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Drugmakers are slow to prove medicines that got a fast track to market really work

Stalled confirmatory trials and lax enforcement plague the Food and Drug Administration's accelerated approval pathway for pharmaceuticals that target urgent medical needs.

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93 days: The summer America lost Roe v. Wade

Shefali Luthra

@shefalil

May 2

A year ago today, a leaked opinion showed us the Supreme Court planned to gut Roe v Wade. It launched an unforgettable summer, where we saw in real time the sweeping impact of losing abortion rights. Here is how it felt to live through those days:

19thnews.org

Twelve Americans — patients, doctors, politicians, activists — relived the uncertainty and chaos of losing federal abortion protections. This oral history chronicles their stories.

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The ‘open secret’ in most workplaces: Discrimination against moms is still rampant

Chabeli Carrazana

@ChabeliC_

Apr 27

🧵: Discrimination against moms is an "open secret" in many workplaces. Workers are still being laid off while on leave, pushed out of jobs or subjected to comments like, “You probably have your hands full at home." There is little legal recourse.

19thnews.org

Moms are laid off while on parental leave and subjected to stereotypes about their competency. But with few legal protections, attorneys say most cases go unreported.

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André Picard

@picardonhealth

Apr 10

Strep is bad right now — an antibiotic shortage is making it worse. Strep throat is more prevalent this year, and amoxicillin manufacturers didn't make enough of the antibiotic to go around, by

npr.org/sections/healt via

@NPRhealth

Strep is bad right now — and an antibiotic shortage is making it worse

Strep throat is more prevalent this year, and amoxicillin manufacturers didn't make enough of the antibiotic to go around.

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KHN Investigation: The System Feds Rely On to Stop Repeat Health Fraud Is Broken

Sarah Jane Tribble

@sjtribble

Mar 28

The Congressman is responding to our findings: The federal system meant to stop health care business owners and executives from repeatedly bilking government health programs fails to do so.

kffhealthnews.org

A months-long KHN examination of the system meant to bar fraudsters from Medicaid, Medicare, and other federal health programs found gaping holes and expansive gray areas through which banned indiv…

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Emily Kwong

@emilykwong1234

Mar 24

Read through this entire thread, and hire these folks.

@NPRinvisibilia

saved my life many times — and

@yowei_shaw

is an audio hero to so many (like THE reason I wanted to stay at NPR at all). If you are upset and have power in the audio industry, reach out to this team about… Show more

Quote Tweet

yowei shaw 邵友薇

@yowei_shaw

Mar 23

Well, looks like my time with NPR is up. Gutted that @NPRinvisibilia the show I love & have given 7+ yrs of my life to has "stopped production". Gutted to not get to work with my brilliant teammates. Gutted that me & some 100 colleagues I admire have lost our jobs. This sucks.

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Geoff Brumfiel

@gbrumfiel

Mar 25

Here’s how bad cuts were at

@NPR

this week: They laid off the person who says “Support for NPR provided by…” Good luck Jessica. ❤️

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Jessica Hansen

@JessActs

Mar 25

More than 100 jobs were eliminated at NPR this week. My job as Announcer and Voice Coach is one of them.

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Sacha Pfeiffer

@SachaPfeiffer

Mar 25

Two painful reporter layoffs on

@NPR

's investigative team this week:

@a_fast

&

@ChiaraEisner

. Both are smart, skilled, talented, and adored by their peers. I hope our terrible loss will be another media outlet's great fortune. Contact me for recommendations of either:

nprextra and 5 others

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Mar 9

So that Walgreens contract

@GavinNewsom

is backing out of over the abortion pill? I found it. Looks like it was worth $54 million OVER FIVE YEARS. Seems more symbolic than punitive to a co with a $30 billion (with a b!) market cap. caleprocure.ca.gov/pages/LPASearc

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Mar 8

I wondered what they'd say after hearing

@selenasd

on

@npratc

last night! I think this is the contract listing: caleprocure.ca.gov/PSRelay/ZZ_PO. Looks like it goes back to Dec. 2017. So $54 million OVER 5 YEARS? Not much to a company with a $30 billion (with a b) market cap.

