Page 14 - Demo

Appendix
Emphasizing Emphasizing the the Emphasizing Emphasizing the the Poste Poste Poste Accept
Unmet Unmet Need Need Unmet Unmet Need Need FD
Nns
FDAM
In de
Wea
Bridg real-w
Abstract and Poster - ISPOR Patient-centered Patient-centered Patient-centered Patient-centered techniques techniques determining determining their and techniques techniques determining determining their and Endpoints Endpoints Endpoints Endpoints D
Decision Tool
Wearables Pilot Study
offering alternative alternative solutions offering offering alternative alternative solutions DM
An
es th th De DMD Prevalence Tool
Inte eliable
n n regions
Internal White Papers
Quantifying Quantifying the the Impact Quantifying Quantifying the the Impact Ab Po Ab Ac Re
In int Coll
clini clini clini patients patients Abstract Poster Manuscript e e on Caregivers Caregivers on on Caregivers Caregivers An
evidence-based model to to provide r r r Delphi Paensetiml ates of DMD cases – critical tool i i i ti i ti i i i Utility analysi limitati of SRP
Plan Plan Abstra Abstra Abstr AUbstitlritayctv aPlPuoleassntenarer edMkaeny udsrcivreiprst in Plananleysdis Published estimates mea
Abs Plan Prev Prev e e e e e e e e e e e e Quan comm
Ma Ma Ma urpopsaetide eviden of SRP-90In01d edvemloopnmsternates need In In development
Env
Critical Appraisal Tool
Patie Patient Utilities and Health and humanistic humanistic impact impact of of DMD on and ng ng ng th Abstra In In In In ed Accept
Abstra Accept
Critic Review
Accepted
ntere Ab Proactively Proactively preparing preparing for ICER evaluation of Proactively Proactively preparing preparing for ICER evaluation evaluation ble to Conctivity comm
comm
commonly used tools to to to to assess
of of of of of of clinical
and and market market and and Multi-phase Multi-phase study study to to assess
the and Multi-phase Multi-phase study study to to assess
the and humanistic humanistic impact impact of of DMD on and caregivers Collaboration with Roche to seek consensus on on on on caErvegidiverns ce ce ce Synthesis
Synthesis
Synthesis
Evide Evide ates in limitationFsDtAhMat AmPayrensegnattiavtieolyn FDAMA Presentation
evidence Abstract and Poster - SMDM
Accepted
Replication of of RAebpstlriaccattianodnPosfteErx-oISnPdOyRsE-U512019
ICER Model
ICAEccRepMtedofdoreplublication SRP-9001 by by understanding understanding evaluation evaluation SRP-9001 SRP-9001 by by understanding understanding evaluation evaluation Review
of strengths and weaknesses of lphi lphi Panel that la la ust evidence Collaboration with with to seek consensus on on on on aboration aboration with with RAobcshtreact andkPcosntseern n2020 clinically meaningful outcomes in in in in in patients cally cally meaningfuPlosut ms iintteDdMtDo pcongrnetss measured by by the the sured sured by by the the NSAA Abstract andAPbosttrearct - ISPOR 2020 tract tract and and PosteArccepted Planned
ned
Manuscript Report
nuscript nuscript nuscript In development
(manuscript being rep rep Planned
ned
ned
into report)
e FDAMA Presentation
In development
patients nts nts Manuscript Abstract andPlPanonsteedr - - act act and and Poster Poster - - AAN 20 clinicall ingful outcomes in in in in DMD Poster Poster submitted submitted to to congress
congress
r r r r r r submitted submitted to to congress
congress
FDAMA Presentation
Presentation
A A A Presentation
Presentation
In development
velopment
velopment
Above the Claims Analyses Patient Utilities Utilities and and Health Health States States nt nt nt nt Utilities Utilities and and Health Health States Evidence Synthesis
Synthesis
nce nce Synthesis
Synthesis
Utility values values are are key key drivers drivers in in in cost-effectiveness cost-effectiveness values values are are key key drivers drivers in in in cost-effectiveness cost-effectiveness Prevalenmceaosufr einNStAhAeUS alence of DMD US QuantifyingAthbestprarecvtalnendcPeoasntedrattritionratesin tifying tifying the prevalence and and attrition attrition rate commerciallPylanndeMd edicaid-insured edicaid-insured ercially and and Medicaid-insured DMD analysis Published Published estimates show significant
s s s s s s s s s s s s s s s s s s s s s s s s s Published Published ePsrtiemvaatelsesnhcoew osigfnDifiMcanDt in in in the US limitations that may negatively impact impact evaluation evaluation ons ons egatively egatively impact impact evaluation evaluation of daenmtifoyinsgtrtahtespnreveadlefnocrenaenwd attrition r r r r r r r r r - -900 e e e e e e e e e e e e e e e e e e trates need for new evidence commercially and Medicaid-insured DMD ce patients Abstract Abstract and and and Poster Poster - - ct ct ct and and and Poster Poster - SMDM
