New re­port her­alds a rev­o­lu­tion in can­cer R&D trends - and the stick­er shock that comes with it

There’s a rev­o­lu­tion play­ing out in can­cer R&D.

Over the last decade drug de­vel­op­ers fo­cused on on­col­o­gy have teamed with reg­u­la­tors on de­sign­ing a short­er R&D path for new can­cer drugs, us­ing tri­al de­signs that have sped clin­i­cal work and slashed the num­ber of pa­tients need­ed to demon­strate safe­ty and ef­fi­ca­cy.

With the num­ber of new check­point com­bos in the clin­ic es­ca­lat­ing rapid­ly, the late-stage pipeline of new can­cer drugs has ex­plod­ed, grow­ing 7.7% in just the last year to 631 new mol­e­cules. The two ini­tial PD-1 ap­provals of Op­di­vo and Keytru­da alone spawned 135 com­bi­na­tion stud­ies. And the work over­all is chang­ing the stan­dard of care in the field — at a high cost for the pay­ers foot­ing the bill.

Those are all con­clu­sions laid out in a new re­port from Quin­tiles­IMS, which con­ve­nient­ly de­tailed the ma­jor trends in can­cer R&D just a cou­ple of days ahead of the bell­wether AS­CO con­fer­ence in Chica­go.

Over the past 5 years, 68 new can­cer drugs have been ap­proved, mark­ing a sig­nif­i­cant im­prove­ment in out­comes for pa­tients, par­tic­u­lar­ly in the area of metasta­t­ic dis­ease, notes Mur­ray Aitken, the au­thor of the re­port and ex­ec­u­tive di­rec­tor of the Quin­tiles­IMS In­sti­tute.

Mur­ray Aitken

Aitken high­lights the role of the FDA’s 2012 Break­through Ther­a­py Des­ig­na­tion ini­tia­tive in help­ing change the path of can­cer drug R&D, along with read­ier use of fast track sta­tus and pri­or­i­ty re­views. With reg­u­la­tors will­ing to as­sist rapid drug de­vel­op­ment plans, the num­ber of pa­tients en­rolled in Phase III stud­ies dropped from 671 18 years ago to 188 in 2016 as in­ves­ti­ga­tors fo­cused on small­er sub­pop­u­la­tions.

Tri­al du­ra­tion in Phase III has been halved, drop­ping from 2000 days in the late ‘90s to a lit­tle more than 1000.

Drug de­vel­op­ers haven’t over­looked the op­por­tu­ni­ty. The num­ber of new drugs in the can­cer pipeline has swelled by 45% over the past decade, with a grow­ing num­ber of these ex­per­i­men­tal drugs us­ing bio­mark­ers to pre­dict pa­tient re­spons­es; 87% of these drugs count as tar­get­ed ther­a­pies.

None of this is free.

Glob­al spend­ing on can­cer drugs swelled from $107 bil­lion to $113 bil­lion in just one year. Over the past 5 years the an­nu­al growth rate in spend­ing on can­cer ther­a­pies has been 8.7% per year, sig­nif­i­cant­ly high­er than the 4.9% rate of the 5 years pri­or to that. And by 2021 to­tal glob­al spend­ing on can­cer drugs and sup­port­ive care will hit $147 bil­lion.

“We are see­ing pric­ing con­ces­sions,” Aitken told re­porters in a con­fer­ence call on Wednes­day, in­tro­duc­ing the re­port. Those con­ces­sions have re­duced the lev­el of net price growth for can­cer drugs to 3.6% last year, down from the 5% rate they record­ed in 2014 and 2015.

As new com­bi­na­tion drugs wind their way through the clin­ic, there’s been con­sid­er­able un­ease in bio­phar­ma about the cost im­pli­ca­tions of meld­ing ex­pen­sive new ther­a­pies in­to one pow­er­ful cock­tail. That de­bate is just be­gin­ning.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.