FDABAA-18-00123N_0002.pdf

FDABAA

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Agency Administr

Issuing O Administr 20857

Researc Announc

Announc

Eligible A single en Developm academic

Researc research developm to accom

Types of

Notes: Re Prospect submitted leading to year (whi

A-18-00123 iew Info

Name: De ration, 1090

Office: De ration, Offic h Opportu cement for cement Ty

Applicants tities or tea ment Cente c institution h Opportu and develo ment activit mplish its m f instrumen egarding Fu tive propose d after that o a possible ich ends Se rmation epartment o 03 New Ha epartment o ce of Acquis nity Title:

the Advan pe: Broad s: This BAA ams from pr ers (FFRDC s.

nity Descr opment for ies awarde ission to pr nts that ma unding ers are enco date will be e invitation eptember 30 of Health an mpshire Av of Health an sitions & G

Food and ced Resea

Agency An

A is open to rivate secto

Cs) (see pag ription: Fo r regulatory ed under th rotect and p ay be awar ouraged to e accepted, to submit a

0th) award.

nd Human S venue, Silve nd Human S rants Servi

Drug Adm arch and D nnounceme o ALL resp or organizat ge 4 for FF od and Dru y science. F is BAA will promote the rded: Procu submit whi but cannot a full propo

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F ood and Dr Maryland, 2 ood and Dr ishers Lane

Broad Age nt of Regul urces. Offe erally Funde bility require tration solic pates that r dvance sci our nation ontracts by March 30 nteed a tech otential for a

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rug 20993 rug e, Rockville ency atory Scien erors may in ed Researc ements) and cits the adv research an entific know

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-00123 e, MD nce nclude ch and d vanced nd wledge pers w scal

INTRODU

Part I: Re

Part II: R

Part III: Pr

Section

Section

Section

Section

Section

Section

Part IV: P

Award De

Part V: At

Attachm

Attachm

Attachm

Attachm

UCTION

esearch Are eporting R roposal Pr n 1: The Ap n 2: Stage 1 n 3: Quad C n 4: Stage n 5: Full Pr n 6: Genera

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F CONT

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FDABA

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This Broad Food and D 35.016(c). T industry’s a improveme specific top full and ope "The Comp

The FDA p availability medical an animals thr FDA has w based regu health care consumer p biologics, s many other

In the US, F consumers responsible safer and m consumers to make the working to decisions b available to same time

The core re its regulato products; to manufactur in innovativ novel medi utilize these of our natio

The BAA is private sec and academ extent that

Agency Ann Drug Adminis The purpose and academi ents in techn pics as descr en competitio petition in Co rotects and of safe med d public hea rough assuri worked to red ulatory agenc e systems, a product prote such as bloo r consumer g

FDA-regulat each year — e for advanc make medici and health e best possi protect Ame based on the o ensure pro fostering an esponsibility ory activities o post-marke re, distributio ve science h cal products e new scient on.

s open to all tor organiza mic institutio such partici

Advancing nouncement stration, is is e of this BAA a’s capabilit ology, mater ribed in this on and in ful ontracting Ac promotes th ical products alth needs. F ng the availa duce the har cy and a crit nd economy ection agenc d products a goods.

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— products t ing the publ nes and dev care provide ble decision ericans from e best availa ducts meet t d advancing of FDA is to

— from pre-et product su on and marke ave provided s, and to imp tific advance responsible tions, Feder ons. Non-U.S pants compl

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INTRO

g Regulator t (BAA), whic ssued under A is to provid ties to advan rials, proces document.

ll compliance ct of 1984" a e health and s and foods FDA also pro ability of safe rm from all r tical compon

y. FDA was cies, and is and vaccines s account for that touch th ic health by vices safer a ers get the a s about thei tobacco-rela ble scientific the highest q g innovation o protect con -market revie urveillance t eting of toba d new techn prove food sa es to accomp sources. Of rally Funded S. organizatio ly with any n

AA-18-00123

ODUCTIO

ry Science ch sets forth r the Federa de a mechan nce the state sses, method Proposals s e with the pr and subsequ d safety of a and promot otects and p e animal dru egulated tob nent to the su created in 1 now respons s, drugs, me r about 25 ce he lives of ev helping to s and more effe accurate and r use of med ated death a c data and u quality stand in the produ nsumers by a ew of efficac to review of p cco products nologies to d afety and qu plish its miss fferors may i Research a ons and/or i necessary no

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and Innova h research ar l Acquisition nism by whi e of the art a ds, devices, selected for a rovision of P ent amendm ll Americans ting innovatio promotes the ug products bacco produ uccess of th 906 as one sible for ass edical device ents of every very America peed innova ective. At th d science-ba dical product and disease sing the bes dards for con ucts it regula applying the cy and safety product qua

s. In the last discover, ma uality; FDA m sion to prote include sing and Develop ndividuals m ondisclosure

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ation reas of inter n Regulation ich FDA can and achieve or technique award are th Public Law 98 ments.

s through en on that addr e health and and food. S ucts. FDA is he nation’s p of our natio suring the sa es, foods, co y dollar spen an daily. FD ations that m he same tim ased informa ts and foods . FDA must st tools and nsumers, wh ates.

e best possib y of many of ality; to regula t few years, nufacture an must keep pa ect and prom le entities or ment Cente may participa e agreement

AA-18-00123

est for (FAR) part n utilize es in he result of 8-369, nhancing the resses unme safety of

Since 2009, a science-ublic health, n’s principal afety of osmetics, an nt by Americ DA is make foods e, FDA help ation they ne

s. FDA is t make methods hile at the ble science t f its regulate ation of the rapid advan nd assess ace with and mote the hea r teams from rs (FFRDCs ate to the ts, security e et l d can ps eed to ed-ces d alth m s), regulations circumstan

Federally F Governmen direct comp the followin

1. Clearly sector.

2. Provide authorit with ind and con

Historically concerns, S concerns, V Business c proposals a

The purpos of interest a

1. Modern

2. Stimulat

Product

3. Support

4. Ensure

5. Harness

6. Impleme

7. Facilitat and Glo

8. Strength

Underst

9. Strength

Multiple aw awards und of funds. A

The Govern proposals r proposers.

to be neces options. Ad portions of negotiation proposals i s, export con ces.