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Liz Kreutz

@ABCLiz

Mar 8

.@GavinNewsom’s office just released some details on what it means for California to break ties w Walgreens. This says the state is not renewing a contract worth about $54m that allows CA to buy prescription drugs from Walgreens for use mostly by the department of corrections:

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‘How Will I Pump?’: When Your First Work Trip After Maternity Leave Is to Ukraine With President...

Sabrina Siddiqui

@SabrinaSiddiqui

Mar 8

When I found out I would be one of just two journalists traveling with President Biden to Ukraine, my first question for the White House was how I would be able to pump. My essay about going to Kyiv fresh off maternity leave:

wsj.com

For a Journal reporter and working mom, covering a historic trip to a war zone carries special meaning, and logistics.

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Ameet Sarpatwari

@AmeetSarpatwari

Mar 2

🚨New Study Alert🚨 Critical evidence when considering Pfizer and Moderna's attempts to increase the price of 'their' mRNA Covid-19 vaccines.

Quote Tweet

Hussain Lalani, MD MPH

@DrHussainL

Mar 2

NEW STUDY: From 1985 to 2019, the United States invested at least $337 million of public funding into critical research & development that directly led to mRNA Covid-19 vaccines.

We document the specific public

that were crucial for success in @bmj_latest !

1/

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Feb 28

A pregnant woman was devastated to learn one of her twins wouldn't make it to birth. Then, it got worse. "You can't do anything in Texas, & I can't tell you anything further in Texas, but you need to get out of state." New from

@selenasd

@nprscience

To safeguard healthy twin in utero, she had to 'escape' Texas for abortion procedure

When Lauren Miller found out one of her twins had a fatal condition, she discovered her doctors in Texas would only say: You need to leave the state. She went to Colorado for a selective reduction.

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🚨 JOB ALERT 🚨 Join

as our health policy editor. Work with me,

,

,

and

and a bunch of super talented reporters on some of our most important coverage. recruiting.ultipro.com/NAT1011NATPR/J

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Stacie Dusetzina

@DusetzinaS

Aug 4, 2022

"It is a really big deal" - me on

@UpFirst

@NPRHealth

talking about the drug pricing legislation that is waiting for the Senate vote. Great piece by

and with

@tricia_neuman

.

ASEAN Meeting Amid Taiwan Tensions, Victor Orbán At CPAC, Prescription Drug Prices : Up First

China held large scale live-fire military drills in six zones around Taiwan, while the US Secretary of State met with the ASEAN ministers in Cambodia. Hungary's far-right leader Viktor Orbán is...

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pink.pharmaintelligence.informa.com

Sarah Karlin-Smith

@SarahKarlin

Aug 1, 2022

ICYMI...US will pay 75% more for updated Moderna COVID booster shots despite much more limited data packages. $MRNA $PFE $BNTX

US Bivalent COVID Booster Purchases Would Cover About Half Of Population At More than 50% Price Hike

The government is paying a premium for updated vaccines despite the much smaller data packages that will be available at the time of a potential authorization and rollout. The administration also...

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And listen to the episode about this issue on #nprshortwave:

The Accelerated Approvals Process: Are Drugmakers Fulfilling Their Promises? : Short Wave

The Food and Drug Administration allows faster drug approvals based on preliminary study data if the drug fulfills an unmet medical need. But the speedy approval comes with a promise that the...

But there's no firm requirement for these studies to be ongoing at the time of accelerated approval, which means regulators, doctors and patients can be waiting YEARS for answers. Check out my story for

@npr

here:

Drugmakers are slow to prove medicines that got a fast track to market really work

Stalled confirmatory trials and lax enforcement plague the Food and Drug Administration's accelerated approval pathway for pharmaceuticals that target urgent medical needs.

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When two of its follow-up drugs, Vyondys 53 and Amondys 45, received accelerated approval, their confirmatory trial was already initiated with 75 percent enrollment, according to the company. That trial is expected to wrap up on time - a few months AHEAD of the Exondys 51 trial.

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Sarepta said it hadn’t figured out its confirmatory trial design until AFTER Exondys 51 received FDA approval, but learned from the experience.

