The new drug reg­u­la­tor con­fronts ma­jor chal­lenges

Modern Healthcare - - NEWS - By Steven Ross John­son

An opi­oid abuse epi­demic fu­eled by pre­scrip­tion drugs. Pub­lic out­cry over high drug prices. A huge back­log of generic drug ap­pli­ca­tions. In­dus­try stake­hold­ers press­ing for faster ap­provals. For­mer clin­i­cal re­searcher Dr. Robert Califf re­cently took over a U.S. Food and Drug Ad­min­is­tra­tion that is head­ing into an era of un­prece­dented chal­lenges and change. The de­ci­sions he will make over the next few years—as­sum­ing he holds on to his job in the next ad­min­is­tra­tion—will re­de­fine how drugs, med­i­cal de­vices, to­bacco, food safety and con­trolled sub­stances are reg­u­lated in the 21st cen­tury.

For that rea­son, Mod­ern Health­care has cho­sen Dr. Califf to top the list of the 50 Most In­flu­en­tial Physi­cian Ex­ec­u­tives and Lead­ers for 2016.

Pres­i­dent Barack Obama’s ap­pointee got off to a rocky start. Though his nom­i­na­tion re­ceived bi­par­ti­san sup­port, sev­eral Demo­cratic law­mak­ers at­tempted to block his con­fir­ma­tion in an ef­fort to get the FDA to take a more ac­tive role in try­ing to curb the wide­spread use of pre­scrip­tion opi­oids. These reg­u­lated prod­ucts are the ma­jor driver be­hind a grow­ing abuse epi­demic re­spon­si­ble for killing well over half the 48,000 peo­ple who died from drug over­doses in 2013. Some con­sumer groups chal­lenged his con­nec­tions to reg­u­lated in­dus­tries while at the Duke Univer­sity School of Medicine, where he served as a clin­i­cal re­searcher and ad­viser to nu­mer­ous drug com­pa­nies. They ex­pressed doubts about how he will use his power to shape and even­tu­ally im­ple­ment the 21st Cen­tury Cures Act, the House-passed over­haul of the rules for ap­prov­ing drugs and de­vices that is de­signed to get med­i­cal break­throughs to mar­ket sooner.

To face these chal­lenges and more, Califf in­her­ited a short­handed agency whose work­force was al­ready strug­gling to keep up with the rapid changes tak­ing place in the sciences of drug and de­vice devel­op­ment. “If you want to ac­tu­ally get re­sults from a

21st Cen­tury Cures, you have to get the most qual­i­fied peo­ple,” said Dr. David Gortler, a for­mer FDA se­nior med­i­cal of­fi­cer and drug-safety ex­pert at the con­sul­ta­tion site FormerFDA.com. “You have to poach those peo­ple from academia and you have to poach them from in­dus­try. Right now, they’re wait­ing for those peo­ple to knock on the door.”

In an exclusive in­ter­view with Mod­ern Health­care (See Q&A, p. 30), Califf said strength­en­ing the FDA work­force is his No. 1 pri­or­ity. That re­quires at­tract­ing top ta­lent to the agency, as well as re­tain­ing cur­rent em­ploy­ees by mak­ing the agency a more ap­peal­ing place to work.

“The FDA must eval­u­ate in­creas­ingly com­plex in­for­ma­tion to make good de­ci­sions on be­half of the pub­lic,” Califf said. “So we need the best peo­ple, and that in­cludes keep­ing the pipeline full, but also keep­ing peo­ple happy when they work here.”

Com­plain­ing about the ad­e­quacy of the FDA work­force is a con­stant re­frain in Wash­ing­ton. But the job of pro­tect­ing the pub­lic from un­safe or in­ef­fec­tive drugs and de­vices will only get tougher should Congress cre­ate a reg­u­la­tory en­vi­ron­ment that gen­er­ates less-de­fin­i­tive clin­i­cal trial data for new drug and de­vice ap­pli­ca­tions, and asks the agency to make its de­ci­sions more quickly.

Among the pro­posed pro­vi­sions is a change that would al­low a drug to be ap­proved with a “break­through ther­apy des­ig­na­tion,” based on ev­i­dence from its early-stage test­ing. An­other pro­vi­sion would al­low man­u­fac­tur­ers to get ac­cel­er­ated ap­proval for a drug based on its ef­fect on a sur­ro­gate end­point—a lab test re­sult like low­ered choles­terol that pre­dicts health ben­e­fits, but hasn’t yet been shown in clin­i­cal tri­als to im­prove pa­tient out­comes.

Given the prospect of greater sci­en­tific un­cer­tainty, crit­ics won­der whether Califf will re­main im­par­tial and hold the line on safety and ef­fi­cacy stan­dards. They cite his long-stand­ing fi­nan­cial ties with drug com­pa­nies while di­rect­ing the Duke Clin­i­cal Re­search In­sti­tute. “The at­ti­tudes he’s formed will lead him to make de­ci­sions that weigh in fa­vor of in­dus­try in­stead of pub­lic health,” charged Dr. Michael Carome, di­rec­tor of the Health Re­search Group for the ad­vo­cacy or­ga­ni­za­tion Pub­lic Cit­i­zen.

Crit­ics of some of the re­form mea­sures pro­posed by Congress, in­clud­ing for­mer FDA Com­mis­sioner Dr. Mar­garet Ham­burg and her for­mer top deputies, are more con­cerned about the agency’s abil­ity to sim­ply keep up with changed reg­u­la­tory stan­dards. The agency will need ad­di­tional re­sources to train ex­ist­ing and new per­son­nel to keep up with evolv­ing reg­u­la­tory science if it is go­ing to play its tra­di­tional role of guid­ing the in­dus­try through the process of de­vel­op­ing new drugs and de­vices with­out caus­ing un­ex­pected harm.

“It’s im­por­tant for peo­ple to un­der­stand the FDA is not an ob­sta­cle to progress but re­ally can help progress come about,” said Dr. Joshua Sharf­stein, an as­so­ci­ate dean at the Johns Hop­kins Bloomberg School of Pub­lic Health and a for­mer deputy FDA com­mis­sioner. “FDA is some­times the agency peo­ple love to hate—but usu­ally that comes out of a place of not un­der­stand­ing.”

Given the elec­tion year un­cer­tainty, Califf will prob­a­bly use the next six months to chart fu­ture plans. The one ex­cep­tion is an ini­tia­tive on opi­oid abuse, which has be­come a ma­jor is­sue on the cam­paign trail. With the Obama ad­min­is­tra­tion end­ing in less than a year, ex­perts say Califf will move cau­tiously on other is­sues, in hopes of max­i­miz­ing his chances of be­ing re­tained by the next ad­min­is­tra­tion— by who­ever wins the elec­tion.

“I would be sur­prised if there were many ground­break­ing po­si­tions that could be con­tro­ver­sial po­lit­i­cally,” said Marc Scheine­son, a part­ner at the Wash­ing­ton, D.C.-based law firm Al­ston and Bird, who pre­vi­ously served as as­so­ci­ate com­mis­sioner for leg­isla­tive af­fairs at the FDA.

“The FDA must eval­u­ate in­creas­ingly com­plex in­for­ma­tion to make good de­ci­sions on be­half of the pub­lic. So we need the best peo­ple, and that in­cludes keep­ing the pipeline full, but also keep­ing peo­ple happy when they work here.”

DR. ROBERT CALIFF

AP PHOTO

#1 DR. ROBERT CALIFF

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