Funded Rese nt/National la petition limita ng conditions demonstrate a letter on o y establishin ustry, and th nditions.

Black Colle Small Disadv Veteran-Own oncerns, an and to join ot se of this BA as listed here ize Toxicolo te Innovation t Developme t New Appro FDA Readin s Diverse Da ent a New P e Developm obal Health a hening Socia tanding hening the G wards are an der this BAA ll funding is nment reser received in r The Govern ssary. If war dditionally, F proposals fo s may be op n phases wi trol laws, an earch and D aboratories, ations and c s:

e that the pro official letter ng their eligib heir complia ges and Uni vantaged Bu ned Small B d HUB Zone ther entities

AA is to solici e and furthe gy to Enhan n in Clinical ent and Patie oaches to Im ness to Evalu ata through I

Prevention-Fo ment of Medic and Security al and Behav

Global Produ ticipated. Th A will depend subject to go ves the right response to t nment also re ranted, porti DA reserves or award. In pened with th th options fo nd other gove evelopment military edu cannot propo oposed work head from th bility to prop nce with the iversities (HB usiness conc usiness con e Small Busi as team me it proposals er described nce Product Evaluations ent Outcome prove Produ uate Innovat Information S ocused Food cal Counterm y vioral Scienc uct Safety Ne he amount o d on the qual overnment d t to select fo this solicitati eserves the ions of resul s the right to the event FD hat propose or continued erning statut

Centers (FF ucational inst ose to this BA k is not othe heir sponsor pose to Gove e associated

BCU), Minor cerns, Wome cerns, Servi ness concer embers in su that focus o in Part I of th

Safety and Person es uct Manufact tive Emergin Sciences to d Safety Sys measures to ce at FDA by et f resources lity of the pro discretion an or negotiation ion, and to m right to cond lting awards o accept prop DA desires t

r. The Gove work at the tes applicab

FRDCs) and titutions) are AA in any ca erwise availa ring organiza ernment soli sponsoring rity Institutio en-Owned S ice-Disabled rns are enco ubmitting pro on one or mo his announc nalized Medi turing and Q ng Technolo Improve He stem to Prot o Protect Aga y Enhancing made availa oposals rece nd availability n all, some, make awards duct discuss may be seg posals in the to award onl ernment rese end of one

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ble under the d Governmen e subject to a apacity unles able from the ation citing t citations and agreement ns (MI), Sma Small Busine d Veteran-Ow ouraged to s oposals.

ore of the fol cement.

icine to Impr

Quality ogies ealth Outcom tect Public H ainst Threats g Audience able for indiv eived and th y.

one, or none s without dis sions if it is la gregated into eir entirety o ly portions o erves the rig or more of t

AA-18-00123

e nt entities (e applicable ss they mee e private the specific d compete and terms all Business ess wned Small ubmit lowing areas rove mes Health s to U.S.

vidual contra he availability e of the scussions wi ater determi o pre-priced r to select o of a proposal ht to fund he phases.

e.g., et s s ct y ith ined nly l, To be eligib financial re integrity, or and equipm

This BAA is https://www

This BAA is issuance th fbo.gov wh for updates ble for award sources, abi rganization, e ment.

s available o w.fbo.gov s a continuo hrough the c en they occu s and amend d, a prospec ility to comp experience, on the follow usly open an losing date

ur. Intereste dments.

ctive recipien ly with the p operational ing websites nnouncemen specified in d parties are nt must meet erformance controls, tec s:

nt valid throu fbo.gov. Am e encourage t certain min schedule, p chnical contr ughout the p mendments ed to periodic

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nimum stand rior record o rols, technic period from t to this BAA cally check t

AA-18-00123

ards pertain of performan cal skills, faci he date of will be poste these websit ning to nce, ilities, ed to tes

Part I:

Through th following r seeks to a consistent preparatio

1. Modern

FDA seek evaluate p developme

1.1 Deve

1.1.1 E

a re

1.1.2 D

p e

1.1.3 P

1.1.4 A

in

1.1.5 In

1.1.6 In

1.1.7 D

n

1.1.8 D

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1.2 Ident

and c

1.2.1 E

a

Resear his BAA, FD research are achieve throu t with resear on and subm nize Toxicol s to improve product safe ent. Areas o lop better m

Evaluate and accurately re eactions;

Develop new potential influ emergence o

Promote a assessment proteins, path

Assess and c nflammatory adverse even nitiate in vitr associated w and the onse nitiate in vitr exposure to m

Develop mod new device m

Develop met accurately as ify and eval clinical eval

Evaluate the assays corre rch Area

DA seeks to s eas of interes ugh this BAA rch and deve mission instru logy to Enh e the toxicolo ty and effica of interest in models of h d promote th epresent hum animal mod uence of dise of adverse ev better und data at m hways, and c characterize y factors that nts (“off-targ ro and in vivo with exposure et of tobacc ro studies to medical prod dern method materials.

