小型市场不可用和延迟的根本原因——国家分析

European Access Hurdles Portal& the Root Causes AnalysisSmaller markets Revised Country AnalysisMay 2025 Approach:We have followed a multi-step approach to understandthe root causes of unavailability and delay in smaller markets Approach:We have applied this framework to understand patternsof filing and availability in each smaller market Why might there be delays in filing of a newly centrally approved medicine? Summary results:We find that the reasons for delays in filing insmaller markets are multi-faceted and driven by the local context In Malta, there is a low reimbursement rate (2%) of innovativemedicines and a low filing rate (9%) •Either MAHs or Medical Consultants can filefor inclusion on the Government Formulary List (GFL)•Pharmacists within theDPAcompileHTAsincludingclinical and budget impactassessments for review by the GFLAC•The GFLAC and ACHCB review outputs from the DPA an issue bothtechnical and financial recommendationsto theMoHon whether an innovative product should be included on the GFL•Along with theMinister for Finance,theChief Medical OfficerandSenior Health Officials,theMoHis responsible forsetting out prioritizationbefore the CPSU are responsible forprocurement, negotiatingwithMAHsandissuing tenders New perspective on Root Causes 1.There are nodirect barriersimpacting the timing of filing in Malta, but in practice, payers reference the completed assessments and decisionsmade in other European countries2.The Maltese P&R system is chronicallyunder-resourced, leading todelays in decision-making,budgetary constraints seespecific therapyareas prioritizedfor procurement3.There is asignificant lack of transparencyas to aproduct’s statusafter filing and a significant backlog, discouraging filling4.The tenders are largelysingle-winner, price-onlyand lasting3-4 years, meaning innovative medicines can be frozen out of the publicreimbursement market •It isnot necessary for medicinesto havefiledand be listed on the GFL to bid for a tender, creating thepossibility for reimbursed patient accesswithout filingandgoing through the standard reimbursement system•TheMaltese Community Chest Fundoffers public access for products which are not supplied via standardreimbursement route anddoes not require filing•TheExceptional Medicines Treatment Committee (EMTC)assess and approve medicines on a named patientbasis for rare disease medicines not currently on the GFL In Malta, it is not necessary to file a medicine for P&R in order toachieve broad patient access How can we interpret this considering our understanding of the market? What does the Portal data tell us? o15% availability through an alternative public channelis likelyan underrepresentationof company willingness to provideaccess; it partially represents the difficulty for innovativemedicines to win tenders (class level, winner-takes-all, pricecriterion, can be competing against generics) We expect that the impact of local filing requirements isunderrepresented in the Portal data for Malta What does the Portal data tell us? How can we interpret this considering our understanding of the market? oWhilst there is noformalrequirement that makes it impossibleto file, there areinformalpreferences. As a country of only500,000 people, payers would prefer to leverage assessmentsfrom other countries for efficiency A major issue impacting filing decisions in Malta is likely not beingfully captured by the current options in the Portal How can we interpret this considering our understanding of the market? What does the Portal data tell us? oOur hypothesis is that this issue cannot be picked up by thePortal’s current reasons for non-filing, and may in part explainsome of thehigh proportion of ‘Other’responses that areconsistently picked up in the data32% It is challenging for companies to make a reasonable business caseto launch in Malta as a result of significant budget constraints How can we interpret this considering our understanding of the market? What does the Portal data tell us? The raw Portal results for Malta will therefore need to be framed witha more nuanced description of the complex barriers to filing In Cyprus, there is a low reimbursement rate (1%) of innovativemedicines and a low filing rate (20%) •Manufacturersmust file for inclusionin the positive reimbursement list of theNational Health System (GESY)•TheMedicines Advisory Committee (MAC)are then responsible for assessing the submissions; although clinical bestpractices are considered, the main focus is onthe budget impactof the innovative product•If the MAC recommends inclusion, the Medicines Reimbursement Advisory Committee (MRAC) willnegotiate an acceptablepricewith the manufacturer for inclusion in the positive reimbursement list New perspective on Root Causes 1.There are nodirect barriersimpacting the timing of filing in Cyprus, but in practice, payers reference the completed assessments and decisionsmade in other European countries2.The MAC areunder-capacityan