Aca­d­e­mics and re­searchers raise con­cerns with FDA’s plan for ‘in­te­grat­ed re­views’

More than 50 aca­d­e­mics and re­searchers from Har­vard, Yale, Johns Hop­kins and oth­er uni­ver­si­ties around the world are call­ing on the FDA to not re­place its orig­i­nal re­views of med­ical prod­ucts with an “in­te­grat­ed re­view” be­cause of the valu­able in­for­ma­tion that would be lost.

The re­searchers claimed that such a shift would de­prive them of in­for­ma­tion and da­ta on the clin­i­cal stud­ies and tri­als sub­mit­ted to the FDA, in­for­ma­tion on the post­mar­ket­ing re­quire­ments, and re­view­er con­cerns with an ap­pli­ca­tion that might not be in­clud­ed in a sum­ma­ry doc­u­ment like the in­te­grat­ed re­view, among oth­er im­por­tant de­tails.

“The FDA should not re­place in­di­vid­ual re­views with an in­te­grat­ed re­view. Rather, an ad­di­tion­al doc­u­ment that sum­ma­rizes the in­di­vid­ual re­views can be pub­lished, as FDA has al­ready been do­ing in pub­lish­ing ‘Sum­ma­ry Re­view’ doc­u­ments,” the re­searchers wrote.

Sim­i­lar­ly, non­prof­it Pub­lic Cit­i­zen said it “strong­ly ob­jects” to the FDA’s pro­pos­al, which was first ex­plained in a June Fed­er­al Reg­is­ter no­tice. “This ill-con­ceived pro­pos­al would, at the least, be a ma­jor step back­wards in agency trans­paren­cy with re­spect to the da­ta that the agency re­lies on when ap­prov­ing new drugs or bi­o­log­ics and the agency’s as­sess­ment of these da­ta,” Pub­lic Cit­i­zen said.

Health tech­nol­o­gy com­pa­ny Flat­iron Health al­so not­ed that some of the sec­tions in the orig­i­nal doc­u­ment ap­pear to be miss­ing in the new in­te­grat­ed sum­ma­ry. “We al­so note that the new doc­u­ment is con­sid­er­ably short­er than the orig­i­nal, which sug­gests that some po­ten­tial­ly use­ful con­tent may be lost in the new for­mat. We urge FDA to en­sure that high­ly valu­able in­sight in­to re­view­ers’ think­ing is re­tained in the new in­te­grat­ed re­view for­mat. We are par­tic­u­lar­ly in­ter­est­ed in en­sur­ing that any ‘con­sul­ta­tive’ re­views from ex­perts out­side the core re­view team are in­clud­ed in the in­te­grat­ed sum­ma­ry re­view or oth­er­wise ac­ces­si­ble,” the com­pa­ny said.

In­dus­try group BIO, how­ev­er, said the new in­te­grat­ed tem­plate is an im­prove­ment over what the agency of­fers now, but al­so cau­tioned the FDA “to strict­ly lim­it dele­tions to the in­for­ma­tion re­peat­ed across re­view doc­u­ments rather than in­for­ma­tion that FDA deems to not be im­por­tant to ex­ter­nal en­ti­ties.”

BIO of­fered the ex­am­ple of the FDA re­cent­ly trim­ming the con­tent of pub­licly post­ed ac­tion pack­ages, which led to the elim­i­na­tion of “a sig­nif­i­cant num­ber” of meet­ing min­utes. In­dus­try group PhRMA al­so said it is im­por­tant for the FDA to en­sure that the com­bi­na­tion of the in­te­grat­ed re­view doc­u­ment and its ap­pen­dices “is no less com­pre­hen­sive than the ex­ist­ing doc­u­men­ta­tion that is post­ed on Drugs@FDA.”

BIO added: “These doc­u­ments for non-mile­stone de­vel­op­ment phase meet­ings, mid­cy­cle com­mu­ni­ca­tions, and late-cy­cle meet­ings are im­por­tant for stake­hold­ers’ un­der­stand­ing of the evo­lu­tion of the FDA’s think­ing dur­ing the de­vel­op­ment pro­gram and through­out the Agency’s re­view of an ap­pli­ca­tion. Like­wise, ad­min­is­tra­tive and cor­re­spon­dence doc­u­ments should con­tin­ue to be made avail­able.”

On the top­ic of the FDA con­tin­u­ing to re­lease spon­sors’ clin­i­cal study re­ports (CSRs), which the agency is con­sid­er­ing end­ing, the aca­d­e­mics and re­searchers al­so dis­agreed with BIO on what to do.

The aca­d­e­mics and re­searchers of­fered their sup­port for the FDA to con­tin­ue its ef­forts to re­lease CSRs, even though on­ly one has been re­leased so far. But BIO and PhRMA said that the re­lease of CSRs should not be pur­sued by the agency.

BIO al­so called on the FDA to work with the Eu­ro­pean Med­i­cines Agency and Health Cana­da to bet­ter align its trans­paren­cy ef­forts.

Com­ments


RAPS: 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.

Author

Zachary Brennan

managing editor, RAPS

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,800+ biopharma pros reading Endpoints daily — and it's free.

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,800+ biopharma pros reading Endpoints daily — and it's free.

Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,800+ biopharma pros reading Endpoints daily — and it's free.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,800+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.