The 21st Century Cures Act:Implementation of Regenerative

Medicine Provisions

GaryH.Gibbons,MD

Director,NHLBINIHAdvisoryCommitteetotheDirector

March28,2017

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Regenerative Medicine: The Promise

§ Potentialtorepairorreplacecellsandtissuesdamagedbyinjury,disease,oraging§ Restorenormalstructureandfunction§ Enhancethebody’sinnatehealingcapacity

§ Widerangeoftechnologies§ Engineeredbiomaterialsandtissues§ Geneeditingorreplacement

§ Manypotentialapplications§ Chronicdiseases§ Acuteinjury§ Congenitaldefects

Regenerative Medicine: Broad Applications Across Disease Areas

§ Spinalcordinjury

§ Musculoskeletalinjuryandtissuedegenerativeconditions

§ Autoimmunediseases

§ Vision

§ Neurogenerativeconditions

§ Diabetes

§ End-organdisease

§ Hemoglobinopathies

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Challenges

Knowledgegaps,technicalhurdles,andoperationalbarriers

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Challenges: Public Trust and Oversight

§ “Hype”inmarketing,untestedandunapprovedRMproductsadvertised§ Clinicsadvertising“NIH-registered

trials”§ “Patient-providedresearch

payments” ($8K-$20K/protocoltreatment)

§ AnumberoftrialsarenotunderIND

§ Need:§ Rigorousscience§ Regulatoryoversight§ Nationalplatformfordiscussionof

thepromiseandchallenges

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Innovation Project: Regenerative Medicine

FromtheAct:

FortheNIH,incoordinationwiththeFDA,toawardgrantsandcontractsforclinicalresearchtofurtherthefieldofregenerativemedicineusingadultstemcells,includingautologousstemcells,…nottoexceedatotalof$30M,asfollows:§ FY2017:$2M§ FY2018:$10M§ FY2019:$10M§ FY2020:$8M

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Regenerative Medicine Funds:Matching Requirement

§ TheActfurtherstipulatesthat:§ Awardsmustbecontingentupontherecipientmakingavailablenon-Federalcontributionsinanamountnotlessthan$1foreach$1ofFederalfundsprovidedintheaward(i.e.,amatchingrequirement)

§ Opportunity:§ Catalyzeafocusedandsystematictrans-

NIHapproachtoadvancingthefield§ WorkwiththeFDAtoaddresspatient

safety,publictrust,andtheintegrityofclinicalresearchinRM

§ ModusOperandi:§ Consultativeandinclusive§ Leverageexistingresourcesand

infrastructurewherepossible

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21st Century Cures Act RM Provisions:Trans-NIH Collaborative Catalysts

RM Innovation Project FundsFiscal Year Authorization

(No Year $) Total Funds w/Matching

Description

2017 $2 million $4 million • NOITP to alert community to new funding opportunities (competitive supplements in FY17 and new projects in FY18-20) and to matching requirements

• Applications for supplemental funds to be solicited through a FOA that includes the matching funds requirement

• Applications to be reviewed by NIH in coordination with FDA, followed by secondary review by Council

2018 $10 million $20 million Solicitation of projects to be informed by expert consultation

2019 $10 million $20 million Solicitation of projects to be informed by workshop consultation

2020 $8 million $16 million Solicitation of projects to be informed by workshop consultation

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RM Innovation Project Work Plan:Criteria

- Abilitytoputsupplementalfundingtogoodandimmediateuse

- Underlyingprojectiswellalong/alreadyunderway

- Matchingfundsavailableattimeofaward- Contributestobreadth/diversityofRMscience- Addressesacriticalknowledgegapand/ortechnicalbarrier

- WillhelptosignificantlybuildoradvancethefieldofRM

- ContributestoregulatoryscienceinRM

§ ForFY17funds,proposalswillbeassessedusingstandardNIHreviewcriteria,aswellasthefollowing:

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ACD Feedback and Dialogue

§ Whatisyoursenseoftheopportunitiesandchallenges?

§ Whatarepriorityresearchtopicsthatweshouldfocuson?