FDA is­sues two guid­ances to ac­cel­er­ate Covid-19 treat­ments

The FDA on Mon­day evening is­sued two guid­ances in­tend­ed to ac­cel­er­ate the de­vel­op­ment of prod­ucts to treat or pre­vent coro­n­avirus dis­ease (Covid-19), lay­ing out rec­om­men­da­tions to help com­pa­nies get to the in­ves­ti­ga­tion­al new drug ap­pli­ca­tion (IND) stage and clin­i­cal tri­al de­sign con­sid­er­a­tions for lat­er-stage stud­ies.

Stephen Hahn

“Ac­cel­er­at­ing the in­ves­ti­ga­tion of safe and ef­fec­tive ther­a­pies that could ben­e­fit peo­ple af­fect­ed by the COVID-19 pan­dem­ic is one of the FDA’s high­est pri­or­i­ties. We are com­mit­ted to max­i­miz­ing our reg­u­la­to­ry flex­i­bil­i­ty and us­ing every tool at our dis­pos­al to speed the de­vel­op­ment and avail­abil­i­ty of these med­ical prod­ucts and be­lieve these new guid­ances will help in­no­va­tors and re­searchers do just that,” said FDA Com­mis­sion­er Stephen Hahn.

The new guid­ance fol­lows the launch of FDA’s coro­n­avirus treat­ment ac­cel­er­a­tion pro­gram (CTAP) in March and the Na­tion­al In­sti­tutes of Health’s (NIH) Ac­cel­er­at­ing Covid-19 Ther­a­peu­tic In­no­va­tions and Vac­cines (AC­TIV) pub­lic-pri­vate part­ner­ship in April.

As of Mon­day, FDA said there are 144 ac­tive clin­i­cal tri­als of ther­a­peu­tics for Covid-19 and that it is aware of more than 450 de­vel­op­ment pro­grams that are still in plan­ning stages.

Pre-IND guid­ance

The first of the two guid­ances fo­cus­es on walk­ing drug­mak­ers through the pre-IND process to pro­vide them with feed­back and help them get their prod­ucts in­to clin­i­cal tri­als in a quick and ef­fi­cient man­ner.

“To fa­cil­i­tate this, we are urg­ing spon­sors to sub­mit a pre-IND meet­ing re­quest that al­lows ear­ly and thor­ough re­view and dis­cus­sion be­tween the spon­sor and FDA, which can lead to more rapid re­view of sub­se­quent IND and as­sur­ance of sub­ject safe­ty,” FDA writes, not­ing it has as­sem­bled a mul­ti­spe­cial­ty, mul­ti­dis­ci­pli­nary team with­in the Cen­ter for Drug Eval­u­a­tion and Re­search for re­view­ing drug de­vel­op­ment pro­pos­als for Covid-19.

FDA ex­plains that dur­ing the Covid-19 pub­lic health emer­gency, it is con­sol­i­dat­ing its pre-IND meet­ing re­quest and pack­age de­vel­op­ment process “in­to a sin­gle step.”

In most cas­es, FDA says it will re­view and re­spond to pre-IND meet­ing re­quests in writ­ing on­ly and that re­quests will be pri­or­i­tized based on “com­plete­ness of the sub­mis­sion and sci­en­tif­ic mer­it.”

FDA al­so says that spon­sors with ac­tive INDs for drugs cur­rent­ly in de­vel­op­ment or those that have sub­mit­ted pre-IND meet­ing re­quests or INDs to FDA to re­pur­pose an al­ready-ap­proved drug for Covid-19 should sub­mit a new pre-IND meet­ing re­quest cross-ref­er­enc­ing any ex­ist­ing ap­pli­ca­tions for the prod­uct in­stead of amend­ing an ex­ist­ing sub­mis­sion.

Ad­di­tion­al­ly, FDA ex­plains that spon­sors should be­gin dis­cus­sions about in­ves­ti­ga­tion­al prod­ucts for Covid-19 through pre-IND meet­ing re­quests as op­posed to pre-emer­gency use au­tho­riza­tion (pre-EUA) re­quests.

