EFPIA CRA-不可用和延迟的根本原因

Executivesummary1 Theunprecedented speed of innovation exhibited over the last five years and the promiseof the industry pipeline2provide an important opportunity to improve outcomes for patients.There is common agreement that the value of innovation isrealised onlywhen patientsbenefit from advances in treatment.However,asignificant number of medicinesare notavailable across allEuropean Union (EU)markets.3 EFPIAhas for many yearslooked at the length of time it takes for medicines to bemadeavailable. As illustrated by the most recent data in the PatientW.A.I.T.Indicator2025Survey,the average time to reimbursement for innovative treatments across EU27countrieshas reached597days, ranging from 158days in Germany to1,110days inRomania.The median time to availability is 532 days, with even more pronounceddisparities between countries-ranging from 56 days in Germany to 1,201 days inRomania.4There are patient access inequities within Europe, with significant differencesacross countries in the number of products available atany giventime and that the timetaken prior to national reimbursement also varies significantly from one country to another.The industry shares concern about these delays and recognise thatdelays and theunavailability of medicines harm patients.These concerns areimportant context for thedebate regarding the impact oftheEU’sGeneral Pharmaceutical Legislationand theproposed Biotech Actand whetheritwillimproveaccess to medicines for patients in theEU. Over the pastsixyears,EFPIA hasdocumentedthe root causesofaccess inequalityandfound 10interrelatedfactors that explainunavailability and delay(defined aslength of timefrom European marketing authorisation to availability atthememberstate level)withregardto innovative medicines, building on the W.A.I.T.analysis.5Thesefactorsarerooted inmedicines access systems and processes in theEU member statesand the correspondingimpact on commercial decision-making. They include a slow regulatory process,lateinitiation of market access assessment,duplicativeevidence requirements, reimbursementdelays,andlocal formulary decisions.Becausethe root causes are multifactorial, they canbe solved onlyby stakeholders working together. This yearwe includetwoadditional sections, oneonroot causes of unavailability and delayin smaller European markets(where smaller markets are definedasthose with a populationofless than 4 million and lower levels of filing)6andan additional analysis on regulatoryapproval assessments in the EU and comparator countries to monitor the potential impactof the US Most Favoured Nation (MFN) drug pricing policy on the availability of, and delaysin access to, new molecules across global markets. The industry considers that the root causes of unavailability and delay could be addressedthrough collaborative work withmemberstates,theEuropean Commission,and otherstakeholderson proposals to improve availability and reduce delays.This workmustoccurfrom the beginning of the process, includingproposals to speed up the regulatory processto deliversafe,high-quality diagnostics, vaccines,and treatments to patients as fast aspossible.The industry is committed to working with the European Commission toachievetheseshared goalsand ensurepatients across Europe have reliable access to life-savingmedicines. Thisrequirestargeted, evidence-based policy solutions.The industrysupportstheEuropean Commission’s ambition to ensure timely access to critical medicines throughthe Critical MedicinesActand the proposed Biotech Act. Finding workable solutions to improve patient access to medicines will require multi-stakeholdercollaborationandconsideration ofthemultifactorialnature of root causes ofunavailability and delay.EFPIA and its members have worked on a series ofspecificproposals to improve patient access to innovative medicines and reduce inequalities acrossEurope.The following are some of these proposals: •Working together to speed up the regulatory process to deliver safe andhigh-quality diagnostics, vaccines, and treatments to patients as fast aspossible.There is a shared aspiration to reduce regulatory approval times inEurope and bring them in line with international best practice.Several areas foraction within the existing legislative framework exist to address this: encourage theuse of new types ofclinical trials; allow greater use of data from real-world use ofmedicines; allow ongoing dialoguebetween the developer and the regulator abouta treatment throughout the development continuum.The proposed Biotech Actcould also improve the clinical trial environment, if appropriately designed andimplemented.Accelerating the regulatory approval timeline is even more importantgiven the potential impact from MFN. •Thecreation of a portal where marketing authorisation holders (MAHs) canprovide timely information regarding the timing and processing ofpricingand reimbursement (P&R)applications in the 30 European countries.Theportal builds on the industry’s commitment to submit P