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ANovaLeidosEnsaiosClnicosPSGRADUAOSOBREDIREITODOMEDICAMENTO

AnaMariaNogueira19fevereiro2015

EnsaiosClnicosContexto Regulamentar

PSGRADUAOSOBREDIREITODOMEDICAMENTOAnaMariaNogueira19fevereiro2015

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Medical Director, MSD Portugal. President of the Board of the Competence in Pharmaceutical Medicine,

Ordem dos Mdicos.

Member of the Clinical Research working group in APIFARMA Invited Professor, University of Aveiro. PhD Student, Health Sciences Department, University of Aveiro.

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I was MAD when I said yes

Perfect, you can plea insanity

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EnsaiosClnicos

7Placebo

8Aleatorizao

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Ocultao

While, in general, doubleblind designs are considered optimal (), this paradigm maydistort one of the fundamental objectives of phase III studies to compare a new regimenwith the existing standard under realistic clinical conditions

JournalofThrombosisandHaemostasis,6:227229,2007

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What we think success looks like

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What it actually looks like

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ThemostheavilycriticizedpieceofEUlegislationintheareaofpharmaceuticals.

Thenumberofapplicationsforclinicaltrialsfellby25%(2007to2011) Thecostsforconductingclinicaltrialshaveincreased.

Staffneedsforindustrysponsorstohandletheclinicaltrialauthorizationprocesshavedoubled (107%);

Administrativecostsincreased98%increasefornonindustrysponsors Insurancefeeshaveincreasedby800%forindustrysponsors.

Theaveragedelayforlaunchingaclinicaltrialhasincreasedby90%to152days.

ClinicalTrialsDirective2001/20/EC

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18PwC/APIFARMAEnsaiosclnicosemPortugal|Apresentaodosresultadosdoestudo

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30%Investimentoperdido!25M

30%Doentesnoincludos!1.699

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TheInnovationGapLongDevelopmentTimes+LowSuccessRates=HighR&DCosts

Source: Kaitin, Clin Pharmacol Ther, 2010;87:356-361

1950

2050

2015

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28StrategicResearchAgendaforInnovativeMedicinesInitiative2

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Appropriateuseofinnovativemedicinesisessentialtoensuringa

sustainablehealthbudget

Appropriateuseofinnovativemedicinesisessentialtoensuringa

sustainablehealthbudget

PharmacouldhaveavitalroleinEuropesgrowthand

futurecompetitiveness

PharmacouldhaveavitalroleinEuropesgrowthand

futurecompetitiveness

Sustainable Financing

Sustainable Financing

Growth & Competitiveness

Growth & Competitiveness

Why Europe needs a thriving

pharmaceutical industry

Why Europe needs a thriving

pharmaceutical industry

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ByensuringuptakeofinnovationEuropehasthepotentialtofurtherimprovehealthoutcomesandwealth

creation

ByensuringuptakeofinnovationEuropehasthepotentialtofurtherimprovehealthoutcomesandwealth

creation

Health & WealthHealth & Wealth1

Source:EFPIA.Health&GrowthEvidenceCompendium(2014) 35

Source:EuropeanCommission:Europe2020(2012);EuropeanCommission:Research&Innovation Horizon2020(2012)

ObjectivesoftheEurope2020andHorizon2020initiatives

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It was the best of times, it was the worst of times, it was the age of wisdom, it was the age of foolishness, it was the epoch of belief, it was the epoch of incredulity.

Charles Dickens

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BenefciosparaaparaasInstituies&Academia

Melhoriadaqualidadeassistencial Promoodoconhecimentoedesenvolvimentocientfico; Retenoeestmuloainvestigadoresnareadasade; Potenciaodacriarnovasparceriasentreempresas,unidadesdesadee

centrosdeinvestigao,permitindoestimularevalorizaraexcelnciacientficadoPas;

Melhoriadavisibilidadeecompetitividadeanvelnacionaleinternacional,proporcionadomaiorparticipaoematividadesinternacionais;

Contributoefetivoparaacriaoderiquezaparaopas.

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BenefciosparaosDOENTES

AcessoPrecoceaNovoseMelhoresMedicamentos MelhorEducaoemSade MelhoriadaQualidadeAssistencial Papelactivo noDesenvolvimentodeNovasMolculas

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BenefciosparaaIndstriaFarmacutica

Envolvimento dacomunidade cientfica Recrutamento deumapopulao representativa de

doentes Maiorrapidez UtilizaoEficientedeRecursos CriaodeParceriasdeInvestigao

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Formaoprepsgraduadaeminvestigaoclnica ValorizaocurriculardaInvestigaoClnica OrganizaoeLideranaInstitucional Definioecomunicaodeprioridadesestratgicas Definio,comunicaoecumprimentodemtricas InformatizaodeRegistosClnicos Controloegestofinanceira Condiestcnicasnoscentros AlocaodeRecursosHumanosdedicados

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ExecutionEngine

CatalyzingEngine

BehaviorEngine

na arte

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