!"#$%#&!"'()"(*+,#$-((.(/012342(5678((
(
*+9::;<(=(>+3??@19A3@?(B@C2(>4<(4?/9?A<(4A(9D@>4<:4?A<(9AA43?A<(D4(:9?:42(
)3<:@C2<(D4(E9A23:39(F>9?:G(,H9I3?4(/@2(J92I@((
(
(
(
J42:3(B@C2(1@A24(3?13A9A3@?(4A(B@C2(H4(B42H4AA24(D4(/9324(C?(B@3?A(<C2(
>+9::;<(=(>+3??@19A3@?(B@C2(>4<(4?/9?A<(4A(9D@>4<:4?A<(9AA43?A<(D4(:9?:42K(
(
$+4<A(C?(<CL4A(<C2(>4MC4>(>+9<<@:39A3@?(,H9I3?4(/@2(J92I@(<+3?14<A3(/@2A4H4?A(
D4BC3<(<9(:209A3@?(3>(N(9(.(9?<G(:92(ma#fille#Margaux,#décédée#à#l#âge#de#
14#ans#d’une#tumeur#au#cerveau,#n’a#justement#PAS#eu#accès#à#des#
traitements#innovants,(H93<(LC<A4(=(C?(B2@A@:@>4(<A9?D92DG(914:(C?4(1343>>4(
:O3H3@(MC3(D9A93A(D4(P6(9?<(4A(D014>@BB04(B@C2(D4<(9DC>A4<K((
(
%2G(Donner#accès#à#l’innovation#9CQ(4?/9?A<(4A(9D@>4<:4?A<(9AA43?A<(D4(
:9?:42(D@3A(RA24(C?4(E!,%!,&"(G(L4(D3293(HRH4(C?4(PRIORITE#DE#SANTE#
PUBLIQUE.#
(
SC4>MC4<(:O3//24<G(MC3(<@?A(D+93>>4C2<(A2;<(B4C(:@??C<(T((
U*4(:9?:42(24<A4(>9(1ère#cause#de#décès#des#enfants#par#maladieK(
U7(4?/9?A(<C2(..6(D39I?@<A3MC0(919?A(>+VI4(D4(7W(9?<((
U-14:(W66(D0:;<(B92(9?G(B>C<(D+C?(4?/9?A(H4C2A(:O9MC4(L@C2(DC(:9?:42(4?(
X29?:4(
U'3(>+@?(IC023A((Y6Z(D4<(4?/9?A<G(:4(?+4<A(D+9[@2D(MC+C?4(<A9A3<A3MC4(=(W(9?<KKG(
3>(N(9(D4(X%!&"'(),'E-!,&\'(D9?<(:4(B@C2:4?A9I4(<4>@?(>4<(ACH4C2<G((4A((
:4<(4?/9?A<(@?A([34?(A2@B(<@C14?A((D4(>@C2D4<(<0MC4>>4<(<C3A4(9CQ(
A293A4H4?A<(@C(=(>9(H9>9D34K((
(
Il#faut#donc#réussir#à#guérir#mieux#ces#enfants,#ces#jeunes,#et#aussi#les#
guérir#davantageK(
Pour#cela,#les#enfants#et#les#adolescents#doivent#avoir#accès#à#
l’innovation#et#ne#plus#être#les#oubliés#de#la#Recherche.#
(
"?(4//4AG((très#peu#de#fonds(<@?A(9>>@C0<(=(>9(24:O42:O4(B0D39A23MC4K(
-14:(86(ANB4<(D4(:9?:42<(D3//024?A<G(:4>9(24<A4(C?4(H9>9D34(2924G(un#marché#
non#rentable#financièrement#pour#l’industrie#pharmaceutique.#
(
"?(:@?<0MC4?:4G((la#plupart#des#médicaments#donnés#aux#enfants#sont#
off#label,##c’est#à#dire#des#médicaments#d’adulte#non#légalement#
autorisés#chez#l’enfant.###
(
