5 EuropEAn ConfErEnCE on rArE -...
Transcript of 5 EuropEAn ConfErEnCE on rArE -...
A conference organised by : with :
5th EuropEAn ConfErEnCE on rArE DisEAsEs
13-15 May 2010 Krakow
www.rare-diseases.eu
5. Medicines for people Living with a rare Disease
CONTEABOUT T
Acknow
Additio
CHAPTER
Pred
Ha
Improvin
EUn
D
An in
M
How
Pr
Deba
Orphan
The
D
Paed
D
Com
D
Deba
Clinical
Char
M
Patie
M
EMP
M
Patie
M
Deba
WHERE D
ENTS HE 5TH EUROP
wledgements,
onal Funders .
R 8‐ MEDICINE
dictors of Orp
arald E. Heem
ng Access to
etHTA ...........
r Claudia Wild
ndustry Persp
Mr Andras Fehe
w can interacti
rof Hans Geor
ate ................
Drug Develo
EMA worksho
r Kerstin Wes
diatric Commi
r Fernando An
mmittee for Ad
r Fabrizia Bign
ate ................
Trials, Invol
rter for Clinica
Mr Rob Camp,
ent Involveme
Mr Richard We
P’s first steps i
Mrs Greetje Go
ent Engageme
Mr Eric Pelkma
ate ................
DO THEY TALK
PEAN CONFER
, Credits and m
......................
ES FOR PEOPL
han Drug App
mstra, PhD ......
o Orphan Dru
......................
d, Ludwig Bolt
pective ...........
ervary, Head
ion between r
rg Eichler, Sen
......................
opment, Pae
op May 2010 .
termark, Chai
ittee and Paed
ndrés Trelles,
dvanced Thera
nami, Therape
......................
lvement of P
al Trials in Rar
Consultant on
ent in Clinical
est, Behcet Un
in the field of
oossens, Euro
ent in Researc
ans, Tibotec ....
......................
K ABOUT MY D
RENCE ON RAR
main Funders
......................
E LIVING WITH
proval .............
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ugs ...............
......................
tzmann Institu
......................
Market Acces
regulators and
nior Medical O
......................
ediatric Inve
......................
ir of the Comm
diatric Investi
PDCO, Europ
apies: What is
eutic Develop
......................
Patients in C
re Diseases ....
n Patient Grou
Trials ............
nited Kingdom
clinical trials .
pean Myelom
ch: Active Invo
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DISEASE? Inde
RE DISEASES E
s .....................
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H A RARE DISE
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ute of Health T
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ss & External A
d payers facili
Officer, Europe
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estigation Pl
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mittee for Orp
gation Plan ...
ean Medicine
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ment Directo
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Clinical Trials
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up Advancem
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ma Platform, B
olvement for t
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ex ...................
ECRD 2010 ....
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EASE .............
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ans and Adv
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phan Medicin
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es Agency ......
rare diseases?
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ent, Eurordis
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the Future He
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ssessment, Vi
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tis Oncology .
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al Products ...
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A
ACKNOWherol
T
P
TDr
This con
T
BOUT
WLEDGEME
e organisers le:
THE JAGELLIO
EUROPEAN CO
DG HEALTH AProgramme o
EXECUTIVE AG
The responsDiseases lieresponsible f
nference was
T THE 5
NTS, CREDIparticularly w
ONIAN UNIVERS
OMMISSION, P
AND CONSUMEf Community a
GENCY FOR HE
sibility of thees with the sfor any use t
s also suppo
5TH EU
TS AND MA
wish to than
SITY IN KRAKO
PUBLIC HEALTH
ER PROTECTIOaction in the fi
EALTH AND CO
e content anspeakers anthat may be m
orted by:
ROPERAREAIN FUNDER
k the followi
OW
H PROGRAMME
ON ield of public h
ONSUMERS
d programmnd programmmade of the
AN CODISEAS ng persons/o
E,
health
me of the 5thme committeinformation c
ONFERASES E
organisation
h European e. The Execcontained the
RENCECRD 2
s/companies
Conference cutive Agenerein.
Page | 3
E ON 2010
s for their
on Rare cy is not
ADDITIOhe orfollow
A
m
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Bm
H1
TONAL FUNDrganisers of twing institutio
Alexion for t
morning
Orphan Europ
Swedish Orph
Glaxo Smith K
Shire Human
Genzyme Corfellowship pr
May.
Takeda Pharm
Celgene Inter
Bayer Sheringmailing of the
Helsinn for th19 & 20.
DERS he 5th Europons/companie
their donation
pe for their do
han for their d
Kline Pharmac
Genetics Ther
rporation for ogramme and
maceuticals Eu
national for th
g Pharma fore conference r
heir donation
ean Conferenes for their fin
n for the co
onation for the
donation for t
ceuticals for th
rapies for the
their donatiod Genzyme P
urope for thei
heir donation
r their donatreport
for the Votin
nce on Rare Dancial suppor
ffee break o
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the coffee bre
heir donation
ir donation fo
n for the EURPoland for the
r donation for
for the Photo
tion for the f
g Devices for
Diseases ECRDrt:
of 14 May in
k of 14 May in
eak of 15 May
for the lunch
or the lunch of
RORDIS membee cocktail in
r the speakers
o Exhibition.
fellowship pro
the interactiv
D 2010 in Kra
the
n the afternoo
in the mornin
of 14 May
f 15 May
ers’ dinner anthe evening
s’ dinner
ogramme and
ve sessions 15
akow wish to
on
ng
nd the of 14
d the
5, 16,
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thank the
C
PREDICTO
HARALD
ABSTRACOblegdisin poMenostuweReforbioCoformu
1 INTROD
paEUfoaffsathinconthstuHomPrhawhiondecli
FigAcde
S
CHAPT
ORS OF ORPH
E. HEEMSTRA
Pharmerit IntPharmaceutic
CT bjective To engislation has bscusses factors the EU. This stotential orphan ethods A compot‐yet‐authoriseudy included chere collected froesults Orphan dr another orpotechnology pr
onclusion This sr subsequent auch knowledge
DUCTION everal jurdiseases. introducecommon,
atients (equivaU, an orphan dr the diagnosfects fewer ttisfactory mee new produccentives provn the regulatoe US, additioudies. owever, to barketing. Durroducts in 200ave obtained hich, 59 weren the EU, thevelopment onical develop
g. 1. Number ofct (1983 to 199esignations liste
S
TER 8‐
HAN DRUG AP
A, PHD ternational, Rcal Sciences, U
courage the dbeen introducethat may influeudy aims to idedrugs in the EU
parison betweeed orphan drugharacteristics oom the public drug approval wphan drug (Ooducts tended study showed tuthorisation of is available.
risdictions haFollowing th
ed its Regulati, although a fealent to 7 patdesignation wis, preventionhan 5 in 10,0ethod for the ct will be of svided by both ory fees and frnal tax credit
e designateding the past t00, a number omarketing aue authorised foere is also c
of orphan medmet program
f orphan design92) and duringed as still active
‐ MED
PPROVAL
Research perUtrecht, The N
development oed in the USAence the develentify predictorU. n randomly selg designations of the indicationdomain only. was strongly assOR=17.3, CI=5to have a highethat experiencef other orphan
ve put forwahe successfulion on Orphanew differencetients per 10,will only be pron or treatmen000 patients. diagnosis, tresignificant benregulations aree scientific as are availabl
d does not aten years, sincof 1094 orphauthorisation (sor marketing. riticism by sedicinal producof many orph
nations in the Ug the first ten e have been inc
ICINESW
rformed at tNetherlands
of drugs for r(1983) and inopment of newrs for successfu
ected authorisehas been perfn, of the produ
ociated with pr.6–53.1). Furter likelihood of e of a companydrugs. The sam
rd incentivesl Orphan Drn Medicinal Pes exist. In the000 residentsovided by thent of a life‐threMoreover, theatment or prnefit for thoseare also slightadvice in addie for clinical s
automatically ce the introduan designationsee Figure 1). Due to the reeveral authorcts in the EUhan drugs is in
US and the EU dyears of the Ecluded. The nu
S FORWITH A
the Utrecht I
rare diseases, n the EU (200w orphan medicul marketing au
ed and a matchormed. Determuct and of the
revious experiethermore, exisreaching approy in developingme applies for
to stimulate ug Act in thProducts in 20e USA, drugs is) are eligible e European Coeatening or che sponsor shrevention of te affected by ly different. Btion to a periostudies, as we
mean an oruction of the ns have been . In the EU, 6elatively lowers of scientifi is progressinnadequate [3,
during the first EU orphan druumber of US au
PEOPA RARE
Institute for
orphan drug 00). Literature cinal products thorisation of
hed sample of minants in the sponsor. Data
nce of the sponsting syntheticoval status (OR=orphan drugs existing (synthe
the developmhe USA, the 000 [1, 2]. Theindicated for afor orphan dommission if thronically debhould establisthis conditionthat conditioBoth regulatiood of market ell as dedicate
rphan drug wEU Regulationprovided in th615 products r number of oic publicationng too slowly 4].
