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”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

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