Even af­ter the FDA added reg­u­la­to­ry path­ways for drug de­vel­op­ers to win ac­cel­er­at­ed ap­provals for new drugs, the po­lit­i­cal pres­sure in Wash­ing­ton to speed up drug re­views con­tin­ues to grow.

In tes­ti­mo­ny be­fore a House sub­com­mit­tee yes­ter­day, FDA com­mis­sion­er Scott Got­tlieb as­sured law­mak­ers that bio­mark­ers, new tech­nolo­gies and more ef­fi­cient tri­al de­signs made it pos­si­ble to short­en the reg­u­la­to­ry process as he vowed to urge all the FDA to repli­cate the fast pace of the agency’s on­col­o­gy di­vi­sion, which has re­con­fig­ured can­cer drug de­vel­op­ment pro­grams over the past 3 years.

But should drugs ap­proved ear­ly with on­ly part of the da­ta that was once re­quired for an OK be able to fetch the full re­tail price that man­u­fac­tur­ers ex­pect to­day?

Two health pol­i­cy ex­perts say no.

Aaron Kessel­heim

Walid Gel­lad

In an op-ed for The New Eng­land Jour­nal of Med­i­cine, Walid Gel­lad from the Uni­ver­si­ty of Pitts­burgh and Har­vard’s Aaron Kessel­heim ar­gue that any bio­phar­ma com­pa­ny that wins an ac­cel­er­at­ed ap­proval should be sub­ject to cer­tain price re­stric­tions. And they’ve of­fered a few ex­am­ples of how that could work. You could, for ex­am­ple:

— Re­quire drug mak­ers to of­fer pub­lic pay­ers a set dis­count on drugs that get an ear­ly OK ahead of con­fir­ma­to­ry stud­ies. Med­ic­aid could get a statu­to­ry price re­duc­tion on top of the dis­counts it al­ready qual­i­fies for.

— Hold a por­tion of the rev­enue from these drugs in es­crow, un­til they prove they work as as­sumed based on the pre­lim­i­nary da­ta. Drug mak­ers can win it on a pos­i­tive Phase III, or lose it all as the cash is used to re­im­burse pay­ers.

— To avoid any gam­ing of this sys­tem, hik­ing the whole­sale price to make sure sell­ers make what they want from the dis­count­ed fig­ure, man­u­fac­tur­ers could be forced to switch to a cost-plus sys­tem, with set mar­gins.

The au­thors al­so call for a new sys­tem where de­vel­op­ers are held ac­count­able to see­ing their late-stage tri­als through on sched­ule. A sys­tem of re­wards and penal­ties can be put in place for com­pa­nies as they set out to achieve spe­cif­ic mile­stones in their stud­ies. And no more long run­ways, they say. New tri­als should start with­in months of an ac­cel­er­at­ed OK. And these con­fir­ma­to­ry stud­ies should be ex­pect­ed to wrap in a rea­son­able amount of time, not ex­tend for years in­to the fu­ture.

We be­lieve there should be plans in place to be­gin con­fir­ma­to­ry tri­als with­in 3 months af­ter ap­proval, with track­ing of tri­al progress through Clin­i­cal­Tri­als.gov. Though the rar­i­ty of the dis­ease and oth­er fac­tors might rea­son­ably af­fect tri­al ac­cru­al times, there should al­so be mean­ing­ful reper­cus­sions for miss­ing mile­stones such as hav­ing a pro­to­col in place or hit­ting re­cruit­ment tar­gets, cul­mi­nat­ing in with­draw­al of the drug if the tri­al is un­nec­es­sar­i­ly de­layed for an ex­tend­ed pe­ri­od. The FDA can, un­der cur­rent law, as­sess fi­nan­cial penal­ties or with­draw an ac­cel­er­at­ed-ap­proval drug from the mar­ket if the man­u­fac­tur­er fails to con­duct its con­fir­ma­to­ry tri­al or fails to do so with “due dili­gence,” a bench­mark that the FDA can fur­ther clar­i­fy with stake­hold­er in­put.

Even more con­tro­ver­sial­ly, they sug­gest that an eco­nom­ic im­pact study should be used to eval­u­ate these drugs af­ter one or two years on the mar­ket, to see if the val­ue of a drug giv­en an ac­cel­er­at­ed ap­proval is lost to the fi­nan­cial tur­moil it can cause.

As far as the in­dus­try is con­cerned, there isn’t any­thing here that would slip un­der the radar. It would all be fought tooth and nail. Ag­gres­sive gov­ern­ment reg­u­la­tions re­strict­ing prices and gov­ern­ing tri­als is anath­e­ma to bio­phar­ma, which much prefers vol­un­tary re­straint in the US. But as the de­bate over drug prices con­tin­ues to boil in Wash­ing­ton DC, it’s an­oth­er set of “so­lu­tions” like­ly to trig­ger fresh de­bate at a time ac­cel­er­at­ed ap­provals may just be get­ting start­ed.

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.