Chart­ing a new drug re­view path­way

Why the reg­u­la­tors at the FDA must think sur­geons

The Washington Times Daily - - COMMENTARY - By Peter J. Pitts Peter J. Pitts, a for­mer FDA as­so­ciate com­mis­sioner, is pres­i­dent of the Cen­ter for Medicine in the Pub­lic In­ter­est.

On a visit to her pre­na­tal physi­cian, Mar­garet Boeme and her hus­band re­ceived un­wel­come news — their un­born baby had a spinal tu­mor. Sev­eral doc­tors rec­om­mended the Boemes ter­mi­nate the preg­nancy, but the cou­ple re­fused to give up. Fi­nally, they found doc­tors will­ing to per­form a risky open fe­tal pro­ce­dure to re­move the tu­mor. Twelve weeks later, Mrs. Boeme gave birth to a beau­ti­ful baby girl.

When sur­geons take these risks, it’s in­spir­ing. But when the FDA does some­thing sim­i­lar — in par­tic­u­lar, re­think­ing a new drug’s re­view path­way so that pa­tients without any op­tions can have one — some con­sider it scan­dalous.

That’s just wrong. Like a sur­geon, the FDA should fol­low proper pro­ce­dures, al­ways. But those pro­ce­dures should al­low for ad­vances in reg­u­la­tory sci­ence — such as the pa­tient voice.

In mid-Septem­ber, the FDA ap­proved Ex­ondys 51, the first drug specif­i­cally in­di­cated for Duchenne mus­cu­lar dys­tro­phy. Duchenne mus­cu­lar dys­tro­phy is a rare dis­ease that strikes boys as young as three, at­ro­phy­ing their mus­cles and even­tu­ally tak­ing their lives. While clin­i­cal tri­als were in­com­plete, they did demon­strate that the drug car­ries few side ef­fects and holds the po­ten­tial to slow the pro­gres­sion of Duchenne mus­cu­lar dys­tro­phy.

For pa­tients, the FDA’s ap­proval rep­re­sented a “wa­ter­shed mo­ment.” Un­til this drug ar­rived on the scene, Duchenne mus­cu­lar dys­tro­phy suf­fer­ers had no vi­able treat­ment op­tions what­so­ever. Now, they have a chance to live to adult­hood. Yet oth­ers in­sist the FDA made a mis­take.

Gi­ant in­surer An­them, for in­stance, claimed the FDA shouldn’t have ap­proved the medicine since it did not have suf­fi­cient ev­i­dence. As a re­sult, An­them is re­fus­ing to cover it. The de­ci­sion will de­prive hun­dreds of pa­tients of their only chance to reach adult­hood.

Two doc­tors even went so far as to say that the FDA ap­proval rep­re­sents a “wor­ri­some model” for fu­ture tar­geted drug ap­provals. They urged the FDA not to ap­prove drugs just be­cause they “show pos­i­tive ef­fects on the body . . . without ac­tu­ally mak­ing any mea­sur­able dif­fer­ence in pa­tients’ health.”

In the case of med­i­ca­tions for com­mon ill­nesses, these doc­tors are cor­rect: There is no com­pelling need for ex­pe­dited ap­proval.

But for or­phan dis­eases — se­ri­ous and life-threat­en­ing dis­eases that af­flict small num­bers of pa­tients — ex­pe­dited ap­proval makes every dif­fer­ence.

The FDA should con­tinue its rig­or­ous scru­tiny be­fore grant­ing ap­proval to new med­i­ca­tions. But when pa­tients with rare dis­eases plead with the FDA to clear a drug that’s proven not to be harm­ful and holds the po­ten­tial to be help­ful — well, then it would be cruel if FDA pol­icy pro­hib­ited it.

The ap­proval of Ex­ondys 51 does not mean a free pass for bad sci­ence. Rather, it shows that the FDA is rightly sup­ple­ment­ing its scrupu­lous re­view process with a dose of com­pas­sion for pa­tients. By bal­anc­ing the need for speed, ac­cu­racy, and im­proved pub­lic health, the agency has taken the next step to­wards a 21st cen­tury, en­tre­pre­neur­ial reg­u­la­tory sys­tem and po­si­tioned it­self as an in­no­va­tion ac­cel­er­a­tor.

Like Mar­garet Boeme’s sur­geons, the FDA should take a chance when the sit­u­a­tion de­mands it — and like her sur­geons, the FDA should be ap­plauded for do­ing so.

IL­LUS­TRA­TION BY GREG GROESCH

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