ten years of theg act (2000‐20thorised orpha
PLE LIVE DISE
nsor in obtaininc entities com=3.9, CI=0.9–16is an importanetic) molecules
ment of drugEuropean U
e systems hava maximum odrug designatithe product isbilitating condsh that theren, or if such eon [1, 2]. Morons provide a exclusivity. Hoed subsidies f
will be authon on Orphan he US and 112have orphan orphan drugsns, who stateand the qua
e US Orphan D009). Only orphan drugs has be
Page | 5
VING EASE
ng approval mpared to 6.6). nt predictor s, for which
gs for rare nion (EU) e much in of 200,000 on. In the s intended dition that exists no exists, that eover, the reduction owever, in for clinical
orised for Medicinal 2 products status, of approved
e that the lity of the
rug han een
co(se
FIGURE 1: c
Thanremreon
2 MATER
Aomw(cowpTof
3 RESULTF3af(Tsesa
rrected for indee www.FDA.go
comparison of or
he above obsend marketing sult in an auarketing authsults of two sn rare diseases
RIALS AND MA case‐controorphan medimedicinal prowere sampled(N=386), andcharacteristicsof the producwere calculateproduct. The same anaoutside the pfurther be dis
TS From the star31 orphan mauthorised fofor two indi(unauthorisedThe results ofshown in Tabexperience ofsponsor was aauthorisation
dications extensov/orphan and
rphan drug desig
ervations give authorisation
uthorisation? horisation for tudies in the cs to new orph
ETHODS ol analysis hacinal productoducts. Cases d from the cd matched 1:s were obtainct, and characed for the cha
alysis has beepublic domaincussed here d
rt of the EU Remedicinal prodr four indicatiications, resud) products wef the univariatble 1. The assf the sponsorassociated wit(OR=16.2, C
sions to make iwww.EMA.euro
nations and auth
strong impetn of an orphaThe aim of torphan drugcontext of a Phan drug deve
as been perfts were comwere sampledcohort of des: 3 on the dned from the cteristics of thance of obtain
n performed fn, and was pedue to the fact
egulation on Oducts obtaineons, and two ulting in 36ere sampled tte comparisonsociation was with drug deth a 16‐fold inI=5.5–47.4), w
in comparable opa.eu).
horisations betw
tus to the quean drug. In othis study wags in the EuroPhD thesis on elopment” [5,
formed in whmpared to thed from the pesignated, but data of desigpublic domainhe sponsor. Oning marketing
for US orphanerformed in ct that the man
Orphan Medid marketing other producapproved o
to serve as con between auts strongest foevelopment. ncrease in the whereas prev
to the number
een US and EU
stion of whatother words: was to discuss opean Union the developm6].
hich the chare characteristeriod April 20not yet aut
gnation of thn; characterisdds ratios (ORg authorisatio
n drugs, althocollaboration nuscript has n
cinal Productauthorisationcts (sunitinib aorphan indicantrols. thorised and or those charPrevious authchance of autvious authoris
r of EU authori
determines swhat makes predictors of or the United
ment of orphan
racteristics oftics of not y00 up to Octohorised orphhe case. Datatics of the indR) and 95% coon for a design
ugh this analywith the FDAnot yet been p
s (April 2000)n. One produand dasatinibations. A to
not‐yet‐authoacteristics thahorisation of athorisation cosation of oth
ised orphan dr
successful devan orphan def successfully d States, basen drugs “From
f European ayet authoriseober 2006 anan medicinala on three gdication, charonfidence intnated orphan
ysis included A. This analyspublished.
) up to 1 Octoct (imatinib)
b) have been aotal of 60 d
orised orphanat were relatan orphan drompared to noher drugs and
Page | 6
ugs
velopment esignation obtaining ed on the m research
authorised ed orphan d controls products groups of racteristics ervals (CI) medicinal
data from is will not
ober 2006, has been
authorised designated
drugs are ted to the rug by the o previous d previous
daapsca
Ta
E
I
P
T
Tcsmatsi
Ta
PrOt Ty
4 DISCUS
TwoaCosmohss
designation oand 8.0 (2.5associated wiproduct is pasynthetic enticompared to and busulfan, ble1. Main resu
Experience oOtheOtheOthe
ndication groMeta
PharmaceutiOral
Type of prodExistiPrevi
The results frcharacteristicssuccessful mamedicinal proauthorisation tendency towsynthetic entinnovative synble 2. Multivar
redictor ther orphan
ype of produ
SSION This study revwith marketinof drug prodauthorisation Companies thobtaining mastraightforwamedium sizedobservation ishave acquiredstage. The EMsponsors of d
f another pot5–25.7) respeth higher chaartly associatities [e.g. newbiologic produ were associaults of the univ
of the sponsoer products aer orphan druer orphan druoup abolism (vs. ocal formulat(vs. parenteruct ing syntheticiously autho
rom the mults were relatearketing authoducts authoof other orp
ward an associtities comparnthetic entitieiate analysis of
drugs appro
ct
veals two indeng authorisatiduct in develfor orphan drhat have sucrket authorisard; however d enterprises s verified by td the necessaMA has addrdesignated orp
ential orphanectively. Furthances for authted with maw chemical enucts (OR=2.0, ted with highariate analysis
or pproved ugs designateugs approved
oncology) ion ral)
c molecules (rised
tivariate moded, are shown horisation, naorised and thhan productsiation for the red to bioteces compared tf predictors of m
Valueoved No
Yes BiologNew Existi
ependent chaon: the experopment. Firsrugs was idencessfully brouation for consa majority o(SMEs) with lthe fact that ary experienceressed this isphan drugs. A
n drug by the shermore, prehorisation in trket authorisntities (NCEs)]CI=0.7–6.1), er chances of
ed d
(vs. biologics
el, which assin Table 2. Tamely the exhe type of ps by a sponsotype of prodchnology proto biotechnolomarketing auth
e
gical Molecular Enng synthetic
racteristics ofrience of a cost, we showentified as the mught an orphsecutive orphof all orphan imited prior emany formere by bringingssue by offerAnother impo
sponsor yieldevious authothe EU (OR=4sation. Althou] such as imaexisting synthf market autho
Case(n=3
s)
essed whethehis model yiexperience of product. The or (OR=17.3, Cuct, with an O
oducts, while ogy products (orisation of orp
ntity molecule
f an orphan mompany in deved that expemost importanhan drug to han drugs modesignations
experience onr SMEs with og on board exring protocol ortant incentiv
ed odds ratiosrisation outs4.0, CI=1.1–14ugh the assotinib was notetic entities, sorisation (OR=
e 36)
Con(n=
31 30 28
9
21
15 8
er any of theelded two indethe sponsor association wCI=5.6–53.1). OR (CI) of 3.9no associati
(OR=1.9, CI=0phan medicinal
OR (CI) 1.0 17.3 (5.61.0 1.9 (0.5‐73.9 (0.9‐1
medicinal prodveloping drug rience of a nt predictor othe market ire than 17‐fos is being den orphan drugone or more axperienced maassistance anve to overcom
s (CI) of 11.5 side the EU 4.5). Finally, thociation for it statistically such as arsen=3.3, CI=1.1–1
ntrol =60)
OR
26 28 13
5
12
15 4
e statistically ependent prer having othewas strongesMoreover, w
9 (0.9–16.6) foion was obs.5–7.7). products in Eu
6‐53.1)
7.7) 16.6)
duct that are ag products andcompany in
of market authincrease theiold. These reseveloped by sg developmenapproved orpanagement and scientific me this hurdle
Page | 7
(3.2–42.2) was also he type of innovative significant ic trioxide 10.6).