hods that fa ssess human luate bioma uations:

accuracy (sp ectly predict p s of Inte support adva

st. This sect A. Offerors s elopment wo uctions are c hance Produ ogic and pha acy by condu clude:

uman and a he use of ce man suscep els that bett ease progre vents;

erstanding multiple leve cell/organ fu molecular t t may be ass et” drug effe o studies to e to tobacco co related di identify pote ducts.

ds for biocom cilitate the u n adverse re arkers and e pecificity and potential hum erest anced resea tion present should propo ork as define contained in uct Safety armacologic ucting interna animal (whe ell- and tissu ptibility than ter mimic dis ssion and di of toxicity els of biolo unction;

argets, host sociated with ects);

identify pote products or iseases; and ential marke mpatibility an use of cell- a esponse to in endpoints t d sensitivity) man and ani arch and dev ts the technic ose a Statem ed in FAR 35 Part III.

c tools used al and collab ere applicab ue-based ass animal mod seases to be isease co-m mechanism ogical organ genetic and h rare and u ential biomar r tobacco pro d rs of harm a nd biological and tissue-ba ngredients in hat can be

) with which imal risk;

FDABA

velopment st cal objective ment of Work

5.001. Prop

to minimize borative rese ble) adverse says that m dels to adve etter underst morbidities on ms by eval nization incl d nexpected rkers of harm oduct constit associated w risk evaluat ased assays n dietary sup used in non animal mod

AA-18-00123

trategies in t es that FDA k (SOW) tha osal risk and earch and e response:

ore erse tand the n the uating safe uding gene m tuents;

with tions for s that more pplements n-clinical dels and in vi the at is ety es, itro

1.2.2 A

d v

1.2.3 E

1.2.4 L

1.2.5 E

a

1.3 Use

1.3.1 Im

th

1.3.2 D

s ta

1.3.3 D

o

1.3.4 D

r

1.3.5 D

c

1.3.6 D

1.3.7 D

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1.3.8 D

1.3.9 D

d

Assess conc determine ho vary across d

Evaluate qua esonance im metabolomic esponses of

Leverage pre performance

Evaluate the alterations in and develo mprove the v he prediction

Develop, vali substructures argets, and t

Develop clini or device effe outcomes;

Develop com isk, safety a

Develop com materials scie corroborate p

Develop and nform compu decision-mak

Develop com will potential products and

Develop data overall regul and analysis egulatory op

Develop com safety of ingr drugs and ot ordance bet ow the perfo different orga antitative ima maging, com

cs) for identif f a chemical.

ecision medi , disease dia role of the m metabolism p computat value of che n of human r idate and im s to a wide r toxicity mech cal trial simu ects, patient mputer mode nd efficacy;

mputer mode ence, or me post-market apply data uter model d king.

mputer mode ly enter the d users’ dem a analysis te atory data s capabilities perations.

mputer mod redients in d her dietary s tween anima rmance of th an systems a aging (e.g. p puted tomog fying new bio icine and bio agnosis and microbiome i m or other me tional meth mical Struct risk.

mplement app range of info hanisms;

ulation mode characterist els of cells, o els that integr chanistic saf findings in d mining, know development els for asses e market by mographic att echniques an quality and s to derive e els of cells dietary suppl supplements al and huma hese biomark and human positron emis graphy) and omarkers an omarkers for progression in contributin echanisms a ods and in ture-Activity proaches to ormation abo els that can r tics, and dise rgans, and s rate pharma fety data to p different pati wledge build t, clinical risk ssing the ris considering tributes and nd perform d support ma enhanced an

, organs, an ements, inc s.

n biomarkers kers and the populations ssion tomogr other advan nd predictors r predicting m n.

ng to advers and identify silico mode

Relationship link chemica out product r reveal intera ease variabl systems to p acokinetic, ph predict clinic ent populatio ding, and dat k prediction, sk of new to g potential r usage patte data profiling athematical, nalytical res nd systems cluding poten

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s of toxicity a eir interpreta ; and raphy, magn nced approa s of efficacy medical dev se responses biomarkers.

eling:

p (SAR) mod al structures isk and safe actions betw les influencin predict produ harmacodyn cal risk and ons; and ta visualizat and regulat obacco prod risks to use erns.

g in order to statistical m sults for hum to predict ntial interact

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and ation may netic aches (e.g.

and adverse vice s through dels in s and ety, disease ween drug ng uct namic, ion tools to tory ducts that rs of the o improve modeling man drug risk and tions with

2. Stimul

Produ

FDA seek personaliz trials. Area

2.1 Develo

2.1.1. R

2.1.1.1 R

le

2.1.1.2 D

2.1.2 Id

a o fo

2.1.3 D

P a th p m s in a A

B C late Innovat ct Developm s to develop zed medicine as of interes op and refin

Refine clinica such as miss enrichment, a

Refine or dev everaging da nsurance cla

Develop and designs and dentify and e areas where osteoarthritis studies (endp or stem cell-

Develop nove or orphan in neonatal trial

Pilot researc FT), priority adequacy of he findings f populations.

marketing ex scientific judg ntent is to id and safe and A. Assessing appropria B. Identifying C. Identifying program t

