Dear Pres­i­dent Trump: Don’t de­stroy the FDA we know and re­spect

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On Tues­day, a group of Big Phar­ma ex­ecs from Mer­ck, J&J, Am­gen, Eli Lil­ly and Cel­gene gath­ered to pay court to Pres­i­dent Don­ald Trump at the White House. Trump didn’t dis­ap­point ei­ther his ad­mir­ers or his crit­ics, launch­ing in­to a full throat­ed ha­rangue on high drug prices while vow­ing to the col­lec­tion of CEOs that he was about to erase a large num­ber of the reg­u­la­tions on drug de­vel­op­ment to speed the ar­rival of a new gen­er­a­tion of drugs at a low­er cost.

He promised a rev­o­lu­tion at the FDA.

“We’re go­ing to be cut­ting reg­u­la­tions at a lev­el peo­ple have nev­er seen be­fore,” Trump said. Peo­ple can still be pro­tect­ed, he said, “but in­stead of 9,000 pages it can be 100 pages. And you don’t have to dou­ble up and quadru­ple up. We have com­pa­nies that have more peo­ple work­ing on reg­u­la­tions than they have work­ing at the com­pa­ny.”

The CEOs in at­ten­dance nod­ded in com­pli­ance, voic­ing their sup­port for the move — while like­ly be­ing par­tic­u­lar­ly keen on tax re­form that would boost prof­its and al­low them to repa­tri­ate bil­lions in cash.

But his com­ments didn’t play so well with biotech ex­ecs. I asked for some feed­back from drug de­vel­op­ers I know, and the re­sponse was an un­equiv­o­cal ‘no thanks.’

From their per­spec­tive, the FDA has al­ready made ma­jor changes that al­low for faster drug de­vel­op­ment. If Trump is talk­ing about shred­ding agency stan­dards and green-light­ing fast-and-dirty ap­provals for drugs with no clear proof of ef­fi­ca­cy and safe­ty, he’s threat­en­ing every de­vel­op­er with in­tegri­ty.

In Trump’s brave new world, pay­ers will be prompt­ed to throw up even high­er walls against new drugs with scant da­ta back­ing them up.

But here’s what they had to say, in their own words.


Michael Gilman, se­r­i­al biotech en­tre­pre­neur:

I will try not to de­volve to a full-on rant here, but in my view it’s nuts to as­cribe a ma­te­r­i­al por­tion of the time and cost of drug de­vel­op­ment to FDA “reg­u­la­tions.” Yes, there are plen­ty of those, but they are large­ly in the ser­vice of pro­tect­ing the safe­ty of pa­tients and clin­i­cal tri­al sub­jects. What makes drug de­vel­op­ment long and ex­pen­sive is the need to prove, be­yond sta­tis­ti­cal doubt, that your damn drug works. That’s not on the FDA — that’s on the un­der­ly­ing bi­ol­o­gy of the dis­eases we’re try­ing to crack. Low­er­ing the bar for proof is invit­ing both cat­a­stro­phe for pa­tients and even more wast­ed mon­ey in a sys­tem that is forced to pay for drugs that don’t work — and may even in­flict harm.

Crazy shit every­where you turn.


John Maraganore, CEO Al­ny­lam:

We need to main­tain stan­dards for both safe­ty AND ef­fi­ca­cy, and work with the FDA to iden­ti­fy nov­el path­ways and clin­i­cal tri­al de­signs that can speed in­no­v­a­tive med­i­cines to pa­tients. We’ve been do­ing this with FDA over the last decade with FDA­SIA, 21st Cen­tu­ry Cures, and PDU­FA VI. This will con­tin­ue in the fu­ture as we learn how to in­te­grate the pa­tient voice, re­al world ev­i­dence, and “big da­ta” in­to the drug de­vel­op­ment process. But the bot­tom line is that pa­tients and physi­cians need to know that the med­i­cines they take are safe AND ef­fec­tive. And these days, pay­ers al­so need to have ev­i­dence for a drug’s ef­fi­ca­cy and val­ue for re­im­burse­ment.


