gao-Regenerative Medicine Therapeutic Applications, Challenges, and Policy Options-此为英文文档.docx

UnitedStatesGovernmentAccountabilityOffice

ReporttoCongressionalCommittees

July2023TECHNOLOGY

July2023

RegenerativeMedicine

TherapeuticApplications,Challenges,andPolicyOptions

GAO-23-105430

Thecoverimagedisplaysastylizedrepresentationofahumanbody,circledbyiconsrepresentingkeyregenerativemedicinetechnologies.

Coversource:GAO;Anttoniart/derariad/greenvector/mariia/microone/texvector/(images).|GAO-23-105430

TECHNOLOGYASSESSMENTRegenerative

TECHNOLOGYASSESSMENT

RegenerativeMedicine

TherapeuticApplications,Challenges,andPolicyOptions

WhatGAOfound

Regenerativemedicineoffersthehopeofbeingabletorestoreorreplacecell,

tissue,andorganfunctionsaffectedbydisease,injury,oraging.Thismayeventuallyhelpmanageorcuremanyconditionsthatarecurrentlyconsideredchronic,

untreatable,orterminal.

ExamplesofDiseasesandRegenerativeMedicineTherapiesThatMightAddressThem

GAOidentifiedmanychallengesthatmayaffectthedevelopmentanduseofregenerativemedicinetechnologiesandtherapiesincluding:

Challengesrelatedtostandardization.Standardsarerules,conditions,guidelines,oragreed-uponpracticesthatareadoptedwithinanindustrytoprovidedevelopers

withacommonframeworkandpromoteconsistency.Developingregenerativemedicinestandardsischallengingbecausethesetechnologiesandtherapiesarecomplexandrapidlyevolving.Inaddition,standardsrequireconsensusfrom

stakeholders,whichmaybedifficulttoobtain.

Challengesrelatedtoregulation.TheFoodandDrugAdministration(FDA)ensuresthesafety,efficacy,andsecurityofhumanmedicalproductsintheU.S.through

regulation.Regenerativemedicinefaceschallengesrelatedtoregulation,includingdifficultynavigatingacomplexregulatoryframework,uncertaintyoverwhich

regulatorypathwayismostappropriateforcertainemergingtechnologiesandtherapies,andstaffingshortagesatFDAandcollaboratingagen