ed Accepted
Abstract Abstract and and and Poster Poster Poster Poster - - AAN 2020 Poster Poster Poster Poster subm ss Abstract and and Poster Poster Poster - - ct ct and and Poster Poster Poster - - ISPOR E o ed In developm Accepted
FDAMA Presentation
Abstract and and Poster Poster - - ct ct and and Poster Poster - - ISPOR E Accepted
for for publication publication ed ed for for publication publication VaVluaelu-der-idvreivnen DeDceiscioisnio-mn-amkiankging
DiffDeiffrenretinatitiantigng 0-
Innovative Innovative Innovative Innovative Reimbursement Reimbursement Reimbursement Reimbursement Solutions Solutions Solutions Solutions Co Abstract and Poster - AMCP En ConsistentClyornespisotretinntglytrhepsotratitunsgothf acce en makin
Los
A lit Pres Ma eviden Abstra On hol
White In deve evidence for rare diseases MonthlyMRoenptohrlytsReports On hold
De re ch Ex s s s s Long-Ltoernmg-tNeartmurNalaHtuisrtaolrHyistory regularly to to to to to better understand health system Leveraging Leveraging long-term long-term population population data data from from market accmesasrkdetvaeclocpemssednetvsetlopinmteerntasl and RWochietecoPlaleRpaoegcruhescolleagues
Differentiating
PlannedPlanned ManuscMriapntuscriptPresented
Planned
Discrete Event Simulation
age
and varaianbdilivtiaersiaabcilriotisesspaactiroensstspatients
A literature review to describe the variability in AbstracAtbasntdraPcotsatnedargPeoastelorssofambulationbygenotype AbstractandPoster
Abs Abstract and Poster - ISPOR EU 2019
Manuscript - Muscle and Nerve
Submitted to journal
Emai
Ensurin develo activity
Email Alerts
Ex sta sta PRO PRO da Ab Ab e e e e e e e toEnhsiguhrilnigghtinttheernpaoltceonltilealgbuenseafirteoafleimrtperdotvoinngew
to highlight the the potential Pla D
Manuscript Manuscript - - Value Value in Health Manuscript Manuscript - - Value Value in Health Sub ement hdeaelvtehlopumtceonmtsesrew: iSthRPe-ff9e0c0ti1vme Leveraging Leveraging long-term long-term population population data data from m In development
Work PrWodourkctiPvriotyduMctiodvietly to to address lack lack of of rigorous rigorous Mani ess
ess
ess
ess
lack lack of of rigorous rigorous rous long-terlomngst-utedrimessttoudeiesscrtoiobfedAeRsWmcrtibraeujeRlcaWttiortoireansjebctyorGiesenotype ories
PlannedPlanned among patiaemnotsnwg ipthatiDeMntDs wanitdhtDheMirDcarnedgitvheerisr Illustrate thIleluwstorarkteptrhoeduwcotirvkitpyraondducinticvoitmyeanlodssincome activity
health outcomes with AbstractaAndbsPtorasctetra-nIdSPOosRte2r02-0ISPOR2020 Poster subPmoistttedr stuobcmonittgreedssto 38
CONFIDENTIAL
Effective Effective Effective Effective An
An
SLR SLR to to validate validate commonly commonly cited cited birth An
SLR SLR to to validate validate commonly commonly cited cited h In development
development
final final version version target target end end of of year
year
Contextualizing Contextualizing Budget Contextualizing Contextualizing Budget Communications Communications Communications Communications reports
Plan Planned
Planned
Planned
Planned
Planned
Planned
Itema Itema ItemandcpusryrcehnotmlyerteriimcbanuarslyesdiscohfrothneicPtehdesrQapLies(e g g inc rts Abstract Abstract Abstract and and Poster Abstract Abstract Abstract and and Poster Poster Report
Report
Report
Report
Asse in patie Abstra Presen Internal Internal analysis analysis that that visually demonstrated Internal analysis analysis that that visually demonstrated In In Development Development In Development Development Manuscript Manuscript Manuscript Manuscript financial financial impact impact of of of one-time one-time gene therapies
Prevalence Tool
Tool
valence valence Tool
Tool
An
evidence-based model to provide reliable eevisdtiemncaete-bsaosfe dcealsteos p–rocrviitidcearletloiaobl lien regions
regions
timates of DMD s s s s s s s s s s s – critical tool in regions
regions
that lack lack robust robust evidence evidence at at at lack lack robust robust evidence evidence Abstract and and Poster Poster Poster - stract stract and and Poster Poster Poster - - AAN 0 Poster Poster Poster submitted submitted to to congress
congress
ster ster ster submitted submitted to to congress
