FDA and EMA push ahead with joint ef­forts to kick-start pe­di­atric can­cer drug de­vel­op­ment plans

As part of an ef­fort to ease the process­es around pe­di­atric can­cer drug de­vel­op­ment, the FDA and Eu­ro­pean Med­i­cines Agency on Wednes­day un­veiled a new tem­plate to pro­vide com­pa­nies with an eas­i­er way to seek sci­en­tif­ic ad­vice from both agen­cies.

The FDA and EMA have more close­ly aligned their time­lines for the re­quired sub­mis­sions of plans for pe­di­atric de­vel­op­ment of new can­cer drugs, which has opened up an op­por­tu­ni­ty for spon­sors to seek pre­lim­i­nary sci­en­tif­ic ad­vice from both reg­u­la­tors si­mul­ta­ne­ous­ly.

The push comes as can­cer re­mains the lead­ing cause of death from dis­ease in chil­dren, even de­spite ad­vances that have pushed the 5-year sur­vival rate to about 85%, of­fi­cials from both the FDA and EMA wrote in an ar­ti­cle pub­lished in the Jour­nal of Clin­i­cal On­col­o­gy last Sep­tem­ber.

De­spite some progress, there’s still a con­sid­er­able lag be­tween when a new can­cer drug is used in adult tri­als and when it can be used for chil­dren.

“From 1997 to 2017, the me­di­an time from first-in-hu­man stud­ies of new can­cer drugs ul­ti­mate­ly ap­proved by the US Food and Drug Ad­min­is­tra­tion (FDA) to ‘first-in-child stud­ies’ re­port­ed­ly was 6.5 years,” EMA and FDA of­fi­cials wrote.

To try to plug that gap, the agen­cies are work­ing to­geth­er be­cause in many cas­es, the sci­en­tif­ic ra­tio­nale for the ear­li­est re­quired stud­ies in­clud­ed in the FDA’s ini­tial pe­di­atric study plans (iP­SPs) and the EMA’s pe­di­atric in­ves­ti­ga­tion plans (PIPs) and the jus­ti­fi­ca­tions for full or par­tial waivers of those pe­di­atric stud­ies are ex­pect­ed to be sim­i­lar or even iden­ti­cal.

Now, the two agen­cies are of­fer­ing a side-by-side tem­plate out­lin­ing the com­mon is­sues re­quest­ed for dis­cus­sion by the EMA and FDA with re­gard to iP­SPs and PIPs, with sec­tions ex­plain­ing non-clin­i­cal and clin­i­cal stud­ies, qual­i­ty de­vel­op­ment, and time­lines.

Both agen­cies call on spon­sors of can­cer drugs to be­gin pe­di­atric stud­ies with­out de­lay, al­though both agen­cies al­so ex­plain cir­cum­stances when de­lays in child tri­als may be nec­es­sary to en­sure suf­fi­cient adult da­ta has been es­tab­lished.

“Giv­en the glob­al na­ture of can­cer drug de­vel­op­ment and the rel­a­tive rar­i­ty of child­hood can­cer, which im­pacts study pop­u­la­tions for clin­i­cal tri­als, the de­mand for in­ter­na­tion­al col­lab­o­ra­tion in study de­sign and con­duct has in­ten­si­fied,” the FDA said in a state­ment. “We have rec­om­mend­ed that new PIPs and iP­SPs for new can­cer prod­ucts be sub­mit­ted to their re­spec­tive agen­cies si­mul­ta­ne­ous­ly to pro­mote glob­al co­or­di­na­tion and in­ter­na­tion­al re­search col­lab­o­ra­tion.”

Aduhelm OK 'bit­ter­sweet' for ALS ad­vo­cates; Con­trast­ing Covid-19 vac­cine read­outs; GSK joins TIG­IT bat­tle; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With the busiest days of June now behind us, we’re starting to think seriously about the second half of the year. In August, we have scheduled a special report where Endpoints will compile a list of the 20 most influential R&D executives in biopharma. Know a luminary who should definitely be included? Nominate them now.