->@2<(MC4((>+3??@19A3@?(<4(D014>@BB4(/@2A4H4?A(D9?<(>4<(:9?:42<(D4<(9DC>A4<G(
914:(D4(A2;<(?@H[24C<4<(?@C14>>4<(H@>0:C>4<G((
(
%?(4<A3H4(MC+9CL@C2D+OC3G(en#Europe,#seulement#10%#des#enfants#en#
rechute#ont#accès#à#l’innovation.#
(
(
Pourtant,#l’année#2016#est#l’année#pour#AGIR#afin##d’accélérer#
l’innovation#et#cela,#pour##principalement#2#raisons#:#
#
(
U>9(7424(4<A(MC4(la#médecine#de#précision#démarre#à#peine#chez#
l’enfant#T(3>(/9CA(MC4(:4>9(s’accélère(4A(MC4(>+@?(<@3A(B2RA(=(B@C1@32(B2@B@<42(
D4<(A293A4H4?A<(:3[>0<(>4<(B>C<(9D9BA0<(9CQ(9?@H9>34<(D4<(ACH4C2<(D4<(
4?/9?A<K()@?:(il#nous#faut#les#médicamentsK(
(
U>9(5;H4(4<A(MC4(:+4<A(4?(5678(MC4(?@C<(D41@?<(/9324(D4<(B2@B@<3A3@?<(B@C2(
MC4(l’environnement#règlementaire#européen(<@3A((B>C<(/91@29[>4(9C(
D014>@BB4H4?A(D4(H0D3:9H4?A<(B0D39A23MC4<(B92(>4<(3?DC<A234><(]("?(N(
9L@CA9?A(=(>9(/@3<(D4<(obligations#et#des#incitationsK(
(
(
*9(H0D4:3?4(D4(B20:3<3@?G(MC3(<4([9<4(<C2(>4<(20<C>A9A<(D4<(B@2A293A<(
H@>0:C>9324<G(>4(<0MC4?^9I4(D4(>9(ACH4C2G(B42H4A(D4(H34CQ(:@HB24?D24(>4<(
9?@H9>34<(H@>0:C>9324<(4A(D@?:(D4(H34CQ(:3[>42(>4<(A293A4H4?A<K((
%?(1@3A(:O4_(>+9DC>A4(MC4(:42A93?4<(H@>0:C>4<(<@?A(A2;<(B2@H4AA4C<4<G(
:@HH4(>+4<A(9C<<3(>+3HHC?@AO029B34K((
J93<(très#peu#de#ses#nouvelles#innovations#sont#accessibles#aux#
enfants#ou#le#sont#très#tard.##
(
-C(?3149C(4C2@B04?G(>4(réseau#ITCC#est#un#acteur#majeur#du#
développement#des#innovations#thérapeutiques(B@C2(>4<(4?/9?A<(4A(
9D@>4<:4?A<(9AA43?A<(D4(:9?:42(4A(D0B>@34(<@?(9:A313A0(D9?<(75(B9N<K((
,&$$(209>3<4?A(>9(H@3A30(D4(<@?(9:A313A0(4?(X29?:4G(D9?<(77(:4?A24<(
D+@?:@>@I34(B0D39A23MC4<(20B92A3<(<C2(A@CA(>4(A4223A@324K((
"?(X29?:4G(<C2(>4<(5(D42?3;24<(9??04<G(566(4?/9?A<(4A(9D@>4<:4?A<((4?(
24:OCA4(D+C?4(ACH4C2(@C(>+C?4(>4C:0H34((@?A(B92A3:3B0(=(C?(4<<93(B20:@:4(
,&$$(9/3?(D+91@32(9::;<(=(D4(?@C149CQ(H0D3:9H4?A<K((
-/3?(D+9::0>0242(>+3??@19A3@?G(>+9H[3A3@?(D+,&$$(4<A(T(
((
7`(D4(B2@B@<42(=(B>C<(D+C?(4?/9?A(<C2(5(4?(24:OCA4(?@?(:C29[>4(D+C?(:9?:42G(