congress
Abstract - stract stract - - ISPO 0 Accepted
cepted
cepted
Report
port In development
development
(manuscript (manuscript being being repurposed repurposed development
development
(manuscript (manuscript being being repurposed repurposed into report)
report)
o o o o report)
report)
FDAMA Presentation
Presentation
AMA AMA Presentation
Presentation
Emphasizi Unmet Ne In development
development
development
development
ctive Communication Pa Patient & Caregiver Dy
Patient-Pleavtielnsti-mleuvlealtisoimn tuoladtieotnertmo idnetbeurmrdienne obfurden of of DMD tential tential Dcoysta-edffeInctitvernvesies owf
treatm treatm and and potential cost-effectiveness of Asse Reim den of Assessment
of New Stud the treatments Study
to to capture parent and and child perception of of of the the the impact of DMD on on daily living to to further
Reimbursement Models Abs Abstract Poster Poster Poster Manuscript Impact Impact of of Gene Gene Therapies
Impact of of Gene Gene Therapies
Planned
Planned
FDAMAPresentation Planned
Planned
Co To a a mar pric with suffi cap financial impact impact of of of one-time one-time gene therapies
currently reimbursed chronic therapies
g FDAMA FDAMA Presentation
Presentation
Humira)
in patientHsuwmitihraD)MD
In In Development Development In Development Development Abstract and Poster - MSG 2019
Presented
Internal White White Papers
Papers
rnal rnal White White Papers
Papers
Delphi Panel Discrete Discrete Event Event Simulation
Simulation
Discrete Discrete Event Event Simulation
Simulation
On On hold
hold
On On hold
hold
idence idence idence idence Evidence Evidence Reviews
Reviews
Incidence Evidence Evidence Reviews
Reviews
vali vali vali vali vali validate validate validate validate the the the the the the the the PROMIS
PROMIS
PROMIS
item item banks banks banks vieWwhsite PWapheitre Paper In In In development
development
development
final final final version version version target target target end end end of of of of of year
year
year
of of of of of year
year
year
Cost of Innovation Study
To assess
all of of the costs of of bringing a a a a a a a drug to to capital underg
undergoing review and integration
Assessment
of of of Existing Abstra Abstra Abstra Abstract Abstract - - - ISPOR ISPOR 2020 a a a a market manufacturing/production to to set a a a a a a a a a a a a a a a Accept
Accept
Accept
Accepted
Accepted
e e e e e e e price such that a a a a a a a a a a a a a a a a a manufacturer is made whole o to to with a a a a a a a a a a a a a a a a a a a reasonable profit and to to ensure
a a a a a a a a a a a a a a a a a a a Manus Manus Manus Manuscript Manuscript - - J J Neuromusc Disord o of sufficient rate o of return to to attract investmeKnOtL fe fe fe KOL KOL feedback feedback received received received on on on on manuscript manuscript M M M ecision ecision Tool
Tool
Above the the Claims Claims Analyses Analyses ove ove the the Claims Claims Analyses Analyses Novel GMAX Communicatio Novel Communications ovel ovel GM mmunications mmunications Decision Tool
Tool
Patient-ce Endpoints d challenges and and opportunities
Wearables Pilot Pilot Study
Study
rables rables Pilot Pilot Study
Study
Bridging the the gap gap from from clinical
clinical
trial trial wearable wearable to to ing ing the the gap gap from from clinical
clinical
trial trial wearable wearable to real-world dEevnicveisronmental Scanning orld orld devices
Environmental Scanning Scanning ironmental ironmental Scanning Scanning tinCuoonutisnmuounsitomroinngitofrcinogmopfectiotmorpaectititvoirtyactivity neawndinfeowrminfornmatiot nmtahyaitmmpayctimpaarkcet tmarket ssacocfeSsRsPo-9f C sortium and Expert
Continuous monitoring of competitor a a a a a a and new information that may impact access of SRP-9001 Critical Appraisal Appraisal Tool
Tool
Assessment
Assessment
al
al
al
al
al
al
Appraisal Appraisal Tool
Tool
Assessment
Assessment
Bridging the gap from clinical
trial weara real-world devices
Review
of of of of of strengths strengths and and weaknesses weaknesses of of of of of of of of strengths strengths and and weaknesses weaknesses of of of onclyomusmedontolyolussteodatsosoelsstqouaslisteysosfqculianlictaylof clinical
ceefvoidr eranrceedfiosrearaseres diseases - nCsonrtisourmtiuamndaEnxdpEexrtpert
gEanggeamgenmtent Value-driv