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In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Who are the lu­mi­nar­ies dri­ving the biggest ad­vances in bio­phar­ma R&D? End­points News is ask­ing for your nom­i­na­tions for a spe­cial re­port

In biopharma, driving a drug to market is the ultimate goal — but none of that happens without a strong research and development program. At the most successful companies, those R&D efforts are spearheaded by true innovators in the field who are always looking for that next novel mechanism of action or breakthrough safety profile.

Now, Endpoints News is asking you to tell us who those guiding lights are.

Bris­tol My­ers breaks the bank on Ei­sai's fo­late re­cep­tor ADC drug, lay­ing out more than $3B+ for rights

For years, innovation in oncology has been a crapshoot with Big Pharma — the whales at the table — dropping the big bucks for the key to the next generation of tumor fighters. Bristol Myers Squibb hasn’t exactly made a name for being an innovator in the space, but that doesn’t mean it won’t splash in when it sees a potential winner.

Now, with a massive check in hand, the drugmaker is willing to put its intuition to the test.

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Michael Chambers (L) and John Ballantyne

Dana­her strikes deal to buy boom­ing next-gen man­u­fac­tur­er Alde­vron for $9.6B

Life sciences conglomerate Danaher Corp. $DHR has struck a deal to buy the fast-growing Aldevron, one of the world’s top manufacturers of hotly sought-after plasmid DNA, mRNA and recombinant proteins for the burgeoning world of vaccine and drugmakers pushing some game-changing technologies.

Buyout talks set the stage for Danaher to settle on a $9.6 billion cash pact to acquire the private Fargo, ND-based company — a key supplier for a disruptive new Covid vaccine as well as a host of gene and cell therapy and CRISPR gene editing players — founded by Michael Chambers and CSO John Ballantyne as a crew of 2 back in 1998.

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Spring reg­u­la­to­ry agen­da: What’s com­ing soon-ish from the FDA

The FDA’s lack of a permanent commissioner does not seem to be halting its progress to propose and finalize dozens of new regulations, with the latest batch covering everything from adverse event reporting to supplemental application submissions to annual reports for INDs.

Overall, FDA expects to release more than 40 new proposed regulations and finalize another 24 in the coming months and years.

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Sen. Joe Manchin (D-WV) on a call with HHS Secretary Xavier Becerra, June 9 (Al Drago/Pool via AP Images)

Sen. Manchin to Biden: Don't nom­i­nate Wood­cock for FDA com­mis­sion­er

While Sen. Joe Manchin’s opposition to Janet Woodcock as Biden’s choice for FDA commissioner has been known for months, the West Virginia Democrat spelled out more detailed concerns in a new letter to President Biden on Thursday.

“Dr. Woodcock is not the right person to lead the FDA,” Manchin, an influential centrist, writes, while noting the agency has been without permanent leadership for about five months.

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Med­ic­aid com­mis­sion to Con­gress: In­crease re­bates for ac­cel­er­at­ed ap­proval drugs

As the FDA continues to approve more new drugs under its accelerated approval pathway, the non-partisan Medicaid and CHIP Payment and Access Commission (MACPAC) is telling Congress to increase the statutory Medicaid rebates for such drugs until their clinical benefits have been verified.

Higher rebates for drugs with accelerated approvals, a move opposed by the biopharma industry, would mean lower net prices, lessening their financial burden on the health care system while incentivizing the companies to speed the verification of the drugs’ clinical benefits in confirmatory trials. Once those benefits are confirmed, the companies would return to the lower rebates when the accelerated approval is converted into a full approval, MACPAC suggests.

Opin­ion: CMS needs to do FDA’s job and lim­it the use of Bio­gen’s pricey new Alzheimer’s drug

Now that the FDA has approved Biogen’s expensive new Alzheimer’s drug with little clinical benefit potential, but plenty of potential to speed the bankruptcy of the government’s health care program for seniors, it’ll be up to the Centers for Medicare and Medicaid Services to limit payment for it and drive the conversation on access.

While such access decisions are normally reserved for the FDA, the agency abdicated its responsibility because of the wide label and because it’s allowing Biogen to take almost a decade to figure out if aducanumab actually works.