Jonathan Hirsch, Syapse founder and president

The re­al-world ev­i­dence project at the FDA adds Syapse to its list of part­ners in a push past tri­al bound­aries

The FDA is mak­ing good on its plans to ex­pand the amount and type of re­al-world ev­i­dence it has on hand to help guide its over­sight of mar­ket­ed drugs and push far past the bound­aries of clin­i­cal da­ta.

The On­col­o­gy Cen­ter of Ex­cel­lence is team­ing up with San Fran­cis­co-based Syapse to ex­pand be­yond elec­tron­ic health records — or EHRs — in build­ing a mul­ti-sourced pro­gram in­volv­ing da­ta drawn from EHRs and reg­istries, as well as mol­e­c­u­lar da­ta from test­ing labs. 

They al­so plan to eval­u­ate “re­al-world end­points for sol­id tu­mors and hema­to­log­i­cal ma­lig­nan­cies, char­ac­ter­iz­ing the us­age and clin­i­cal im­pact of mol­e­c­u­lar test­ing, un­der­stand­ing out­comes and ad­verse events in pa­tients re­ceiv­ing pre­ci­sion med­i­cines rel­a­tive to clin­i­cal tri­al pop­u­la­tions, and in­cor­po­rat­ing pa­tient-re­port­ed out­comes in­to RWE.” And they’ll be work­ing with on­col­o­gists on joint out­comes re­search. 

Ex-FDA com­mis­sion­er Scott Got­tlieb made the use of re­al-world ev­i­dence a key part of the agency’s man­date, out­lin­ing plans to gath­er da­ta on mar­ket­ed drugs to bet­ter eval­u­ate pro­posed la­bel changes as well as hunt for un­known safe­ty fac­tors that should guide pre­scrib­ing.

Sean Khozin FDA

“Re­al-world ev­i­dence from well-de­signed stud­ies meet­ing ap­pro­pri­ate da­ta qual­i­ty stan­dards can help to in­form de­ci­sion-mak­ing and pro­vide in­for­ma­tion re­gard­ing the im­pact of new ther­a­pies in re­al-world pa­tient pop­u­la­tions, par­tic­u­lar­ly those not rep­re­sent­ed in clin­i­cal tri­als,” said Sean Khozin, as­so­ciate di­rec­tor for on­col­o­gy reg­u­la­to­ry sci­ence and in­for­mat­ics in the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “This is es­pe­cial­ly crit­i­cal in pre­ci­sion med­i­cine, where un­der­stand­ing all of the fac­tors that may dri­ve safe­ty and re­sponse is both im­per­a­tive and dif­fi­cult to cap­ture at scale us­ing tra­di­tion­al clin­i­cal tri­als.”

Syapse joins CO­TA and Brigham and Women’s Hos­pi­tal in work­ing with the agency on re­al world da­ta. The FDA and Brigham and Women’s Hos­pi­tal are ex­pand­ing a pact reg­u­la­tors have with Ae­tion to see if they can use RWE — one of the buzzi­est of acronyms in bio­phar­ma — to pre­dict the re­sults of ran­dom­ized clin­i­cal tri­als that have yet to wrap up. And they’re us­ing a batch of 7 up­com­ing stud­ies to test its ac­cu­ra­cy.

“Ad­vanc­ing a deep­er un­der­stand­ing of re­al-world end­points and an­a­lyt­i­cal method­olo­gies is crit­i­cal to as­sur­ing that all stake­hold­ers can have con­fi­dence in the qual­i­ty of ev­i­dence pro­duced and ac­cel­er­at­ing the use of RWE in reg­u­la­to­ry de­ci­sion-mak­ing,” not­ed com­pa­ny founder and pres­i­dent Jonathan Hirsch in a pre­pared state­ment. “The net­work of health sys­tems that Syapse rep­re­sents of­fers a unique op­por­tu­ni­ty to learn from on­col­o­gy pa­tient jour­neys and pop­u­la­tions not well rep­re­sent­ed in tra­di­tion­al clin­i­cal tri­als.”

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.
SpringWorks

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.