BIO, Al­ler­gan and Roche call for FDA flex­i­bil­i­ty with pa­tient ex­pe­ri­ence da­ta

In­dus­try group BIO and bio­phar­ma com­pa­nies Al­ler­gan and Roche are call­ing for the FDA to be flex­i­ble in its ap­proach for us­ing pa­tient ex­pe­ri­ence da­ta (PED) and in al­low­ing the use of such da­ta in la­bel­ing.

BIO said that it “strong­ly be­lieves” that in or­der for a broad adop­tion of PED, any up­com­ing guid­ance doc­u­ments on pa­tient-fo­cused drug de­vel­op­ment “should em­pha­size the FDA’s will­ing­ness to ex­er­cise reg­u­la­to­ry flex­i­bil­i­ty and ac­cep­tance of in­no­v­a­tive de­signs and ap­proach­es for col­lect­ing PED.”

Danielle Friend

For ex­am­ple, the draft guid­ance on gath­er­ing pa­tient in­put, ac­cord­ing to Danielle Friend, di­rec­tor of sci­ence and reg­u­la­to­ry af­fairs at BIO, is “high­ly fo­cused on sta­tis­ti­cal re­quire­ments for col­lec­tion of sub­jec­tive mea­sures and al­though rep­re­sen­ta­tion of the pop­u­la­tion is para­mount, there is al­so a need to bal­ance such rep­re­sen­ta­tive­ness with sta­tis­ti­cal va­lid­i­ty, as the re­quire­ment of many stra­ta for rep­re­sen­ta­tion could have a neg­a­tive im­pact on sta­tis­ti­cal pow­er.”

Sim­i­lar to BIO, Al­ler­gan urges the FDA to con­sid­er the need for flex­i­bil­i­ty with re­spect to the method­olog­i­cal re­quire­ments and op­por­tu­ni­ties for en­gage­ment to pro­vide spon­sors with clar­i­ty to in­form ev­i­dence gen­er­a­tion in a time­ly man­ner.

BIO al­so calls for flex­i­bil­i­ty on sam­pling meth­ods and in col­lect­ing da­ta for small­er pa­tient pop­u­la­tions, such as with rare dis­eases. The in­dus­try group ad­di­tion­al­ly re­quests that the FDA de­lin­eate be­tween col­lect­ing PED to in­form clin­i­cal stud­ies (e.g., de­vel­op­ment of a clin­i­cal out­come as­sess­ment tool or to in­form clin­i­cal study end­points) and PED col­lect­ed with­in a clin­i­cal study meant to in­form a reg­u­la­to­ry de­ci­sion.

Roche’s Genen­tech, mean­while, calls on the agency to fur­ther clar­i­fy when PED can be la­bel-en­abling.

Janet Jenk­ins-Showal­ter

“As dif­fer­ent types of pa­tient ex­pe­ri­ence da­ta serve dif­fer­ent pur­pos­es, we urge the FDA to de­scribe rea­son­able stan­dards that en­able the in­clu­sion of fit-for-pur­pose pa­tient ex­pe­ri­ence da­ta with­in dif­fer­ent sec­tions of drug la­bel,” Janet Jenk­ins-Showal­ter, head of US reg­u­la­to­ry pol­i­cy at Genen­tech, writes.

Al­ler­gan al­so calls on the FDA to sub­stan­ti­ate the dif­fer­ences in ev­i­den­tiary stan­dards for PED for reg­u­la­to­ry-de­ci­sion mak­ing vs. la­bel­ing.

Jenk­ins-Showal­ter adds: “We would al­so high­light the im­por­tance of cre­den­tial­ing and shar­ing pa­tient ex­pe­ri­ence da­ta deemed of suf­fi­cient qual­i­ty to in­form drug de­vel­op­ment pro­gram de­sign (e.g., bur­den of dis­ease, bur­den of ex­ist­ing treat­ments) that is col­lect­ed out­side the con­text of an IND pro­gram. It is im­por­tant that these reg­u­la­to­ry qual­i­ty da­ta are shared with the broad­er com­mu­ni­ty of Spon­sors, mea­sure­ment sci­en­tists, pa­tients, and reg­u­la­tors such that clin­i­cal sci­ence is able to ad­vance to­wards the de­vel­op­ment of out­comes that are mean­ing­ful to pa­tients.”

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email for more in­for­ma­tion.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.