FDA slaps regs on fe­cal trans­plants

In­creased steps for C. diff treat­ment draw mixed re­ac­tions from providers

Modern Healthcare - - THE WEEK IN HEALTHCARE - Mau­reen McKin­ney

When Dr. Michael Ed­mond learned that the Food and Drug Ad­min­is­tra­tion had tight­ened its reg­u­la­tions gov­ern­ing fe­cal trans­plants, he promptly can­celed 10 sched­uled ap­point­ments for the pro­ce­dure.

Most of those were for el­derly pa­tients who had been on the an­tibi­otic van­comycin for weeks or even months to stave off the symp­toms of their Clostrid­ium dif­fi­cile in­fec­tions, said Ed­mond, a pro­fes­sor of in­ter­nal medicine in the di­vi­sion of in­fec­tious dis­eases at Vir­ginia Com­mon­wealth Univer­sity, Rich­mond.

“Th­ese pa­tients are very, very sick and the an­tibi­otic ther­a­pies we have for them are not re­ally ef­fec­tive,” he said. “They are stuck.”

For physi­cians who per­form fe­cal trans­plants, an in­creas­ingly re­lied-upon treat­ment for pa­tients with se­ri­ous, re­cur­rent cases of C. diff, the days of fly­ing un­der the radar of fed­eral over­sight are over.

The FDA re­cently an­nounced that the use of “fe­cal mi­cro­biota for trans­plan­ta­tion” meets its def­i­ni­tion of a bi­o­logic prod­uct, mean­ing doc­tors who want to per­form trans­plants must sub­mit an in­ves­ti­ga­tional new drug ap­pli­ca­tion, or an IND.

The move ran­kled some physi­cians, in­clud­ing Ed­mond, who worry that the ad­di­tional pa­per­work, 30-day wait­ing pe­riod and other re­quire­ments of an IND could hin­der ac­cess to the pro­ce­dure, which uses donor fe­ces to re­pop­u­late good bac­te­ria in the guts of pa­tients with C. diff.

“It’s un­for­tu­nate for pa­tients be­cause it puts up an­other bar­rier to get a pro­ce­dure that they re­ally need,” Ed­mond said. “It’s go­ing to mean a moun­tain of pa­per­work, writ­ing pro­to­cols, writ­ing ex­clu­sion and in­clu­sion cri­te­ria, and ex­plain­ing what I’ll do if some­one has a side ef­fect. I just can’t do it.”

Rates of C. diff have soared to “his­tor­i­cally high lev­els,” ac­cord­ing to data from the Cen­ters for Dis­ease Con­trol and Preven­tion, even as providers have made gains in con­trol­ling other types of health­care-as­so­ci­ated in­fec­tions. C. diff is linked to 14,000 deaths an­nu­ally, ac­cord­ing to the CDC. Fe­cal trans­plants have been shown to be ef­fec­tive in treat­ing stub­born cases of the bug, with many physi­cians re­port­ing suc­cess rates of 80% to 95%.

And in Jan­uary, the New Eng­land Jour­nal of Medicine pub­lished the re­sults of the first ran­dom­ized con­trolled trial of fe­cal trans­plants as a treat­ment for C. diff. That study, con­ducted in the Nether­lands, found much higher suc­cess rates for fe­cal trans­plants than for the an­tibi­otic van­comycin. The find­ings were so strik­ing, in fact, that the study was halted be­cause of con­cerns that it was un­eth­i­cal to pre­vent pa­tients in the van­comycin group from re­ceiv­ing trans­plants.

Af­ter can­cel­ing all of his up­com­ing fe­cal trans­plant ap­point­ments, Ed­mond ad­vised his pa­tients that they could still use at-home en­ema kits, a choice he pre­dicted some would opt for if they can’t find a physi­cian will­ing to do it. “It will still hap­pen, just not in a con­trolled en­vi­ron­ment,” Ed­mond said.

But other physi­cians wel­comed the FDA’s over­sight, ar­gu­ing it would lead to ev­i­dence-based stan­dards and greater pro­tec­tions for pa­tients.

“I looked at it as a very pos­i­tive thing,” said Dr. Charles Poster­nack, chief med­i­cal of­fi­cer at 394-bed Boca Ra­ton (Fla.) Re­gional Hos­pi­tal, which has per­formed fe­cal trans­plants since late 2012. “The FDA rec­og­nizes that this treat­ment is im­por­tant and, in many cases, life-sav­ing for pa­tients. They just want it con­trolled.”

Poster­nack bris­tled at sug­ges­tions that the FDA’s IND re­quire­ments were oner­ous and would put undo bur­den on physi­cians. The process does take some ef­fort, he said, but he ar­gued that it quickly be­comes sec­ond na­ture. He also said the FDA’s ex­panded role would foster col­lab­o­ra­tion and ev­i­dence shar­ing among sites that per­form the pro­ce­dure.

“We plan on ful­fill­ing all of the re­quire­ments, get­ting our in­ves­ti­ga­tors cre­den­tialed and be­com­ing a re­search cen­ter for this treat­ment,” Poster­nack added.

The FDA con­tended that it has al­ways had au­thor­ity over the pro­ce­dure and that it pre­vi­ously ad­vised in­quir­ing physi­cians of their need for an IND. But af­ter a two-day work­shop on fe­cal trans­plants, held this month, the agency so­lid­i­fied its re­quire­ments, said Cur­tis Allen, an FDA spokesman.

The FDA of­fers guid­ance to physi­cians on how to com­plete the ap­pli­ca­tion, Allen said, adding that the agency can also au­tho­rize emer­gency INDs, in some cases.

“In an emer­gency sit­u­a­tion, the re­quest to use fe­cal mi­cro­biota for trans­plan­ta­tion (FMT) may be made via tele­phone or other rapid means of com­mu­ni­ca­tion, and au­tho­riza­tion may be given by the FDA of­fi­cial over the tele­phone,” Allen said in an e- mail. “In th­ese sit­u­a­tions, known as emer­gency INDs, treat­ment may be­gin prior to FDA’s re­ceipt of the writ­ten IND sub­mis­sion that is to fol­low the ini­tial emer­gency re­quest.”

Dr. Paul Sax, clin­i­cal di­rec­tor of the di­vi­sion of in­fec­tious dis­eases at Brigham and Women’s Hos­pi­tal, Bos­ton, said he wasn’t sur­prised to hear of the FDA’s newly clar­i­fied stance, given that the agency’s pri­mary man­date is to en­sure pa­tient safety. But he did call the news ironic, be­cause fe­cal trans­plants have been used ex­ten­sively with­out an IND for some time, with very high suc­cess rates.

“I’m not crit­i­cal of their de­ci­sion be­cause we want some­thing like this to be done safely and ap­pro­pri­ately,” Sax said.

“I’m just hop­ing it won’t be an im­ped­i­ment.”

Newspapers in English

Newspapers from USA

© PressReader. All rights reserved.