Faster, prici­er and of­ten worth­less: Study slams reg­u­la­tors for wave of ques­tion­able can­cer drug OKs

The ex­plo­sion of new can­cer drugs that’s oc­curred over the past decade in­cludes some ma­jor stand­outs that in­clude ther­a­pies that have reg­is­tered some jaw-drop­ping re­sults in clin­i­cal tri­als. But when re­searchers at King’s Col­lege Lon­don and the Lon­don School of Eco­nom­ics took a step back and ex­am­ined 68 can­cer in­di­ca­tions ap­proved in Eu­rope over a 5-year pe­ri­od through 2013, they found that many were OK’d on on­ly flim­sy da­ta de­rived from un­re­li­able tri­al de­signs and at least 10 of these OKs have nev­er proved to have any re­al val­ue for pa­tients.

Kick­ing back at in­dus­try’s crit­i­cism of groups like NICE, which of­ten balk at cov­er­ing high-priced can­cer drugs, the bio­phar­ma crit­ics say that the vogue of fast-track­ing on­col­o­gy ther­a­pies has put a long line­up of drugs in­to reg­u­lar use, even though the man­u­fac­tur­ers haven’t pro­duced sol­id ev­i­dence of ef­fi­ca­cy four or more years lat­er.

Their study was pub­lished in the BMJ.

Court­ney Davis

The two au­thors — Court­ney Davis at King’s Col­lege in Lon­don, and Huseyin Naci of LSE Health — say 39 in­di­ca­tions (57%) were sanc­tioned “with­out ev­i­dence of a sur­vival or qual­i­ty of life ben­e­fit.” And they ex­co­ri­at­ed the use of sur­ro­gate end­points, say­ing they are un­re­li­able at best.

While 24 of these 68 in­di­ca­tions of­fered ev­i­dence for in­creased sur­vival, the av­er­age was less than 3 months. And on­ly 7 of 68 pro­vid­ed da­ta demon­strat­ing that they im­proved qual­i­ty of life.

“When ex­pen­sive drugs that lack clin­i­cal­ly mean­ing­ful ben­e­fits are ap­proved and re­im­bursed with­in pub­licly fund­ed health­care sys­tems, in­di­vid­ual pa­tients may be harmed, im­por­tant re­sources wast­ed, and the de­liv­ery of eq­ui­table and af­ford­able care is un­der­mined,” the re­searchers say. They added that while reg­u­la­tors have the au­thor­i­ty to pull a drug back from the mar­ket if it doesn’t demon­strate ef­fi­ca­cy, they don’t do it.

Can­cer Re­search UK cau­tioned, though, that the sys­tem on ap­prov­ing and pay­ing for can­cer drugs has un­der­gone some change in re­cent years, with a greater em­pha­sis on some of the weak spots high­light­ed in the study.

Of course, many of these same drugs are al­so ap­proved in the US. And the au­thors’ list of ques­tion­able drugs cov­ers some block­busters. It in­cludes:

→ Avastin, OK’d for ovar­i­an can­cer by the EU in 2011, they say, slap­ping re­searchers for switch­ing from over­all sur­vival to pro­gres­sion-free sur­vival.

Vinay Prasad Wash­ing­ton Post

→ Her­ceptin al­so comes in for crit­i­cism, with the re­searchers flag­ging an ap­proval for breast can­cer long be­fore it failed to demon­strate a sur­vival ben­e­fit.

→ Cel­gene’s Po­m­a­lyst was OK’d in Eu­rope af­ter reg­u­la­tors crit­i­cized their com­par­i­son of the drug com­bined with low dose dex­am­etha­sone against high dose dex­am­etha­sone.

“The ex­pense and tox­i­c­i­ty of can­cer drugs means we have an oblig­a­tion to ex­pose pa­tients to treat­ment on­ly when they can rea­son­ably ex­pect an im­prove­ment in sur­vival or qual­i­ty of life,” in­dus­try crit­ic Vinay Prasad told CRUK. The find­ings sug­gest “we may be falling far short of this im­por­tant bench­mark”

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.

