Page 15 - Demo
P. 15

Abst
t t t t ti t t Manuscript Val
Val
Val
EU 2019
Valu Value Videos
Plan cla cla a a a a to track in p with DM
their limitations limitations and and and their limitations limitations and assess
Pre
solutions White PapeWr hite hite POD
esentati
Impact of of DMD DMD Impact of DMD Health Utilities Utilities Index Index Analyses Analyses Utilities Utilities Index Index Analyses Peer-reviewed Journals Journals viewed viewed Journals Abstract Abstract Abstract Poster Poster Poster Manuscript In In development
mpromising mpromising competitive competitive advantage
Multi-faceted study to to characterize HRQoL Congress Activities Activities Media Media s s s s s s Activities Activities Media cost-effectiveness show significant
CustoentegmPeany
Customized P P P P P P Manu Manu Manu Form Form Planne Planne Planne the risk
for new g g models Develo Develo pati Assessment
Abstra Abstract Abstract and Post meas PRO stra stra areappr votalintrDia
and and and and and and and and States HUI HUI scores scores and and and and and and and and NSAA DMD on on families families and and wed wed evidence evidence is is is is available available as needed without compromising competitive competitive advantage
advantage
hefeasi
PR AnalysssiersFD
for ICER evaluation evaluation of of for ICER evaluation evaluation of Disease Agno
mitted tt tt t t Outlin Outline developed
Deter n n n n n n n n n of the the the ng ng ng if if the the the Com Com SRP- In d ment
PlannrtFina
quality of of clinical
Planned
Consistently reporting the status of market access developments to internal and colleagues
and and Abstract Abstract Abstract Abstract and and Poster Poster - - HTAi HTAi 2021
2021
assess
the economic economic and and and teddsrtivuedrystofcuhtialritiacetse raiznedHpRoQssoiLb le le correlation
correlation
Strategic publication plan plan to to ensure
ensure
assess
the economic economic and and DMD on on on families families and and and and utilities and and and possible correlation
between HUI HUI scores scores and and and NSAA NSAA ublication ublication plan to ensure
peer-reviewed evidence evidence is is is available available as as needed needed caregivers 2 2 Abstracts Abstracts Posters Posters - MDA MDA Posters - MDA impact evaluation Add Forms Forms for for for for DM
DM
cont
cont
Development of co usein
ansfer fr
Exondys-51 Disea Disea Ab nd Poste Pos Pos evaluation evaluation Models the attributes a a a assessment criteri
Abstract and Poster Poster - - AMCP AMCP Nexus Nexus 2020 2020 Abstract Abstract and and Poster Poster - - AMCP AMCP Nexus 2020 of of of of Abstract Abstract subAmbsitttreadctosucbomngittreesds to to congress
Abstr Abstract Poster M use use in in DMD DMD trials u u u Cl nd nd Poste Poste Analys Plan Ec Ec Economic Cos
Model
Quantifying the Impact of alth on Caregivers Multi-face drivers of Multi-phase study to assess
the economic and and humanistic impact of on caregivers Abstract Poster Manuscript 2 Abstracts In developm Manuscript Planned
mized PROMIS
ROMIS
s s for DMD DFDAMA Presentation
pmInednetvoelfocpomepnutteradaptivetestsfor mputer adaptive adaptive tests for Planned
script Payer S S Payer Segmentation
dCustomized PROMIS
Forms for Development of computer adaptive testspafotirents tr tr tr use in e e Videos
Addressing the risk
of attrition and and its impact on on on contracting models by understanding how patients transfer from one payer to to to another
DMD trials using PROMIS
PROMIS
item item banks banks sing sing PROMIS
PROMIS
item banks between and and ents transfer from one one payer payer to to another
another
om om one one payer payer to another
ract and Poster - - AMCP AMCP Nexus Nexus Claims r r r - AMCP Nexus ned
aims Analysis to to to Define the the Peer-reviewed Journals Congress Activities Media Strategic publication plan to ensure
peer-reviewed evidence is is available as needed without compromising competitive advantage
trials using PROMIS
item banks Addressing contractin Abstract a a a Planned
Manuscrip Planned
