ChinaOrphanDrugsEmergencyAidProjectDuringCOVID-19

ActionReport

ChinaOrphanDrugsEmergencyAidProjectDuringCOVID-19ActionReport

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ProjectOverview

TheCOVID-19pandemicbrokeoutinChinaattheendof2019.ItstartedfromHubeiProvinceandquicklyspreadacrossthecountry.TheChinesegovernmentproactivelytookmeasurestofightagainstthepandemic,whichincludingthelockdownofWuhanfromJanuary23thtoApril8th,2020,aseriesofrestrictionsontraveling,andredirectionofmedicalresourcesandsupplies.Ontheonehand,thesemeasureshavepreventedthespreadofCOVID-19.Ontheotherhand,therarediseasecommunityfacedanevenmorechallengingsituationofaccessingtreatmentsandhealthcare.

InJanuary2020,theIllnessChallengeFoundation(ICF)receivedacallforhelpfromtheorganizationofTuberousSclerosis(TS)patients,expressingthattheircommunityisinanemergencyofdrugshortage.BecauseChina'smedicaladministrationhasnotapprovedsomemethodsoftreatmentforTS,ChinesepatientswithTShavetobuydrugsoverseas.AffectedbytheCOVID-19crisis,internationaltravelandlogisticshavebeenrestricted.Inthiscase,TSpatientsinChinacouldnotobtainthenecessarydrugsthroughpreviouschannels.Thedruginterruptionmaycauserelapse,mayevenbelife-threatening.ThissituationdidnotonlyoccurintheTSgroup.JustfromtheendofJanuarytoearlyFebruarythisyear,theICFreceivedmorecallsforhelpfromdifferentrarediseaseorganizations.

OnFebruary11,2020,theICFandChinaAllianceofRareDiseases(CHARD)1formedajointtaskforceandlaunchedthe"ChinaOrphanDrugsEmergencyAidProjectDuringCOVID-19".BoththetaskforceandtheProjectwereactivefor3months(fromFebruary11thtoApril10th,2020),whichweretheworstperiodofthepandemicinChinaandwhenrarediseasepatientsweremostinneed.

Duringthesethreemonths,thetaskforceliaisedwithallparties,andrespondedtothestrongestneedsfromtherarediseasecommunity.Inparticular,thetaskforceprovidedsolutionsto382rarediseasepatientswhowereendangeredbydrugshortage.Moreover,toresolvetheissuesofparent-childcommunicationandlackingofpersonalprotectiveequipmentduringCOVID-19,thetaskforcedistributedtheboardgame“Para

���������������������������������������� ������TheChinaAllianceforRareDiseases(CHARD)isapprovedbytheNationalHealthCommission;ledbyPekingUnionMedicalCollegeHospital(PUMCH),ChinaPharmaceuticalInnovationandResearchDevelopmentAssociation(PhIRDA),ChinaHospitalAssociation(CHA),ChineseResearchHospitalAssociation(CRHA);iscomposedofmedicalinstitutions,universities,scientificresearchinstitutes,andindustry;isanationalvoluntary,non-profit,cooperativeplatform

TiBaby”2to152familieswithrarediseases,aswellas1390facialmasksto156familiesand5rarediseasesorganizationsintotal.

ProjectDetails

ProjectProcedure

ThetaskforcewascomposedofthestaffofCHARDandtheICF,withthedivisionof���������������������������������������� ����2“ParaTiBaby”isa,interactivefamilyboardgamedevelopedbytheICFforchildrenwithrarediseasesaged4-9,toencouragechild-parentcommunicationandtoreducepsychologicalstressofthefamily.Formoreinformation:https://mp.weixin.qq.com/s/drOfwT3JyX_cIgRbMH0o8g�

ProvideEmergencyAid�

CHARDliaisedwithPUMCHandICF,discussedthesolutionforeachverifiedcase.CHARDandPUMCHconnectedlocalhospitalsformedicalresources,andICFconnectedwithpatientsandpatientorganizations.�

Follow-up�

ICFcontactedeachverifiedapplicantaccordingtopriority,andcontactedpatientorganization/supportgroup(iftherewasoneforsuchspecificraredisease),thenreportedbacktoCHARDwithverifiedandconformedinformation.�

CategorizeandPrioritize�

ICFverifiedcaseinformation,categorizedandprioritized,andreportedbacktoCHARDfordoublecheckingby5p.m.everyday�

CaseRegistration�

CHARDcollectedinformationofallapplicants(patientswithneeds)withtechnicalsupportfromPUMCHandreportedtoICFtwiceaweek.�

labor,timelycommunication,andorderlyconnection.CHARDstaffdesignedandimplementedanonlineregistrationtogatherpatients’basicinformation,whiletheICFdisseminatedtheprojectinformationinpatientcommunity.

Aftertheprojectwasstarted,Sincetheprojectstarted,CHARDexportedpatients’informationonadailybasis,andtheICFverifiedthepatients’informationandcollectedfeedback.Eachpatient’sinformationwasreviewed.Iftherewasapatientorganization/supportgroupforthespecificdisease,ICFwouldconfirmtheurgencyoftheneedsandlearnthecurrentsituationofthediseaseasawhole,suchasthereasonscausingdrugshortage,andwhethertheorganization/supportgrouphadresponsemeasures.Whereafter,ICFcontactedthepatientstoverifyandconfirmtheirneeds.Telephoneinterviewswerecarriedoutintermsofthepatients’conditions,reasonscausingdrugshortage,thehospitalsanddoctorstheynormallyvisited,thetypesandspecificationsoftheirdrugs,howlongtheycouldlastwithremainingdrugs,howseriousdruginterruptionwouldaffectthemandifitwaslife-threatening,andthecostofthedrugs(reimbursement/out-of-pocketratio).ThentheICFcategorizedandprioritizedaccordingtotheurgencyofneeds,listedthenamesofcompaniesmanufactured/suppliedtheneededdrugs,andthenumberofpatientsineachspecificdisease.Themosturgentcasesweremarkedindividually,followedupclosely,andresolvedwithin1workingday.

CHARDconnectedwithhospitalsandcompaniesforsolutions.CHARDcarefullyanalyzedtheunderlyingcausesofeachdrugshortagecase:wasitduetologisticsinterruption(nointernationaldeliveries,thelongertimefordeliveries,etc.),thedecreaseofhospitalsandmedicalprofessionals(medicalresourcesredirectedtoinfectedpatients,ortoseverelyaffectedareas),thefactthatthepatientwasinaseverelyaffectedarea(HubeiProvince),orinabilitytopay(unabletocomplywiththereimbursementprocedures,joblossduetothepandemic,etc.).Then,CHARDprovidedtargetedsolutionsforeachcase.

Forpatientswhowerenotabletotravelforhospitalvisits(Scleroderma,PropionicAcidemia,Acromegaly,etc.),thetaskforcecontactedhospitals[includingPerkingUnionMedicalCollegeHospital(Beijing)andtheFirstPeople’sHospitalofJinzhou(HubeiProvince)]tocoordinateprescriptions.Meanwhile,thetaskforceactivelycommunicatedwithlocaldrugsupplychannelsandprovidedpatientswithinformationonlocalaccesstotreatments.

Forpatientswhowereinseverelyaffectedareasandonlockdown,thetaskforcecontactedDTPpharmacies.Ifneededdrugswereinstock,thetaskforcecoordinatedwithlogisticscompaniesfordelivery;Iftheywereoutofstock,thetaskforcecontactedandcoordinatedwithbothdrugsuppliersandlogisticscompaniesforsolutions.Forexample,inthecasesofpatientswithAngelmanSyndromeinHubeiProvince,DTPpharmaciessentthelife-savingmedicinestothreecitiesinHubeiProvincebyexpressdeliveryattheworsttimes.