R (CI)
11.5 (3.2‐48.0 (2.5‐216.2 (5.5‐4
3.6 (1.0‐1
4.0 (1.5‐1
3.3 (1.1‐14.0 (1.1‐1
significant edictors of er orphan st for the we found a or existing erved for
rope
associated d the type obtaining
horisation. r odds of sults seem small and nt [7]. This han drugs t an early advice to e is EMA’s
2.2) 5.7) 7.4)
2.4)
0.6)
0.6) 4.5)
dgmTifumibcbs
5 CONCLIadciMhp
ACKNOWTECv
REFEREN[1] US F
22 A[2] Regu
orph[3] Jopp
Phar[4] Jopp
May;[5] Heem
Euro[6] Heem
Utrec[7] Wäst
orph[8] EMEA
dedicated SMgood steps fomaze in the dThe other indit shows that four times higup to Octobermetabolic disinto drug probiotech producelecoxib andbe one of theshare of orpha
LUSION In conclusion,already existdesignations. companies ininexperiencedMedicinal Prohas to ‘grow’process.
WLEDGMENTS This is a modEuropean UniClin Pharmacovan Weely, Ha
CES FDA (1983) Thpril 2010 ulation (EC) Nan Medicinal i R, Bertele macol 61:355i R, Bertele V;67(5):494‐50mstra HE, de Vpean Union. Emstra HE, Fromcht, The Nethtfelt M, Fadeean drugs. J IntA (2005) SME
ME office, whicorward to guievelopment pependent prethe odds of agher comparer 2006 were oorders. This foducts is geneuct. This grou sildenafil, fore reasons whyan drugs base
, we have fouting (small) The EMA a
n their orphad companies.oducts seem t’ and acquire
dified and abbion” that was ol (2008) 64:5ans A. Büller a
he Orphan Dr
No 141/2000 Products. OffV, Garattini 5–360 V, Garattini S02 Vrueh RL, vanEur J Clin Pharm research onerlands, 2010el B, Henter JI tern Med 260E Office. http:/
ch addresses tde smaller anprocess. edictor that wauthorisation ed to biologicaof biological ofinding is not erally considep also includer which devely the relativelyed on existing
und that expemolecules arddresses thisan drug dev The incentivo be helpful fe expertise on
breviated versoriginally pub545–552). Thand Hubert G.
ug Act, Public
of the Europeficial Journal oS (2006) Orp
S. Orphan dru
Weely S, Bülrmacol. 2008 n rare disease0. (2006) A jour0:1–10 //www.emea.
the regulatorynd relatively i
was identified wfor products al products. Oorigin; of theseunexpected, ered much mes orphan druopment was y young Europsynthetic ent
erience in there associateds with severvelopment. Thves forthcomfor companiesn the specific
sion of the arblished in thee author wisM. Leufkens,
c Law, 97–14
ean Parliameof The Europephan drug de
ug developme
ler HA, LeufkeMay;64(5):54es to new orph
rney of hope:
europa.eu/SM
y needs of SMinexperienced
was the type based on exisOnly 6 of the 3e, 5 are used as the develo
more straightfougs based on only a matterpean Regulatitities.
e drug develod with markral helpful inhere seems ming from thes developing c peculiarities
rticle “Predicte European Johes to acknowwho co‐autho
1.http://www
nt and of thean Communitevelopment i
ent is not ta
ens HG. Predi45‐52 han drug deve
lessons learne
ME/SMEbackg
MEs [8]. Thesed companies t
of product. Asting synthetic31 orphan drufor enzyme reopment of exiorward than existing appror of an indicaton on OMPs h
pment procesket authorisacentives to sto be a steee European orphan drugss of the orph
tors of orphaurnal of Clinicwledge Remcored the origin
w.fda.gov/orph
e Council of ties 2000 s progressing
king off. Br J
ctors of orpha
elopment. The
ed from studie
ground.htm. A
e incentives sethrough the r
Although not sc entities mayugs approvedeplacement thisting synthetthe developmoved therapietion extensionhas yielded su
ss and develoation of orpsupport inexep learning Regulation os, although thhan drug dev
n drug approcal Pharmacolco L.A. de Vrunal article.
han/oda.htm.
16 December
g too slowly.
Clin Pharma
an drug appro
esis; Utrecht U
es on rare dis
Accessed 22 A
Page | 8
eem to be regulatory
significant, y be about in the EU herapy for tic entities ment of a es, such as n. This can uch a high
opment of han drug perienced curve for n Orphan e industry velopment
oval in the logy (Eur J ueh, Sonja
Accessed
r 1999 on
Br J Clin
acol. 2009
oval in the
University,
seases and
pril 2010
Authoris
Session 9Mrs Lia v
FIGURE 2: sHans Georg
EUNETHworking prioritisat
DR CLAUTECHNOL
n prepareprese
EUNetHTa large nu
INTRODUHealth Teyears agoten years
HTA is a missues recontains
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sed but not alwon improving
9 was co‐chairan Ginneken,
speakers and chag Eichler, Claudi W
TA group on Motion of HTA
DIA WILD, LULOGY ASSESSM
aration of thisentative of myTA is a Europeumber of HTA
UCTION: WHA
echnology Asso in Canada, ms.
multidisciplinalated to the assessment o
Health proble
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red by Prof HaEuropean My
airs, session on imWild, Andras Feh
onitoring em
UDWIG BOLTZMENT, VIENN
s presentationy agency in Aan project anA agencies in E
T HTA IS ABO
sessment is a more recently
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em and curren
ble to patientsphan drugs, w
ans Georg Eichyeloma Platfo
mproving access hervary and Lia va
erging/new t
ZMANN INSTINA
n, I found mysAustria, and asd therefore I Europe.
OUT method that
y in other cou
hat summarisealth technolog
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s! From the Euwe now need t
hler, Senior Morm, Netherlan
to orphan drugsan Ginneken
technology de
ITUTE OF HEA
self in two diffs a EUNetHTAneed to refle
t has been intntries like Au
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uropean Pharmto consider im
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evelopment a
ALTH
ferent roles: aA representatct the opinion
troduced in hestria where it
n about the mmatic, transp
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maceutical Fomplementation
r, European M
and
as a ive. n of
ealth relativelt has been dev
medical, sociaparent, unbias
cess to Orph
orum recommn through inte
colla
Medicines Age
ly recently, soveloped durin
l, economic ased, robust m
Page | 9
an Drugs
endations ernational aboration.
ncy and
ome thirty ng the last
nd ethical manner. It
T S C C O S
Its aim isachieve b
The intenthat is be
Several Hliving in tthe sameassessmeThis is hig
Several iNetwork EUnetHTApartners.
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s to inform tbest value. De
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Page | 21
f with cost with health
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payers
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to start a 2009. The European
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ks on one sideon ground thatrs could not co“there isn’t swe want to mthe regulatorrpose of the is needed. ties me of marketa situation whgement stratek side, it is vebenefits’ side
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that go into e current mave (Relative Efss Analysis, Ber states place
cross Europe
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arketing authoris
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me to a curve
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as to be some spontaneousregisters, obss request a coof conditional
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ve similar to t
al
Page | 22
sequences sment.
al yet, but possible”.
e to grant A common
follow‐up reporting ervational ompany to approval,
evidence t between ave a good
he way to t how the ook at the e appraisal o less so).
the one in
Benefit‐rilarge degcost struc
Finally, aand econvalue. Th
DEBATE Question
Responseaccept oupoint theearly stagThat is a the basis relevant o
ResponseAustraliahave a loof Life insdo?” And
It is not aProgressifrom the some proshow thewill pay fleast not
Question
Responseevidence won’t be
Responsepayers tobased onindicationaccording
Responsenot prediwill go mpatient gmore orpmarket ex
isk evaluationgree. Full HTActures are so d
ccess to orphnomic issues. is cannot gua
1. What shou
e 1, HTA: we utcomes that ere might be pges of clinical matter of meof surrogatesoutcomes like
e 1, regulatorn payer who ot of sympathystruments, wd I have a lot o
an easy question Free Surv HTA organisaogresses by heir cards and sor it. How couquestion the
2. What will b
e 2, HTA: A slbased medicany huge dise
e 2, industry: o realise is than the value thn. There is recg to the value
e 2, regulatorsict, and thoseainstream; thgroup into maphan conditioxclusivity.
n is now harmA and even modifferent acro
han drugs is a Collaborationrantee but wi
uld be the prio
must approaare not patie
possibilities totrials. Almostthodological ws and surrogate Overall Surv
r: we are alresaid ”How cay for that. Whe don’t knowof sympathy fo
ion. We have ival and we aations that wharmonising dsay “If this druuld you? You doutcome, the
be the picture
lightly provoccine will live aease group an
Direction in tat it is not a se drug is gencognition that generated by
s. I would like e who predicthe better we uany substrata.ns. There nee
monised. Relatore societal poss Europe. Th
societal goal n between thill likely facilita
ority aspect wh
ach methodolent relevant lo cooperate ust none of theweaknesses otes like Progreival.