isAtbostrDacetfiandePtohster - AMCP Nexus onomic Costs of of DMD DMD fin in in the the the US US ts ts of of of DMD DMD in tin in in the the US Planned
idating the the feasibility of of using available available US US US biMlityanoufsucsriinpgtavailableUS
ims data to track track performance and and outcomes outcomes pPelarfnonremdance and outcomes atients with DMD D
stract and Poster - - AMCP AMCP 2020 2020 r r r r - AMCP 2020 ter ter submitted to congress
congress
o o congress
Claims Analysis to Define the the Replication of sIeCEARgnMoosdtieclModel
stic Model
stPhreoaimctipvaecltyopfrevpaaryringfdoisreICaEseR-sepvealcuifiaticonof ti i i i i fi i ti -specific
a a a a a a a offering alternative solutions Paper e e e e e e e e developed
developed
writing writing underway underway writing writing underway ct ct and Poster - - AMCP AMCP 2021
2021
er er - - AMCP 2021
PODCI
eorsftarnadintigoneavlaUluSativoalnue
al
al
US value meent criteria rmining their limitations and Determini measures
ned
Validating ti t t claims da dat in patients Abstract a a Poster sub Value Videos
Economic Costs of DMD fin in the the US Validating the feasibility of using available US claims data to track performance and outcomes in patients with DMD Abstract and Poster Poster - AMCP 2020 Poster Poster submitted to congress
Abstract and Poster - AMCP Nexus 2020 Presented
FDAMA Presentation
In development
o - Disease Agnostic Model
Models the impact of of varying disease-specific attributes and limitations of of traditional US value PRO Do Compilatio SRP-9001 in DMD pi Final Repo assessment criteria CI
Analysis isWhite Paper mining if the traditionally traditionally used used PODCI
PODCI
traditionally traditionally used PODCI
Outline developed
writing underway ures
are appropriate for for DMD DMD opriate opriate for DMD Abstract Abstract and Poster Poster - act act act Poster Poster Manuscript anuscript ed Abstract Abstract submitted to congress
Abstract Abstract and Poster - Abstract Abstract submitted to congress
- Nexus 2020 Abstract Abstract Abstract Abstract submitted to congress
eSRsPta-9tu0s01of SRP-9001 ameVwa
A A synthesis of of asAsessysnmtheenstistooflassustielsizsemdebnyt tools utilized by akersd elic
pies agnedn idencoeff-b
k MeNte
Abstract Abstract Abstract Abstract and and Poster Poster - HTAi 2021
AbstractMsubomnitttheldytoRceopnogrretss
taondinternal and An
accurateAunadcecr history of dihseisatsoery needmessangeesdanm
nd Poste Abst
and Studaiensd Studies
SLR of the deSvLeRloop respiratory dereclsipnier es oinffporom
from mittPeodst
the benefit of improving
Presented
Pres Presen Presen Presen Presen Presen develop in Health Manuscript- Orph Manu ment
In de
athrkeerat paicecsess
Pro Model
ComparinCgoDmispearsiengPrDoigsreeasseioPnrogression
Soc Societal Prefe
stuQdyuato to to soimcipe nd treaset
Natural HNiastouryalEHvidsteonrcyeERveidveienwces Reviews
Rates for RDaMte
caregivers nd PAobsstet
loss
ndAPbostre AbstractanAdbPsotsrat Prese Prese Prese Prese effective therapies
t- Value Man n n n to indi pre AbstractanAdbPsotsrat congress
menItn de
differeffin
Presented
Presen Presen t- t- J Clin Neurol
Manuscript- J Clin Clin Neurol
Neurol
Abstract and Poste Ab Pre
CONFIDENTIAL
39
Budget g g g g visually demonstrated the the visually demonstrated the the the the for gen e e Stud Vign
gene therapies
therapies
vs Utiliti Pati seffortt mentI ntad
Conti reimbur
barrie
barrie
t t Manuscript On hold
ic ic Assessment
Assessment
Tool
Economic Assessment
Assessment
Tool
Tool
Therapies
Therapies
Continuous effort Contin developing financi develo for for gene therapies
gene therapies
therapies
vs vs therapies
(e g g g ealth sta Estim rspectiv
societal perspective to to describeclinical describeclinical course course ssion
ssion
with with granular granular detail
Value Soc a a a a Discrete Cho a D
Submitted to journ Submi ngage Paye er Wh Analog Analog Resea Resea Work of of of of of of Anal Anal Anal FDA FDA and progression with with granular granular detail
detail
DMD DMD Evidence Evidence Toolkit