Forpatientswhowereunabletocomplywiththereimbursementproceduresduetothepandemic,thetaskforcecontactedhospitalsandmedicalprofessionals,coordinatedtoprescribelongtermrepeatprescriptionsforthepatientstoreducehospitalvisits.Thetaskforcealsogatheredanddisseminatedthereimbursementcoverageinformationoflocalhospitalsandpharmacies,andmonitoredtheirdrugstorage.InthecasesofAcromegaly,CHARDcoordinatedwiththedrugsuppliers,whosharedinformationontheirnationalsalesnetworkandthereimbursementcoverageforhospitalsnationwidetoAcromegalypatientorganizationsothatthepatientorganizationcouldhelptheirmemberstogainaccesstothedrugsduringCOVID-19.

Thetaskforceprioritizedthemostlife-threateningcases,followedupwithhospitals,drugcompanies,pharmacies,andpatientorganizations/supportgroupstomonitortheprogressandoutcome.Afterthat,thetaskforcerevisitedeachpatienttoensuretheiremergencywascopedwith.

Throughmultiplechannels,thetaskforcecoordinatedwithmultipleparties,includingpharmaceuticalcompanies,commercialcompanies,DTPpharmacies,industry/academicassociations,consultingfirms,etc.

Duringtheimplementationoftheproject,thetaskforcealsodiscoveredthatmanypatientsandfamiliesfacedotherdifficultiesinthepandemic,suchastheshortageofpersonalprotectionequipmentandparent-childcommunicationbarriers.Inresponsetotheseproblems,thetaskforcedistributed1,390facialmasksto156familieswithrarediseasesandfiverarediseaseorganizations.Meanwhile,152rarediseasefamiliesalsoreceivedthe“ParaTiBaby”boardgamesfromthetaskforce.

ProjectOutcomes

• Providedsolutionsto382patientswithrarediseasesfacedwithdrugshortage,andpublishedinformationontreatmentaccessibilityandmedicalinsurance,benefitingmorepatientswithvariousneeds.

• Distributedtheboardgame“ParaTiBaby”to152familieswithrarediseases,anddistributed1,390facialmasksto156familiesandfiverarediseaseorganizationstoreducepsychologicalstressandchancesofinfection.

• Elevenpatientorganizations,threehospitals,dozensofcompanies,industry/academicassociations,andDTPpharmacieswerelinked,amongwhichnolessthan30interestedpartiesintotal.

• FivemediaoutletsincludingCCTV,BeijingNews,XinhuaNet,BeijingNewsBroadcasting,andDisabilityinChinareportedontheProjectindetail.

NotableCases

CaseA:GettingMedicationsforPatientswithScleroderma.

FuSuPian,FuSongPian,andFuKangPianwerethreeessentialmedicationsthatpatientswithSclerodermaneededtotakecontinuously.Themostuseddrug,FuSuPian,wascompoundedandhand-madebyPekingUnionMedicalCollegeHospital(PUMCH).Patientsfromalloverthecountrycametothehospitaleverythreemonthsfortheirroutinefollow-upsandrepeatedprescriptions.However,duringCOVID-19,trafficcontrols,travelrestrictions,combinedwiththefearofcross-infectioninthehospitals,madeitverychallengingforpatientstogotoPUMCHandgettheirprescriptions.AftertheICFreceivedseveralcallsforhelpfrompatientswithScleroderma,thetaskforcestarttowork.TheICFverifiedtheneedsoftheChineseOrganizationforScleroderma.Meanwhile,CHARDreachedouttoPUMCH,seekinghelpfromDr.ZhangTao,thedoctormostmentionedbySclerodermapatients.CHARDaskedDr.ZhangTaotoconfirmthepatients’medicalrecords,verifytheirneeds,andissueprescriptionsremotely.Thenthetaskforceassistedindeliveringthedrugstopatients.Duringthisperiod,CHARDcoordinatedwithPUMCH’sDermatologyDepartment,OutpatientDepartment,andHospitalPharmacytosetupaGreenPassage(expeditedservices)forpatientswithScleroderma.Over150patientswithSclerodermareceivedtheirmedicationsin2batches.

