Thumbs up: Can­cer drug R&D is ex­plod­ing, get ready for the pric­ing de­bate to fol­low


I ar­rived in Chica­go last night, still try­ing to sort out how to pri­or­i­tize the news com­ing out of AS­CO this week­end. In the lead up, I had a chance to do a pair of sto­ries that looked at new re­ports cov­er­ing the rapid pro­lif­er­a­tion of PD-(L)1 com­bos along with a 7.7% one-year growth rate in late-stage can­cer drug stud­ies, com­ing in at 631 piv­otal-stage ef­forts.

This didn’t hap­pen by ac­ci­dent.

Over the last few years we’ve seen a tru­ly rev­o­lu­tion­ary change in the way on­col­o­gy drugs are de­vel­oped. Backed in par­tic­u­lar by the FDA’s break­through pro­gram, now lib­er­al­ly ap­plied along with fast-track sta­tus and pri­or­i­ty re­views, the bio­phar­ma in­dus­try is com­pet­ing like nev­er be­fore to carve out new ter­ri­to­ry in can­cer treat­ment.

I well re­mem­ber a lot of the ini­tial hoot­ing from some an­a­lysts at the idea that the FDA’s BTD pro­gram would make a dif­fer­ence. But no one is hoot­ing now. The agency — and in par­tic­u­lar Richard Paz­dur — can­did­ly em­braced an open-door ap­proach on can­cer R&D. And that’s the path FDA com­mis­sion­er Scott Got­tlieb wants the rest of the FDA di­vi­sions to fol­low.

Richard Paz­dur

When a bu­reau­cra­cy sets new goals, things change. So I’ll be ex­pect­ing lots of fresh ac­tion on the BTD front over the next few years, for all kinds of dis­eases.

There are bil­lions of dol­lars at stake here, which is as it should be. The greater the pay­back, the greater the R&D in­vest­ment and the greater the re­wards for pa­tients, who are see­ing some im­por­tant and some­times dra­mat­ic im­prove­ment in the way many can­cers are treat­ed.

Out of this rev­o­lu­tion, though, will come plen­ty of added pres­sure on drug mak­ers to find a way to rea­son­ably price these new drugs and the com­bos com­ing down the pipeline. Af­ter get­ting side­lined at the Trump ad­min­is­tra­tion, changes on pub­lic pol­i­cy around drug pric­ing may well get lost in all the shout­ing in DC these days.

But you can be sure that pay­ers haven’t lost their fo­cus, es­pe­cial­ly as six-fig­ure com­bos start to make the rounds. So the in­dus­try will need to have a more co­her­ent ap­proach to pay for per­for­mance.

On­col­o­gy now rep­re­sents the po­ten­tial that drug R&D has in sig­nif­i­cant­ly im­prov­ing the stan­dard of care in a short pe­ri­od of time. Next, it has to lead the way on mar­ket-based so­lu­tions to the pric­ing de­bate.

That next part could prove to be much, much more chal­leng­ing.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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