Continuing Continuing outline outline development
development
Continuing outline outline development
development
DMD PROs
Poster M M M on on findi Abstr Expa Expa Abstra Abstract DMD DMD Evidence Evidence Toolkit
Toolkit
Planned
work a a a a a a a a a a a a a a a to to to to a a a a a a a a a a a a a a a a Plann Plann qua qua Evolving Evolving Evolving synthesis synthesis synthesis of of of of of of all all all the the the the the the the the evidence evidence evidence to to to e e e e e e e e e e e e e e e e e e e e e e e e e e e e e e e e e gener
a a a a a a is is is Evolving Evolving synthesis synthesis of of of of all all the the the the the the evidence evidence to to articulate articulate articulate the the the the the the the the the benefits benefits benefits of of of of of of of SRP-9001 SRP-9001 for for for DMD DMD and srteusde
a a a a a to a a a a a - - articulate articulate the the the the benefits benefits of of of SRP-9001 SRP-9001 for for DMD DMD of on on on Slide Slide Slide Deck Deck Deck Poster M M Slide Deck Abstract Poster Poster Manuscript Planned
attri Health Utilities Index AnalysesPeer-re Multi-faceted study to characterize HRQoCLo ngres between HUI scores and and NSAA drivers of utilities and and possible correlation
2 Abstracts Posters - Strategic p p peer-revie without co eIrn dSeeveglompmenttation tation resMsaingustchreiptrisk of of attrition attrition and and and its its impact impact on on on on on of of attrition attrition and and and and its impact on on on raPcltianngemd odels odels by by understanding understanding how how by by understanding understanding how O ers
ers
Analysis Value Fr
decision-m rieCntoacmle se Progressiogenne thera sented
PRO Dossiers
p p p p p f f the evidence supporting eDviedtenrcmeinsuinpgpiofrttihnegtraditi CIdevelop 9001 PRO strategy and its temgyeansudriets aimrepalepmpreonptaritiaote MD pivotal trial l
l
l
l
l
l
Abstract Poster Manuscript l
l
Report
Planned
Presented
FDAMA Pr AMA Presentation
on eCveolmopmilaetinotn of the evidence supporting SRP-9001 PRO strategy and its implementation in DMD pivotal trial Final Report
lourekFWrahmitewPoaprkerWhitePaper
- misiitoanti-omnsakwehrse nliampiptalitieodntsowhen applied to to to to eratrheefratpailedsisaenadsersa reanfdatal diseases and Value Framework White Paper A synthesis of assessment tools utilized by decision-makers limitations when applied to to gene therapies
and and rare fatal diseases and and offering evidence-based solutions Societal Preferences
DsafondrDBMDandBMD
Natural History Evidence Reviews
earsinegd esvoilduetinocnes-based solutions fmtehnetdoefvcealrodpiommeynotpoaftchayradniodmyopathy andimportant
and Studies
aintopraytidenctlsinweitinh pBaMtiDenttoshweiltph BMD to to to to helpresearch a a a a a a a a a a a a a a a a a a a a a Rate offering ev litdaetitveermstiundeya es most otyrStwaLnRhteontfoptshroieocrdiietitvzyeinwloghpdeminsepnartsieorfiticazrindgiodmisyeoapsaethy and marercenhstpaoinrpadtitotrneysadtmecelinteoinptipoantisents with to to help Networ
Proactively preparatio efficacy of Qualitative study to to determine attributes most important
to to society when prioritizing disease research and treatment options
Work Productivity Model
sutrantediunngdoefrtshteandaitnugraolf the natural inform rat rat isorfedqiusieraesdetois sruepqpuoirretdutnomseutpport unmet from DMD Illustrate the the work productivity and and income loss
deescsoangeosmaicndeveacluoantioomnis luations
among patients with and and their careAgbivsetrsact a a a a a a a a a a a a a a a ecrt-aSnMdDPoMst2e0r1-9SMDM2019 Presented
to highlight the the the potential benefit of improving
tehdealth outcomes with effective therapies
Payer E Continuou barriers to ecrt-aInSdPOPoRsEteUr2-0I1SP9OR Abstract and Poster tePdoster submitted to congress
anet
script- Orphane
Manuscript - - al
tteOdntohojoldurnal
grrAeanstseaisocnocufarpnardtoehguorenwsdsteihorsentyaandndiffdinehgroowf theyndatiffuerarl
DMhiDstory of disease is is is required to to to support unmet Poster sub need messages and and economic evaluations