CaseB:TheStoryofFengQinfen.

Feng,apatientwithErdheim-ChesterDisease(ECD),araretypeofNon-Langerhans’cellhistiocytosis(alsoknownaslipogranulomatosis),wasinhersixtiesandhadsufferedalot.Likemanyotherpatientswithrarediseaseswhorequirelong-termcontinuoustreatments,Fengneededrecombinanthumaninterferonα2ainjectioneveryotherday.FengvisitedPUMCHregularlyforhertreatments,eventhoughshelivedinNanjing,JiangsuProvince,whichwashundredsofmilesawayfromthehospitalthathadthecapacityoftreatingECD.Itwastime-consumingandexpensiveforherandherfamilytogotoBeijingtogetthetreatments,aslongasshecouldgetthetreatmentsforherraredisease,alleffortswereworthwhile.

WhentheCOVID-19hitthewholecountryduringtheChineseSpringFestivalholidayseason,trafficrestrictionswereissuedandcitieswereclosedoff.Feng’smedicinesfromherlastprescriptionwerecomingshort.Eachday,Fengandherfamilycrossedtheirfingersthatthepandemicwouldendsoon,whichwouldmakethetravelingtoBeijingpossible.Onthecontrary,thesituationwasgettingworse.Feng’sdaughterwenttoalllocalpharmaciesandhospitalsbutfoundnorecombinanthumaninterferonα2ainjectionsinstock.

Inthemomentofbeingpanicanddesperate,Feng’sdaughtersawthenewsofthe

Project.Sheappliedforhermother,wasinterviewedbythetaskforce,andwaitedanxiouslyforasolution.Justinadayortwo,thetaskforcecalledbacktoFeng’sdaughterwiththegoodnewsthattherewererecombinanthumaninterferonα2ainjectionssuppliesinNanjing,JiangsuProvince.However,itturnedouttobeadifferentdosageform,whichwasmuchtothedisappointmentofFeng’sdaughter.Shecouldnotdoanythingbuttowaitforfurtherinformation.Soonafter,CHARDconfirmedthattherewerethesametreatmentsthatFengusedinNantong(about250kilometersawayfromNanjing,whereFenglived),JiangsuProvince.TheICFconfirmedwithFeng’sdaughterthatshewasabletotraveltoNantong.ThenthepharmaceuticalcompanywhichmanufacturedthetreatmentsmadesurethetreatmentswouldbedeliveredtoaspecificpharmacytimelyforFeng’sdaughtertopickup.Atlast,allsettledsatisfactorily.

ConclusionandReflection

1.Thecomplexityoftherarediseasewasfurtherrevealedthroughthepandemic,thesolutionofwhichmustbefoundbythecollaborationofallparties.

Thejourneytosolverarediseaseproblemshadalwaysbeendifficult.It’sacomplicatedsystematicissue.Duringsuchaperiodofthepandemic,allaspectsofcomplexitywereexposedmoreclearly.Forexample,althoughthetaskforcecontacteddrugcompaniesandsuppliers,localpharmacies,andonlinemedicalserviceproviders,neededtreatmentsforpatientswithHemophiliainHubeiProvincecouldn’treachthem.ColdChainLogisticsforthedeliveryofthetreatmentswasn’tpossible.Itwasagainstlockdownpoliciesandtooriskyforpatientstogotohospitalsorpharmacies.Everypartyinvolvedhaddifferentconcernsandinterests,whichwasbothtrueandfrustrating.But,nomatterwhat,thecomplexityoftherarediseaseproblemmadeitmoreclearthattheonlywaytotacklethisproblemwastobringinallstakeholdersandcollaborate.

2.Thedifferentperspectivesofallpartiesledtodifferentactions.

