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 trends: EMEA analysis EMEA analysis The 2007 trends: Francesco Pignatti, MD Safety and Efficacy Sector, Unit Pre-authorisation Evaluation of Medicines for Human Use, EMEA
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 � 2
Analysis InfoDay InfoDay 2005 2005 Analysis 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) – – feedback feedback analysis, reports) � Low scores Low scores for long for long Clockstop Clockstop , , list A list A products products � (Q), small small company company , , orphan products orphan products (C) (C) (Q), Bo Aronsson, EFPIA InfoDay 2005 3
(Co) Rapporteurs’ Questionnaires at Day (Co) Rapporteurs’ Questionnaires at Day 70- -80 80 70 � Measure satisfaction with dossier Measure satisfaction with dossier � (0 dissatisfied to 10 satisfied) (0 dissatisfied to 10 satisfied) Qu ua al li it ty y N No on n- - Cl li in ni ic ca al l Q C cl li in ni ic ca al l c La ay yo ou ut t 0 – – 1 10 0 0 – – 1 10 0 0 – – 1 10 0 L 0 0 0 An na al ly ys si is s 0 – – 1 10 0 0 – – 1 10 0 0 – – 1 10 0 A 0 0 0 Clinical Global Score Ov ve er rv vi ie ew ws s 0 – – 1 10 0 0 – – 1 10 0 O 0 0 Score Su um mm ma ar ri ie es s 0 – – 1 10 0 0 – – 1 10 0 0 – – 1 10 0 S 0 0 0 St tu ud dy y r re ep po or rt ts s 0 – – 1 10 0 0 – – 1 10 0 0 – – 1 10 0 S 0 0 0 Bo Aronsson, EFPIA InfoDay 2005 4
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 ( 5
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 � 2003 2004 2006 Negative (%) 9 (26) 7 (23) 17 (27) Negative (%) 9 (26) 7 (23) 17 (27) Positive (%) 26 (74) 24 (77) 47 (73) Positive (%) 26 (74) 24 (77) 47 (73) All (%) 35 (100) 31 (100) 64 (100) All (%) 35 (100) 31 (100) 64 (100) 6
Compliance Compliance 2003 2004 2006 Total 2003 2004 2006 Total No. No. applications applications (N=35) (N=31) (N=64) (N=130) (N=35) (N=31) (N=64) (N=130) ≥ 1 form (%) ≥ 1 form (%) 100 97 94 97 100 97 94 97 2 forms (%) 2 forms (%) 87 76 75 78 87 76 75 78 7
Product Characteristics Product Characteristics 2003 2004 2006 Total 2003 2004 2006 Total (N=35) (N=31) (N=64) (N=130) (N=35) (N=31) (N=64) (N=130) Part A/B A/B A 9(26) 6(19) 18(28) 33(25) Part A 9(26) 6(19) 18(28) 33(25) B 26(74) 25(81) 46(72) 97(75) B 26(74) 25(81) 46(72) 97(75) ATC L 14(40) 6(19) 11(17) 31(24) ATC L 14(40) 6(19) 11(17) 31(24) J 6(17) J 6(17) 7(23) 13(20) 26(20) 7(23) 13(20) 26(20) A 5(14) 4(13) 8(13) 17(13) A 5(14) 4(13) 8(13) 17(13) N 1(3) 7(23) 6(9) 14(11) N 1(3) 7(23) 6(9) 14(11) Other 9(26) 7(23) 26(41) 42(32) Other 9(26) 7(23) 26(41) 42(32) 8
Product Characteristics Product Characteristics 2003 2004 2006 Total 2003 2004 2006 Total (N=35) (N=31) (N=64) (N=130) (N=35) (N=31) (N=64) (N=130) Orphan Yes 10(29) 10(29) 8(26) 8(26) 15(23) 33(25) Orphan Yes 15(23) 33(25) No 25(71) 23(74) 23(74) 49(77) 49(77) 97(75) No 25(71) 97(75) Scientific Advice Yes Yes 8(23) 8(23) 11(35) 22(34) 22(34) 41(32) Scientific Advice 11(35) 41(32) No 27(77) 20(65) 20(65) 42(66) 42(66) 89(68) No 27(77) 89(68) 9
