Study: Just half of post­mar­ket­ing com­mit­ment tri­als are pub­lished

A new study pub­lished Mon­day in BMC Med­i­cine finds that on­ly about half of the clin­i­cal tri­als re­quest­ed by the FDA as part of post­mar­ket­ing com­mit­ments for new­ly ap­proved drugs and bi­o­log­ics are pub­lished in peer-re­viewed jour­nals.

The study al­so found that in­for­ma­tion for near­ly half of the post­mar­ket­ing com­mit­ment stud­ies sub­ject to re­port­ing re­quire­ments un­der sec­tion 506B of Fed­er­al Food, Drug, and Cos­met­ic Act (FD­CA) was not up to date.

Con­duct­ed by re­searchers at Yale Uni­ver­si­ty, Uni­ver­si­ty of Con­necti­cut and Uni­ver­si­ty of Cal­i­for­nia, San Fran­cis­co, the study re­viewed post­mar­ket­ing com­mit­ments for the 110 new drugs and bi­o­log­ics ap­proved from 2009-2012.

Un­like post­mar­ket­ing re­quire­ments, which drug­mak­ers must con­duct as a con­di­tion of ap­proval, post­mar­ket­ing com­mit­ments are agreed to by drug­mak­ers at the time of ap­proval and are not re­quired un­der statute or reg­u­la­tion.

More than half (55.5%) of the 110 ap­provals had one or more post­mar­ket­ing com­mit­ment(s), with 33 re­quir­ing a new clin­i­cal tri­al. Of those, 27 were sub­ject to Sec­tion 506B re­port­ing re­quire­ments.

For the stud­ies sub­ject to re­port­ing re­quire­ments, 12 (44.4%) were nei­ther closed nor had an up-to-date sta­tus in pub­licly avail­able data­bas­es on the FDA’s web­site.

The study did find, how­ev­er, that near­ly all the clin­i­cal tri­als re­quest­ed as part of post­mar­ket­ing com­mit­ments were reg­is­tered on Clin­i­cal­Tri­als.gov (90.3%), most of which were marked as com­plet­ed or ter­mi­nat­ed (82.1%). Of the com­plet­ed or ter­mi­nat­ed stud­ies all but one had re­port­ed re­sults.

How­ev­er, the au­thors found that most of the stud­ies (81.8%) that had re­port­ed re­sults on Clin­i­cal­Tri­als.gov re­port­ed their re­sults af­ter the sched­uled sub­mis­sion dead­line.

On­ly about half of the stud­ies that were el­i­gi­ble for pub­li­ca­tion (i.e. com­plet­ed or ter­mi­nat­ed in Clin­i­cal­Tri­als.gov or sub­mit­ted, ful­filled or re­leased ac­cord­ing to the FDA) were pub­lished in peer-re­viewed jour­nals.

“Among the 29 reg­is­tered or un­reg­is­tered stud­ies for which pub­li­ca­tion would be ex­pect­ed based on the most re­cent sta­tus pro­vid­ed by the FDA, phar­ma­ceu­ti­cal com­pa­nies, or on Clin­i­cal­Tri­als.gov, just un­der half were pub­lished in a peer-re­viewed jour­nal (14 of 29 (48.3%)),” the au­thors write.

And while post­mar­ket­ing com­mit­ments on­ly make up 19% of the postap­proval com­mit­ments and re­quire­ments im­posed by the FDA, the au­thors say the da­ta gen­er­at­ed by post­mar­ket­ing com­mit­ments “may be a po­ten­tial­ly im­por­tant source of in­for­ma­tion about drug and bi­o­log­ic safe­ty and ef­fec­tive­ness af­ter mar­ket ap­proval.”

Study


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.

So­cial im­age: Shut­ter­stock

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

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The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

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The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

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