A pharmacist at Whalen's Drug Store, 55 E 42nd Street, is shown putting up a sign advertising the fact that they now have the wonder drug, penicillin, in stock. However, New Yorkers soon learned that the potent germ killer cannot be bought across the counter like liver pills but only by prescription and that it must be administered by a physician. (March 1945 — via Getty Images)

FDA sur­vey finds Amer­i­cans don’t re­al­ly un­der­stand drug ap­provals

On­ly 25% of Amer­i­cans un­der­stand that a new drug ap­proved by the FDA does not nec­es­sar­i­ly mean the drug will help most peo­ple who use it, ac­cord­ing to the re­sults of a sur­vey con­duct­ed in 2017 and pub­lished last week in Phar­ma­coepi­demi­ol­o­gy & Drug Safe­ty.

How­ev­er, FDA’s sur­vey of a na­tion­al­ly rep­re­sen­ta­tive group of 1,744 US adults did find that more than half un­der­stood that the reg­u­la­tor ap­proves pre­scrip­tion drugs when the drug of­fers ben­e­fits that out­weigh the risks, al­though 17.5% thought that an FDA-ap­proved drug will cure the con­di­tion for which it’s pre­scribed.

“Fur­ther in­ves­ti­ga­tion in­to pub­lic knowl­edge of FDA’s OTC [over-the-counter] drug and di­etary sup­ple­ment ap­proval over­sight yield­ed ad­di­tion­al ev­i­dence of a lack of un­der­stand­ing re­lat­ed to the ap­proval of such prod­ucts. We found that 69.3% of re­spon­dents thought that FDA ap­proves all over‐the‐ counter drugs. One quar­ter of re­spon­dents (24.9%) be­lieved FDA ap­proves di­etary sup­ple­ments and an­oth­er quar­ter (24.5%) did not know,” the sur­vey’s au­thors found.

In ad­di­tion to the find­ings on the ap­provals, the re­searchers al­so sought to un­der­stand how the pub­lic views FDA’s role in reg­u­lat­ing di­rect-to-con­sumer (DTC) pre­scrip­tion drug ad­ver­tis­ing.

FDA re­searchers found less of an un­der­stand­ing with DTC ads, not­ing that 31% in­cor­rect­ly thought that FDA ap­proves DTC ad­ver­tis­ing, and less than half in­cor­rect­ly thought that risk or ben­e­fit state­ments in ads are FDA‐ap­proved.

Al­though the re­searchers said the sur­vey aligns with pre­vi­ous sur­veys, they al­so said it sug­gests “that some of the mis­con­cep­tions held by con­sumers may have a neg­a­tive im­pact on pub­lic health. For in­stance, we found that 42.9% of con­sumers were not able to ac­cu­rate­ly re­port that FDA‐ap­proved pre­scrip­tion drugs may cause harm. This is con­cern­ing be­cause some ad­verse events re­quire dis­con­tin­u­a­tion or dose mod­i­fi­ca­tion. More­over, ad­verse event re­port­ing is crit­i­cal to phar­ma­covig­i­lance and un­der‐re­port­ing of ad­verse events is a con­sis­tent prob­lem world­wide.”

They al­so ex­plained how there is an op­por­tu­ni­ty to en­hance con­sumers’ un­der­stand­ing of drug ap­provals, FDA’s role and over­sight of pre­scrip­tion drug ad­ver­tise­ments.

As far as po­ten­tial ways to im­prove the pub­lic’s un­der­stand­ing on drug ap­provals, one of the sug­ges­tions is to place ban­ner ads on gen­er­al health ref­er­ence web­sites, like Drugs.com, Web­MD.com and may­oclin­ic.org, with short mes­sages of­fer­ing key facts about the ap­proval process and reg­u­la­tion of ad­ver­tise­ments.

First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. 

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Skin tu­mors in mice force Pro­tag­o­nist to halt lead pro­gram, crush­ing stock

Protagonist Therapeutics just can’t catch a break.

Six months after the Newark, CA-based biotech unveiled grand plans to launch its lead candidate for blood disorders into a Phase III trial, the FDA has slapped the program with a clinical hold. The halt — which applies to all trials involving the candidate, rusfertide — comes after skin tumors were discovered in mice treated with the drug, according to Protagonist.

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Take­da scores a win for a rare type of lung can­cer, gear­ing up for a show­down with J&J

Four months after J&J’s infused drug Rybrevant scored the industry’s first win in a rare type of non-small cell lung cancer (NSCLC), Takeda is following up with an oral option for the small but desperate patient population.

The FDA granted an accelerated approval to Takeda’s oral TKI inhibitor Exkivity (mobocertinib) in metastatic NSCLC patients with EGFR exon 20 gene mutations who had previously undergone platinum-based chemotherapy, the company announced on Wednesday.

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