Pub­lic funds help late-stage de­vel­op­ment of about 25% of new drugs, study finds

One in four new drugs ap­proved in the US be­tween Jan­u­ary 2008 and De­cem­ber 2017 re­ceived di­rect fund­ing from pub­lic re­sources for late stage re­search or through spin-off com­pa­nies cre­at­ed from pub­lic re­search in­sti­tu­tions, a study pub­lished in the BMJ on Wednes­day found.

As the drug pric­ing de­bate has ac­cel­er­at­ed in re­cent months, the de­bate over whether pub­lic or pri­vate en­ti­ties do the ma­jor­i­ty of drug de­vel­op­ment work has con­tin­ued, with the gen­er­al as­sump­tion that the Na­tion­al In­sti­tutes of Health (NIH) funds much of the ear­ly-stage re­search (the Con­gres­sion­al Re­search Ser­vice ex­plained in April how NIH aids ba­sic re­search that trans­lates in­to phar­ma­ceu­ti­cal de­vel­op­ment) while bio­phar­ma­ceu­ti­cal com­pa­nies most of­ten fund the lat­er clin­i­cal stages that lead to the ap­proval and mar­ket­ing of new drugs.

Re­searchers at the Pro­gram On Reg­u­la­tion, Ther­a­peu­tics, And Law (POR­TAL) at Har­vard Med­ical School in­ves­ti­gat­ed 248 nov­el drugs in the 10-year win­dow and found that 62 (25%) had doc­u­ment­ed late-stage re­search con­tri­bu­tions from a pub­licly sup­port­ed re­search in­sti­tu­tion or spin-off com­pa­ny. Most of the drugs were iden­ti­fied as hav­ing pub­licly spon­sored re­search or spin-off con­tri­bu­tions through patent da­ta avail­able through the Or­ange Book.

In ad­di­tion, 48 of the drugs (19% of all new ap­provals) had ev­i­dence of di­rect pub­licly sup­port­ed re­search, and all but one re­ceived fund­ing re­lat­ed to the drug’s “ini­tial dis­cov­ery, syn­the­sis or oth­er key in­tel­lec­tu­al prop­er­ty lead­ing to a patentable in­ven­tion.” Of these 48 drugs, 38 (80%) had at least one patent held by a pub­licly sup­port­ed re­search in­sti­tu­tion or spin-off com­pa­ny.

“These pub­licly spon­sored drugs were more like­ly to re­ceive ex­pe­dit­ed reg­u­la­to­ry des­ig­na­tion and be first in class, sug­gest­ing high ther­a­peu­tic im­por­tance,” the re­searchers wrote.

And the oth­er 14 (6%) drugs de­vel­oped by spin-off com­pa­nies were based whol­ly or in part on pub­licly sup­port­ed re­search, ac­cord­ing to the study. For a spin-off ex­am­ple, the study dis­cuss­es Gilead’s he­pati­tis C treat­ment So­val­di (so­fos­bu­vir), which orig­i­nat­ed at Phar­mas­set (ac­quired lat­er by Gilead), but was orig­i­nal­ly a spin-off com­pa­ny based on fed­er­al­ly fund­ed re­search from Emory Uni­ver­si­ty in At­lanta.

“Iden­ti­fi­ca­tion of drugs with late stage, pub­licly sup­port­ed re­search con­tri­bu­tions, par­tic­u­lar­ly those for which such in­sti­tu­tions hold key patents, could rep­re­sent a use­ful pol­i­cy lever. Such drugs in­clude nusin­ersen (Spin­raza, for spinal mus­cu­lar at­ro­phy; list price US$750,000 (£610,400; €685,000) in the first year of use), eliglu­s­tat (Cerdel­ga, for Gauch­er dis­ease; $310,250/year), and en­za­lu­tamide (Xtan­di, for prostate can­cer; $129,000/year). The prices of these drugs, each of which re­lied on sub­stan­tial aca­d­e­m­ic de­vel­op­ment, have been crit­i­cized in the US and all are sub­stan­tial­ly low­er in oth­er coun­tries,” the au­thors note.

They al­so ex­plain how us­ing so-called “march-in” rights, which could al­low the gov­ern­ment to use a patent­ed prod­uct for its own pur­pos­es and which pres­i­den­tial hope­ful Eliz­a­beth War­ren has said she would use, could help fight “crit­i­cal drug short­ages or ex­treme price hikes.”

The au­thors wrote in the con­clu­sion: “These find­ings pro­vide ad­di­tion­al da­ta for the on­go­ing de­bate on sup­port for pub­lic sec­tor bio­med­ical re­search, and the best ways to take these key con­tri­bu­tions in­to ac­count in de­ter­min­ing the own­er­ship of and fair prices for new drugs, es­pe­cial­ly those priced at very high lev­els.”


RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

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.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.