Global Score (All Years, N=130) Global Score (All Years, N=130) .3 .2 Density .1 0 0 2 4 6 8 10 Satisfied Dissatisfied Global Score (All Questions) Average score per questionnaire (all questions) 10
Global Score by Period Global Score by Period .5 2003 2004 2006 . P= 0.1703 0 0 2 4 6 8 10 . Global score per questionnaire (all questions) 11
Score Part of Dossier by Period Score Part of Dossier by Period .5 Quality .5 Non-clinical .5 Clinical 2003 2004 2006 . . . P= 0.5324 P= 0.1243 P= 0. 5785 0 0 0 0 2 4 6 8 10 0 2 4 6 8 10 0 2 4 6 8 10 . . . 12
Overview by Period Overview by Period The scientific overview was sufficiently critical The scientific overview was sufficiently critical Quality .5 Non-clinical .5 Clinical 2003 2004 2006 . . P= 0.0078 P= 0.1076 0 0 0 2 4 6 8 10 0 2 4 6 8 10 . . 13
Global Score by ATC Code Global Score by ATC Code .5 J N A Other . L P= 0.3732 0 0 2 4 6 8 10 . Global score per questionnaire (all questions) 14
Score Part of Dossier by Orphan Desig Desig. . Score Part of Dossier by Orphan .5 Quality .5 Non-clinical .5 Clinical Orphan No . . . P= 0. 3033 P= 0.4051 P= 0.0433 0 0 0 0 2 4 6 8 10 0 2 4 6 8 10 0 2 4 6 8 10 . . . 15
Trend in Clinical Score by Orphan Trend in Clinical Score by Orphan 2003 2004 2006 .5 .5 .5 .5 Non-orphan Orphan . . . P= 0.0892 P= 0.0326 P= 1.0000 0 0 0 0 2 4 6 8 10 0 2 4 6 8 10 0 2 4 6 8 10 10 . . . . 16
Part A (Biopharmaceuticals) Part A (Biopharmaceuticals) New Chemical Entities Biopharmaceuticals 10 Clinical Score 5 0 0 5 10 0 5 10 Quality Score 17
Global Score and Clock- -stop stop Global Score and Clock 2003 2004 600 400 Cumulative Clock Stop 200 0 0 1 2 3 4 5 6 7 8 9 10 2006 600 400 All periods 200 r =-.36 0 0 1 2 3 4 5 6 7 8 9 10 Global Score (All Questions) bandwidth = .8 18
Score Part of Dossier by Outcome Score Part of Dossier by Outcome .5 Quality .5 Non-clinical .5 Clinical Positive Neg. Withdr. . . . P= 0.0348 P= 0.0022 P= 0.0001 0 0 0 0 2 4 6 8 10 0 2 4 6 8 10 0 2 4 6 8 10 . . . 19
Outcome Outcome Multivariate logistic regression Multivariate logistic regression OR R 95 5% % C C. .I I P> >z z O 9 P 20 00 03 3 C Cl li in ni ic ca al l s sc co or re e 1. .2 27 7 1. .0 07 7 1. .5 51 1 0. .0 00 07 7 2 1 1 1 0 O Or rp ph ha an n . .5 52 2 . .1 12 2 2. .3 32 2 0. .3 39 94 4 2 0 20 00 04 4 C Cl li in ni ic ca al l s sc co or re e 1. .5 56 6 1. .1 17 7 2. .0 08 8 0. .0 00 02 2 2 1 1 2 0 O Or rp ph ha an n . .1 11 1 . .0 01 1 . .8 85 5 0. .0 03 35 5 0 20 00 06 6 C Cl li in ni ic ca al l s sc co or re e 1. .2 27 7 1. .1 12 2 1. .4 43 3 0. .0 00 00 0 2 1 1 1 0 O Or rp ph ha an n . .4 45 5 . .1 12 2 1. .6 68 8 0. .2 23 35 5 1 0 Al ll l C Cl li in ni ic ca al l s sc co or re e 1. .3 31 1 1. .2 20 0 1. .4 44 4 0. .0 00 00 0 A 1 1 1 0 O Or rp ph ha an n . .3 36 6 . .1 15 5 . .8 85 5 0. .0 02 20 0 0 20
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 orphan status are � associated with positive outcome associated with positive outcome 21
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