arct and and Poster Abstract and and Poster Poster - Abstract a a Presented
Manuscrip In develop Manuscrip In develop sement sement of SRP-9001 rs
rs
to to to reimbursement of of SRP-9001 SRP-9001 nted Presented
Abstract and Poster - Presented
Manuscript- Submitted to journal
ment
r r r Engagement
o o o o o o o gather gather insight insight into potential nuous effort to to to to gather gather insight insight into into potential potential Qualitative Abstract a a info progression and how they differ r ra-cWt - - WMS 2020 f oerc tocongress
Abstract and Poster ra-cItSaOnQdOPLo2s0te2r0- ISOQOL 2020 Presented
White Pap Continuous effort to to to gather insight into pIontdeenvtieallop
velopment
Abstract and Poster Poster - WMS 2020 Poster Poster submitted to congress
at-AwabnostaralkyctsMaisnedtPao-satenra- lIySOsiQsOL 2020 Presented
Network Meta-analysis
Proactively developing best practices in in in preparation to indirectly compare safety and efficacy of different DMD gene therapies
Abstract and Poster - MSG 2019
Presented
White Paper In development
target final version end of year
Manuscript On hold
Presented
ented
ented
uinscHreipatl-thValue in in Health velopment
d ta aincgtivbelsytdperavectilocpeisnignbest practices in in tin in rpeaMcrtalaytincouonsmctropipaitnr-edVsiraelfcuetelyinacnoHdmeaplatrhe safety and tcDaIncMydDoefvgedelnioffepemtrheennrtatDpMiesDgenetherapies
rM-ManSuGsc2r0ip19t-JClinNeurol stract and Poster - - - MSG 2019
seIntdeedvelopment ite Paper Payer Engagement
rget rget final final version version end of year
evelopment
target final final version version end end of of year
year
- barriers to to reimbursement of Manuscrip On hold
rch Analog Research FDAMA Continuous effort to stay current of shops shops and Tools
MA Workshops and and Tools
Tools
ogCoRnetsexatrucahlizing Budget to to stay current of of uoIums peffaocrt tofstGayecnuerreTnht eofrapies al
al
al
and and payment payment models ping financial and and payment payment models models developing financial and payment models y
y
to to Measure
ette Study
to to Measure
Measure
for gene therapies
in fin in the the US es ent Utilities te te te utlility utlility values from a a a a a a a Societal ate ate health state utlility utlility values values from from a a a a a a a a a a a e e e e e e e e e e e e e e e to to describeclinical describeclinical course FDAMA Workshops and Tools
inIntthernUaSl analysis that visually demonstrated the the the the the e e e e e e e e e e e e e e e e e e e e e therapies
therapies
in fin in in fin the the the the the US Vignett
financial impact of one-time gene therapies
therapies
therapies
vs currently reimbursed chronic therapies
therapies
(e g g Humira)
Estimate h societal pe and progre Abstract Patient Vignette Study
to Measure
Patient Utilities Econom Estimate health state utlility values from a a a a a a a a a a a ssofociretTalrpeearstpmecetinvet:to describeclinical course ietal Values for Treatment:
iacned Eprxopgrersismionewnith granular detail
anuscript Expanding
acDt MPoDsteEr vMidaenunsceripTtoolkit
ed qualitative Evolving synthesis of of of all the the the evidence to study of of of th th th th iscrete Choice Experiment
ngAsbfsrotrmaccto Pmopsltetre dManuscript
nding nding on findings from completed
quPalantinteadtive preference
litative work a a a a a a a a a a a a a a a a quantitative preference
preference
al
al
al
public public regarding regarding disease y
of the general public public regarding regarding disease disease arch priorities
butes and research priorities
priorities
anuscript a a a a Discrete Choice Experiment
Economic Assessment
Tool
Societal Values for Treatment:
Expanding
on findings from completed
qualitative work a a a a a a a a a quantitative preference
study of the general public regarding disease attributes and research priorities
Abstract Poster Manuscript Planned
- articulate the the the benefits of of Slide Deck Continuing outline development
for D
Abstract Planned
   11   12   13   14   15