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.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

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Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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