eady in the man a regulatorhen I listen to what they saor that positio
battled for haare proud thawe are far behdisagreementsug comes to mdon’t see the e endpoints, a
e in 10 years fr
cative responsany longer. Beny more.
he future willsin for a moleerating. You t each use of y that use.
to quote thist do not haveunderstand dis. Therefore, ieds to be a re
tive efficacy creferences whe greatest ga
in the EU. Ohe regulators ate access to
where regulato
logical issues ike surrogatesing only surro drugs analysof quality of lifession Free Su
middle of this r ever authoro our CHMP may. Are they von.
alf a decade “at we did it ahind, we’re sts we have. Wmarket in threresults; you d
and the metho
from now?
se... In ten yeecause of the
definitely beecule to havedon’t have toa molecule h
s Chinese philoe knowledge”.sease processn an ideal woethinking on
could also be ill be far morein could be in
rphan drugs aand the payorphan drugs
ors and payers
earlier. For e markers (e.gogates that ared benefited fe tools. Almourvival are som
cultural and rise a drug wimembers, I wovalid? Do they
“Should we mhead of the Atill using surroWe should getee years fromdon’t know wodology of the
ears from nowe many bioma
personalisede several indico renegotiate as a certain v
osopher who . I would agreses the more world, if sciencthe part of le
harmonised ae difficult to h the relative e
are linked to yers could hav.
s should work
example less g. Progressionre validated afrom Quality ost all orphan metimes not a
Darwinian dethout qualityould hear” Buty give the info
move from objAmericans.” Aogate endpoint the payers now, we will hat the cost we trial.
w everything arkers pushed
medicine andcations. Each every time yoalue and that
said “Those wee with my cowe will stratife progresses,egislators for
across Europeharmonise beeffectiveness
unique methove considerab
together?
and less HTAn Free Survivand incorporat of Life measdrugs are appassociated wi
ebate. I havey of life informt look, all theormation we t
ective responAnd now we’rnts. We can oon board so not guarantewill be. But we
will be orphad into the ma
d what is impindication beou go back wt it should be
who have knowolleague. Orpfy what we th, we will haveexample rega
Page | 23
e to a very ecause the area.
odological ble added
A agencies al). At that te them in urements. proved on th patient
heard an mation?” I se Quality think they
nse rate to re hearing only make that they
ee that we e would at
an and no rket there
portant for ing priced
with a new rewarded
wledge do han drugs ink as one e so many arding the
Question this affec
Responseaccepted
Regulatoand you hbeing ma
Industry. thing do t
Question HTA evide
Responseinvested organisat
3. Clinical trict the evidence
e 3, HTA: I tota much too oft
r: what do yohave to offer andated by the
In this case, to. This was d
4. Other neeence analysed
e, HTA: I 100%in HTA reportion of care.
ials last shorte you need?
ally agree. If tten.
u mean by mthem somethe ethics comm
I agree Progrdone in an eve
ds than drug d.
% agree. Sincerts is for drug
ter, and trials
the trial stops
uch too oftenhing. Three wmittees.
ression Free Serolimus trial.
reimburseme
e HTA is mainlg and techno
’ participants
too early, we
n? If there is pwill be cross‐ov
Survival has to
ent exist, e.g.
y used for druology reimbur
s can cross‐ov
e don’t know e
rogression yover for ethica
o replace oth
evaluation of
ug reimbursemrsement, almo
ver from one a
enough about
ou cannot keel reasons, and
er endpoints;
f long term ca
ment decisionost never for
arm to the ot
t the drug. Cro
ep a patient ond that is exact
; it is ethically
are. There is h
ns, all the pubr the evaluati
Page | 24
ther. Does
oss‐over is
n the drug tly what is
y the right
hardly any
blic money on of the
This s
Session 4Agency, a
THE EMAf
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Page | 25
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Page | 26
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Page | 27
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Page | 28
of unmet
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ment with
actice, e.g. d efficient
access to
nteraction
in
Research)
drugs for
rm
bel”)
Potential
a i i d
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FIGURE 11: indication
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Figure 12: P
US paediatric
risks:
adverse effecinefficacy (unimproper formdelay in acces
ovement in rec
pplications apre, 42% were d potentially re). The CHMP
only a third of n
then, signification from adu
Pharmacokinetic
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ts (over dosinder dosing) mulation ss to useful m
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pproved in thnot applicablbe used in chP had no legal
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does change wit
harmaceutical
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edicines
he first ten ye to children hildren, only p tool to enfor
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n Act or BPCA)
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om 1995 to 2006
nes for paedition…
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6 benefited from
iatric populat
Page | 29
ncies
entralised ers, drugs or children
a paediatric
tions. And
In adults,around 2dose neeeliminatioto adultswould be
But shouefficacy)?with no e
MEDICIN
The inforSummaryand valid
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WITH TH
Now, westimulate
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PRINCIPLHow are combines
(a
, the eliminat20 to 25 houreds to be redon is more ras’ ones. If onee calculated fo
ld one study e? The Paediatexaggeration r
NES EVALUATI
rmation on Qy of Product Cated by regul
he drug is apprn medicines, m patients’ or
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roach is to enmain example
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E REGULATIO
e have the Re needed rese
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LES OF THE PAcompanies sts obligations a
Is under deve(PIP), in otheradult formula
In thgene
Is on the mar
tion of a drugrs (see figureduced to achipid, thereforee would extraor one or the o
every single dtric committeregarding the
ON FOR REGIS
Quality, SafetyCharacteristicsators at the ti
roved via the an Europeanrganisations v
on is made atofitable to regr children, dis
nsure that thes in the EU:
s (EU regulatioediatric populaame into forc
institutions tbut its implem
ON FOR PAEDI
Regulation onarch on them
1st time on Jus (e.g. nationa
AEDIATRIC REimulated to dand incentives
elopment (art r words a prosation cannot bis case the inceric products’ ket but still un
g called diazep). In some paieve the desie the absorbeapolate and aother paediat
rug in every pee tries to reqdemand.
STRATION y and Efficacys, the Patient ime of market
centralised prn Public Assesvia the Patient
t the request gister/developseases of pove
e required me
on in operatioations (the pae on the 26 Ja
o take the inmentation has
ATRIC DRUGS
n paediatric Mm. It came into
uly 2007. It is l drug agencie
EGULATION: Ho the work ths/rewards. Th
7): in this casespective plan be accepted)centive is six m(competitors)nder data pro
pam from theaediatric popured blood leved dose needsadjust simply ric group.
paediatric subquest the nec
y in normal Leaflet and thting authorisa
rocedure (singssment Repots’ and Consum
of applicantsp drugs or generty…?
edicinal prod
on since 2000)ediatric regulanuary 2007
nitiative and dproven to be
S Medicines (Ro force on the
composed ofes), three pati
HOW DOES IT
hey would nothey depend on
e it is compulfor clinical stu
months exten) entry on theotection (art 8
e body (calledulations, elimvels. In others to be increaaccording to
b group for evcessary inform
conditions ofhe Packaging cation, and is th
gle procedurert (EPAR) witmers’ Workin
s, generally dnerate data to
ucts become
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do it themseldifficult.
egulation ‐EC26th January
f five CHMP mients’ represe
WORK? t spontaneousn whether the
sory to submiudies in childr
nsion data proe market will b) and if (usual
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very aspect (phmation for th
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e for all 27+3 mth a “summarg Party at the
rug companieo meet health
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ves. This is a
C‐ No 1901/22007.
embers, 22+2ntatives and t
sly do? The Pae medicine…
t a paediatricren (if not, the
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) is relatively ower and therldren and infequivalent blt, then the w
harmacokinethe necessary
on label”) meormation that
member statery for the pue EMA is publi
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the develope
1901/2006 +
also a possibi
2006 + 1902
2 experts fromthree health c
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e drug is appry six months) PIP submitted
Page | 30
rapid and refore the fants), the ood levels rong dose
tic, safety, subgroup,
eaning the t is known
es), life for blic” with shed.
ey do not as orphan
ers. There
lity in the
/2006) to
m national care
ulation
n plan for the
roved,
A Paediatthe rewaof its impexpected
The rewalead to an
CONSEQUCompaniesubjects.
