What's the hard cost of a piv­otal drug tri­al? A lot less than you might ex­pect

Of all the num­bers cit­ed by bio­phar­ma com­pa­nies to de­fend drug costs, $2.6 bil­lion — the es­ti­mat­ed cost of bring­ing a pre­scrip­tion drug to mar­ket — is by far the most pop­u­lar and res­o­nant. But per­haps less well-known was the Tufts re­searchers’ es­ti­mate of the av­er­age cost of Phase III tri­als, the most ex­pen­sive and ar­guably im­por­tant com­po­nent to scor­ing an OK: $255 mil­lion in 2014 dol­lars.

A new study is out to chal­lenge that.

Thomas Moore

Af­ter plug­ging de­tails about 101 new drugs ap­proved be­tween 2015 and 2017 in­to a clin­i­cal tri­al cost es­ti­ma­tor cre­at­ed by IQVIA, the au­thors con­clud­ed that the es­ti­mat­ed me­di­an piv­otal tri­al cost was $48 mil­lion, with an in­terquar­tile range of $20 mil­lion to $102 mil­lion. When you look at the 225 in­di­vid­ual tri­als, the me­di­an cost was $19 mil­lion (IQR $12 mil­lion to $33 mil­lion).

Per­haps more im­por­tant­ly, the re­search pa­per, pub­lished in the BMJ, of­fered a break­down of the fac­tors con­tribut­ing to costs.

Chief among them is the num­ber of pa­tients re­quired to es­tab­lish the treat­ment ef­fects — which var­ied wide­ly from just 4 to 8,442. The num­ber of tri­al clin­ic vis­its (2-166) al­so mat­tered, as did the type of treat­ment.

Tri­als for dis­eases where com­mer­cial drugs that have been proven to be ef­fec­tive typ­i­cal­ly re­quire more pa­tient en­roll­ment, they not­ed. Led by Thomas Moore, the sci­en­tists work at the In­sti­tute for Safe Med­ica­tion Prac­tices, a non­prof­it that mon­i­tors drug safe­ty. On av­er­age, the tri­als with an ac­tive con­trol group had 653 pa­tients, com­pared with 547 for place­bo-con­trolled stud­ies and 145 for un­con­trolled ones.

In a nod to the FDA’s in­creas­ing will­ing­ness to speed through new meds serv­ing pa­tients with few op­tions, the au­thors not­ed that 45 out of 101 drugs were ap­proved with a sin­gle tri­al — which trans­lat­ed to a low­er me­di­an cost of $28 mil­lion (IQR $13 mil­lion to $62 mil­lion). Those bur­dened with a repli­ca­tion re­quire­ment of two tri­als had an es­ti­mat­ed me­di­an cost of $45 mil­lion (IQR $28 mil­lion to $69 mil­lion).

While by no means rep­re­sen­ta­tive of the full pic­ture, the num­bers can un­der­cut phar­ma’s ar­gu­ments about the high cost of de­vel­op­ing drugs, since the piv­otal tri­als ac­count for a sig­nif­i­cant por­tion of the R&D spend­ing.

Ex­pect the usu­al de­bates and dis­cus­sions to fol­low. As the au­thors them­selves ad­mit, the num­bers are mere­ly their best es­ti­mates and not ac­tu­al costs. They al­so weren’t able to as­cer­tain all tri­al fea­tures, falling back on de­fault val­ues that could be in­ac­cu­rate. Since it fo­cused on CRO costs, it ex­clud­ed spon­sor costs for tri­al de­sign, mon­i­tor­ing or pro­vid­ing the drug it­self.

Drug de­vel­op­ers might al­so add that in pric­ing new drugs, they fac­tor in failed projects — vir­tu­al­ly al­ways out­num­ber­ing the suc­cess­ful ones — and cost of cap­i­tal.

Still, the es­ti­mat­ed costs are mod­est for es­tab­lish­ing what amounts to the path guid­ing a drug to thou­sands or mil­lions of fu­ture pa­tients. Count­ing by per pa­tient en­rolled, the cost would just be $41,413. From the BMJ pa­per:

Note that these costs-per-pa­tient for these tri­als are some­times sim­i­lar to what phar­ma­ceu­ti­cal com­pa­nies charge for these same drugs to treat a sin­gle pa­tient or a hand­ful of pa­tients af­ter mar­ket­ing ap­proval.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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