9C(H@3?<(C?(?@C149C(H0D3:9H4?AG(9C(H34CQ(4?(9<<@:39A3@?G(
(5`(9::0>0242(>+3?A2@DC:A3@?(D4<(?@C149CQ(H0D3:9H4?A<(4?(B24H3;24(>3I?4(
D4(A293A4H4?A(a4A(?@?(9AA4?D24(>9(24:OCA4`(B@C2(>4<(:9?:42<(D4(H9C193<(
B2@?@<A3:K(
(
SC4><(H@N4?<(4?(X29?:4(B@C2(N(9223142(b((E>C<34C2<(3?3A39A314<(@?A(
D0H92204<(T((
(
*4(B>9?(:9?:42(PG(4?(:209?A(8(:4?A24<(>9[4>>3<0<(D+3?14<A3I9A3@?(B20:@:4(
B0D39A23MC4(ales#CLIPP`G(9(A@CA(D+9[@2D(24?/@2:0(>9(:9B9:3A0(=(D014>@BB42(
D4<(3??@19A3@?<K((
(
"?<C3A4G((l’essai#MAPPYACTG(<@CA4?C(B92(>4(Ec!$(4A(B92(>9(X@?D9A3@?(-!$G((
9(D0H9220(20:4HH4?A(4A(propose#aux#enfants#en#rechute#un#portrait#
moléculaire#complet##
#
P66(B9A34?A<(<42@?A(3?:>C<(<C2(P(9?<K#
*4<(B@2A293A<(H@>0:C>9324<(<@?A(209>3<0<(=(dC<A914(!@C<<N(4A(=(>+,?<A3ACA(
$C234K(
(
J-EEe-$&((<+3?<:23A(D+93>>4C2<(D9?<(C?4(D0H92:O4(4C2@B04??4G(914:(
B>C<34C2<(B2@I29HH4<(DC(HRH4(ANB4(D9?<(D+9CA24<(B9N<(4C2@B04?<G(:4(MC3(
B42H4AA29(=(A42H4(une#grande#base#de#données#clinico-biologiqueK(
(
L’on#pourra#donc#bientôt#mieux#connaître#mieux#les#anomalies#des#
tumeurs#des#enfants#mais#il#faudra#alors#être#capable#de#proposer#le#
traitement#le#mieux#adapté#avec#les#médicaments#les#plus#
prometteurs.#
(
)+@C(>9(nécessité#d’accélérer#l’accès#à#des#essais#cliniques,#de##repenser#
la#manière#de#les#concevoirK(
(
"A(4?(:4(<4?<G(>4(B2@I29HH4("'J-!&(MC3(<+3?A;I24(D9?<(>4(B2@I29HH4(-:<0(
a9::;<(<0:C23<0(9C(H0D3:9H4?A`(DC(E>9?(:9?:42(P(B3>@A0(B92((>+,#$9(4<A((C?(
[4>(4Q4HB>4(D+9::;<(=(>+3??@19A3@?K((
(
ESMART#sera#une#première#mondiale#dans#les#cancers#de#l’enfant#T((
,>(B2@B@<429(9C(H@3?<(76(H0D3:9H4?A<(3??@19?A<(B2@14?9?A(D4(B>C<34C2<(
3?DC<A234><(019>C0<(4?(BO9<4(B20:@:4(aH0D3:9H4?A(<4C>(4A(4?(9<<@:39A3@?`(
D9?<(C?(<4C>(4A(HRH4(B2@A@:@>4K(
(
C’est#un#programme#transversal,#qui#permettra#aux##enfants#en#
rechute#ou#en#échec#thérapeutique#d’avoir#un#traitement##ciblé,#
quelque#que#soit#leur#tumeur,#solide#ou#leucémie.#
(