Steve Holtz­man, CEO, Deci­bel:

Most (at least the good ones) biotech ex­ec­u­tives val­ue a strong, sci­ence-based FDA; hence, I don’t think you will see any sup­port for cuts in FDA reg­u­la­tion.  Most of the CEO’s I know were pret­ty ap­palled by the Sarep­ta ap­proval as, giv­en the da­ta, it seemed to be ap­proval of a very ex­pen­sive, safe place­bo that rais­es pa­tients’ hope in­ap­pro­pri­ate­ly.  Is there some red tape to­day? Sure.  Is it out­weighed by the vi­tal func­tion played by the Agency to safe­guard the health and well-be­ing of the pop­u­lace?  You bet.

Cut­ting FDA staffing dras­ti­cal­ly would be prob­lem­at­ic as it will slow down the (thor­ough) re­view of po­ten­tial­ly im­por­tant new med­i­cines.  I stress the word “thor­ough” in the fore­go­ing sen­tence.  If the Ad­min­is­tra­tion cuts staffing, and then turns up the pres­sure to ap­prove drugs more quick­ly, that will be tan­ta­mount to a com­pro­mise of the sci­en­tif­ic na­ture of the reg­u­la­tion.  It will drift to­ward the stat­ed goal of some in the Ad­min­is­tra­tion to lim­it­ing re­views to mat­ters of safe­ty while leav­ing to the mar­ket the eval­u­a­tion of ef­fi­ca­cy.  Again, most biotech ex­ecs do not fa­vor this ap­proach (see Sarep­ta, above).  Al­so, Right to Try, is not sup­port­ed by the in­dus­try.  It pos­es a threat to con­duct­ing well con­trolled reg­is­tra­tional tri­als.


Bassil Dahiy­at, CEO of Xen­cor:

Steve said it beau­ti­ful­ly and I agree com­plete­ly.  I’d ar­gue that a strong, sci­ence-based FDA helped cre­ate the biotech­nol­o­gy in­dus­try by cre­at­ing a way for doc­tors, and fi­nan­cial mar­kets, to know if a med­i­cine ac­tu­al­ly worked (ef­fi­ca­cy too!) and was worth try­ing or in­vest­ing in.  And now for pay­ers to know if it is worth pay­ing for.  In­di­vid­ual doc­tors and pa­tients will have no way of de­duc­ing from the mi­cro­cosm of their own ex­pe­ri­ence how a new drug im­pacts a dis­ease; very very few sit­u­a­tions are as eas­i­ly in­ter­pret­ed as “you take an an­tibi­ot­ic and your ear in­fec­tion goes away in a cou­ple days.” And we don’t want to mar­ket things that don’t work.

The FDA seems very en­gaged with all of the new ini­tia­tives that are be­ing tried to­day.  Want to help biotech in­no­va­tion, small com­pa­nies and cre­ate more new med­i­cines by re­duc­ing reg­u­la­to­ry bur­dens?  Fund the agency more, hire more re­view­ers and let the FDA pay high­ly skilled sci­ence and med­ical re­view­ers com­pet­i­tive­ly to the mar­kets for their la­bor.


Je­re­my Levin, CEO, Ovid:

I would on­ly add:

First­ly, that over the last sev­er­al years there has been mas­sive changes in the per­for­mance of the FDA and the re­la­tion­ship with them has dra­mat­i­cal­ly im­proved.  They are the part­ners to our in­dus­try and be­have as such to in­sure the safe­ty AND the ef­fi­ca­cy of de­vices and med­i­cines.  In part the rea­son why Amer­i­ca has lead­er­ship in de­vel­op­ing med­i­cines is not just that we have great sci­en­tists, clin­i­cians, fi­nanc­ing and an in­fra­struc­ture to sup­port and en­cour­age in­no­va­tion, it is al­so be­cause we have the best reg­u­la­to­ry process in the world.  Work­ing with it we will move in­creas­ing­ly to es­tab­lish how to lay the foun­da­tion for what val­ue a drug brings.

Sec­ond­ly, with­out rig­or­ous sci­en­tif­ic and med­ical proof to es­tab­lish that a drug is both Safe and Ef­fec­tive, we risk tak­ing a step back in­to a time when peo­ple sold col­ored wa­ter for can­cer treat­ments and pa­tients be­came the un­wit­ting tools of un­scrupu­lous mar­ke­teers.  Pro­pos­als that walk us back to that time, are counter to in­no­va­tion and lead­er­ship in the world of med­i­cine.  They are not just bad for sci­ence and clin­i­cal med­i­cine and dan­ger­ous to pa­tients, they risk be­ing un­eth­i­cal and bad for busi­ness and the in­dus­try.