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When I asked Sarepta about this in May, the company said the first part of the trial was already complete and it was enrolling for the second, but declined to tell me how many children were enrolled so far.

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But the study didn't START until July 2020, and isn't expected to wrap up until November 2024. (Actually, until recently, its expected completion date was listed as 2026.)

clinicaltrials.gov

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne...

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) - Full Text View.

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Exondys 51 got accelerated approval in 2016, had to complete the confirmatory study in November 2020 and submit it by May 2021. accessdata.fda.gov/drugsatfda_doc

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I asked Sarepta about its muscular dystrophy drug as part of my accelerated approvals reporting project that pubbed this month for

@nprscience

, but that part wound up getting cut because, well, the story (below) was already pretty long. npr.org/sections/healt

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M

@bio_clouseau

$SRPT will be submitting its 4th accelerated approval BLA since 2016. Yet to complete a confirmatory study for any of its previous 3 AA's investorrelations.sarepta.com/news-releases/

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Rachel Sachs

@RESachs

When FDA granted Sarepta's first accel approval, in 2016, they asked for confirmatory trials to be completed by late 2020. But results are not expected for another few yrs. FDA should be given (as it has requested) more authority to require these trials be underway at approval.

Quote Tweet

M

@bio_clouseau

$SRPT will be submitting its 4th accelerated approval BLA since 2016. Yet to complete a confirmatory study for any of its previous 3 AA's investorrelations.sarepta.com/news-releases/

Show this thread

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Kerry Dooley Young

@KDooleyYoung

#mustread Excellent deep look at the "broken bargain" for faster

@US_FDA

action on accelerated approvals. Kudos to

Quote Tweet

Would you take a drug if it was still awaiting a confirmatory study to show it really worked? What if I told you some #pharma companies take years to even START those studies? And make millions of dollars in the meantime. My latest for @npr: npr.org/sections/healt

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ICYMI,

's piece is a fantastic deep dive into the accelerated approval program - its origins, its transformation over time, and how its latest challenges pose problems for patients, physicians, and payers alike.

Quote Tweet

Would you take a drug if it was still awaiting a confirmatory study to show it really worked? What if I told you some #pharma companies take years to even START those studies? And make millions of dollars in the meantime. My latest for @npr: npr.org/sections/healt

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Ameet Sarpatwari

@AmeetSarpatwari

Deep dive into flaws in the accelerated approval process featuring insights from

@gregggonsalves

and

@akesselheim

among others. One line: "But for nearly 18 years on the market, Clolar's most important confirmatory clinical trial remained incomplete." 1/2

Quote Tweet

Would you take a drug if it was still awaiting a confirmatory study to show it really worked? What if I told you some #pharma companies take years to even START those studies? And make millions of dollars in the meantime. My latest for @npr: npr.org/sections/healt

Show this thread

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You can also listen on #nprshortwave

The Accelerated Approvals Process: Are Drugmakers Fulfilling Their Promises? : Short Wave

The Food and Drug Administration allows faster drug approvals based on preliminary study data if the drug fulfills an unmet medical need. But the speedy approval comes with a promise that the...

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And if you don't have the time to read our whole story, we've got you covered with this 5 takeaways version:

5 things to know about the FDA's flawed approach to accelerated drug approvals

An NPR investigation found stalled confirmatory trials and lax enforcement are plaguing the FDA's accelerated approval of drugs for urgent medical needs.

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You can also listen on #nprshortwave

The Accelerated Approvals Process: Are Drugmakers Fulfilling Their Promises? : Short Wave

The Food and Drug Administration allows faster drug approvals based on preliminary study data if the drug fulfills an unmet medical need. But the speedy approval comes with a promise that the...

Would you take a drug if it was still awaiting a confirmatory study to show it really works? What if you had cancer and were running out of time? My latest investigation for

@npr

tackles accelerated approvals. Check it out!

Drugmakers are slow to prove medicines that got a fast track to market really work

Stalled confirmatory trials and lax enforcement plague the Food and Drug Administration's accelerated approval pathway for pharmaceuticals that target urgent medical needs.

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