But someoccurringis reason a paediat
As so malimited, eexpectati
Agreeing outside it
T
There shodata base
RESULTS Up to noimplemenbelow sh
RewaIs on the mar
paedIs an orphan m
Incen
tric Investigatrd. It must beplementation d significant be
ard may be obny indications
UENCES AND
es cannot claIf the PDCO f
etimes it is ng both in adultto believe thatric developm
any new drugespecially forions and healt
on (establishts control:
The results of Wha
PIPs normally Wha Or wcomp
ould be timelyes, etc.
ow, April 20nting a PIP (dows the numb
ard of six monket without pdiatric use mamedicine (art ntive of 2 extr
tion Plan (PIPe submitted tocan be agreedenefit, etc.).
btained if the s.
ISSUES im any longefinds it of bene
not easy to dts and childreat its mechanent is, at least
gs are simultar less prevaleth needs.
hing) the PIP i
f the investigat if the plan isy include clinict if they are n
what if there apany)? Should
y coordination
10, no orpharug developmbers of PIPs/w
nth extensionrotection (artrketing autho37): compulsora years of ma
), pre‐approvo the PDCO ed; a waiver is
PIP is implem
r that they arefit for the ch
distinguish ben, e.g. a drug ism of action t, “encourage
neously invesent conditions
s a (binding)
ation (for orphs not considercal trials and cnot accepted bre trials alreadd the PDCO try
n with the res
an drug has ment is a long waiver submit
n data protectt 30) and if deorisation (PUMory, if under darket exclusivi
ved by the Paearly in the degranted in so
mented, even
re not intereshildren, they a
etween what may be devewould apply ed”.
stigated, the s. The PDCO
responsibility
han medicinesred sufficient clinical trials aby them? dy in progressy to amend in
st of involved
been granteprocess, implted by year.
tion veloped for c
MA) granted development,ity
ediatric Commevelopment foome circumsta
in cases wher
ted in studyinre obliged to
t is an “adulteloped in adulequally well t
availability of tries to prio
y of the PDCO
s) are assessedto grant the inre approved b
s in some MS ncomplete tria
parties. This r
ed the 2 extementing a d
hildren accord
to submit a P
mittee (PDCOor adults (endances (e.g. adu
re the results,
ng a certain indo so.
t only” indicats for an adulto some paedi
f subjects for oritise accord
O but some di
d by the CHMndication targby NCAs
(normally spoals?
requires timel
tra years of evelopment p
ding to a PIP
PIP
O), is always nd of phase I). ult only disea
, once obtaine
ndication for
ation or a “ct cancer. Whiatric cancer?
r clinical trialsding to perce
rectly related
MP. geted?
onsored by th
ly transparen
market excluplan takes tim
Page | 31
eeded for A deferral se, lack of
ed, do not
paediatric
condition” at if there Normally,
s becomes ived drug
d tasks are
e
cy, shared
usivity for me). Figure
FIGURE 13:
But sponaffect on
CONCLUSThe new diseases.
That this requires tscrutiny.
COMMIT
DR FABRThe contetheir auth
EC REGUADVANCE
he neof su
validate ttreatmen
The publithe EU leforce on 2009.
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Source EMA, pre
sors developly children an
SIONS regulation aimIt will genera
is, in fact, efftransparency But the begin
TTEE FOR ADV
IZIA BIGNAMent does not hor.
ULATION 1394ED THERAPIESew EU regulatuch products their use exisnts, of segmen
ic was consultevel, with a s30 Decembe
ulation on ad: some tissuee the final “co
eliminary results
ing orphan dd 55% affect
ms to improveate abundant
ficiently transland good coo
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VANCED THER
I, THERAPEUpresent the o
4/2007 ON AS (CAT) tion on advancwere under sted across Enting the mark
ted in 2002, 2specific, harmr 2008, and t
dvanced ther engineered pombined med
s from 2007 to Ap
rugs do applyboth adults an
e the situatioresearch, bure
lated into releordination beomising.
RAPIES: WHAT
TIC DEVELOP
fficial view of
ADVANCED T
ced therapiesdevelopmentEurope, with ket and of und
2004, and 200monised and cthe EMA Com
apies covers products, or cicinal product
pril 2010
y for PIPs. Obnd children (o
n of medicineeaucracy and
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T IS RELEVAN
MENT DIRECT
f the EMA or o
HERAPIES AN
s was necessat, but differea risk of hindermining ind
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a scope thacell or gene tt”. However, o
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T TO RARE DI
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ric populationthe industry.
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SEASES?
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MITTEE FOR
asing numberes on how tonts’ access topment.
ll stakeholderamework. Therapies (CAT) w
oth medical be associatedf biotech prod
5‐20% of rare
ns, including f
ared responsuld evolve un
f
r o o
rs wanted clee regulation cwas created i
devices and d to medical dducts (e.g. rec
Page | 32
e diseases
for orphan
ibility that der public
ar rules at came into in January
medicinal devices to combinant
insulin ounder thAdvancedregulatedpharmace
The CATCHMP mfrom eacrepresentfrom Erepresentprofessiois an alte
EXPERTISAll togeththe areabelow an
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TH CARE PROF
are professionhair/vice‐chairon applicatioor for the Cessification prore reimbursed
ate 2009‐2012EGAN (Europe
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Figure
ed to cover the figure al aspects.
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NVOLVEMENT
embers of themmittee. Theyvanced theraQuality/Non‐Ce rapporteur ther member
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IVITIES vote on produpart of the re‐registratioAs any other Cguidelines. Th
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Figure 15: C
The CAT drafts
provide advico, the preparemit of the CA
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s patient ber) and Nick
Page | 33
AT activities
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ce for the ration and AT.
rocedures, teams for ts already , they can, for travel
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ISSUES AIssues
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mbers of adorphan drugg over the yy innovative chanisms of aand often lifeher chances efit evaluation
y and haemomains whereveloped.
ORDIS (Europerizia Bignami la (alternate)
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of products anof action) and costly invdvanced thermall market (
out of the gambeing)
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n drugs designneered produe therapies.
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matology are e advanced th
ean Organisat(member) and.
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vestments apies) for the time
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ge rarity and ces: ach to determn follow‐up of
ork at the EU
nations by theucts, 19 cell
rapies that is regularly se products completely ddress very diseases to a positive
the main herapies are
tion d
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Fide
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igure 16: advanceesignation, by ye
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IGURE 17: clinicaesignated as orp
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red so evaluation
ncentives and
anced therapie
al domains of ahan drugs
h an orphan drugg to category
of their econ
support
es (or 7% of a
advanced therap
Page | 34
g
omic
all orphan
pies that are
Overall, aspecificalKingdom,Italy (figu
Who is ddiseases?enterprisprofit orgTelethon designateproducts,envisagedsector.
INCENTIVFor advan
Scientific
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These are
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advanced therlly those that, Netherlandure below).
developing ad? Five smses develop 1ganisations lik(Italy) hold
ed gene th, and specid to support
VES FOR INDU
nced therapie
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ease patients,onsiderable ho that for Orpment?
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What additioauthorisationWhich infrastcentres) How to facilit
rapies are mot have an orpds, France, G
dvanced theramall and m16% of themke Genethon around 30%
erapy orphaific incentivet efforts in th
USTRY AT EMAes:
ction for SMErs
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til 2011/2012
ction*
ction for 1st y
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health interes
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ns to be answ
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FIGURElocated
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munity is prov
ly debilitatingmic research applies to adv
nvisaged? (res
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nce to the new
Kingdom, Germve countries
E 18: countries d
t before Dec 2
ven)
g and often lifand advancevanced thera
search grants,
uring Practice
w regulatory r
many and Fraare leading i
where advanced
2008
fe‐threateninged therapies. pies: namely
tax credits, e
s platforms, s
equirements?
ance. When con equal term
d therapies dev
g diseases, haTherefore a how to facili
extension of
specialised cli
?
Page | 35
onsidering ms: United
velopers are
ave always reflection itate their
marketing
nical trials
t
CONCLUSRare Dise“non‐clas
Advancedkeen to eDisease C
DEBATE Question
Responseauthoriseare authodraw theassessed
Question2new drugDutch eth
Responseissues. Sotrials in achildren ithe sametheir view
Question efforts foresearch,children,
Question helpful. Inor the EU
Question advancedresearch that is leadministrenvisage
How to attracHow to suppprocedures? How to accelethreatening dHow to ensur
SIONS eases (the whssical conditio
d therapies arexplore innovaCommunity in
1: what is the
e 1: with theed all across torised nationae public attenyet.