• Safety withdra such as

• Timelin

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• Clinical attribut advoca tion in Clini ment and Pa p new tools e and to mo st include:

ne clinical tr al trial design sing data, mu and adaptive velop statisti ata from exte aims, electro validate sta data analys evaluate imp optimal end s in humans points and d -derived ther el trial desig dications, de ls);

h to assess review (PR) pre-market from smaller The impact xclusivity, pri gment availa entify factors d effective us g the adequa ate analyses g appropriat g factors eith that can ass of the drugs awals, advers s a boxed wa nes, achieve ation of nove l trial popula es (such as acy involvem cal Evaluat atient Outco and approac dernize and rial designs n and statist ultiple endpo e designs;

cal clinical t ernal source onic health re tistical progr es;

proved clinic dpoints are la and animals duration), for rapies);

ns and endp esigns and e the impact o ), and Break efficacy and r clinical trial of incentives ority review able under e s or metrics se post-appr acy of curre and tracking te comparato her common sess:

s in the post-se events re arning or res ment of mile el or innovati ation sizes an available na ment, funding tions and Pe omes ches needed advance th s, endpoints tical methods oints, compo rial designs es such as h ecords and p ram package al endpoints acking (e.g., s, for gene th ophthalmic points for sp endpoints fo of Accelerate through (BT d safety asse populations s for the res vouchers an existing regu that may fur roval. Appro ntly availabl g and ors for asses n for all or pa

-approval pe eported, or s stricted indic estones or co ve clinical tr nd diversity;

atural history g sources, dr ersonalized d to catalyze e science a s and analy s of analysis osite endpoi and data an historical stud pre-clinical s es for innova s and related efficacy and herapy, for tr indications, pecial needs r pediatric tr ed approval T) designatio essments an s to larger m pective prog nd the applic lations need rther enhanc aches includ e data sourc ssing impact articular to e eriod (e.g., h serious label cation) osts during d rial designs a drug, disea y studies or rug class or

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d Medicine t e the develo nd conduct ysis method s to address ints, patient nalyses meth dies, patient studies;

ative clinical d biomarkers d safety end ransplant-re for tumor va

(e.g., small rials includin

(AA), and F ons to help a nd the gener more diverse grams such a cation of flex ds to be asse ce drug deve de:

ces to condu ts.

each expedite igher numbe ing changes drug develop and data an ase, or progr registries, pa disease pre

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to Improve opment of of clinical ds:

issues hods for t registries, l trial s for trials in dpoints for elated accines, and trials g

Fast track assess ralizability of as xibility and essed. The elopment uct ed ers/rates of s for safety, pment alyses am atient-ecedent) f

2.1.4 C

2.1.5 D

2.1.6 D

2.1.7 Id

2.2 Levera

2.2.1 D

2.2.2 U

d c d b

2.2.3 D

tr

2.2.4 S

2.3 Identif

2.3.1 F

a g

2.3.2 D

2.3.3 D

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2.4 F

R

• Pricing

• In parti addres flexibilit

Continue to r enhance the

Develop prac patient-match

Develop edu clinical outco eported outc and performa dentify and e and conduct.

age existing

Develop qua

Utilize large, differences in chronic disea different subs clinical param biomarkers.

Develop new rials) for reg

Survey existi sources such fy and qual

Facilitate ide and efficacy, progression a guide dosing

Develop and systems biolo

Develop robu e.g. algorith across the va

Facilitate An Resistance and access cular for orp s unmet med ty for rare di refine the us effectivenes ctical method hed medical cational mat ome assessm comes, clinic ance outcom evaluate goo g and future ntitative mod pooled clinic n specific po ase states, s sets of disea meters and o w methodolog ulatory decis ng and deve h outside of ify biomark ntification an pharmacod and prognos g); and evaluate no ogy, and hig ust technique ms, workflow ariability with ntibacterial sibility post-a phan drugs, e dical needs sease drug e of modelin ss of clinical ds to determ products terials to enh ments (and t cian-reported mes.

od practices e data:

dels and me cal trial data opulations an sex, race and ases, improv outcomes, a gies to harne sion-making elop new sta US studies, kers and stu nd qualificati ynamic resp sis, and phar ovel approac h throughpu es to evalua ws, software hin a desired

Drug Deve approval; and effectiveness in the rare d developmen ng and simul studies; and mine the abso hance FDA’s their resultin d outcomes, of patient in easures of di asets to ident nd subpopula d ethnicity, p ve understan nd evaluate ess big data atistical meth historical st udy endpoin ion of new a ponse - dose rmacogenom ches for biom ut methods.

ate the ability

e) used to fit d population lopment an d effectivene s of program disease popu nt and appro lation in clini d olute or com s capacities g endpoints , observer-re nvolvement i isease progr tify potential ations (e.g., pediatrics an nding of relat clinical utilit a or real-worl hods for syn udies, and r nts:

and improved e selection, d mics (to pred marker ident y of patient-m implants an nd Address

FDABA

ess post-app ms and incen ulation and F ovals ical trial des mparative eff to conduct r ), including p eported outc in clinical stu ression; and l trial endpoi stage of dis nd age group tionships be ty of potentia ld data (e.g.

thesizing da registries.

d biomarkers disease seve dict safety an ification, inc matching pro nd surgical g

Antibacteri

AA-18-00123

proval ntives to FDA's use o ign to fectiveness o review of patient-comes, udy design ints, explore sease,

ps) and etween al pragmatic c ata from vari s for safety erity, nd efficacy o luding -omic ocesses uides ial Drug of of e clinical ous or cs, A

2.4.1 E

to

2.4.2 A

fa

2.4.3 E

2.4.4 A

2.5 Fa

Illn

Se

SM

the a b an su

2.5.1 Th

tha Se inte tre

2.5.2 Th

stu ran stu ab

2.5.3 Th

for co

2.5.4 Th

wit inc

Antibacterial combatting a new antibact rial design. F

Evaluate pote such as enro ools, clinical

Advance the acilitate anti development and patients

Evaluate stra drugs such a

Advance the acilitate Dru ness.