“While we are en­cour­aged to see the rapid de­vel­op­ment of po­ten­tial ther­a­pies for COVID-19, in most cas­es the ef­fec­tive­ness of nov­el or re­pur­posed ther­a­pies is un­known at the pre-IND stage. Au­tho­riza­tion of drugs through the EUA mech­a­nism in­volves an un­der­stand­ing that the known and po­ten­tial ben­e­fits out­weigh the known and po­ten­tial risks of the drug for the di­ag­no­sis, treat­ment, or pre­ven­tion of an ap­pro­pri­ate dis­ease or con­di­tion. In gen­er­al, drugs be­ing stud­ied for treat­ment or pre­ven­tion of COVID-19 have in­suf­fi­cient da­ta for FDA to make such a de­ter­mi­na­tion,” FDA writes.

The guid­ance goes on to pro­vide spe­cif­ic rec­om­men­da­tions for the con­tent of pre-IND meet­ing re­quest sub­mis­sions; non­clin­i­cal, clin­i­cal and qual­i­ty con­sid­er­a­tions; and ad­di­tion­al rec­om­men­da­tions for an­tivi­ral and in­hala­tion drugs.

Clin­i­cal de­vel­op­ment

In the sec­ond guid­ance, FDA pro­vides rec­om­men­da­tions for clin­i­cal tri­al de­sign for Phase 2 and 3 clin­i­cal tri­als in­tend­ed to es­tab­lish safe­ty and ef­fi­ca­cy for ther­a­peu­tic or pro­phy­lac­tic drugs and bi­o­log­ics for Covid-19.

FDA says the guid­ance is fo­cused on the de­vel­op­ment of di­rect an­tivi­ral drugs or prod­ucts with im­munomod­u­la­to­ry ac­tiv­i­ty and notes that the guid­ance does not ap­ply to pre­ven­ta­tive vac­cines or con­va­les­cent plas­ma.

“FDA an­tic­i­pates this guid­ance will help spon­sors to ef­fi­cient­ly de­sign stud­ies that may lead to the re­view and po­ten­tial ap­proval of safe and ef­fec­tive drugs and bi­o­log­i­cal prod­ucts to ad­dress the COVID-19 pan­dem­ic,” FDA writes.

Specif­i­cal­ly, the guid­ance pro­vides rec­om­men­da­tions for study pop­u­la­tion, tri­al de­sign, ef­fi­ca­cy end­points, safe­ty and sta­tis­ti­cal con­sid­er­a­tions.

FDA says it “strong­ly rec­om­mends that drugs to treat or pre­vent COVID-19 be eval­u­at­ed in ran­dom­ized, place­bo-con­trolled, dou­ble-blind clin­i­cal tri­als us­ing a su­pe­ri­or­i­ty de­sign,” and that stan­dard of care should be ap­plied in all treat­ment arms.

“Spon­sors should ad­dress an­tic­i­pat­ed off-la­bel use of any oth­er drugs, de­vices, or in­ter­ven­tions that might be used to man­age COVID-19,” FDA writes.

FDA opens the door to de­cen­tral­ized or plat­form clin­i­cal tri­als and re­quests that spon­sors con­sid­er­ing such stud­ies con­tact the agency.

FDA al­so pro­vides a list of what it con­sid­ers to be im­por­tant clin­i­cal out­come mea­sures for treat­ment tri­als, in­clud­ing all-cause mor­tal­i­ty, res­pi­ra­to­ry fail­ure, need for in­va­sive me­chan­i­cal ven­ti­la­tion and sus­tained clin­i­cal re­cov­ery. For pre­ven­tion tri­als, FDA says the pri­ma­ry end­point should be the oc­cur­rence of lab­o­ra­to­ry-con­firmed SARS-CoV-2 in­fec­tion or SARS-CoV-2 in­fec­tion with symp­toms with­in a pre­spec­i­fied time­frame.


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