summary key points€¦ · to provide access to transplant for all americans in need of a...
TRANSCRIPT
WrittenTestimonyofDr.JeffreyW.Chell
beforetheHouseCommitteeonEnergyandCommerce
Hearingon“ExaminingPublicHealthLegislation:
H.R.2820,H.R.1344,andH.R.1462”
June25,2015
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SummaryofKeyPoints
• The NMDP/Be The Match appreciates the continuing bipartisan support to reauthorize the C.W. Bill Young Cell Transplantation Program and the National Cord Blood Inventory. ItiscriticallyimportanttoreauthorizetheseprogramsbeforetheirauthorizationsexpireattheendofSeptember2015.
• SincethelateCongressmanBillYoungwasfirstinspiredby11‐year‐oldBrandyBlywhodidnotsurviveherbattlewithleukemiaandhetestifiedbeforetheCommerceCommitteein1987,significantprogresshasbeenmadetoprovideaccesstotransplantforallAmericansinneedofalife‐savingbonemarroworcordbloodtransplant.
• Today,theNMDP/BeTheMatchisthegloballeaderinhelpingpatientsseekingacureforoneofthemorethan70differentdiseasesorconditions,includingbloodcancersandsicklecelldisease.Cellulartherapyistheirbesthopeforacure.
• TheBeTheMatchRegistryhasgrowntoincludenearly12.5milliondonorsandmorethan209,000cordbloodunitsandthroughinternationalrelationships,wehaveaccesstomorethan24.5millionpotentialdonorsand622,000cordbloodunitsworldwide.
• Between1988and2005,theone‐yearsurvivalrateforthesepatientshasincreasedfromjustover40percentto70percent.Accesstomultiplecellsourcesallowsustofindthebestmatchpossibleforpatients.
• In2014,wefacilitatedmorethan1,200unrelatedtransplantsforpatients18oryoungerusingbonemarroworcordblood.Currentpediatricresearchfocusesnotonlyonmalignancies,butalsooncuringnon‐malignantdiseasessuchcertainmetabolicdiseasesandhemaglobanopathieslikesicklecelldiseaseandthalassemia.
• Thecalculatedneedforunrelatedtransplanthasincreasedby25percentsince2005.Muchofthereasonisduetotheexpansioninthenumberofindicationsfortransplant.
• Thenumberoftransplantsforminoritypatientshasincreasedfrom253in2000to990in2014.
• Thenon‐matchbarrierstoaccessandcarehaveaprofoundaffectonourabilitytomaketransplanttherapiesaccessibletoall.
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GoodmorningMr.Chairman,andotherdistinguishedmembersofthe
Subcommittee.MynameisDr.JeffreyChellandIamtheChiefExecutiveOfficeof
theNationalMarrowDonorProgram(NMDP)/BeTheMatch.WeoperatetheC.W.
BillYoungCellTransplantationProgram(Program)throughfourcompetitivelybid
contractswiththeHealthResourcesandServicesAdministration(HRSA).These
contractsincludetheSinglePointofAccessandOfficeofPatientAdvocacy,theBone
MarrowCoordinatingCenter,andtheCordBloodCoordinatingCenter.Collectively,
thesecontractsallowNMDP/BeTheMatchtooperatethenationalBeTheMatch
Registryandprovidelife‐savingbloodandmarrowtransplantsusingindividual
adultdonorsandumbilicalcordbloodunits.AndwiththeMedicalCollegeof
Wisconsin,NMDP/BeTheMatchalsoholdsthecontractfortheStemCell
TherapeuticOutcomesDatabase,knownastheCenterforInternationalBloodand
MarrowTransplantResearch(CIBMTR)ofwhichIamtheexecutivedirector.
Tobegin,IwouldliketothankChairmanPitts,RankingMemberGreen,andallofthe
MembersoftheHealthSubcommitteeforinvitingmetospeakwithyoutoday.On
behalfof565NetworkpartnerorganizationsandeveryoneattheNMDP/BeThe
Match,wealsowanttothankyouformaintainingtheCongressionalcommitmentto
patientsfightingbloodcancersandotherdisorderswhoseonlyhopeforacureisa
bonemarroworcordbloodtransplant.
