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.


Zachary Brennan

managing editor, RAPS

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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