Feds of­fer up a new bonus plan for the R&D work they of­ten pay for

As the near­ly 1,000-page 21st Cen­tu­ry Cures Act is im­ple­ment­ed in phas­es, the full im­pact of the law is just be­gin­ning to be re­al­ized, and for one pro­vi­sion, the law has cre­at­ed a new pro­gram that re­wards bio­phar­mas for de­vel­op­ing prod­ucts that the gov­ern­ment pur­chas­es and of­ten pays to de­vel­op.

Back­ground

Un­der sec­tion 3086 of the Cures Act, the FDA must es­tab­lish a new pri­or­i­ty re­view vouch­er pro­gram for ma­te­r­i­al threat med­ical coun­ter­mea­sures — or MCMs — which are prod­ucts that may be used in the event of a po­ten­tial pub­lic health emer­gency, like an an­thrax or ricin at­tack, or a nat­u­ral­ly emerg­ing dis­ease like SARS or oth­er nat­ur­al dis­as­ter.

If a com­pa­ny wins ap­proval for such a ma­te­r­i­al threat MCM, FDA will award the com­pa­ny a pri­or­i­ty re­view vouch­er that can be used or sold to an­oth­er spon­sor to ex­pe­dite by four months FDA’s re­view of an­oth­er prod­uct that would oth­er­wise not re­ceive pri­or­i­ty re­view from the reg­u­la­tor.

The vouch­ers and ex­pe­dit­ed re­views are a hot com­mod­i­ty for com­pet­i­tive phar­ma­ceu­ti­cal com­pa­nies try­ing to bring their prod­ucts to mar­ket first — some have al­ready been sold for as much as $350 mil­lion.

This is the third PRV pro­gram cre­at­ed by Con­gress — the oth­er two were de­signed to in­cen­tivize the de­vel­op­ment of new rare pe­di­atric and trop­i­cal dis­ease treat­ments. And al­though all three pro­grams are dis­tinct, all the vouch­ers award­ed by FDA com­pete with one an­oth­er, mean­ing that gen­er­al­ly the val­ue of the vouch­ers can go down if there more be­come avail­able for sale.

But what makes this pro­gram unique is that the US gov­ern­ment — usu­al­ly via the Bio­med­ical Ad­vanced Re­search and De­vel­op­ment Au­thor­i­ty (BAR­DA), Na­tion­al In­sti­tutes of Health and FDA grants — fre­quent­ly pays for such prod­ucts to be de­vel­oped and of­ten is the on­ly pur­chas­er of such prod­ucts along­side oth­er gov­ern­ments. Ob­vi­ous­ly, no con­sumer mar­ket ex­ists for ma­te­r­i­al threat med­ical coun­ter­mea­sures.

FDA says it in­tends to is­sue guid­ance to ad­dress MCM-spe­cif­ic is­sues and the agency has al­ready is­sued guid­ance to ad­dress ques­tions re­lat­ed to the trop­i­cal dis­ease PRV pro­gram and the rare pe­di­atric dis­ease PRV pro­gram.

Ex­am­ples

No MCM pri­or­i­ty vouch­ers have been award­ed by FDA yet, but Soli­genix, a com­pa­ny with no FDA-ap­proved prod­ucts mar­ket­ed, is de­vel­op­ing an MCM vac­cine, known as Ri­Vax, to pre­vent the ef­fects of ex­po­sure to the dan­ger­ous poi­son known as ricin.

On Mon­day, the com­pa­ny an­nounced that NIH’s Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases has ex­er­cised an op­tion to fund GMP-com­pli­ant Ri­Vax bulk drug sub­stance and fin­ished drug prod­uct man­u­fac­tur­ing, which is re­quired for fu­ture pre­clin­i­cal and clin­i­cal safe­ty and ef­fi­ca­cy stud­ies.

To date, Soli­genix has re­ceived $21.2 mil­lion from NI­AID, and if all con­tract op­tions are ex­er­cised, the com­pa­ny and the Uni­ver­si­ty of Texas South­west­ern, where the vac­cine pro­tein orig­i­nat­ed, will have re­ceived al­most $25 mil­lion to de­vel­op Ri­Vax thanks to a se­ries of grants from both NI­AID and FDA. A spokes­woman from Soli­genix con­firmed to Fo­cus: “De­vel­op­ment of heat sta­ble Ri­Vax, which in­cludes man­u­fac­ture, has been ful­ly fund­ed to date by NI­AID.”

If ap­proved by FDA, Ri­Vax, which has al­ready re­ceived an or­phan drug des­ig­na­tion, would like­ly be pur­chased by the US Cen­ters for Dis­ease Con­trol for its $7 bil­lion Strate­gic Na­tion­al Stock­pile and the com­pa­ny would win a cov­et­ed PRV.

An­oth­er ex­am­ple of a prod­uct that would have won such an MCM PRV if the pro­gram had been cre­at­ed ear­li­er and was al­so de­vel­oped with the help of BAR­DA and NI­AID funds is Emer­gent BioSo­lu­tions’ an­thrax vac­cine, known as Bio­Thrax. Last De­cem­ber, Emer­gent signed a $911 mil­lion con­tract with CDC to sup­ply the Strate­gic Na­tion­al Stock­pile with al­most 30 mil­lion dos­es of the vac­cine through 2021.

An Emer­gent spokesman told Fo­cus that the com­pa­ny does in­vest in re­search and de­vel­op­ment and has four prod­uct can­di­dates in its pipeline that could have PRV po­ten­tial, in­clud­ing a dengue ther­a­peu­tic in Phase 1, a Zi­ka ther­a­peu­tic, a broad-spec­trum an­tibi­ot­ic be­ing de­vel­oped for burk­holde­ria pseudo­ma­llei and a ther­a­peu­tic for pan-filovirus­es.


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

Author

Zachary Brennan

managing editor, RAPS

Brent Saunders [Getty Photos]

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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

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