erious Menta MI include: (1 e science of better unders d benefits in bstance abu e developm at could be u ection 2.1, bu erest also ar eatment optio e developm udy new or e ndomized tri udies within t out the appr e identificati r SMI drug d nsider and s e developm th drugs inte cluding pedia drug resista antibacterial erial drugs t FDA is intere ential innova ollment strate endpoints, science of i bacterial dru t for special with renal o ategies to en as the use of science of a g Developm al Illness (SM

1) facilitating clinical trial standing the n specific po use and depe ent and refin used for SMI ut should co re specific a ons for pedia ent or enhan existing drug als, includin these netwo ropriate use ion and qual evelopment specifically b ent of appro ended for SM atric patients ance is a ma drug resista to treat patie ested in the ations in clin egies, data c and new sta in-vitro, anim ug developm populations r hepatic dy nrich enrollm f rapid diagn antibacterial ment and Ap

MI) is a majo g the develop design, inclu e performanc pulations, in endence. F nement of cl I drug develo nsider and s pproaches t atric patients ncement of c gs for SMI fo g pragmatic orks, platform of medicatio lification of b . This includ be tailored to oaches to be MI or to enco s.

ajor threat to ance are to: ( ents and (2) a following top nical trial des collection str atistical analy mal model, a ment, includin such as pat sfunction ment in clinica nostic tests drug susce ppropriate U or threat to p pment of new uding identif ce of availab ncluding thos DA is interes inical trial de opment. Th specifically b that could be s with SMI.

clinical trial n r patients of c design trials ms, and/or re ons for SMI a biomarkers o des the topic o SMI drug d etter identify ourage increa public healt

(1) facilitate advance the pic areas:

sign for new reamlining, d ytic approac and/or pharm ng studies fo tients with u al trials for n ptibility testi

Use for Pat ublic health.

w therapies fication of bio ble therapies se with como sted in the f esigns, endp is includes t be tailored to e used to su networks, pl f all ages. T s, or observ egistries to in and to facilit or other stud cs listed abo development patients wh ased compli

FDABA

th. FDA’s ro the develop e science of antibacteria drug develop ches macokinetic s ocused on d nmet need, new antibact ng ients with S

. FDA’s role to treat pati omarkers, a s, including t orbid diagno following top points, and a the topics lis o SMI drug d pport the de atforms, and This includes vational or na nform patien tate new dru dy endpoints ove in Sectio t, including p o would ben iance with S

AA-18-00123

les in pment of clinical al drugs pment studies to rug children terial

Serious Men es in address ents, (2) adv and (3) devel the specific r oses, such a pic areas:

analysis met sted above in development evelopment o d/or registrie s the conduc atural history nts and pres ug developm s that could b on 2.3, but s pediatric pat nefit from tre SMI drug trea ntal sing vancing loping risks as thods n

t. Of of es to ct of y scribers ment.

be used hould ients.

eatment atment,

3. Suppor innovative research.

3.1 Enab

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echnology, to egration of m ontinuous pro anufacturing

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effects of con a batch appro

S Biomedica manufacturin ificant poten facturing pro ufacturing of of biotechno bioreactors, cial scale has abling areas otential impa armaceutical antify the im or pilot prod enhanced in of critical qu anufacturing ools, or appr multiple cont ocesses for system, inje ecific novel m pact product mplex drug s ulatory ques of research c of the propo try, control s oduct dosage ytical metho

Improve Pro tion of novel to improve p e:

valuation of ntinuous ma oach) on pro al Advanced ng (CM) as a tial to impro ocesses. Alt f small-molec ology produc end to-end s not been re s of research acts of the p l industry, co provement m duction if rel

-line proces uality attribut g for complex roaches (inc tinuous oper homogeneo ection moldi material and failure rates substances a stions related could include sed enabling strategy, and e forms:

ods for comp oduct Manu technologie product manu f novel and anufacturing oduct quality

Research a an emerging ve agility, fle hough the c cule drug pr cts (e.g. mo continuous ealized.

h could inclu roposed ena ontrol strateg metric for im evant:

s analytical tes x dosage for luding mode rations ous productio ng, and prin manufactur s; and and complex d to drug qu e the followin g technology d/or regulato plex drug sub ufacturing a es to product ufacturing a improved m

(manufactu y.

and Develop g technology exibility, cos continuous in roducts has noclonal ant manufacturi de the follow abling techn gy, and/or re mplementatio technologie rms (e.g. mo eling approa on of final do ting).

ing technolo x drug produ ality.

ng, but prop y on readine ory evaluatio bstances or

FDABA

and Quality t developme nd quality th materials an ring using a ment Author y within the p t, and robus nput of active been met w tibodies) by ng from reag wing, but pro ology on rea egulatory eva on of CM at a s that can e odified relea ches) that st osage forms ogies to dete uct dosage fo posals should ess for broad n for comple products

AA-18-00123

ent and hrough active nd continuous rity (BARDA pharmaceuti stness in the e pharmace ith some suc means of gents to dru oposals shou adiness for b aluation of C a commercia nable real-ti se, biotechn treamline th s (e.g., strip f ermine orms, espec d clearly des d implement ex drug subs e

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al scale me nology e film cially scribe ation in stances

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Develop and and metrics f op new ana nvestigate fe nuclear magn Raman spec other regulat should be inc

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ety and perf nd effectiven g of medical manufacturing es to incorpo ogies, includi ractical in-pro manufacturin hods:

d value of us ance (NMR), evaluating p , and evalua nto product a various anal ence product rch to suppo ure analytica ods and tools biological b uticals (phar patches, inh

