美国国会报告,发展个体化医学,XXXX

PCASTReport:PrioritiesforPersonalizedMedicinePresident’sCouncilofAdvisorsonScienceandTechnology(PCAST)Member2001-2009Chair,SubcommitteeonPersonalizedMedicine1.StudycommencedJanuary20072.ReportPublishedSeptember2008,metwithPresidentandCongressionalStaffSeptember16and17,20083.’sCouncilofAdvisorsonScienceandTechnology(PCAST)PersonalizedMedicineSubcommitteehostedmorethantenmeetingsandworkshopsover110individualsparticipatedRepresentativesfrom:Academicinstitutions,diagnostic,DTC,serviceandimagingcompaniesBiotech,pharma,tools,ITcompaniesInsurancecompaniesandprovidersPatientadvocatesVC’s,tradeandprofessionalassociations,gov’tagenciesPolicyAreasStudiedbyPCAST1.RegulationoftherapiesanddiagnosticsbyFDA,CMS;2.ReimbursementoftherapiesanddiagnosticsbyCMSandprivateinsurancecompanies;3.Genomicdiagnostics,intellectualpropertyandrelatedemergingpatentissues(PTO);4.Patientprivacy;5.Informationtechnologyandissuesofelectronicpatientrecordsandassociateddata/databases;6.Economicsofpersonalizedmedicine;7.Personalizedmedicinetechnology/tools;8.Patient,physicianandpubliceducation.PCASTFindingsHighlevelofpolicyinterestattributabletopromiseofimprovedpatientcareanddiseaseprevention,pluspotentialtopositivelyimpactincreasingcostofhealthcareanddecreasingrateofnewmedicalproductdevelopment;Abilitytodistinguishpatientsmostlikelytobenefitfromtreatmentorsufferharmshouldimprovequalityofcareandresultincostsavings;Abilitytostratifypatientsbydiseasesusceptibilityorlikelyresponsetotreatmentcouldreducesize,durationandcostofclinicaltrialstofacilitatetreatment,diagnosticandpreventionstrategiesdevelopment.PCASTFindingsIdentifiedspecificpolicyactionsrelatedtogenomics-basedmoleculardiagnosticswithgreatestpotentialtoaccelerateprogressinpersonalizedmedicine;Paralleldevelopmentsingenomics-linkedtherapeuticsalsoimportant,thoughpaceofchangeinmoleculardiagnosticsmostrapidandpolicyhurdlesgreatestinthelatteratpresenttime;PCASTFindings--continuedNarrowedpolicyrecommendationstothreeareas:technology/tools,regulationandreimbursement;Otherareas,whileveryimportanttolongtermprogress,deemedlessurgentdueto:Significantongoinggovernmentactivity(informationtechnologyandprivacy)Earlystageofpersonalizedmedicinedevelopment(physicianandpatienteducationandeconomics)orNeedformorecomprehensivepolicyrecommendationsbeyondthescopeofpersonalizedmedicine(intellectualpropertyandprivacy).PCASTFindings:TechnologyandToolsChallengestovalidatinggenomic/clinicalcorrelationsnecessarytoadvanceproductsintoclinicaluse;Concernsforimbalancebetweendiscoveryandvalidation.PCASTRecommendations:TechnologyandToolsFederalgovernmentshoulddevelopstrategiclong-termplanbycoordinatingpublicandprivatesectoreffortstoadvanceR&D;HHSshouldleadefforttocreatepublic/private“PersonalizedMedicineR&DRoadmap”;NIH,DOEandotheragenciesshouldevaluaterelativefundingfordiscoveryversustranslationalresearchrelevanttopersonalizedmedicine;NIHshouldcoordinateprocesstoidentifyandprioritizediseasesandcommontherapiesthatwouldbenefitfromapplicationsofgenomics-basedmoleculardiagnostics.PCASTRecommendations:TechnologyandToolsFederalgovernmentshouldmakecriticalinvestmentsinenablingtoolsandresourcesmovingbeyondgenomicdiscoveriestopersonalizedmedicineproductsandservicesofpatientandpublicbenefit;NIHshouldspearheadpublicandprivateeffortstodevelopanationwidenetworkofstandardizedbiospecimenrepositories;NIHshouldpromotemethodsforvalidatingtheclinicalutilityofmoleculardiagnosticsbasedongenomiccorrelationswithdisease;NIHshouldestablishlargeUSpopulationcohortforinvestigatinggeneticandenvironmentalhealthimpacts.PCASTFindings:RegulationFDAprogressviaGuidancestodefiningapproachtoregulationofgenomics-basedmoleculardiagnostics,withfollowingoutstandingissues:RiskdefinitionfordeviceversusinformationStandardsforstudydesignandproductperformanceCoordinationbetweenCLIA(CMS)andFDARegulatoryapproachtoco-developmentCriteriaandproceduresfortherapylabelingwithdiagnosticinformationRegulatoryapproachtoClinicalDecisionSupportSystemsPCASTRecommendations:RegulationFDAshouldimplementmoretransparent,iterativeapproachtoregulation:FinalguidanceforIVDMIAsshouldclarifyriskdefinitionFDAandCMSshouldclarifyrolesandpotentialregulatoryredundancyFDAshouldfinalizedraftco-developmentpaperFDAshouldclarifycriteriaandproceduresforincorporatinggeneticdataonlabelsfortherapeuticsFDAshouldissueGuidanceforregulationofautomatedClinicalDecisionSupportSystemsPCASTRecommendations:RegulationFDACriticalPathshouldbefundedtoincludesupportofpersonalizedmedicineprogress:Biomarkerstofacilitateproductdevelopment,clinicaltrialdesignandvalidationformoleculardiagnosticsshouldbepriority;Reagan-Udallshouldbefundedandexpandedtoincludeventurecapitalandmoleculardiagnosticcompanyrepresentation.PCASTRecommendations:RegulationIndustryshouldadoptproactiveandconstructiverolewithFDAtofulfillitsregulatoryresponsibilities:Whenpossible,responsestodraftguidancesshouldincludealternativeapproaches;Industryshould