Rare dis­ease clin­i­cal tri­als: FDA awards $15M in grants

Af­ter re­ceiv­ing 89 clin­i­cal tri­al grant ap­pli­ca­tions, the FDA on Tues­day se­lect­ed 12 projects that will be fund­ed with more than $15 mil­lion over the next four years.

Amy Aber­nethy FDA

The an­nounce­ment of the new fund­ing comes as ap­prox­i­mate­ly 30 mil­lion Amer­i­cans are af­fect­ed by 7,000 known rare dis­eases, but on­ly a few hun­dred treat­ments ex­ist for these rare dis­eases.

Two-thirds of the 2019 grants will go to stud­ies af­fil­i­at­ed with uni­ver­si­ties, two-thirds will al­so go for stud­ies of rare can­cers and three-quar­ters of the new awards fund stud­ies en­rolling chil­dren, in­clud­ing chil­dren as young as one month, ac­cord­ing to the FDA.

For in­stance, Cincin­nati Chil­dren’s Hos­pi­tal Med­ical Cen­ter won a three-year, $750,000 grant for a phase 1 study of a treat­ment for a type of pe­di­atric brain can­cer. Sim­i­lar­ly, New Jer­sey-based Tar­get­ed Ther­a­py Tech­nolo­gies won a $660,000 grant over three years for a phase 1 study of a treat­ment for retinoblas­toma, which the FDA said is a rare can­cer in the eye most com­mon­ly af­fect­ing young chil­dren.

Re­searchers at the Uni­ver­si­ty of Al­aba­ma at Birm­ing­ham al­so won a $750,000 grant over three years for a phase 1 study of an on­colyt­ic en­gi­neered her­pes sim­plex virus ther­a­py for the treat­ment of pe­di­atric brain tu­mors.

Janet May­nard FDA

The largest award — $2 mil­lion over four years — went to Co­lum­bia Uni­ver­si­ty Health Sci­ences for a phase 2 study of dai­ly vi­t­a­min D for the treat­ment of sick­le-cell res­pi­ra­to­ry com­pli­ca­tions.

“To date, the Or­phan Prod­ucts Clin­i­cal Tri­als Grants Pro­gram’s grants have sup­port­ed re­search that led to the mar­ket­ing ap­proval of more than 60 treat­ments for rare dis­eases,” said FDA Prin­ci­pal Deputy Com­mis­sion­er Amy Aber­nethy.

Janet May­nard, di­rec­tor of the FDA’s Of­fice of Or­phan Prod­ucts De­vel­op­ment, al­so not­ed that since the grant pro­gram was cre­at­ed in 1983, The FDA has pro­vid­ed more than $400 mil­lion for more than 600 new clin­i­cal stud­ies.

Rare Dis­ease Grants


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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

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