A-regulated p ess quality o

sp) and pote e.g. jerky pet ods for pred ntamination high-throug d chemical co rility; and ethods for m hat are not a formance of ness of antim devices.

g processes orating medic ing quality a ocess monit ng processe sing improve mass spect product qual ate whether t assessments ytic technolo ts;

ort developm al methods g s to detect a behavior of e rmacoprinte halation deliv products.

of glycerin de ntial contam t treats for a icting and m n of product ghput method ontaminants microbial inac menable to reusable de microbials, s s to ensure p cal device d nd risk man toring system es.

ed analytical trometry, or n lity of pharm these improv s;

ogies for det ment and eva give consiste and measure engineered n d products), very systems evelop meth minants in the nimals, drug monitoring m ts:

ds to detect, s and validat ctivation/rem conventiona evices by imp sterilization a

FDABA

product quali evelopment agement.

ms, methods technologie near infrared maceutical ag ved technolo termination o aluation of ne ent reproduc e the physica nanomateria , and comple s, and target ods to ident e various gly gs for human edical devic

, identify, an te their utility moval al proving and

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ity s, es like d or gents and ogies of the “simila ew assays cible al structure, ls, additively ex dosage ted drug tify the qualit ycerin grade ns and anima ce clinical d y arity” of y ty s used als).

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In July 20 and Drug User Fee generic dr commitme regulatory

GDUFA. T

(GDUFA I

3.4.1 P

3.4.1.1 D

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3.4.2 C

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3.4.2.2 Im

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ve scientific

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Based Pharm y (e.g., nasa based BE m based BE m ternatives to BE studies fo inical endpo combination identified d ric drug-dev s for bioequi harmacolog or complex g ate generic

Drug User Fe n Act (Public to enhance

y. To support d the public i rch on gene

Generic Drug the FY 2018

Drugs methods fo eneric drug q ons, or dosag cterization of redients ce characteri and colloidal d animal stud eneric produc nce (BE) met abuse deterr nasal studies macokinetic al, inhalation, ethods acros ethods acros the use of fo or inhaled co int BE studie s differences i vice combin valence and y and bioeq generic dru c drugs ee Amendme c Law 112-1 public acce t this goal, F in order to c eric drugs for g User Fee A 8 topic areas or generic dru quality and e ge forms chemical com ization to sup l drug produc dies to evalua cts thods for long rence of gen s

(PBPK) mod dermal, oph ss all topical ss all ophtha orced expirat orticosteroids es for locally-in the user-nation produ d substituta quivalence g products

FDABA

ents (Title III 144)). The G ss to safe, h

FDA agreed create an an r each year Amendments s are as follo ug substitutio effectiveness mpositions, m pport demons cts ate immunog g-acting injec eric solid ora dels of drug a hthalmic) dermatolog almic product tory volume s

-acting nasa

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AA-18-00123

of the Food Generic Drug high-quality in the GDUF nual list of covered by s of 2017 ows:

ons.

s.

molecular stru stration of genicity risk o ctables al opioid prod absorption v ical products ts in one secon l products n the uation tion to optim

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c th d m ntegrate pre models for d standards

Expand the drug produc Classificatio nequivalent

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Develop new characteristic herapeutics derived prod manufacturin edictive dis decision-ma scientific u cts to suppo on System o

t) formulatio thods that w alence stud and utilizat elated to gen rug substitu lify Pain-As Pain in Food ecular bioma to serve as agents to alle atory Datab

NA barcode s to improv rformance of curity are so being increa e with one a ogical enviro nhance perfo d the impact ness to Eva e new and e th external p ment tools f w approaches cs of product (e.g., engine ucts, for clin ng in medica solution, PB aking about nderstandin ort the expa of Class 3 b ons will allow FD y submissio tion pattern neric drug a ution sociated Bi d Producing rkers (proteo surrogate e eviate pain in ase for Spe sequence d e the cyber f Digital Hea ome of the fa asingly used nother secu nments ach ormance and t of software aluate Innov emerging tec partners. Are for novel the s such as in t safety, qua eered tissue ical applicat al products m

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DA to lever ons, electro s and drug approval an iomarkers t g Animals omic or geno ndpoints in a n food anima ecies Identif database for rsecurity of alth and med astest growin in networke rely and acc ieve the des d security of e modificatio vative Emer chnologies th eas of interes erapies:

vitro and in ality, and pot es or cell the tion in regen medical prod

K/Pharmaco ug bioequiv le of excipie e Biopharm to non-Q2 ( age large d onic health safety and nd post-mar that are Ass omic) that ca assessing th als such as fication species ide medical de dical device c ng areas imp ed environme curately. To sired public h f medical dev ns on device rging Techn hrough active st include:

vivo method tency when e erapy produc nerative med ucts.

FDABA

odynamic (P valence ents in gene aceutics (quantitativ data sets (s records, quality data rket surveill sociated wit an be qualifi he capacity o cattle, pigs, ntification evices cybersecurit pacting med ents and are ensure thes health impac vices and in e performan nologies e research i ds to identify evaluating n cts, including dicine, additi

AA-18-00123

PD)

eric ely uch

a) for ance th Therapeu ed against of and goats.

ty: Digital He ical devices e expected t e technolog ct, research teroperabilit ce.

ntramurally y measurabl new g stem cell-ve utic ealth o ies is ty, and

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fa

4.1.3 In

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4.1.4 E

4.1.5 D

4.1.6 E

4.2 Deve

solut abuse

4.2.1. P

fe

4.2.1.1. E

4.2.1.2. R

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4.2.3.1. P

in d re

Develop new ast-paced sc ntegrate an novel genom echnologies

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Develop met materials

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Perform rese emerging pa continuum of eatures.