E@C2((H4AA24(4?(B>9:4("'J-!&G(3>(9(/9>>C(9>>42(:O42:O42(>4<(H@>0:C>4<(*"'(
E*f'(-)-E&""'(9CB2;<(D4<(>9[@29A@324<K((
g4(<93<(JH4(FC_N?(MC4(1@C<(N(914_(B9<<0(DC(A4HB<G((1@C<(4A(9C<<3(1@A24(
:@>>9[@29A23:4(#9A9>34(c@@I(*9[@C24AG(L4(1@C<(4?(24H42:34K(
g4(24H42:34((9C<<3(>4<(0MC3B4(D4(dC<A914(!@C<<NG(B2@H@A4C2(DC(B2@L4AG(4A(
?@A9HH4?A(>4(E2(h9<<9>G(>4()2(F32I3A(d4@42I42G(93?<3(MC4(>4<(>9[@29A@324<(MC3(
@?A(D0L=(9::4BA0(D+N(B92A3:3B42K((
g+4?:@C29I4(>4<(9CA24<(3?DC<A234><(MC3(@?A(0A0(<@>>3:3A0<(=(24L@3?D24(:4(
B2@I29HH4G(
4A(9C<<3(>4<(9CA@23A0<(2;I>4H4?A9324<G(MC3(134??4?A(D4(24:41@32(>4(B2@L4A(D4(
>+0ACD342(4A(D@??42(>4C2(9::@2D(D9?<(>4<(H43>>4C2<(D0>93<K((
(
C’est#un#programme#suffisamment#porteur#d’espoir#pour#les#familles#
qui#mérite#de#ne#pas#perdre#de#temps.#((
(
)4(?@A24(:iA0G(Imagine#for#Margo#s’est#engagé#à#verser#1#million#d’euros#
pour#booster#le#démarrage#du#projet#ESMART,#car#nous#savons#trop#
bien#comme#le#temps#est#précieux#quand#un#enfant#est#en#rechute.(
(
*+3??@19A3@?(<4(D014>@BB4(9C<<3(4?(7424(>3I?4(D4(A293A4H4?AG(?@A9HH4?A(
B@C2(>4<(ACH4C2<(DC(A2@?:(:020[29>G(MC3(4<A(>9(ACH4C2(9C(B>C<(H9C193<(
B2@?@<A3:K((
-(dC<A914(!@C<<NG((>+4<<93#BIOMEDE#D4(,&$$G(B2@B@<4G(9C(D39I?@<A3:G(A2@3<(
AO029B34<(:3[>04<(<4>@?(>4<(20<C>A9A<(D4(>9([3@B<34K((
-14:(D4(B>C<G(C?(B@2A293A(H@>0:C>9324(9/3?(D+@234?A42(>4<(9CA24<(A293A4H4?A<(
9C(H@H4?A(D4(>9(B2@I24<<3@?(ACH@29>4K(
(
*9(création#des#CLIPP(93?<3(MC4(>4(B2@I29HH4(Acsé#–#ESMART(H@?A24?A(
MC4(le#plan#cancer#3#a#été#un#accélérateur#de#l’accès#à#l’innovation#en#
onco-pédiatrie.##
"?(5678G(>4(B2@I29HH4(PAIR#pédiatrique(aE2@I29HH4(D+9:A3@?(3?A0I20(D4(
!4:O42:O4`G(<@CA4?C(B92(>9(*3IC4(:@?A24(>9($9?:42(4A(>9(X@?D9A3@?(-!$G(
:@HB>;A42@?A(:4(D3<B@<3A3/(4?(<@CA4?9?A(>9(!4:O42:O4(/@?D9H4?A9>4(4A(
A29?<>9A3@??4>>4K(
(
h@3>=(D@?:(D4<(initiatives#qui#démarrent,#ENFING(B@C2(MC4(>4<(4?/9?A<(?4(
<@34?A(B>C<(>4<(@C[>30<(D4(>9(24:O42:O4K((
(
J93<(ce#n’est#qu’un#débutG(3>(/9CA(continuer#à#construire#ensembleK((
(
(
