Clin­i­cal tri­al spon­sors have to dis­close decade’s worth of un­re­leased da­ta, fed­er­al judge rules

A decade’s worth of un­re­leased tri­al da­ta may soon see the light of day.

A New York fed­er­al judge ruled this week that the FDA and the NIH have for years mis­in­ter­pret­ed a law that would re­quire com­pa­nies, uni­ver­si­ties and oth­er clin­i­cal tri­al spon­sors to re­lease tri­al da­ta from stud­ies com­plet­ed be­tween 2007 and 2017. The rul­ing cov­ers drugs and med­ical de­vices that were ex­per­i­men­tal when the study was com­plet­ed but have since been ap­proved, po­ten­tial­ly putting hun­dreds of spon­sors out of com­pli­ance if they don’t put their re­sults on clin­i­cal­tri­als.gov.

The FDA had been in­ter­pret­ing a ‘fi­nal rule’ added to 2007 law, known as the Food and Drug Ad­min­is­tra­tion Amend­ments Act, to mean that spon­sors on­ly had to re­port re­sults for tri­als that were com­plet­ed af­ter the law rule was pro­mul­gat­ed in 2017. Plain­tiffs said it was retroac­tive.

“The court has set aside that er­ro­neous in­ter­pre­ta­tion of the law and has said that the statute means what it has al­ways said,” Christo­pher Morten, the lawyer for the plain­tiffs, told End­points News. “So our hope here is that tri­al spon­sors are go­ing to start, fi­nal­ly, af­ter years of non­com­pli­ance, re­port­ing some of that miss­ing da­ta to the pa­tients.”

The FDA and the NIH did not put out state­ments fol­low­ing the rul­ing and did not im­me­di­ate­ly re­spond to re­quests for com­ment.

FDAAA was the law that re­quired spon­sors to reg­is­ter most tri­als on clin­i­cal­tri­als.gov, and in 2018 the change went in­to ef­fect re­quir­ing that com­pa­nies post re­sults with­in a year of the tri­al’s com­ple­tion. The gov­ern­ment in­ter­pret­ed it to mean on­ly the re­sults of tri­als com­plet­ed af­ter the law went in­to ef­fect.

The FDA and NIH’s ex­e­cu­tion of that rule, though, has been the sub­ject of ma­jor scruti­ny in the last few months. In­ves­ti­ga­tions in The Lancet and in Sci­ence pub­lished in Jan­u­ary found that tri­al spon­sors have wide­ly ig­nored the re­port­ing re­quire­ments since 2018. The Lancet study, by Ben Goldacre, found on­ly 41% of over 4,700 tri­als re­port­ed on time.  An­a­lyz­ing a sim­i­lar dataset, Sci­ence’s Charles Piller found 45% non­com­pli­ance and iso­lat­ed a group of 30 “ha­bit­u­al of­fend­ers” who col­lec­tive­ly failed to re­port the re­sults 67% of the time.

Ad­vo­cates for greater trans­paren­cy say this kind of ob­fus­ca­tion hurts the field by giv­ing doc­tors and re­searchers on­ly a par­tial glimpse at the over­all re­sults. It can even be dan­ger­ous, Morten ar­gued. He cit­ed Vioxx, the painkiller Mer­ck pulled off the mar­ket in 2004 af­ter stud­ies showed an in­creased risk of stroke. Had Mer­ck dis­closed pre-ap­proval tri­al re­sults soon­er, Morten said, the risk could have been caught soon­er.

The case was brought by for­mer as­so­ciate FDA com­mis­sion­er Pe­ter Lurie and NYU jour­nal­ist Charles Seife, who ar­gued a mis­in­ter­pre­ta­tion of the rule has im­ped­ed their work more broad­ly. The rul­ing takes the re­port­ing re­quire­ments pre­vi­ous­ly ap­plied on­ly to tri­als com­plet­ed since 2018 and ap­plies them to those com­plet­ed since 2007.

“That’s hurt pa­tients who lack the op­por­tu­ni­ty to learn about the drugs they take, it’s hurt doc­tors that lack in­for­ma­tion on the drugs they pre­scribe, and it’s hurt re­searchers like Charles and Pe­ter,” Morten said.

It’s not clear, though, how the rul­ing will be en­forced. The plain­tiffs were over­ruled on a sep­a­rate is­sue, in which they ar­gued the FDA and NIH were not abid­ing by a statute re­quir­ing them to give pub­lic no­tice of non­com­pli­ance to com­pa­nies not abid­ing by the FDAAA rules.

The gov­ern­ment could yet ap­peal the rul­ing but it’s not yet clear if they will.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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Stronger to­geth­er? Boehringer and Mi­rati team to put first KRAS-KRAS com­bo in the clin­ic

Researchers are still waiting to see how much any of the vaunted KRAS drugs now in the clinic can, after decades of preclinical research and some early human studies, help patients. But while they do, two of the leading developers will look to see whether a KRAS-KRAS combo might pose a better shot than any KRAS alone.

Boehringer Ingelheim and Mirati have signed a collaboration to combine Mirati’s closely-watched lead KRAS inhibitor, MRTX849, in a clinical trial with the pan-KRAS blocker that Boehringer has quietly developed with high expectations behind their flashier contenders.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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Christian Itin, Autolus CEO (Autolus)

#ES­MO20: Au­to­lus pro­vides glimpse of next-gen­er­a­tion CAR-T pro­gram, show­ing ear­ly pos­i­tive safe­ty da­ta

CAR-T therapies were hailed as a breakthrough when Novartis received the first FDA approval for Kymriah back in 2017. Though highly effective at treating certain types of blood cancers, CAR-Ts are also associated with severe and potentially deadly side effects, including lethal instances of cytokine release syndrome.

With this in mind, Autolus Therapeutics is looking to take a crack at a safer CAR-T and presented Phase II cohort data for its AUTO3 program at virtual ESMO 2020. The data showed that, among the 35 patients in the cohort being treated for r/r diffuse large B cell lymphoma, there were no instances of Grade 3 or higher CRS. Eight individuals saw Grade 1 inflammation while another four patients reached Grade 2.

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Sean Bo­hen's break from bio­phar­ma is over. The ex-As­traZeneca CMO has re­tired his Big Phar­ma jer­sey and is now — hap­pi­ly — run­ning a lit­tle biotech

The last I had heard about Sean Bohen, he had stepped out of his high-profile job as chief medical officer at AstraZeneca at the beginning of 2019 as CEO Pascal Soriot triggered a broad-ranging R&D shakeup. And then, earlier this week, I got a chance to catch up.

It turns out that Bohen decided at the time that he would not just jump into a new job in the booming biopharma business. As an oncologist, he had worked on the big programs at AstraZeneca, and before that he was at Genentech. That was good for a ticket to just about anyplace in the big biopharma world. But he felt it was time to stop and think things through.

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Covid-19 roundup: UP­DAT­ED: Amid calls for trans­paren­cy, Mod­er­na re­leas­es full PhI­II pro­to­col; As­traZeneca tri­al halt thought to be 'con­firmed trans­verse myelitis,' per CNN

Prominent scientists, public health experts and industry critics have been calling for more transparency from vaccine makers, including the release of the companies’ Phase III trial protocols. On Thursday, Moderna became the first major developer to do so.

“It is usually not what industry does,” CEO Stéphane Bancel told Endpoints News in an interview in advance of their R&D day today. “But given the need there is for the scientific community and the medical community and the media to understand how the vaccine is being developed, to make sure people are confident in the safety of the process, in the governance with scientific independence in the medical world and the like, we’re dropping tomorrow the full protocol. It is unredacted.”

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