2: the paediags. But in thehics committe
e 2: it is a reaometimes chila given countin other coune grounds. A Cws, but this re
3 (comment)or paediatric d focus on peand encourag
4 (commentn fact the only
U may fail in its
5: the goal id therapies wbeing stoppe
et say injecteration can bethis?
ct new compaport academi
erate advancediseases? re economic re
hole communons”.
re already beative strategie particular.
e risk of treatm
e newly adopthe EU. Treatmally or when ttion on the r
atric regulatioe Netherlands ee did not app
al problem, aldren are recrry and to accntries. The ideClinical Trial Felies entirely o
): the Framewdrugs and adculiarities of
gement to SM
t): SMEs are vy incentive is ts attempt to s
is to market twill probably ed because of ed once in ae financially r
nies into this c research, a
e therapies de
eturn on inves
nity and the C
nefiting fromes for drug de
ment tourism
ted Regulatioment tourismthey are not risks when pu
on is to stimuthe law is ve
rove a gene th
and not only ruited in very cept the drug ea is to set upFacilitation Gron member sta
work Programvanced therapchildhood in
MEs (with a goa
very involved the Certificatisupport advan
therapies thathave a lifelo
f the absence a patient liferewarded wit
area? academia and
evelopment fo
stment on inn
COMP) have
this experienevelopment in
with advance
on, when an m already existreimbursed evurchasing adva
ulate and to rery restrictiveherapy trial fo
in the EU. Thpoor countrieonce it is ap
p a global ethroup meets atates, not the
me of DG Respies via the fall research
al to spend 15
d in advancedion and this isnced therapies
t provide retung effect in of a business? Investors dth the curren
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paved the w
nce and the cn collaboratio
ed therapies?
advanced thts for classic verywhere. Thanced therapy
regulate paede regarding reor Duchenne d
he FDA held aes. It can be qproved, drug ical standard t regular inteEMA.
search at the following meaaspects, supp
5% of the rese
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urn on investmpatients who
s model: how do not see hnt reimbursem
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bined with an
ay for develo
ommittee forn with all stak
erapy will bepharmaceutiche CAT has puy products th
diatric researcesearch in childystrophy in ch
a meeting recquestioned hothat has thento approve orvals in the E
European Coasures: fundinport to researarch budget f
owever the RNew incentive
ment and an ao will receive
can we makeow a lifelong
ment policy. H
s’ access to r
pies for sever
n “affordable”
opment of th
r advanced thkeholders and
e authorised, cal products wublished a stahat have not
ch and develoildren. For exachildren.
cently to discow ethical it isn been develoor reject trialsU, trying to c
ommission is sng to advancerch on off labfor SMEs).
Regulation is es should be e
affordable prthem. I am
e profit from g effect withHow do HTA
Page | 36
regulatory
re and life‐
price?
erapies in
herapies is d the Rare
it will be when they tement to been fully
opment of ample the
cuss these s to refuse oped with based on coordinate
supporting ed therapy bel use in
not really envisaged,
rice. Many aware of a therapy
h a single A agencies
Responseprocess. foresees
Question products How to en
Question
The resdeclar
Session 2EBE/Euro
FIGURE 19: Camp, Eric
CHARTER
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R FOR CLINICA
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ical trials for rcruitment of aClinical Trialevant patienthow to identifwith regulatorntify progress
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nt study condctively particip
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AL TRIALS IN
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mmitment,
ch the CAT yee EUNetHTA s in a near fut
s for the deveclusivity and pers on this ma
ucted by EUROpate in clinica
r Alastair Ken
sion 22 on clinicaeetje Goossens
RARE DISEAS
PATIENT GRO
R INTERACTIO
are often extrd volunteers en as a pre‐tons, for examd/or patients nts and deadof trials for ra
ment face high
ase, e,
et, as advancestakeholder fure.
elopment of gpatent, and starket?
Clinical Tria
ORDIS showed al trials. Are pa
nt, Director G
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e field of rare
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ents in Clinic
atient groups artners to clinations ready t
nd Dr Erik Ta
Page | 37
regulatory d. The CAT
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Page | 38
sors, POs,
gh‐quality
articipants
e scale via
ts by their
s
ts of the ute to the d the on of a an when who do not
d in an ents, nt
e
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h a view
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nisations
o sign the
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PATIENT
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Behcets’
ABOUT BAbout 6078 000 tomore predisease: Tit is mosblindness
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BEHCET S600 knowactive me
TREATME
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OUR ROL
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Any collaboratestified to byon the EURORD
IS ‐ FACILITATEURORDIS will hEURORDIS mayinterfering in EURORDIS devcontribute to Additional docollaboration”
INVOLVEMEN
HARD WEST, By name is Rsince my diaazathioprine
disease is nam
BEHCET DISEA00 patients ho 311 000 in evalent than Turkey, Iran, Ist prevalent. s.
manifestationse body; non s, Neuro Behis, and demen
SYNDROME IN
wn sufferers lembership.
ENT AND CAR
no known cas. Proposed tr
esearch is notthe use of a f the disease.
LE IN CLINICAL
www.eurordis
M
ation betweey the “AgreemDIS website.
TING THE APP
help the spony also assist inthe study itseelops traininclinical trials.ocuments aim” ‐ can be ava
NT IN CLINICA
BEHCET UNIT
Richard West,agnosis I’ve tae, and steroid
med after a Tu
ASE ave this diseTurkey (100 ‐rheumatoid raq, Russian cIn Japan, Beh
s are ulcers ofdeforming a
hcet’s (that antia.
N THE UNITED
ive in the UK
E: WHERE WE
ause and no eatments incl
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L RESEARCH
s.org/IMG/pdf
en the Sponsment of Under
LICATION OF
sor identify En the setting‐elf. g sessions ai
med at these ilable on EURO
AL TRIALS
TED KINGDOMI am a patie
aken some 65s.
urkish dermat
ase in the U‐ 400 per 100poly‐arthritis
countries, Chihcet’s disease
f the mouth, tarthritis, Erythaffect 5% of
D KINGDOM. Behcet Socie
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lude steroids a
there has beeug Alfa Interfe
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sor and the rstanding”, wi
THE CHARTEuropean POs ‐up of the co
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collaborationORDIS website.
M ent living with5 000 tablets (
tologist Hulus
K (1 in 100 00 000 inhabitas. It is knowna, Japan aree is the seco
to the extenthema nodosuBehcet pati
ety UK starte
agnostic test and other dru
en only one kneron as it has
RY_FINAL.pdf
Patient Organill be made pu
R interested in ollaboration b
ping PO repre
ns ‐ glossary6,.
h Behcet’s dis(my estimates
hi Behcet.
000 inhabitanants). In Turkwn as the Sil the Countrieond highest c
patients cannum, chronic fents), causin
d over 25 yea
and we are ugs that suppr
nown clinical ts shown in oth
f
nisation, accoublic to all stak
collaboratingbetween spon
esentatives b
specifics of
sease. I was ds), including t
ts), but key, it is k Road s where ause of
not eat, legs, fatigue, uveitig migraines,
ars ago. It has
experiencingress the immu
trial in the UKher countries
ording to thekeholders and
g with them nsors and POs
better unders
agreement, “
diagnosed in thalidomide, c
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Page | 39
e Charter, d included
s, without
stand and
“fiches de
1995, and colchicine,
anywhere artial/total zures and
and a very
lems with
0 years, to hange the
For this tmembersdatabasethe otherlength. Ittwo week
The Behcinjector shospital ebank.
LESSONS By being offered itpopulatioin the UK
I can recoparticular
With theidea rececentre ofmonths Commiss
WHERE A
W
cc
Structureauthoritiegroup.
Another developmof the Beaspects, people sulot of atti
WHO CA
O
et
It is impo
trial, recruitms in its databae took part in r half are on a was only whks, and some
cet Society gasyringes so tevery two we
LEARNT FRO
involved thets help ‐ blooon, a genetic tK. Nowadays, t
ognise sitting r area we wor
se experts, weived a lukewf expertise, wpatients havioning author
ARE WE NOW
We have an Birmingham aBased on the centres, the Ncompletely an
e, ambition, ces and the in
action that dment and validehcets’ diseasefrom the lessuffered with ditudes and de
N HELP? Other patientEURORDIS Charexpertise will the moment.
ortant to atten
ent in the Unase to providthe trial whicalpha interferen the trial sthad to travel
ave £ 3000 fohat volunteeeeks. This £ 10
M THIS TRIAL
e Behcet’s Sood samples gtesting projectwelve of the
here John Dark in, is that p
we started to warm welcomewith their owne been workrities.