TheindustrywasnotasmotivatedthroughouttheProjectwiththeirinterestsbeingconcerned.Patientorganizations/supportgroupshaddifferentexpectations.DuringtheProject,allpartiesworkedcloselytofightagainstthisemergency,whichwasalsoanopportunityofre-thinkingexpectations,values,andboundaries.Asapatientorganizationwiththevisionofknittingallpartiestogetherandempoweringthepatientcommunity,ICFshouldre-evaluateitsroleinsolvingrarediseaseproblemsandwhichwaytoleaditsactionsandpartners.Forexample,ICFsharedtheavailabilityandinformationofaspecificdrugwithpatientorganizationsandthepatientcommunity.However,someorganizationsandpatientsconsideredtheinformationasmedicaladvice,whichtheyshouldnotbeusedasmedicaladvice.Theyexpectedtorelymoreon

theICFtomakehealthcaredecisions,whichcouldbeindeedriskyandneededtobehandledwithcaution.WhattheICFprovidedshouldonlybeinformationondrugavailability,whilemedicaladviceshouldbemadebyprofessionals.Patientorganizationsandpatientsshouldbemoreautonomousandresponsiblefortheircommunity’sortheirhealthcare.Generally,patientsweremoreknowledgeableabouttheirdiseases.ThiswasadiscussionworthtobebroughtupbytheICFinthehopesthatpatientorganizations/supportgroupscouldbeempoweredonhowtofilter/verify/deliverinformationtotheircommunity.

3.Newlearningofpatientorganizations

TheICFworkedwithvariousraredisease-specificpatientorganizations.Thedevelopmentofthesepatientorganizationswasverydifferent.Somewereregisterednon-profitorganizationswithseveralfull-timestaff(developedorganizations).Someweresupportgroupsoperatedwithafewvolunteers(developingorganizations).Developedorganizationshadstrategicplanning,projects,operatingteam,funding,andwereabletomobilizemoreandwidercommunity.However,duringtheProject,theICFgainedmoreinsightintodevelopingorganizations.Eventhoughsomeoftheorganizationsonlyoperatedbyvolunteers,theystillmanagedtoreachouttotheircommunityandresolvetheirurgentneeds.Forexample,theChineseOrganizationforSclerodermawasayoung,growingorganization,whichonlyhadonefull-timestaff(founder,asclerodermapatient).DuringtheProject,theChineseOrganizationforSclerodermamanagedtomobilizetheirpatientsasvolunteerstodividelaborandcooperatetorespondtotheemergingneeds.Itdidnotnecessarilyhavetobearegisteredorganizationwithfull-timestafftodothingsright.Aslongasyouweredetermined,devoted,andcaredfortherealneedsofthecommunity,onepersoncoulddoalot.Intheend,notonlythedrugshortageforindividualpatientswereresolved,butalsoaGreenPassagewhichwouldbenefitthewholecommunitywassetup,withsupportfromthedoctors,hospital,andCHARD.ItwasenlighteningfortheICFonfuturecooperationwithpatientorganizations:whatcharactersoftheorganizations/leadersshouldbevaluedmostwhentakingaction.

Forfutureactionsandprojects,theICFshouldengagemorepatientorganizations,establishcollaborationwithmorepartiesbesidesCHARD,andcreatemoreopportunitiesforcommunicatingwiththepatientsforallICFstaff,toimproveempathyandservicequality.Withtheseexperiencesandthoughts,theChineserarediseasecommunityshouldmoveforwardinsolidarity.

Pictures

MemberoftheProject’staskforcewenttoPekingUnionMedicalCollegeHospitaltocollectdrugsforpatientsaroundthecountryinFebruary2020.

MedicalprofessionalsfromthePekingUnionMedicalCollegeHospitalcheckeddruginventoryanddetails.

Patientreceivedthemedication.

Drugswerepackedandreadyfordelivery.

InFebruary2020,whenthepandemicwasmostsevereinWuhan,volunteerswenttoapharmacyclosedtotheWuhanSeafoodMarkettogetdrugsforpatients.

ReportwrittenbytheIllnessChallengeFoundation.SpecialthankstoMs.LILingandMs.YINGXiaoyufortranslatingthisreportintoEnglish.

Formoreinformation,pleasecontact:HelenZhouzhoushuang@chinaicf.org