Global Blood's brave gambit

The stock price of Global Blood Therapeutics closed 16 percent higher Wednesday — a sign investors are satisfied with the benefit observed in sickle cell disease patients treated with the biotech’s experimental drug voxelotor in a Phase 3 clinical trial.

But scale back on that stock chart and Global Blood shares are still lower than they were at the beginning of June. The stock is also down almost 30 percent from its all-time high reached in February.

So, let’s call the Global Blood story unsettled. That could change, pending the outcome of the biotech’s risky regulatory gambit. As detailed by Adam Feuerstein, the biotech is talking with the FDA, trying to convince regulators that voxelotor data collected to date warrants an accelerated (read: speedy) approval filing. The company does not want to spend any more time conducting the second part of the voxelotor Phase 3 clinical trial, as previously planned.

If the FDA says yes, Global Blood’s stock could revisit or eclipse that February high. Sickle cell patients would get access to voxelotor much faster. But if FDA says no? The stock is heading to new lows and sickle cell patients will be forced to wait.

We finally found a question that split readers down the middle

Yesterday, in light of a thorough ProPublica story about the FDA’s increasing flexibility with drug approvals, we asked readers whether the agency was becoming too business-friendly, creating a system that speeds up the process for good and bad drugs alike.

And the result was nearly down the middle. Exactly 50.2 percent of responders said no, the FDA is doing the right thing, while 49.8 percent said yes, the agency has gone too far to appease the industry it regulates.

That's interesting because Readout subscribers tend to be people in and around the drug industry, which is to say human beings with a vested interest in drugs getting approved in a timely fashion. The fact that this audience is so evenly divided on the question of FDA leniency, even on such a laughably unscientific poll, is perhaps a tea leaf worth reading.

Speaking of fast FDA approvals

They might be more lucrative than anyone thought.

Yesterday, the federal government said that state Medicaid agencies must reimburse for drugs approved under the FDA’s accelerated program, under which companies can get treatments on the market without proving their clinical benefit.

Here’s why that’s important: When people freak out over the FDA approving new drugs on scant evidence, they are often consoled with reminders that payers serve as gatekeepers of the marketplace, only shelling out for treatments that actually work. But that explanation only makes sense if those payers can say no, and now Medicaid, a huge force on the marketplace, must be a yes man.

Read more.

'Try Kymriah, for one easy payment of $474,999.99'

Remember the Trump administration’s confusing, possibly impossible demand that drug companies put the prices of their products in TV ads? Turns out it’s very popular.

According to a poll from the Kaiser Family Foundation, 76 percent of Americans are in favor of the idea. And that cuts across party lines: 83 percent of Democrats, 73 percent of independents, and 72 percent of Republicans said they want to see dollar figures in their nightly gavage of drug ads.

All that said, there still remains the ontological question: What even is “price?” Would the ads disclose list prices, the daunting figures that don’t reflect rebates or discounts and aren’t actually paid by the vast majority of patients? Or would it list net prices, the bottom-line costs that vary widely from patient to patient depending on insurance?

Either way, “it’s completely worthless, because it has zero informational value,” one pharma consultant told STAT’s Ed Silverman. “Companies won’t determine pricing based on numbers in an ad campaign, but on what payers are willing to pay.”

Read more.

Global Blood's brave gambit

We finally found a question that split readers down the middle

Seeking young scientists with big futures

Speaking of fast FDA approvals

'Try Kymriah, for one easy payment of $474,999.99'

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