? application inand London inAlstrom sociNHS will provnd we are in f
committed pedustry. By ap
derives from odation, we ene even amongs severe to tdepression whpression is no
t organisationrter on clinicahelp boosting
nd conference
ited Kingdome them with ch is still on‐gron. The prototarted that vo100 or 200 m
or the train fars could self‐0 000 donatio
? ciety could aiven to try toct where the Sleading consu
art who gave meople are gen
develop the e as each of t expertise. Thking on the
n the commisn the UK ety model, thvide the doctoact evolving t
eople are guapplying for thi
our role in thnrolled all 600g doctors, as ihe more devhich had not bow mentioned
s, umbrella gral trials: we hg our relation
es, even for a s
m was immedithe informat
going: half remocol was discuolunteers realimiles to do so.
ares so that v‐inject alpha on represente
approach medo identify genSociety gave £ultants for this
me advice somnerous with th
idea of centrthe experts whis was not thconcept of
ssioning group
he Behcet’s Soors and healthowards a new
arantees of cis funding, I t
his clinical tria0 members in it described tvastating aspebeen acknowld during consu
roups, health have not signens with the ph
society like ou
ately a concetion on the trmain on their ussed with paised they wer
volunteers cointerferon ated a fifth of w
dical teams inetic markers £ 1000 to sees disease in th
me years ago heir time to he
es of expertiswas considerinhe opinion of centre of ex
p to run thre
ociety offers th care profesw “company”.
credibility to hink we chan
al was to devour databasehe awful realiects. For examedged by docultations.
authorities, ded yet as we armaceutical
urs.
rn. The Behcerial. 80 voluntprevious trea
atient represee asked to go
ould attend tht home withowhat the Behc
nvolved with based on dise if these resuhe UK are part
and I think thelp you with t
se for Behcetng he/she wathe patients,xpertise for
e centres of e
to run the admsionals. This w
engage into nged the doct
velop a qualite. Again this city of living wmple this revtors before. T
octors, EURORexpect the neindustry. We
et Society conteers, mainly atment left unentatives, but o to their hosp
heir visits, £1out having tocet’s Society h
Behcet’s resescoveries in thults could be t of our medic
hat is the beatheir advice.
t’s disease. Inas already wo, and in the lathis disease
expertise, in
ministrative swill change o
discussion wtors’ opinion
ty of life survchanged the pwith the diseasvealed that 60This has now c
RDIS and manyext to come ce have put it o
Page | 40
ntacted all from this nchanged, not in full pital every
10 000 for o visit the had in the
earch and he Turkish replicated cal panel.
uty of the
itially this orking in a ast twelve with the
Liverpool,
side of the ur society
ith health about our
ey: for its perception se in all its 0% of our changed a
y others. centres of on hold for
CONCLUSThe Grea
Wherevebenefit fr
EMP’S F
MRS GRE
ABSTRAChe deresea
as “subjeand theirthe deveinvolving can be volunteerarea wheMoreovedocumenexplains hbe gaineinvolvemreach of tskills; it gprobably
1. INTRMp
Mhaf
Hahtpo
2. STRA
2.1.
T
E
SION t Wall of Chin
er we are as prom the exper
FIRST STEPS IN
EETJE GOOSS
CT evelopment parchers, healthects” and hadr advocates helopment of cpatients, a pquite a chalrs and limitedere deadlineser, patient repnt presents thhow a patiented through dent in the devthe group. Thgave them costrong found
RODUCTIONMP (Europeanpatient organi
Multiple Myehave been limand treatmenfurther impro
However, ourand medical ahave a uniquetraining, usefpossibilities oother potentia
ATEGY
EXPLORATIOIn order to understand organised, t
na was built ov
atients’ organrience of thos
N THE FIELD O
ENS, EUROPE
rocess of clinihcare provided a passive rohave been moclinical trials.atient tailoredlenge for pad manpower s, standards apresentatives he case of a yot organisationifferent trainvelopment of hese first prudonfidence andations for mo
N n Myeloma Plisations.
loma (MM) ismited for a lont options areove the access
r organisationarena is a hige and valuableful for the woof partnershipal partners be
ON OF THE MMhave a goodthe other sthe “No Policy
ver a period of
nisations, partse who have b
OF CLINICAL TR
EAN MYELOM
ical trials has ers and regulaole. However, oving more to Many staked system of caatient organito act as an and certain wneed to acquoung patient n can proceedning programclinical trials,
dent steps gavd it created aore extensive c
atform) is a E
s a rare canceng time. Howe improving. to new treatm
n did not wanhly scientific e experientialork. We wantp with other setter and to in
M HEALTH CA
d insight in ttakeholders’ py without Pa
of more than 2
ticularly with been there bef
RIALS
MA PLATFORM
for long beentors. Patientsin the last d
owards an acteholders realiare can be acsations, whoequal partnework principleire enough “lorganisation
d step by step s. The docum, by initially onve the patienta relationship collaboration
European umb
er of the bonewever, a lot ofOne of EMP’ments and me
nt to tackle thenvironment knowledge, tted to proceestakeholders nstall a dialogu
ARE ENVIRON
the health capoint of viewatients” symp
2000 years. Bu
clinical trials, fore us.
M, BELGIUM
n the territory s were only invecennium, pative participatse that by achieved. Howeo often workr in this speces have to beiteracy” in thEMP and theto meet the ement further nly focusing ot representatiof trust within the field of
brella organis
e marrow, a df research ha’s basic goalsedications for
his new terraand, althougthey do not ned in a structin the field, wue with them.
MENT are environmw on patientposium, a mu
ut someone ha
we have to st
of the volved atients tion in ctively ever, it k with ialised e met. is highly scien way EMP haexigent criterexplains hown activities thves further knh the other stf clinical trials
ation for natio
disease for whs been done s is to supporr the disease.
in impulsivelyh patients anecessarily havured way andwe first want.
ment related tinvolvementlti‐stakeholde
ad to lay the f
tart somewhe
ntific environmandled this chria and how litw EMP starthat were withnowledge andtakeholders. s in the future
onal multiple
hich treatmenin the past drt the resear
y. Indeed, thed their repreve the specificd before expted to get to
to MM and t in health cer‐meeting, in
Page | 41
first brick.
ere and to
ment. This allenge. It teracy can ted active in realistic d research These are .
myeloma
nt options ecennium ch and to
e research sentatives c scientific loring the learn the
to better care, EMP n October
2.2.
2.3.
2008 in MaWaldenströmbetween thepoliticians, h
OBSERVATIO
An importanmedications This way, a pcompletes tshould devel
Many areas o
the rtrial)
the r the d the d the e repre advic advic the s
For a young involved in awork, a patieareas that acurrent stren
Another impmeet certainorganisation
ACTION PLAWith these cin the field researchers amedical liter
Indeed, feedpatient infodocuments b
Review of thprocedures aconsider the
Another imppatient invoabsolute req
aastricht. Thm Patiënten, e different pealthcare pro
ONS nt conclusion can be greatlpatient tailorehe scientific lop working re
of useful patie
review of me) review of clinidisseminationdistribution ofevaluation of tesentation byce on the selece on risk mansupport of fun
patient organll of the abovent organisatiare within reangth and poss
portant feedbn criteria in oal structure a
N conclusions, aof clinical triaand the indusature for pati
dback from marmation) are by patients en
e protocols ofare acceptablpsychosocial
portant conclulvement in tquirement tha
is event wathe Netherlapartners in hofessionals, re
on which aly improved bed system of and medical elationships w
ent involveme
edical literatur
cal trial proto of informatiof information the effects ony patients in etection and thenagement pronding for resea
isation such ae‐mentioned on should deach of the paibilities of the
back from theorder to becond a certain le
n Action Planals would be stry. Our initiaents and the r
any patients tnot always sures that the
f clinical trialse and comforaspects or th
usion of the syhe field of clat patient org
as organised ands). The syhealth care: esearchers and
l stakeholderby involving pacare can be aknowledge o
with the indus
ent in clinical t
re for patient
ocols on on trials toand data on nn the quality othics committe developmenogrammes arch and drug
as EMP, it wasareas at oncefine prioritiesatient organise group.
e other stakeome an equaevel of literac
n was set up foto establish
al collaboratioreview of the
told us that theasily undere documents
s by patients irtable for pate issues surro
ymposium walinical trials, ganisations ca
together wymposium serpatients andd the industry
rs agreed is tatient represeachieved. Theof researchertry and with a
trials were dis
ts (informed
patients in lanovel trials wiof life of new ttees t of clinical tr
g developmen
s unrealistic ane. In order to bs and proceedsation’s actua
eholders was al partner in y (see 2.3).
