JohnForman–ICORDPresidentSuppor9ngcountriesemergingtorarediseases

RareDiseasesInterna9onal–Edinburgh25May2016

Aboutme WithJudith,TimothyandHollieTwins,age41 Alpha-Mannosidosis,arareLysosomalstoragediseasewithprogressiveandsevereimpacts InvolvedinmanyrarediseasegroupsinNZandworld-wide CurrentPresidentofICORD

A41yearjourneythatneverends ATer50daysinhospitaland42daysonIVan9bio9cs Anaddi9onalrarediseaseforHollie XGPkidneydisease NoOrphanetorGARDentry,buttreatable!!

IntroducingICORDØ Societysetupin2007(firstICORDconference2005inStockholm)Ø Amul9-stakeholdersocietyfocusedonrarediseasesandorphandrugsØ Membersincludehealthofficials,regulators,researchers,clinicians,industry,academics,pa9ents,advocacygroupleadersØ Hasheld10interna9onalconferencesØ Sweden,Spain,Belgium,USA,Italy,Argen9na,Japan,Russia,theNetherlandsandMexicoØ 2016–CapeTown,SouthAfrica.Ø 2017–China?2018–India?

ICORD’spolicystatement

Ø TheYukiwarisoDeclara9on

Ø PublishedinActaPaediatricaVol101,Issue8,pp805-807,August2012(summaryversion)-Alsoatwww.icord.se(fullversion)

Ø Thedeclara9onprovidesara9onaleandframeworkforlegisla9on,policies,ac9onplans

TheDeclara9on’s6principles

Ø 1.RDsareasignificantpublichealthissue–6to8%Ø 2.Humanrightsandgovernmentdu9esareinvolvedØ 3.RDresearchandproductdevelopmentshouldbesupportedØ 4.AcomprehensiveapproachtorarediseasesshouldbeadoptedØ 5.Theimportanceofpa9entautonomy,consentandinforma9onneedsØ 6.Includepa9entgroupsinpolicyandservices

Thereare12guidancepointstoassistimplementa9onoftheseprinciples

ThedevelopingworldØ OurDeclara9on’sadviceonimplementa9onofrarediseasepoliciesincludesreferencetothedevelopingworld

Ø But“light”onanalysisofthatpoint

Ø Howtoeffec9velypromoteRDpolicyandac9onplansindevelopingworld?

Ø AmajorchallengeforICORD,RDI,WHOandUNagencies

RarediseasesinthedevelopingworldØ Easiertoarguewhenprimarycareandpublichealtharewellestablished

Ø Easierforpoli9ciansandofficialswhenresourcesareplen9ful

Ø Manycountriesrelyonlineardevelopment,i.e.whenthebasicsareinplace

Ø Buttherighttohealthappliestoall,includingthosewithrarediseases

RightsandequityØ Progressiverealisa9onoftherighttohealthmeansworkingfromwhereyouareandprovidingforallsec9onsofthecommunityØ Rarediseasepa9entsindevelopingworldsufferdoubleortripledisadvantagesofpoverty,limitedbasichealthcare,andlackofprovisionfortheirrarediseaseØ Withoutaccesstobasichealthcare,rarediseasepa9entshavehighermortality,greatermorbidityandcompoundedsocialdisadvantageØ Establishingthebasicsimprovesoutcomesforall,includingthosewithrarediseasesØ Thebasicsareanecessaryplamormfordiagnosis,care,preven9onandtreatmentofrarediseases

Modelstoconsider

Ø WHO/WAOPBD,1999.ServicesforthePreven9onandManagementofGene9cDisordersandBirthDefectsinDevelopingCountriesØ Aformulaforparalleldevelopmentofspecialistservices

Ø Offersgoodinsightintowhatcanbedone

Modelstoconsider

Ø Mexico’seffortsfrom2004throughSeguroPopular,agovernment-fundedprogram.Ø 52.6millionpreviouslyuninsuredMexicansenrolledinhealthcareinlessthanadecade.

Ø Achieveduniversalhealthcareby2012.Ø WorldBank2013–“NinecountriesfromacrossLa9nAmericaandCaribbeanhighlightedfortheirhealthcaresuccesses.”

ModelstoconsiderØ Philippines-KalusuganPangkalahatan,UniversalHealthcareØ Since2012,boostedhealthcareexpenditurebycloseto100%throughaSinTaxontobaccoandalcoholØ Healthcarecoverageextendedto82%ofthepopula9on-anaddi9onal45millionpeople

Ø 1,000moredoctors,10,000morenurses,6,000moremidwivesØ Asolidfounda9on-necessaryforthesurvivalandiden9fica9onofrarediseasepa9ents

Combinethemodels

Ø Buildthebasisanddevelopthespecialistservicesatthesame9me

Ø AvoidtheunfairnessofalinearapproachØ Comprehensiveprimaryandpublichealthservicesneedspecial9eslikepathology,clinicalgene9csandlaboratoriestosupportthem

Ø Thesespecialistservicesalsoprovideessen9albasicstoiden9fyandcareforrarediseases

Ø Recognisethedifferentstagesofdevelopmentthatmeandifferentapproachesareneedinthedevelopingworld

“Topdownandboqomup”

Ø UN,WHO,WorldBanksupporttoinfluencegovernments

Ø Partnershipswithresearchandmedicalallies

Ø Buildgrassrootspa9entadvocacy

Ø Workonall3levelsinparallel.Oneortwoonlywillbeverydifficult

AnidealopportunityØ Rarediseaseday2013“Rarediseasesmustbecomeapublichealthpriorityaroundtheworld.Improvedaccesstodiagnosis,accurateinforma9on….accesstohealthcareandsocialservices,andafocusonresearchthatwillleadtotreatmentsorevenacureforpeoplelivingwithararedisorder.”HelenClark,FormerPrimeMinisterofNZ,UNDPAdministratorandCandidateforUNSecretary-General

Bewarethepimalls

Ø Templatemodelswillnotwork.Ø Challengesofcross-borderhealthcareØ Absenceofgene9cservicesØ CriteriaforOrphanetØ Languageandpoli9cal/culturalbarriersØ Verylimitedpa9entadvocacynetworks

Ø Allofthesemeanwecannotjust“transplant”modelsofrarediseasepoliciesandservicesfromotheradvancedhealthcaresystems

Thetakehomemessage

Ø Promoteparalleldevelopmenttoensurefairandequitableprovisionforallsec9onsofthecommunityØ Encouragegovernments,healthplannersandhealthagencies,whilealsobuildingpa9entadvocacyandallianceswithotherrarediseasealliesØ AdaptpoliciestostagesofdevelopmentØ Avoidrelianceontemplatesolu9ons

Thankyou

Ø Tomyfamilyforsuppor9ngmeinrarediseaseadvocacyØ Tomymotherforins9llingmyvaluesaboutcaringforthedisadvantagedØ Toallofyou,whoseworkandinterestshelpsimprovethelivesofthosewithrarediseases