Evaluate whe packaging, s of opioid ana medication a

Research wh packaging, s prevent or de dentify and e storage, deliv deter misuse

Combine find needs and gu storage, deliv deter misuse ts use.

Perform rese deterrent opi abuse, addic data systems esources an

Perform rese of dispensed nclude qualit decreases in eformulated w ways to eva cientific prog understandi mic, proteomi role of digital hods for pre an factors e ment tools ell as produ analgesics earch to enh ckaging, sto f opioid misu ether any ex torage, deliv algesics. Exp adherence pa hether there torage, deliv eter misuse evaluate app very and dis e and abuse dings from 4 uiding princi very and dis e and abuse earch to enh oid product ction, overdo s and metho nd methods i earch to enh prescription tative and/or prescription with abuse aluate gene gress;

ng of produc c, metabolo l health in ne dicting and m ngineering p to evaluate ct formulat ance FDA’s orage, delive use and abu xisting data r very and dis ploratory res ackaging, or are any exis very and dis and abuse o propriate end posal solutio of opioid an

.2.1.1 and 4 ples should posal solutio of opioid an ance FDA’s formulations ose and deat ods available in this area.

ance FDA’s ns and abus r quantitative n volume tha deterrent pr therapy pro ct quality and mic, and oth ew medical t monitoring c principles in e packaging ions, desig understand ery and dispo se, and the requirements posal solutio search shoul r child resista sting data re posal solutio of opioid ana dpoints for s ons approve nalgesics.

4.2.1.2 and w be for indus ons approve nalgesics. Dr understand s after appro th in commu e to study the understand e rates for o e research t at often occu roperties. Ot oducts develo d safety bas her -omic therapies an clinical perfor device desig g, storage, d ned to prev ing of the fe osal solution evidence av s in other fie ons to preve d include ab ant packagin equirements ons approve algesics.

studies unde ed or labeled work with FD stry to follow ed or labeled raft and prov ing of the up oval and thei unities, impro eir impact, a ing of the re opioid produc o better und ur after the m ther relevant

FDABA

oped in this sed on nd diagnostic rmance of d gn and revie delivery and vent or dete eatures of ex ns, how they vailable to su elds may be ent or deter m buse deterre ng.

in other cou ed or labeled ertaken to su d as being ab

DA to define w to have the d as being ab vide to FDA ptake and us ir impact on ove our know and develop elationship b cts. Relevan derstand the marketing of t activities w

AA-18-00123

period of cs.

evices and ew.

d disposal er misuse an xisting and y fit into the upport these applicable f misuse and a ent packagin untries to ha d as being ab upport packa ble to preve what the da eir packaging ble to preve a Final Rep se of abuse patterns of wledge abou new data etween the nt activities w reasons for f opioid prod would include nd e for abuse g, ve ble to aging, nt or ata g, nt or ort for misuse, ut the number would r ucts q

4.2.3.2. P

re s d w

4.2.3.3. D

a li

4.3 FD

drug safe three mai adult inst has show utilization comprehe ability to would inc

4.3.1.

4.3.1.1.

4.3.1.2.

4.3.1.3.

4.3.1.4.

4.3.1.5.

4.3.2.

4.3.2.1.

4.3.3.

qualitative an national drug misuse and a

Perform rese evaluate the esearch to b settings of dr drug-specific would include abuse of spe data collecte

Develop new abuse-deterr and death in nking, and a department a reatment pro

DA is interes ety in pediat n sources:

titutions, an wn that thes n patterns. T ensive pictu assess pos clude:

Identify and

Identify and

Evaluate th disease/co

Evaluate th characteris characteris

Evaluate ge compositio

Evaluate po the size an pharmacie

Provide rob validate the

Develop an pediatric dr

Identify and nd/or quantit g utilization d abuse in com earch to enh impact of ab better unders rug misuse, c trends in po e research t ecific opioid p d from indiv w data resou rent opioid p communitie analyzing da and other me ograms, syri sted in tren tric populat free-standi nd general h e types of i The overall ure of pedia st marketing d have acces d evaluate a he ability to p ondition, geo he ability to p stics (e.g. be stics such as eneralizabilit on of either in otential diffe nd nature of s located in bust data an e methodolo nd provide re rug utilizatio have acces tative resear data reflect a mmunities.

ance FDA’s buse deterre stand factors abuse, and oison contro o better und products and iduals enter rces, metho roducts and es in the U.S ata from pres edical record nge exchan nding drug u tions. In pe ing children hospitals th nstitutions intent is to atric drug o g utilization ss to approp ppropriate v project to su ography).

present both ed size, rural s satellite ou ty of the data nstitution or erences in in the pediatric wholly-owne alyses acros ogies develo eports that w on ss to sources rch to better availability of understand ent formulati s influencing overdose, a ol center utiliz derstand the d formulation ing or being ds, and linka their impac . Examples scription dru ds, administ ge programs use over tim ediatrics, th n’s hospital hat also care may have m o develop a utcomes to n and safety priate source variables nee b-population overall drug l/urban, teac utpatient pha a and flexibi pediatric po patient versu c network, su ed pediatric ss a variety o ped.

would enhan s for pediatri understand f, or access ing of existin ons. Releva g the use of and how thes zation over t accuracy of ns in poison assessed fo ages to adva ct on misuse might includ g monitoring rative claims s, and medic me to provid is requires s, pediatric e for childre markedly d methodolo o improve u y in pediatri es for pediat eded to calc ns of interes g utilization d ching/non-te armacies in p ility to encom opulations.

