anna bucsics presentation

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1 Seite 1 2012 M A. A S S I H T A W E 2012  P 1 A A B, H S D P A A A Seite 2 2012 M A. A S S I H T A W E 2012  P 2 A T   D  P A, MEDEV C, EUHTA . T . S E S

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Page 1: Anna Bucsics Presentation

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Seite 1 2012 M A . A S S I H T A W E 2012 P 1

A

A B , H S

D P A A A

Seite 2 2012 M A . A S S I H T A W E 2012 P 2

A

T D

P A , MEDEV C ,

EU HTA .T .

S E S

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2012 M A . A S S I H T A W E 2012 P 3

.

,

, .

D

Seite 4 2012 M A . A S S I H T A W E 2012 P 4

T B

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Seite 5 2012 M A . A S S I H T A W E 2012 P 5

O D C

• RARITY ( ) M 5 10,000 C ( 250,000)

OR

• RETURN ON INVESTMENT: W

• SERIOUSNESS: L

• ALTERNATIVE METHODS AUTHORISED

• I

F :// . . . / / GB/ /P /2011/06/WC500107046.

Seite 6 2012 M A . A S S I H T A W E 2012 P 6

W ?

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. R

,

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P M O C EH C . W , B H , 7. O 2010

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2012 M A . A S S I H T A W E 2012 P 7

A , , ‖

BIOMARKERS DEFINITIONS WORKING GROUP:BIOMARKERS AND SURROGATE ENDPOINTS: PREFERREDDEFINITIONS AND CONCEPTUAL FRAMEWORK.CLIN PHARMACOL THER 2001;69:89 95.

B D

Seite 8 2012 M A . A S S I H T A W E 2012 P 8

B E F

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R M : :// . / / /D

Maraviroc: Before taking CELSENTRI it has to beconfirmed thatonly CCR5-tropic HIV-1is detectable(i.e. CXCR4 or dual/mixed tropic virus not detected)

using an adequately validated and sensitive detectionmethod on a newly drawn blood sample. The

Monogram Trofile assay was used in the clinicalstudies of CELSENTRI […] Other phenotypic and

genotypic assays are currently being evaluated.

C C:ObjectiveResponse Rate for EGFR-mutation positive patients:71% Objective ResponseRate for EGFR-mutationnegative patients: 1%

A :LA-B*5701BUTIn any patient treatedwith abacavir, theclinical diagnosis ofsuspectedhypersensitivity reactionmust remain the basis ofclinical decision-making

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Seite 9 2012 M A . A S S I H T A W E 2012 P 9

A C

.

,

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I H B E R MSG ., N E J M 2012; 366:883 892,M 8, 2012,DOI:10.1056/NEJM 1113205

Seite 10 2012 M A . A S S I H T A W E 2012 P 10

B

• P :• M A• T D (89/105/EWG)

• N R• D :

• M D D (93/42/EEC)• N R

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Seite 11 2012 M A . A S S I H T A W E 2012 P 11

C D 89/105/EEC 21 D 1988

• L S : D , I C E C

• S : D

• C : D : – T ( 90 + 90 )

– B & – T

Seite 12 2012 M A . A S S I H T A W E 2012 P 12

C D 93/42/EEC 14 1993

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Seite 13 2012 M A . A S S I H T A W E 2012 P 13

THE FOURTH HURDLE

A• Q• E• S

E

• W

?• I

?• I

?

A .

2012 M A . A S S I H T A W E 2012 P 14

D C

1. H P C U T2. D T C

T3. C E

4. S5. C E

E E6. E A7. O A8. S A9. L A . .

. .

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Seite 15 2012 M A . A S S I H T A W E 2012 P 15

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2012 M A . A S S I H T A W E 2012 P 16

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Seite 17 2012 M A . A S S I H T A W E 2012 P 17

D E

C D

M 1 251

M 32 242

M 407 631

E E : 2010 2020 O J RD 2011, 6:62 :10.1186/1750 1172 6 62, C S ( @ . ), T M( @ . ), A H ( @ . )

2012 M A . A S S I H T A W E 2012 P 18

• P O M • T .

A C . A D A 2011 A C . A D A 2011

20,90 2.698,96

Loss of orphan status – orphan price remains…

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Seite 19 2012 M A . A S S I H T A W E 2012 P 19

P

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O D L 2001, E 377 , 500

Seite 20 2012 M A . A S S I H T A W E 2012 P 20

C A

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– WP1: / – WP2: M – WP3:T ( )

• :DG ENT, MS (P , P , P ), EMIN

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Seite 21 2012 M A . A S S I H T A W E 2012 P 21

FC D D D

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Seite 22 2012 M A . A S S I H T A W E 2012 P 22

D : ? E ?

O J R D 2012, 7:74 :10.1186/1750 1172 7 74, W H W (. @ .), A P( @ . ), A S (AS @ .), S S ( . @ . . )

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Seite 23 2012 M A . A S S I H T A W E 2012 P 23

C & C

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T D, ,

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Bi1

Seite 24 2012 M A . A S S I H T A W E 2012 P 24

C C : C D , D D

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C 21 : D ; SM , H G E , A B , W E H L L G , SPC ; DOI: 10.1111/ .13652125.2011.03981.

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Slide 23

Bi1 Who pays for the test?Helicobacter-Eradicaton & Helicobacter-TestHDL/LDL-Assay and statins

Anna Bucsics, 15/10/2012

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Seite 25 2012 M A . A S S I H T A W E 2012 P 25

Challenges to Estimating Efficiency of

Personalised Medicines C D :

• U ,

• S

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• ( ) , ( )

B B :• V B• W /

?

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Seite 26 2012 M A . A S S I H T A W E 2012 P 26

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Seite 27 2012 M A . A S S I H T A W E 2012 P 27

B ?

• C (X ): C $116.000 US

• I 3 7% ALK+ N S C L C

• T . $250 $1.500 P

Seite 28 2012 M A . A S S I H T A W E 2012 P 28

V Z

5.1 D B AF

Vemurafenib isindicated in mono-therapy for the treat-ment of adult patientswith BRAF V600mutation-positiveunresectable or meta-static melanoma (seesection 5.1).

.

Before taking vemurafenib, patients must haveBRAF V600 mutation-positive tumour statusconfirmed by a validated test. In the phase II andphase III clinical trials, eligible patients wereidentified using a real-time polymerase chainreaction assay (the cobas 4800 BRAF V600Mutation Test). This test has CE marking and isused to assess the BRAF mutation status of DNAisolated from formalin-fixed, paraffin-embedded(FFPE) tumour tissue.

Costs: Ca. €100.000per patientper year*

*S A J 2012S PC

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Seite 29 2012 M A . A S S I H T A W E 2012 P 29

...T !