è$@?A3?C42(=(RA24(B>C<(/>4Q3[>4(D9?<(>+9::;<(9CQ(H0D3:9H4?A<(A@CA(4?(
I92D9?A(C?(:9D24(<0:C23<0((
è'91@32(I92D42(4A(D014>@BB42(>+3??@19A3@?(D9?<(?@A24(B9N<(a?@C<(
91@?<(4?:@24(D4<(/9H3>>4<(@[>3I04<(D4(B92A32(=(>+0A29?I42(B@C2(9::0D42(
=(C?(A293A4H4?A(?@?(9::4<<3[>4(4?(X29?:4`((
è#4(B9<(:2042(D+3?0I9>3A0<(<C2(>4(A4223A@324(4A(D@?:(/9:3>3A42(>4(
A29?</42A(D4<(4?/9?A<(D9?<(>4<(0A9[>3<<4H4?A<(@j(>4<(3??@19A3@?<(
AO029B4CA3MC4<(<42@?A(4?(019>C9A3@?G(
èX9:3>3A42(9C<<3(>+3?/@2H9A3@?(B@C2(>4<(B2@/4<<3@??4><(4A(>4<(/9H3>>4<(
<C2(>+4Q3<A4?:4(D4(:4<(4<<93<(T(MC4(>+3?/@2H9A3@?(<@3A(A29?<B924?A4(4A(
:4?A29>3<04((
(
$4<(3?/@2H9A3@?<(B@C22934?A((RA24(B92(4Q4HB>4(24I2@CB04<(9C(<43?(D+C?(
observatoire#des#cancers#des#enfants(MC3(B42H4AA293A(9C<<3(D4(H4<C242(
>4<(20<C>A9A<(D4<(D3//024?A4<(3?3A39A314<(4A(3?/@2H42(>4<(/9H3>>4<K((
(
g4(A42H3?4293(B92(un#autre#point#important#pour#accélérer#l’innovation#;#
l’adaptation#de#la#Règlementation#européenne.##
(
)4BC3<(H93?A4?9?A(5(9?<G(4?<4H[>4G((H0D4:3?<G(:O42:O4C2<(9:9D0H3MC4<G(
3?DC<A234><(DC(H0D3:9H4?AG(20IC>9A4C2<(aH4H[24<(9C(:@H3A0(B0D39A23MC4(4A(
D4(>+9I4?:4(4C2@B04??4(DC(H0D3:9H4?A`(93?<3(MC4(D4<(9<<@:39A3@?<(D4(
B924?A<G(A29193>>@?<(4?<4H[>4(B@C2([@@<A42(>+3??@19A3@?G(D9?<(>4(:9D24(D+C?4(
plateforme#européenne#d’oncologie#pédiatriqueK(
(
#@C<(91@?<(/93A(C?(0A9A(D4<(>34CQ(D4(>9(!0I>4H4?A9A3@?((4C2@B04??4(<C2(>4(
H0D3:9H4?A(B0D39A23MC4G((9/3?(D4(:@HB24?D24(B@C2MC@3G(9>@2<(MC+4>>4(4<A(C?(
<C::;<(D9?<(B>C<34C2<(H9>9D34<(D4(>+4?/9?AG((3>(N(9(des#freins#majeurs(pour#
l’industrie#dans#le#veloppement#dans#les#cancers#des#enfants(T((
(
èD4<(24A92D<(3HB@2A9?A<(D9?<(>4(D0H9229I4(D4<(H0D3:9H4?A<(((
èD4<(D02@I9A3@?<(inacceptables#9C(24I92D(D4((>9([3@>@I34(D4<(ACH4C2<(
B0D39A23MC4<(@C(>4(B@A4?A34>(D4(:42A93?<(H0D3:9H4?A<(:3[>0<(((
èD4<(E>9?(D+,?14<A3I9A3@?(E0D39A23MC4<(<@C14?A(3?9D0MC9A<(@C(3?/93<9[>4<K(
(
*4<(B2@B@<3A3@?<(3<<C4<(D4(:4AA4(B>9A4/@2H4(134??4?A(D+RA24(/@2H9>3<04<(
914:(=(>9(/@3<(T(
(
1 / 7 100%