us outpatien uch as the u off-site clinic of drugs and ce the FDA’ ic drug safet

FDABA

how well lo to, various o ng data syst ant activities poison cont se factors m time. Other f information n center data or substance ance the sci , abuse, add de novel met g programs, s, surveys, s cal examine de a contex integrating c hospitals (

en. Prior in ifferent ped gy to provid tilization, a c populatio tric drug utiliz ulate utilizat t (e.g. Age g data and da eaching, othe pediatric clin mpass chang nt utilization use of satelli cs.

d drug class s knowledge ty outcome d

AA-18-00123

cal, state, o opioid produ tems used to would includ rol call cente may drive ove relevant act n collected o a or in self-re e abuse trea ence of eva diction, overd thods of coll emergency substance a er databases xt for evalua g informatio (or wards) w nternal rese diatric drug de a nd improve ons. Deliver zation data.

tion projectio group, ta based on er pediatric n nics and/or o ges in the data depend te hospital es across tim e regarding data.

r ucts for o de ers in erall or ivities on eported atment.

luating dose lecting, buse s.

ating on from within earch e FDA’s rables ons.

facility network offices).

ding on me to

4.3.3.1

4.3.3.2

5. Harnes

FDA seek

5.1 Deve

and o

5.1.1 Id

d

5.1.2 P

5.2 Analy

5.2.1 R

5.2.2 D

fr

5.2.3 D

5.2.4 L

5.2.5 T

e

5.2.6 E

s

5.2.7 D

a

5.2.8 De

5.2.9 D

d

In o a

Evaluate dru

Evaluate de ss Diverse D s to develop lop and app other regula dentify oppo data analysis concern, suc biomaterials;

Promote nov models, and yze large sc

Refine metho spontaneous healthcare

Develop met rom multiple

Develop data submitted to rom FDA cre

Leverage rea domains in re

Test and vali electronic he

Enhance FDA safety and/or

Develop guid across multip evelop statis

Develop syst designed to r n November opioid crisis.

addiction; 2.

ug safety ou etailed clinica

Data throug p agency info ply simulati atory scienc ortunities an s and mode ch as toxins, and vel clinical tri novel anima cale clinical ods for analy s reports and databases hods to harn e domains a mining met the Agency eated review al-world evid egulatory de date innova ealth records

A’s capacity r effectivene delines for as ple sources stical method tems model reduce the fr r 2017, the F These prior supporting t utcome data al drug safet h Informatio ormation scie on models ce uses:

nd develop c el biologica toxic comp al design us al models/an and noncli ysis of post-m d analysis of and electr ness clinical thods for an such as CD ws and other dence and em ecision-maki tive compute

s) to evaluate y to assess d ess in large e ssessment o ds for assist of opioid cri requency or

FDA Commis rity areas inc the treatmen in both impa ty outcome d on Sciences ences capab for product computer si al systems pounds, pat sing simulatio nimal model nical data s market data, data acce ronic health evidence an alyzing stan DISC SEND d r documents mploy evide ng er models a e safety of n death and ca electronic he of data qualit ting complian sis to asses r lethality of o ssioner anno clude 1. decr nt of those w atient and ou data in neon s to Improv bility. Areas t life cycles mulation an and their thogens, ele on, new stat alternatives sets:

, including d ssible from h records nd evidence ndardized ele datasets and ence synthes nd tools on novel drug pr ause of deat ealthcare dat ty and study nce inspecti ss the effectiv overdose an ounced four reasing expo with opioid us

FDABA

utpatient pe nates.

ve Health Ou of interest in

, risk asses nd modeling responses ectromagnet tistical ata mining o m large e synthesis ectronic data d for extract sis across m clinical data roducts th as an outc tabases y designs for on veness of F nd opioid use r priority area osure and p se disorder;

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diatric popu utcomes nclude:

ssment, to streamli to agents ic energy, a of a ing data multiple

(e.g.

come of prod r synthesizin

DA interven e disorder as to addres reventing ne

3. fostering lations ne of and duct ng data tions ss the ew the to

N q o li im s th

5.3 Comp

5.3.1 D

p p li

5.3.2 D

c

5.3.3 D

te c d e f.

5.3.4 D

5.3.5 D

in a to h development assessing be o better com crisis must be disorder and

New researc experts wher quantitatively overdose dea terature and mplementati should have hat is readily eadily comm support tool f puter Modeli

Develop new potentially en potentially ha mited to exp he users and

Develop nove system toxici contribution t rom a tobac silico, in vitro

Develop nove erms of prob

a. Usage of

b. Race

c. Gender

d. Age

e. Body We . Family str home, ca

g. Occupatio or second

h. Establish

Develop nove

Develop nove otal health ri nto consider associated w obacco prod health risk su t of novel pa enefit-risk. T mmunicate F e built. This overdose d h approache re data is un y how FDA’s ath. Researc d expert opin on of new a the potentia y updatable municated to for the FDA ing and Sim w modeling ap nter the mark arm users an posure to tox d usage patt el methods t ties resulting to adverse h cco product b o, in vivo, and el models fo bability distri tobacco pro ight ructure and r, or other lo n and work dary exposur h tobacco pro el methods t el quantitativ isks of tobac ration all hea with the whol ducts. They s usceptibility ain treatment o inform the DA’s analys model will e eath in the n es are neede navailable, a s opioid inter ch approach nion and qua nd existing F al to produce and maintai policymake in the devel mulation to A pproaches to ket by consid nd non-users xicants, prod terns.

to model the g from chron ealth conditi by integratin d human clin or the demog ibution curve oduct position with ocations) premises (s re such as s oduct modeli to display m ve risk…