Da­ta sug­gest US, UK uni­ver­si­ties fall woe­ful­ly short on re­port­ing clin­i­cal tri­al re­sults

Clin­i­cal tri­al da­ta are used by pa­tients, doc­tors and pol­i­cy­mak­ers to make in­formed choic­es about the ben­e­fits and safe­ty of in­ter­ven­tions — while the meth­ods and re­sults of all tri­als are cru­cial to the pace and di­rec­tion of sci­en­tif­ic progress. How­ev­er, there is a large body of ev­i­dence that sug­gests that com­plet­ed clin­i­cal tri­als are com­mon­ly left un­re­port­ed, and ed­u­ca­tion­al in­sti­tu­tions in the Unit­ed States and the Unit­ed King­dom — ar­guably the two biggest re­gions that breed the bulk of med­ical in­no­va­tion — have emerged as one of the key cul­prits guilty of these vi­o­la­tions.

In the Unit­ed States, Con­gress passed a law in 2007 re­quir­ing tri­al spon­sors — in­clud­ing uni­ver­si­ties — to post the re­sults of cer­tain clin­i­cal tri­als on clin­i­cal­tri­als.gov with­in a year of tri­al com­ple­tion, and a decade lat­er in Jan­u­ary 2017 the rule was fi­nal­ized. Since 2017, 40 lead­ing US uni­ver­si­ties should have post­ed the re­sults of 450 clin­i­cal tri­als — but over a third (31%) of those re­sults are miss­ing, ac­cord­ing to an analy­sis by Uni­ver­si­ties Al­lied for Es­sen­tial Med­i­cines (UAEM) in part­ner­ship with non-prof­it re­search ad­vo­ca­cy group TranspariMED.

The vi­o­la­tors in­clude some of the most ac­tive tri­al spon­sors: For ex­am­ple the MD An­der­son Can­cer Cen­ter, which has on­ly re­port­ed 77% of due tri­als, Mayo Clin­ic (42%), UC San Fran­cis­co (37%), New York Uni­ver­si­ty (21%), and Co­lum­bia Uni­ver­si­ty (17%).

A sum­ma­ry of re­sults by per­cent­age of each uni­ver­si­ty eval­u­at­ed can be seen be­low:

Source: UAEM, TranspariMED

Click on the im­age to see the full-sized ver­sion

Over­all, 140 clin­i­cal tri­als are still miss­ing re­sults and five uni­ver­si­ties are re­spon­si­ble for half of the un­re­port­ed tri­als: Uni­ver­si­ty of Cal­i­for­nia San Fran­cis­co (17 tri­als with­out re­sults), Co­lum­bia (15 tri­als), Mayo Clin­ic (13), MD An­der­son Can­cer Cen­ter (12) and Chica­go (8), ac­cord­ing to the re­port.

These trans­paren­cy vi­o­la­tions are con­cern­ing con­sid­er­ing at least half of the valu­able med­i­cines that ex­ist to­day were orig­i­nal­ly de­vel­oped in uni­ver­si­ty labs with tax­pay­er fund­ing, in­clud­ing al­most all vac­cines, many HIV and tu­ber­cu­lo­sis drugs, and even in­sulin, the re­port not­ed. Be­tween 2010 and 2016, every sin­gle one of the 210 FDA-ap­proved med­i­cines can be traced back to fund­ing from the NIH, ac­cord­ing to a study pub­lished in the of­fi­cial jour­nal of the Na­tion­al Acad­e­my of Sci­ences.

Mean­while, these trans­paren­cy trans­gres­sions are echoed in the UK. Reg­u­la­tions in Eu­rope are sim­i­lar. Any tri­al of of any med­i­c­i­nal prod­uct con­duct­ed since 2004 in an EU coun­try has al­ready been re­quired to reg­is­ter on the Eu­ro­pean Union Clin­i­cal Tri­als Reg­is­ter (EU­C­TR) and since 2012, spon­sors must en­sure that all reg­is­tered tri­als since 2004 dis­close their re­sults to the EMA with­in 12 months of tri­al com­ple­tion. But the de­lays to the EMA’s soft­ware plat­form pushed the fi­nal date for re­sults post­ing by spon­sors to late De­cem­ber 2016.

Ben Goldacre

In a BMJ study pub­lished in 2018 — led by Ben Goldacre, a best-sell­ing au­thor, med­ical doc­tor and re­searcher who fo­cus­es on un­pack­ing the mis­use of sci­ence and sta­tis­tics in his books Bad Sci­ence and Bad Phar­ma — it was found that in Eu­rope, of the 7274 tri­als where re­sults were due, 49.5% re­port­ed re­sults. Tri­als with a com­mer­cial spon­sor (such as a drug de­vel­op­er) were sub­stan­tial­ly more like­ly to post re­sults than those with a non-com­mer­cial spon­sor (68.1% v 11.0%), the analy­sis sug­gest­ed.

Out of his labs at the Uni­ver­si­ty of Ox­ford, Goldacre set up an EU Tri­al­sTrack­er to con­tin­u­ous­ly mon­i­tor the re­port­ing of tri­als. As of 10 Jan­u­ary 2019, Goldacre and his team have iden­ti­fied 8,062 reg­is­tered tri­als that are ‘un­am­bigu­ous­ly’ due to re­port re­sults — but re­sults on just over half  (53.6%) have been post­ed to the reg­istry. The da­ta, which sug­gest­ed that UK uni­ver­si­ties were less re­li­able than drug de­vel­op­ers, sparked the in­ter­est of House of Com­mons Sci­ence and Tech­nol­o­gy Com­mit­tee. UK uni­ver­si­ties could be brought in front of the com­mit­tee if they fail to im­prove their track record, and the com­mit­tee will ask them to ex­plain them­selves in a fol­low-up ev­i­dence ses­sion if im­prove­ments are not made.

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.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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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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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

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.

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.”

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonucleotides, a wide variety of synthetically modified RNA or RNA/DNA hybrids that bind to a target RNA sequence to alter RNA and/or protein expression, have been winning approvals in recent years (e.g. Novartis’ cholesterol drug Leqvio), the regulatory agency is offering new draft guidance for those looking to follow a similar path.

The non-binding guidance, titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry” deals with pharmacokinetic, pharmacodynamic, and safety assessments required as part of oligonucleotide therapeutics R&D.

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