It's OK to just say your drug failed
“Vanda Pharmaceuticals Announces Positive Tradipitant Results in Atopic Dermatitis” is the title of this press release, which goes on to explain that the drug in question “was shown to improve the intensity of the worst itch patients experienced, as well as atopic dermatitis disease severity.”
That sounds quite wonderful — until you get to about 500 words into the release and notice this: The trial's primary endpoint was average itch, not intensity or severity. And on that measure, tradipitant failed.
And it wasn’t particularly close. The p-value on the primary endpoint was .306, quite a distance from the .05 required to merit statistical significance. The problem, according to Vanda, was a high placebo response and “the lack of sensitivity” of the itchiness metric the company itself chose for the trial.
"We are extremely pleased with the outcome of this study,” Vanda CEO Dr. Mihail Polymeropoulos said in the release. The company’s shares dropped about 8 percent as soon as trading resumed Wednesday evening, suggesting investors did not quite share his enthusiasm.
What is the 'real world,' really?
Drug companies are excited about using more "real world" evidence to get their medicines approved. But how, exactly? At an FDA workshop yesterday, pharma execs said they're looking forward to a future filled with more evidence collected outside of the traditional randomized controlled trials — but had few specific ideas to share.
Instead, they had questions: How good do real world data need to be? How would such data be audited? How would patients give their consent for data to be gathered outside of the context of a trial?
They're waiting on answers from the FDA.
"I do think regulatory clarity is a huge piece," said Symantha Melemed, a global product team leader in Eli Lilly's oncology unit. "It's a little bit like Captain Obvious."
Read more from Ike Swetlitz on STAT Plus.
Will nothing get us to take our pills on time?
Investors have been eager to pour their dollars into medication adherence efforts. These include pills with a digital clock built in, mailed reminders, and glow-in-the-dark pill bottles whose makers offer rewards or alerts to friends or family.
Problem is, none of these gimmicks seem to get patients to stick with their meds.
“No one’s built a great business focused particularly around this huge problem,” one CEO told STAT.
Read more from Rebecca Robbins on STAT Plus.
Why PBMs are not fond of transparency
States like California are working doggedly to expose the inner workings of pharmacy benefit managers. PBMs, not surprisingly, are doing all they can to stall them.
“There’s been huge pushback,” a California legislator working on the issue told Bloomberg. “It really makes me suspicious that there’s simply things they don’t want us to know.”
The PBMs like to use Nevada as an example: They say that state's law requiring pharma middlemen to disclose the rebates they get from insulin makers prevents them from effectively bargaining for the diabetes drugs — thereby driving up prices.
By that logic, lack of transparency in the health care system keeps drug costs low. Many a consumer would beg to differ.
- Martin Shkreli jailed for his Facebook threat against Hillary Clinton (AP)
- Biotech stocks: now more forgiving, but riskier. (Bloomberg)
- Allergan CEO worries Americans will say "enough is enough" and embrace single-payer. (The Intercept)