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The 2007 trends: EMEA analysis EMEA analysis The 2007 trends: - - PowerPoint PPT Presentation

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EMEA- -EFPIA INFO DAY EFPIA INFO DAY EMEA 5 February 2007 5 February 2007 Analysis of Performance Indicators for Analysis of Performance Indicators for Initial Applications/Full Applications Initial Applications/Full Applications The 2007

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Analysis of Performance Indicators for Analysis of Performance Indicators for Initial Applications/Full Applications Initial Applications/Full Applications

The 2007 trends: The 2007 trends: EMEA analysis EMEA analysis

Francesco Pignatti, MD

Safety and Efficacy Sector, Unit Pre-authorisation Evaluation of Medicines for Human Use, EMEA

EMEA EMEA-

  • EFPIA INFO DAY

EFPIA INFO DAY 5 February 2007 5 February 2007

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2

Contents

  • Background

Background

  • EMEA analysis EFPIA

EMEA analysis EFPIA InfoDay InfoDay 2005 2005

  • Methods

Methods

  • Results

Results

  • Compliance

Compliance

  • Time trends

Time trends

  • Other factors (ATC, Orphan, Part A/B)

Other factors (ATC, Orphan, Part A/B)

  • Scores and Outcome

Scores and Outcome

  • Summary

Summary

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3

Analysis Analysis InfoDay InfoDay 2005 2005 Conclusions Conclusions

  • Good compliance

Good compliance with Qs with Qs – – validation ongoing validation ongoing

  • Average scores are

Average scores are stable over time stable over time – – Majority Majority satisfaction satisfaction

  • Lowest satisfaction with

Lowest satisfaction with Overview Overview

  • Learning curve

Learning curve with experience (layout, with experience (layout, analysis, reports) analysis, reports) – – feedback feedback

  • Low scores

Low scores for long for long Clockstop Clockstop, , list A list A products products (Q), (Q), small small company company, , orphan products

  • rphan products (C)

(C)

Bo Aronsson, EFPIA InfoDay 2005

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4

Bo Aronsson, EFPIA InfoDay 2005

(Co) Rapporteurs’ Questionnaires at Day (Co) Rapporteurs’ Questionnaires at Day 70 70-

  • 80

80

  • Measure satisfaction with dossier

Measure satisfaction with dossier (0 dissatisfied to 10 satisfied) (0 dissatisfied to 10 satisfied)

Q Qu ua al li it ty y N No

  • n

n-

  • c

cl li in ni ic ca al l C Cl li in ni ic ca al l L La ay yo

  • u

ut t – – 1 10 – – 1 10 – – 1 10 A An na al ly ys si is s – – 1 10 – – 1 10 – – 1 10 O Ov ve er rv vi ie ew ws s – – 1 10 – – 1 10 S Su um mm ma ar ri ie es s – – 1 10 – – 1 10 – – 1 10 S St tu ud dy y r re ep po

  • r

rt ts s – – 1 10 – – 1 10 – – 1 10

Clinical Score Global Score

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5

Selection Criteria Selection Criteria

  • Study periods

Study periods

  • 2006:

2006: 01 01-

  • 06

06-

  • 2005 to 31

2005 to 31-

  • 09

09-

  • 2006

2006

  • 2004: 25

2004: 25-

  • 02

02-

  • 2003 to 31

2003 to 31-

  • 05

05-

  • 2005

2005

  • 2003: 01

2003: 01-

  • 08

08-

  • 2002 to 24

2002 to 24-

  • 02

02-

  • 2003

2003

  • Include

Include

  • All centralised procedures with outcome in study period

All centralised procedures with outcome in study period

  • Exclude

Exclude

  • Double

Double-

  • applications, WHO opinion

applications, WHO opinion

  • Use average scores if 2 rapporteurs completed

Use average scores if 2 rapporteurs completed questionnaires questionnaires

  • Graph Kernel density estimates, non

Graph Kernel density estimates, non-

  • parametric test

parametric test ( (Kruskal Kruskal-

  • Wallis), logistic regression

Wallis), logistic regression

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6

Results Results

  • Included products (2006)

Included products (2006)

  • 64 products (positive, negative or withdrawn)

64 products (positive, negative or withdrawn)

  • Excluded

Excluded

  • 10 Double

10 Double-

  • applications, WHO opinion

applications, WHO opinion

  • Outcome

Outcome

Negative (%) Negative (%) 9 (26) 9 (26) 7 (23) 7 (23) 17 (27) 17 (27) Positive (%) Positive (%) 26 (74) 26 (74) 24 (77) 24 (77) 47 (73) 47 (73) All (%) All (%) 35 (100) 35 (100) 31 (100) 31 (100) 64 (100) 64 (100) 2003 2004 2006

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Compliance Compliance

No. No. applications applications 2003 2003 (N=35) (N=35) 2004 2004 (N=31) (N=31) 2006 2006 (N=64) (N=64) Total Total (N=130) (N=130) 78 78

≥ ≥1 form (%)

1 form (%) 100 100 97 97 94 94 97 97 2 forms (%) 2 forms (%) 87 87 76 76 75 75

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Product Characteristics Product Characteristics

2003 2003 (N=35) (N=35) 2004 2004 (N=31) (N=31) 2006 2006 (N=64) (N=64) Total Total (N=130) (N=130) Part Part A/B A/B A A 9(26) 9(26) 6(19) 6(19) 18(28) 18(28) 33(25) 33(25) B B 26(74) 26(74) 25(81) 25(81) 46(72) 46(72) 97(75) 97(75) ATC ATC L L 14(40) 14(40) 6(19) 6(19) 11(17) 11(17) 31(24) 31(24) J J 6(17) 6(17) 7(23) 7(23) 13(20) 13(20) 26(20) 26(20) A A 5(14) 5(14) 4(13) 4(13) 8(13) 8(13) 17(13) 17(13) N N 1(3) 1(3) 7(23) 7(23) 6(9) 6(9) 14(11) 14(11) Other Other 9(26) 9(26) 7(23) 7(23) 26(41) 26(41) 42(32) 42(32)

