researchers dosed mice with a senescent cell-hunting CAR-T therapy and saw promising results. The mice, which had fibrotic livers, saw their senescent cells nearly vanish after 10 days. Tissue scarring improved, and their liver function bounced back toward normal.

There are loads of barriers between a single mouse study and using such a CAR-T method in humans, including safety, cost, and practicality. But the positive results provide yet more evidence that senescent cells are a viable target, which lends weight to the efforts of companies including Unity Biotechnology and Velabs Therapeutics.

Read more.

Americans don’t trust pharma not raise drug prices during the pandemic

That’s according to the latest Gallup poll, which found that nearly nine in 10 Americans are worried the drug industry is going to take advantage of the coronavirus crisis to increase prices.

As STAT’s Ed Silverman reports, the results were consistent regardless of sex, race, or household income. Through the poll, which surveyed 1,016 adults in all 50 states last month, “the public is saying indisputably that the government should play a role in addressing the cost crisis and of prescription drugs, in particular,” said Dan Witters, research director and principal at Gallup.

We’ll soon find out whether those concerns were valid. Major drug companies customarily take price increases at the start and middle of the year, meaning we’re just weeks away from a regularly scheduled hike. There’s a campaign to convince pharma to skip this round of increases, and there’s a recent precedent for doing so. But so far there’s no indication the industry plans to hold off.

Read more.

June is going to be a big month for biotech IPOs

Nine biopharma companies have already gone public this month, raising more than $3 billion in the process, and there’s another seven slated to pull off debut offerings before June is over.

If everything works out, they’ll raise about $700 million combined. And the performance of their forebears bodes well. Of the nine companies that have gone public this month, all have sold shares either above or at the top of their proposed price ranges. Royalty Pharma made headlines with its record-breaking IPO, but the smaller likes of Vaxcyte, Burning Rock Biotech, and Avidity Biosciences also increased in value after pricing.

The next likely debutante is Forma Therapeutics, a hematology company expected to raise about $200 million in an IPO this week. 

More reads

  • Some Covid-19 trial sponsors never posted other study results in an EU database. Will they hide data again? (STAT)
  • The CEO of one of the world's largest generic drugmakers tells us why he's making a $50 million bet on CBD. (Business Insider)
  • For some biotech employees in the age of Covid-19, the workday starts at the bathroom sink. (Boston Globe)
  • Moderna executive stakes trimmed even before latest lockup lifts. (Bloomberg)

Thanks for reading! Until tomorrow,

Thursday, June 18, 2020


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