Ron Co­hen, CEO of Acor­da:

I would add on­ly that there as in­deed been im­mense progress in FDA ef­fi­cien­cy and ap­provals of new med­i­cines over the past 5 years, and we ex­pect fur­ther progress to be made based on such leg­is­la­tion as the bi­par­ti­san 21st Cen­tu­ry Cures Act. There is al­so a clear move now to­ward re­im­burse­ment for med­i­cines based on out­comes and the val­ue that these out­comes rep­re­sent, which the in­dus­try sup­ports and would like to see fur­ther en­abled by ju­di­cious re­lax­ing of cer­tain reg­u­la­tions that in­hib­it such agree­ments be­tween bio­phar­ma and pay­ers.  In ad­di­tion to the is­sues raised by the oth­ers on this thread, low­er­ing the stan­dards for de­ter­min­ing safe­ty and ef­fi­ca­cy will lead to enor­mous chal­lenges in get­ting drugs re­im­bursed, as pay­ers will have  less da­ta on which to base de­ter­mi­na­tions of out­comes and val­ue. Pa­tients there­fore will be at risk of hav­ing less ac­cess to need­ed med­i­cines.


Bernard Munos

Bernard Munos, Founder of In­no­Think Cen­ter for Re­search in Bio­med­ical In­no­va­tion:

I am afraid that this talk of FDA dereg­u­la­tion is an­oth­er as­sault of ide­ol­o­gy over ev­i­dence. The re­search (done by Hen­ry Grabows­ki and his team at Duke many years ago) is clear: in­no­va­tion is best served by ex­act­ing reg­u­la­to­ry stan­dards. Coun­tries with the high­est stan­dards (US, UK) have fos­tered the most com­pet­i­tive and in­no­v­a­tive bio­phar­ma com­pa­nies. When they ap­prove drugs, they of­ten be­come glob­al brands that dom­i­nate their ther­a­peu­tic cat­e­gories be­cause they are su­pe­ri­or. In con­trast, coun­tries with per­mis­sive regimes (France, Japan) ap­prove more drugs – Trump’s goal – that may get trac­tion in their do­mes­tic mar­kets, but sel­dom sell much out­side be­cause they are mediocre (save for the oc­ca­sion­al ex­cep­tions). In­no­va­tors know that in their guts, and have spo­ken out loud­ly against plans to de­base FDA (e.g., Len Schleifer). Any­one can come up with safe snake oil, but, if that be­comes our reg­u­la­to­ry stan­dard, that’s what we’re go­ing to get. Will it cre­ate a vi­brant in­dus­try? No, it will oblit­er­ate it.

If the pres­i­dent wants to cut costs and speed drug to mar­kets – laud­able goals – cut­ting cor­ners on safe­ty and/or ef­fi­ca­cy stud­ies is not the way to do it. In­stead, he should look at cheap­er/faster ways the col­lect the da­ta need­ed to demon­strate safe­ty/ef­fi­ca­cy. As it hap­pens, new tech­nolo­gies are emerg­ing that do just that (e.g., biosen­sors, apps), and the US has a lead in them. C And FDA has al­so been quick to em­brace them and ap­prove clin­i­cal tri­als that use them. If any­thing, it is the con­ser­v­a­tive phar­ma com­pa­nies which have been drag­ging their feet (or more pre­cise­ly the com­pli­ance and reg­u­la­to­ry groups in these com­pa­nies, which are loath to use “un­proven” reg­u­la­to­ry path­ways.) There is al­so usu­al­ly enough mon­ey to keep do­ing things the tra­di­tion­al way, so why take a “risk”? If the pres­i­dent wants to help, he could prod the in­dus­try to get on with tech­nol­o­gy that can sig­nif­i­cant­ly cut costs. It spends over $100 bil­lion a year in col­lect­ing clin­i­cal da­ta in hos­pi­tals, slow­ly, in the old-fash­ioned way. Any dent we can make in that spend­ing could be quite mean­ing­ful.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



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