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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Amgen VP of R&D David Reese

Am­gen rolls out da­ta for KRAS in­hibitor com­bo study in col­orec­tal can­cer, hop­ing to move on from ug­ly ear­ly re­sults

With the first win for its KRAS inhibitor sotorasib in hand, Amgen is pushing ahead with an aggressive clinical plan to capitalize on its first-to-market standing. The drugmaker thinks combinations — in-house or otherwise — could offer a path forward, and one early readout from that strategy is bearing fruit.

A combination of Amgen’s sotorasib and its EGFR inhibitor Vectibix posted an overall response rate of 27% in 26 patients with advanced colorectal cancer (CRC) with the KRAS-G12C mutation, according to data from the larger Phase Ib/II CODEBREAK 101 study set to present at this weekend’s virtual ESMO Congress.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Eu­ro­pean study finds that Gilead­'s Covid-19 an­tivi­ral remde­sivir shows no clin­i­cal ben­e­fit

Gilead’s remdesivir — or Veklury, as it’s marketed in the US — raked in around $2.8 billion last year as the only FDA-approved antiviral to treat Covid-19. But new data from a European study suggest the drug, which has been given to about half of hospitalized Covid patients in the country, has no actual benefit.

The open-label DisCoVeRy trial enrolled Covid-19 patients across 48 sites in Europe to test a handful of treatments, including remdesivir, lopinavir–ritonavir, lopinavir–ritonavir and interferon beta-1a, and hydroxychloroquine. To participate, patients had to show symptoms for seven days and require oxygen support. A total of 429 patients were randomized to receive remdesivir plus standard of care, while 428 were assigned to standard of care alone.

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Gri­fols drops $1B on Ger­man hold­ing com­pa­ny in con­tin­ued plas­ma push

One Spanish biotech is beefing up its plasma therapy operations, and on Friday, it announced that it’s doing so in a billion-dollar deal.

Grifols is now the largest shareholder of Biotest, a company valued at more than $1.8 billion. By teaming up, the two will try to increase the number of plasma therapies available and increase patient access around the world, Grifols said in a press release.

The company did so by acquiring holding company Tiancheng Pharmaceutical, the Germany-based owner of nearly 90% of Biotest shares, for nearly $1.27 billion. Grifols now owns nearly 90% of Biotest voting rights and almost 45% of the total share capital of Biotest.

Covid-19 roundup: FDA re­veals boost­er ad­comm ques­tion; Eli Lil­ly's an­ti­body cock­tail cleared for pre­ven­tion

The FDA released briefing documents this week from the agency and Pfizer each outlining their arguments for today’s Covid-19 booster shot adcomm, but one thing conspicuously missing was the question on which panel members would be voting. But late Thursday night, regulators published that question.

Adcomm members will be asked whether or not the safety and efficacy data from Pfizer/BioNTech’s original Phase III study “support approval” of a booster shot at least six months after the second dose in individuals older than 16. The question notably excludes the real-world data from Israel and other analyses that Pfizer and the Biden administration had said would be a centerpiece of their arguments for boosters.

A Pfiz­er part­ner wel­comes ex-ADC Ther­a­peu­tics CMO Jay Fein­gold to the team; Amid tough sled­ding, Im­muno­vant choos­es Eli Lil­ly alum as CFO

→ Last week we told you about the CMO revolving door at ADC Therapeutics, as Joseph Camardo replaced the departing Jay Feingold. The next opportunity for Feingold in the CMO slot has opened up at antibody-drug conjugate and mAb developer Pyxis Oncology, which has added several new execs and scientific advisory board members in recent months, including ex-Immunovant CFO Pamela Yanchik Connealy. Before his tenure at ADC, Feingold was Daiichi Sankyo’s VP of US medical affairs and chairman of the Global Medical Affairs Oversight Committee. Within weeks in March, Pyxis struck a licensing deal with Pfizer for two of its ADCs and raked in $152 million from a Series B round.

Multiple antibiotic resistant Pseudomonas aeruginosa bacterium

A new way to in­fil­trate (and de­stroy) some of the dead­liest drug-re­sis­tant bugs

About four years ago, Ruben Tommasi, the gregarious scientific chief of antibiotics startup Entasis, walked into a meeting with his top chemist and top biologist to chew over another batch of unchanging results.

“It felt like we were running the same experiment over and over,” Tommasi told Endpoints News. “We had all sort of come to that point in time where we felt like we were banging our heads against the wall.”

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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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