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IwouldalsoliketothankCongressmanChrisSmith,CongresswomanDorisMatsui,
CongressmanDavidJolly,andCongressmanChakaFattahfortheirleadershipin
introducingH.R.2820toreauthorizetheProgram,aswellastheNationalCord
BloodInventory(NCBI)grantprogram.Theseprogramsareexamplesofhowthe
CongresscaninspireinnovationtobringcurestopatientsacrossAmerica.
WhoWeAre
AsItestifybeforeyoutoday,Icannothelpbutberemindedofanotherhearingthat
tookplacein1987.Onthatday,thelateCongressmanBillYoungcalledonthe
Congresstoestablishanationalregistrywheremen,women,andchildrenwith
leukemiaandotherfatalblooddisorderscouldfindanunrelateddonortosavetheir
lives.HewasjoinedbyDr.RobertGraves,whosedaughterreceivedthefirst
unrelatedbonemarrowtransplantforleukemia,andNavyAdmiralBudZumwalt.
TheCongressheardthatcallandestablishedthenationalregistry.Firsthousedin
theDepartmentoftheNavy,itfounditspermanenthomeasoneofthenation’s
premierpublichealthprogramsatHRSA.
CongressmanYoung’squestwasinspiredbyachild,11‐year‐oldBrandyBly,and
herfamilyracingtosaveherlife.Unfortunately,nooneinherfamilywasasuitable
matchandshedidnotsurvive.Itwasthesimplestatementfromherphysician–
“Wouldn’titbegreatiftherewereawaythatdoctorscouldsearchforadultswilling
todonatetheirbonemarrow?”–thatledtothenationalregistry.
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Sincethatfirsthearingin1987,wehavemadegreatprogress.Today,theNMDP/Be
TheMatchisthegloballeaderinprovidingacuretopatientswithlife‐threatening
bloodandmarrowcancerssuchasleukemiaandlymphoma,aswellasother
diseases.ThroughthecontractwithHRSA,wemanagetheworld’slargest
registry—theBeTheMatchRegistry—ofpotentialmarrowdonorsandumbilical
cordbloodunits,connectpatientstotheirdonormatchforalife‐savingmarrowor
umbilicalcordbloodtransplant,educatehealthcareprofessionals,andconduct
researchsomorelivescanbesaved.Today,childrenlikeBrandyhaveamuch
betterchancetofindthatlife‐savingmatchandultimatelyacure.
But,theC.W.BillYoungProgramasithascometobeknown,ismorethanthe
nationalregistry.ThroughtheProgram,theNMDP/BeTheMatchalsooperatesthe
OfficeofPatientAdvocacy.TheOfficeofPatientAdvocacyassistspatientsandtheir
familiesinnavigatingthecomplexitiesofhealthinsuranceandhelpsthemovercome
logistical,psychosocialandinformationalbarriersthroughoutthetransplant
continuum.Wealsoworkcloselywithdonorandcollectioncentersthroughthe
BoneMarrowCoordinatingCentercontracttorecruitandretainvolunteerpotential
donors,produceacomprehensiveplanfordonorretention,andplanforincreasing
operationalefficiencies.Wesimilarlyprovidefinancialandeducationalsupportto
publiccordbloodbanksasthemanageroftheCordBloodCoordinatingCenterand
provideguidancetoHRSAintheadministrationoftheNationalCordBlood
Inventoryprogramtodetermineoptimalcompositionofcordbloodinventory.And,
wepartnerwiththeMedicalCollegeofWisconsintooperatetheStemCell
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TherapeuticOutcomesDatabase,whichfacilitatesresearchtoimprovepatient
outcomesandfindnewandexcitingwaysbonemarrowandumbilicalcordblood
canbeusedtosavelives.
