价格管制对罕见病药物获取的影响和美国的经验教训.docxVIP

Theimpactofpricecontrolsonrare

diseasemedicinesaccessandlessonsfortheUS

September2025

FINALREPORT

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arCSVcrSSOCatesTheimpactofpricecontrolsonraredisease

arCSVcrSSOCates

TableofContents

ExecutiveSummary ii

1.Introduction 3

1.1Background 3

1.2Methodology 5

2.MFNandrarediseasemedicinesaccess 7

3.AccesstorarediseasemedicinesoutsidetheUS 8

3.1GreaternumberofregulatoryapprovalsofnovelmedicinesintheUScomparedtoEuropeand

Japan 8

3.2Persistentaccesschallengesdisproportionatelyaffectrarediseasemedicines 10

4.ImplicationsofpricingandaccesspoliciesforUSrarediseasedrugaccess 12

4.1Conclusions 14

Appendix:Theimpactofthepolicyenvironmentonaccess 14

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arCSVcrSSOCatesTheimpactofpricecontrolsonraredisease

arCSVcrSSOCates

ExecutiveSummary

Sustaininginvestmentindrugdevelopmentforrarediseasesisinherentlychallengingduetosmall

patientpopulationsandhighfixedcostsfordrugtrials.CertainUSpolicyapproachesacknowledge

thesechallenges;thisincludestheOrphanDrugActof1983,whichincreasesthefinancialincentiveforrarediseasedrugdevelopment,andarecentexemptionformedicineswithmultipleorphan

diseaseindicationsfromMedicaredrugpricenegotiationsintheOneBigBeautifulBillAct.However,contemplatedadministrativepricesettingor“pricecontrols”formedicines,includingexternal

referencepricing(wherepricesinonecountryareusedtoinformpricesinanothercountry),riskcreatingabarriertodrugdevelopment,particularlyformedicinesaddressingorphandiseases.

Inlightofthepolicydebateaboutdrugpricing,CharlesRiverAssociates(CRA)wascommissionedbytheRareDiseaseCompanyCoalition(RDCC)toinvestigatetheeffectofinternationalapprovaland

pricingschemesonrarediseasemedicineaccess.Inthisreportweevaluatehowinternationalprici