or EMP. The Agood workin
on with these protocols of t
he trial documrstandable foare patient‐fr
s useful to maients. Indeed,ounding the q
s that, in ordecertain condian rely on a s
ith CKP (Corved as a co patient orgy.
that access toentatives in the experiential rs. Therefore,academic rese
scussed:
consent, pati
y‐language thin the patietreatments or
ials
nt
nd too ambitibe efficient and step by stepal capacity, ta
that patient the field, su
Action Plan stg relationshippartners wouthe clinical tri
ments (the infor laypersons.iendly.
ake sure that t, treatment puality of life.
er to achieve aitions have tostrong organis
ntactgroep Kommunicationganisations, r
o new treatmhe area of clinknowledge o, patient orgearch teams.
ent informati
ent communitr drugs
ous at that stnd deliver quap to become inaking into ac
organisationuch as having
tated that ourps with the (auld be the revals.
ormed consen. The review
the treatmenprotocols do n
a positive evao be in placesational struc
Page | 42
Kahler en platform regulators,
ments and nical trials. of patients anisations
ion of the
y
age to get ality in the nvolved in count the
s have to g a strong
r first step academic) iew of the
nt and the of those
ts and the not always
aluation of e. It is an cture with
2.4.
3. RESAfterlitera
An aMyecollatrials
smooth comorganisationprinciples of
Active patiensome of theiand to be kn
So, in order exigent critewith the disvolunteer orlarge scale coalitions, ndisease orga
REALISATIONOur membeknowledge athe work.
Some of ouEURORDIS Slearn about tDuring this representativdevelopmenrepresentativ
EURORDIS ofHouÿez chairPlan of the Swork.
EMP’s activorganisation advises on th
Several of onational andin the field.
ULTS r this period acy to start pa
agreement onloma Networboration wous and the trial
munication li, as an equaltheir interloc
nt involvemenir representatowledgeable
to be an equeria. Most patsease and thrganisations hand financialetworking annisations.
N OF THE ACTership of andand literacy in
ur representaummer Schoothe structure training, p
ves in order t. The Summves and a stim
ffered even mred an internSymposium. W
e participatiosome furthe
he role of pati
our collaboratd internationa
of training artnership wit
n collaborationrk) specialiseduld consist of documents, a
nes and distinl partner, hasutors.
nt also requiretives are. Litein the specific
ual partner inient represeney do not nhave more dily stronger und collaborati
TION PLAN d our collabon the field of
tives participol is a uniqueof clinical triaparticipants ito learn from
mer School pmulating envir
more valuableal EMP‐worksWith his tailor‐
on in the Par insight in thent organisat
tors further il workshops o
and instructih the researc
n was signed d in research the review baddressed to
nguished tasks to act accor
es that patienracy is necessc disease field
n health care,ntatives volunnecessarily haifficulties in rumbrella orgaion among d
oration with clinical trials
pated at the e and intensivals, drug develinteract withm each otherproved to beronment for fu
e help to our shop on the d‐made advice
atientPartnerhe area of clintions in clinica
improved theon the topic a
on, EMP rephers and the
with a Europin Multiple M
by our patientpatients (info
ks and responrding to the
nt organisatiosary in order d, clinical rese
, a patient ornteer for the wave this specrealising thesanisations widisease specif
EURORDIS win order to gi
EURORDIS Sve 4‐day proglopment and dh regulators,r’s experiencee an importauture involvem
organisation.development e and guidance
r Project, a nical trials. Thal trials.
eir knowledgeand by their i
presentatives Industry.
pean AcademMyeloma. At at organisationrmed consent
sibilities. Thisstandards, tim
ns are highly to grasp and arch, health p
rganisation hawork becauseific backgroue conditions th paid stafffic organisatio
was a great hve us the nec
Summer Schogramme in wdrug regulato, researcherses regarding ant source oment in clinica
The Health Pof the previoe, EMP was ge
3‐year EU Fis project inve
e by self‐studnteractions w
gathered en
mic Research ga first stage, of the protot and patient
s is because thmeframes and
literate or at discuss comppolitics and so
as to meet qe of their confund at the bthan well esf: hence the ons and umb
help in imprcessary backg
ool, in Barcewhich patient ory processes s and otheclinical trials of informational trials.
Policy Directoously mentionetting prepar
FP7 project, estigates, enf
dy, by followwith other sta
nough backgr
group: EMN (it was agreed
ocol of the EMinformation l
Page | 43
he patient d working
least that plex issues o on.
uite some frontation ase. Also, stablished, need for
brella rare
oving our ground for
lona. The advocates in Europe. r patient and drug n for our
r François ned Action ed for the
gave our forces and
wing other keholders
ound and
(European d that the MN clinical eaflet).
In thHowpatiethe mbrou
Afteractivthe tIn th“EURDiseaenhaalso orga
Besidproje
4. CONThosknowcollapatiebroucoalistakestren
Thesin thmutuand f
ACKNOWThe aSymppolic
REFERE[1] Giele
[2] EURO
LINK TO
he beginning ever, in ordeent organisatimeantime andght all EU‐MM
r this positiveve in the field trial protocol ahe collaboratioRORDIS Charteases” consistsancing a transdetails what anisation shou
des these firstects:
Prov“dem
InforMye
Collaclinic
NCLUSION se first projewledge and iboration is seent friendly aght several mtions. For oeholders stepngth of our or
se first projecthe field of clinual respect anfurther involv
WLEDGMENauthor wishesposium Reporcy without Pat
NCES en, Peter. Eur
RDIS Charter fo
O THE SYMP
of the collar to represenons to join ind we can say M patient orga
e experience,of multiple mand the patienon with the Iner for Collabos of guidelinesparent and ea written Agreld include for
t partnerships
iding informamystify” the surming patientloma aboration witcal trials “Eud
ects gave ouinsight as ween as very frund accessiblemultiple myeur organisatip by step. Weganisation. W
ts allowed thenical trials, tond trust. Thesvement in futu
NTS s to thank Petrt and Françoitients” sympo
ropean Sympo
or Clinical Tria
OSIUM REP
boration, EMnt all European the work (Mthat a positivanisations clo
we also stamyeloma. Hernt documentsndustry, EMP ration betwees, aiming to ieffective dialoeement of Una given clinic
s in clinical tr
ation on clinicubject and betts, via our me
h the EuroperaCT”
r organisatioell as some uitful by the pe to the patieeloma patienton, it was te focused on
We have learne
e different stao get to learnse are strong ure clinical tria
ter Gielen, Tres Houÿez, EURsium and the
osium “No Pol
als in Rare Dis
ORT
MP was the oan MM patienMyeloma Eurove “side effecoser together!
rted up collare also, at thiss. works accorden Sponsors amprove the qogue betweenderstanding bal trial.
rials, EMP is a
cal trials to patter inform thember associa
ean Medicine
n and its reself‐assuranc
participating pents, a win‐wt groups togethe right strn a working aed to walk bef
akeholders to each other aand necessarals.
efpunt ZelfhuRORDIS for his EMP internal
licy without P
seases
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Page | 48
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Page | 49
6, 8
ACKnoWLEDGEMEnTs, CrEDiTs AnD MAin funDErsthe organisers particularly wish to thank the following persons/organisations/companies for their role:
thE JAGELLIONIAN UNIVERSItY IN KRAKOW
EUROPEAN COMMISSION, PUBLIC hEALth PROGRAMME, DG hEALth AND CONSUMER PROtECtIONProgramme of Community action in the field of public health
EXECUtIVE AGENCY FOR hEALth AND CONSUMERSThe responsibility of the content and programme of the 5th European Conference on Rare Diseases lies with the speakers and programme committee. The Executive Agency is not responsible for any use that may be made
of the information contained therein.
This conference was also supported by:
CSL Behring
Novartis
Sigma tau
This publication was coordinated by françois Houÿez, Director of Health policy, Eurordis.
All articles were written by françois Houÿez and shane Lynam, except those written by the speakers (Cristina rusu, pohla Gubo, stein Are Aknes, Annet van Betuw, Harald Hemstra, Kate Bushby, Bruno Dallipicolla, francesc palau, Greetje Goossens, Krystyna Chrzanowska, Marek Twardowski, Anil Mehta, steffen suchert, Elisabeth Hernberg, and Gabor pogany) to whom we are very grateful.
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