TheSuccessoftheProgram
Duringthepast28years,theNMDP/BeTheMatchhasbeenhonoredtoserveasthe
stewardofthiscriticalnationalresource.Today,theBeTheMatchRegistryserves
asthesinglepointofaccessforbothumbilicalcordbloodunitsandadultvolunteer
donors.Thissinglepointofaccessassuresthatthesephysicianswillhaveaccessto
anypotentialdonororumbilicalcordbloodunitregardlessofwherelocatedacross
theglobeinordertoperformadultstemcelltransplantsthatcancuremorethan70
differentdiseasesorconditions,includingbloodcancersandsicklecelldisease.The
BeTheMatchRegistryhasgrowntoincludenearly12.5milliondonorsandmore
than209,000cordbloodunits.Throughinternationalrelationships,theNMDP/Be
TheMatchhasaccesstomorethan24.5millionpotentialdonorsand622,000cord
bloodunitsworldwide.
Forpatientsbattlingthesefatalcancersandotherblooddisorders,theNMDP/Be
TheMatchofferssupportandeducationtohelpthemlivehealthylivesafter
transplant.Weprovidepatientservices,caregiversupport,andfinancialsupport
throughtheBeTheMatchFoundation.Wealsoworkcloselywithtransplant
physiciansthroughoutthecountrybydevelopingandimprovinguponpost‐
transplantguidelinestoimprovesurvivalrates.
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Ourfocusisonpatientsforwhomcellulartherapyisthebesthopeforcureoftheir
diseasesandisoftentheonlytherapyavailablewithanintenttocure.Today,we
areabletotreatpatientswithcancersandpre‐cancers,suchasleukemia,
Myelodysplasia,andlymphomas;bonemarrowfailuredisorders,suchasaplastic
anemiaandimmunodeficiencysyndromes;andgeneticdiseases,suchassicklecell
disease.Totreatthesediseases,weinfusebonemarrow,peripheralbloodstem
cells,orcordbloodcellsintoapatientafterhavingeliminatedhis/hercurrentbone
marrow.Thesenewcellsrestorethepatient’sabilitytomakebloodcellsorprovide
anewimmunesystemtoattackcancercells.Findingthebestmatchpossibleis
importantbecauseifdonorstemcellsarenotthesameHLAtypeastherecipient
theywillrecognizetherecipientasbeingdifferentandattack,leadingtorejection.
Becausewecollectdataonalltransplants,wehavebeenabletoimprovepatient
outcomesandreducerejection.Between1988and2005,theone‐yearsurvivalrate
forthesepatientshasincreasedfromjustover40percentto70percent.Accessto
multiplecellsourcesallowsustofindthebestmatchpossibleforpatients.Initially
focusedonlyonbonemarrow,theProgramtodayalsoallowsphysicianstoselect
peripheralbloodstemcellsandcordblood,aswellasbonemarrow,asthesourceof
theadultstemcellsusedintransplant.
Weareespeciallyproudoftheworkwehavedonetohelpchildreninneedofa
transplant.In2014,theNMDP/BeTheMatchfacilitatedmorethan1,200unrelated
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transplantsforpatients18oryoungerusingbonemarroworcordblood.Our
currentresearchfocusesnotonlyonmalignancies,butalsooncuringnon‐malignant
diseasessuchcertainmetabolicdiseasesandhemaglobinopathieslikesicklecell
diseaseandthalassemia.Theseblooddisorderscanbefatalifleftuntreated.Prior
totransplantationtherapy,childrenwiththesediseaseswouldoftendie
prematurely.
Wearealsolearningmoreabouthowtoimproveoutcomesforchildrenfighting
bloodcancers.Byhavingaccesstoallthreesourcesofadultstemcells,physicians
canselectthebestsourcetomeettheiryoungpatients’needs.Forexample,most
physicianspreferafullymatchedbonemarrowgraftifavailableforallpatients,
includingchildren.Incertaininstances,umbilicalcordbloodisused,especiallyif
thereisnofullymatchedadultdonor.Inthisway,umbilicalcordbloodhas
significantlyextendedtheopportunityforallpatientswhootherwisewouldnot
havefoundanacceptableadultmatch.ThroughtheCIBMTR,researchers
throughouttheworldarefindingnewandexcitingwaysthatbonemarrowandcord
bloodtransplantscanhelpchildrenfightlife‐threateningdiseases.