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Product Characteristics Product Characteristics

2003 2003 (N=35) (N=35) 2004 2004 (N=31) (N=31) 2006 2006 (N=64) (N=64) Total Total (N=130) (N=130) Orphan Orphan Yes Yes 10(29) 10(29) 8(26) 8(26) 15(23) 15(23) 33(25) 33(25) No No 25(71) 25(71) 23(74) 23(74) 49(77) 49(77) 97(75) 97(75) Scientific Advice Scientific Advice Yes Yes 8(23) 8(23) 11(35) 11(35) 22(34) 22(34) 41(32) 41(32) No No 27(77) 27(77) 20(65) 20(65) 42(66) 42(66) 89(68) 89(68)

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Global Score (All Years, N=130) Global Score (All Years, N=130)

.1 .2 .3 Density 2 4 6 8 10 Global Score (All Questions)

Average score per questionnaire (all questions) Dissatisfied Satisfied

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Global Score by Period Global Score by Period

.5

.

2 4 6 8 10

.

2003 2004 2006

P=0.1703 Global score per questionnaire (all questions)

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Score Part of Dossier by Period Score Part of Dossier by Period

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

Quality Non-clinical Clinical 2003 2004 2006

P=0. 5785 P=0.5324 P=0.1243

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Overview by Period Overview by Period

The scientific overview was sufficiently critical The scientific overview was sufficiently critical

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

Quality Non-clinical Clinical 2003 2004 2006

P=0.0078 P=0.1076

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Global Score by ATC Code Global Score by ATC Code

.5

.

2 4 6 8 10

.

L N A J

Other

P=0.3732 Global score per questionnaire (all questions)

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.5

.

2 4 6 8 10

.

Score Part of Dossier by Orphan Score Part of Dossier by Orphan Desig Desig. .

P=0.0433

.5

.

2 4 6 8 10

.

Orphan No

.5

.

2 4 6 8 10

.

P=0.4051 P=0. 3033

Quality Non-clinical Clinical

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Trend in Clinical Score by Orphan Trend in Clinical Score by Orphan

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

.5 .5

.

2 4 6 8 10 10

. .

2003 2004 2006 Non-orphan Orphan

P=0.0326 P=0.0892 P=1.0000

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Part A (Biopharmaceuticals) Part A (Biopharmaceuticals)

5 10 5 10 5 10

New Chemical Entities Biopharmaceuticals

Clinical Score Quality Score

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Global Score and Clock Global Score and Clock-

  • stop

stop

200 400 600 200 400 600 0 1 2 3 4 5 6 7 8 9 10 0 1 2 3 4 5 6 7 8 9 10

2003 2004 2006

Cumulative Clock Stop Global Score (All Questions)

bandwidth = .8

All periods r =-.36

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19

Score Part of Dossier by Outcome Score Part of Dossier by Outcome

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

.5

.

2 4 6 8 10

.

P=0.0348 P=0.0022 P=0.0001

Positive

  • Neg. Withdr.

Quality Non-clinical Clinical

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Outcome Outcome

Multivariate logistic regression Multivariate logistic regression

O OR R 9 95 5% % C C. .I I P P> >z z 2 20 00 03 3 C Cl li in ni ic ca al l s sc co

  • r

re e 1 1. .2 27 7 1 1. .0 07 7 1 1. .5 51 1 0. .0 00 07 7 O Or rp ph ha an n . .5 52 2 . .1 12 2 2 2. .3 32 2 0. .3 39 94 4 2 20 00 04 4 C Cl li in ni ic ca al l s sc co

  • r

re e 1 1. .5 56 6 1 1. .1 17 7 2 2. .0 08 8 0. .0 00 02 2 O Or rp ph ha an n . .1 11 1 . .0 01 1 . .8 85 5 0. .0 03 35 5 2 20 00 06 6 C Cl li in ni ic ca al l s sc co

  • r

re e 1 1. .2 27 7 1 1. .1 12 2 1 1. .4 43 3 0. .0 00 00 O Or rp ph ha an n . .4 45 5 . .1 12 2 1 1. .6 68 8 0. .2 23 35 5 A Al ll l C Cl li in ni ic ca al l s sc co

  • r

re e 1 1. .3 31 1 1 1. .2 20 1 1. .4 44 4 0. .0 00 00 O Or rp ph ha an n . .3 36 6 . .1 15 5 . .8 85 5 0. .0 02 20

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The 2007 trends The 2007 trends

  • Confirm 2005 analysis

Confirm 2005 analysis

  • Good compliance

Good compliance

  • Average scores are stable over time

Average scores are stable over time

  • Majority satisfaction

Majority satisfaction

  • Low scores associated with

Low scores associated with

  • Biopharmaceuticals

Biopharmaceuticals (Q) (Q)

  • Orphan products (C),

Orphan products (C), trend reversing? trend reversing?

  • Clockstop

Clockstop, Negative outcome , Negative outcome

  • High clinical score and non

High clinical score and non-

  • orphan status are
  • rphan status are

associated with positive outcome associated with positive outcome

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Acknowledgments

Project leader and analysis

Bo Aronsson

Data Management

Esther Cozar Calvente Nadia Kresse