2/19/2015

ANovaLeidosEnsaiosClnicos
PSGRADUAOSOBREDIREITODOMEDICAMENTO
AnaMariaNogueira
19fevereiro2015

EnsaiosClnicos
Contexto Regulamentar
PSGRADUAOSOBREDIREITODOMEDICAMENTO
AnaMariaNogueira
19fevereiro2015

2/19/2015

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.

I was MAD when I

said yes

Perfect, you can

plea insanity

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EnsaiosClnicos

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EnsaiosClnicos

Placebo

EnsaiosClnicos

Aleatorizao

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EnsaiosClnicos
Ocultao

While, in general, doubleblind designs are considered optimal (), this paradigm may
distort one of the fundamental objectives of phase III studies to compare a new regimen
with 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

ClinicalTrialsDirective
2001/20/EC
ThemostheavilycriticizedpieceofEUlegislationintheareaofpharmaceuticals.

Thenumberofapplicationsforclinicaltrialsfellby25%(2007to2011)

Thecostsforconductingclinicaltrialshaveincreased.

Staffneedsforindustrysponsorstohandletheclinicaltrialauthorizationprocess
havedoubled (107%);

Administrativecostsincreased98%increasefornonindustrysponsors

Insurancefeeshaveincreasedby800%forindustrysponsors.

Theaveragedelayforlaunchingaclinicaltrialhasincreasedby90%to152days.

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PwC/APIFARMA
EnsaiosclnicosemPortugal|Apresentaodosresultadosdoestudo

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30%Investimentoperdido!
25M
30%Doentesnoincludos!
1.699
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TheInnovationGap
LongDevelopmentTimes+LowSuccessRates=HighR&DCosts

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

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1950
2015
2050

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StrategicResearchAgendaforInnovativeMedicinesInitiative2

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Why Europe needs a

thriving
pharmaceutical
industry

Health & Wealth

Sustainable
Financing

Byensuringuptakeof
innovationEuropehasthe
potentialtofurtherimprove
healthoutcomesandwealth
creation

Appropriateuseof
innovativemedicinesis
essentialtoensuringa
sustainablehealthbudget

Growth &
Competitiveness

Pharmacouldhaveavital
roleinEuropesgrowthand
futurecompetitiveness

Source:EFPIA.Health&GrowthEvidenceCompendium(2014)

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ObjectivesoftheEurope2020andHorizon2020initiatives

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

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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,permitindoestimularevalorizaraexcelnciacientfica
doPas;

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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Behavior
Engine

Catalyzing
Engine

Execution
Engine

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na arte

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(MORE!) try

DoorDoNot.Thereisno
Yoda

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