MoreNeedsToBeDone
However,moreneedstobedone.Theneedfortransplantsisincreasing,especially
amongolderAmericans.Thecalculatedneedforunrelatedtransplanthasincreased
by25percentsince2005.Muchofthereasonisduetotheexpansioninthenumber
ofindicationsfortransplant,aswellasadvancesthatallowolderAmericanstobe
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candidatesfortransplants.Transplantsforpatients51‐64yearsoldaregrowing
fasterthanotheragegroups.NMDP/BeTheMatchfacilitatedtransplantshave
grownby200percentoveralland250percentforminoritiessince2006.
Whilewehavemadesignificantimprovementsintransplantsforracialandethnic
minoritypatients,theretoomoreworkisneeded.Thenumberoftransplantsfor
minoritypatientshasincreasedfrom253in2000to990in2014.Wecontinueour
effortstoexpandthediversityofthenationaladultvolunteerdonorregistryand46
percentofcordbloodunitsontheregistryarefromaminoritydonor.Duringthe
last5yearscordbloodhasbeentheproductsourceforabout21percentofall
transplantsand37percentofminoritypatientswhoreceivedatransplantrelied
uponcordblood.
Federalfundingremainscriticaltocontinuingtoprovideaccesstotransplantation.
Weneedtocontinuetorecruitnewpotentialdonorsbothtoimproveaccessfor
minoritypatientsandtorenewthecurrentlistofdonorswithyoungerdonors.
Graftsfromyoungerdonorshaveshownimprovedclinicaloutcomes.Foreveryone
milliondollarsallocatedbytheCongress,theProgramcanadd10,000adult
volunteerdonorsor750cordbloodunitstothenationalregistry.Preservingthese
fundsthroughthereauthorizationoftheProgramallowsittocontinuetoimprove
thechancesofeveryAmericanneedingatransplanttofindamatchandprovidesthe
criticalinfrastructurethatallowsNCBIcordbloodunitstobeusedtosavelives.
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EventhoughtheNMDP/BeTheMatchhasimprovedtheabilityofthoseneedinga
transplanttofindamatch,thereareotherbarriersthatcontinuetomakeaccess
difficult.Thesenon‐matchbarrierstoaccessandcarehaveaprofoundaffectonour
abilitytomaketransplanttherapiesaccessibletoall.Language,literacy,finances,
insurance,geography,lackofknowledge,andpredispositionbygeneral
hematologistsandoncologiststowardsnon‐transplanttherapiesallhaveanimpact.
TheNMDP/BeTheMatchcontinuestoworkwithpatients,physicians,community
leaders,andotherstoaddresstheseproblemsaswell.
Conclusion
TheongoingCongressionalcommitmenthasmadetheseadvancespossibleand
turnedthetragiclossof11‐year‐oldBrandyBlyintohopefortensofthousandsof
Americans.OneofthoseAmericansisHadleyMercer.Whenshewasjustsix
monthsold,Hadleywasdiagnosedwithacutemyeloidleukemia(AML).Aftertwo
roundsofchemotherapy,herparentsbegantoconsiderabonemarrowtransplant
asanalternativetreatmentoptionforHadley.Theyconsultedvariousphysicians,
whoallagreedthathavingabonemarrowtransplantwouldbeherbestchanceof
survival.Thefamilyheldabonemarrowdriveandregisteredmorethan1,000
peopleinfivehours.Afewmonthslater,Hadleywasmatchedwithayoungmanin
histwenties.Nowalmosttwo,Hadleyisalivebecauseofher“donorangel”.Sheis
alsoalivebecauseofyourcontinuedsupportfortheC.W.BillYoungCell
TransplantationProgram.
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TheNMDP/BeTheMatchhasneverforgottentheimportanceofthephysician’s
simplestatementthatinspiredCongressmanYoungandeverydayweareinspired
bythepeoplewemeet,youngandold,whoareseekingtofindamatchandundergo
atransplant.
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Appendix:StatisticalChartsandGraphs
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