veDloepvmeloenptmoefnat tohfinaktthaink thanatk mtheaettmsDeetscision- gurlaerglyultaorlbyetttoebreuttnedreursntdaenrdsthaenadlthesaylsthemsystem allcehnaglelesnagnedsoapnpdoortpupnoitirteusnities
ctAabnsdtrPaocsttaenrd-APMosCtePrN-
d d d On hold
Engagement
g g g g Development of a a a a a a a think tank that meet
PaWpehrite PapeLrong-term Natural History loInpmdevnetlopment
Leveraging long-term population data fro Manitoba Canada to to to address lack of rigo long-term studies to to to describe RW traject and variabilities across patients to sLofsAsmofbAumlatibounlatibyonGebnyoGtyepneotype
erAatluitrerraetvuirewretoviedwesctoribdestchreibveartihaebivliatyriianbility in atalgoessaot floasms bouf lamtiobnublaytigoennboytygpenotype
traAcbtsatnradcPtoasntderP-oIsStPeOrR-
enPtredsented E t t t t t t t t t t t R skforce
nuMscarinputs-cMriputsc-l E l
l
l
l
l
l
AEmleratisl AlMeratnsuscript Planned
pEexrpt ePrRtOPRTaOskTfaosrkcfeorce
SRP-9001 Innovativ Reimburs Solutions peErtxpeenrgtaegnegmaegnetmtoendtetoerdmeitneermbeinset best tistitactialstimceatlhmodetohlogdioeslofogrieasnfoarlyazninaglyPzRinOg PRO tadinatcaliiniccallintircialstrials
stract stract Poster Poster Manuscript Manuscript Abstract Poster Poster Manuscript Manuscript nned
nned
Planned
gEintseurninagl cinotllerangaulecsoallreeaaglueersteadretoanleerwted to to new pmdevnetslorpem: SeRnPt-s9r0e0:1SmRPa-r9k0e0t 1acmceasrsket access activity
n miSttuebdmtiottjeodurtnoajournal
to determine best statistical methodologiesfor analyzing
data in in clinical
trials Abstract Poster Manuscript Planned
tient tient & & Caregiver Caregiver Patient & & Caregiver Caregiver M id ad ad Interview Interview Dyad Interview Interview y
y
y
y
to to to capture capture parent parent and and child child perception perception of of Study
to to to to capture capture parent parent and and child child perception perception of of of of impact impact of of DMD ily living to to to to further
the the the the impact impact of of of of oIndcaiidlyelinvicneg Etovfiudrethnecr e e e e e e e e e e e e e e e e e e e e e Re
date date the the the the PROMIS
PROMIS
banks validate validate the the the PROMIS
PROMIS
item banks banks s Report
An
SLR to validate commonly cited birt tract tract Poster Manuscript Abstract Positnecri dMenacneursecproiprts
ned
Planned
In Development FDAMA Presentation
In Development a Patient-level simulation to determine bur ssment
ssment
ofDNMeDwand potential cost-effectiveness of of of Assessment
of of New bursementrteaMtmoedntesls Reimbursement Models White Paper In development
final version target end Abstract and Poster ssment
ssment
ofMEaxniussticrnipgt DMD P P P Assessment
of of of Existing PROs
st st of of Innovation Innovation Study
Study
Cost of of of of Innovation Innovation Study
Study
Planned
nd nd psychometric psychometric analysis analysis of of of the the PedsQL Item and psychometric psychometric analysis analysis of of the the nts nts with with DMD in patients with with ct ct and and Poster Poster - - MSG 20 Abstract and and Poster Poster 1 - ted Presented
ssess
ssess
all all of of of of of the the costs costs of of of of of bringing bringing a a a a a a a a a a drug drug to to To assess
all all of of of of the the costs costs of of of of bringing bringing a a a a a a a a a a a a drug drug to to to to ket ket manufacturing/production manufacturing/production to to to set a a a a a a a a a a a a a a a a a market manufacturing/production manufacturing/production to to to set set a a a a a a a a a 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 such such that that a a a a a a a a a a a a a a a a a a a a a manufacturer manufacturer is is made made whole price such such that that a a a a a a a a a a a a a a a a a a a a a a a a manufacturer manufacturer is is made made whole whole a a a a a a a a a a a a a a a a a a a a a reasonable reasonable profit profit and and to to to ensure
ensure
a a a a a a a a a a a a a a a a a a a a a with a a a a a a a a a a a a a a a a a a a a a reasonable reasonable profit profit and and to to to to ensure
ensure
a a a a a a a a a a a a a a a a a a a a a cient cient rate rate o o of of return return to to to to attract attract investment
investment
sufficient rate rate o of of return return to to to attract attract investment
investment
ital capital ct ct - ISPOR 20 Abstract - - ed Accepted
cript cript - J Neuromu Manuscript - - op edback edback received KOL feedback received received on on on manuscript oing oing review review and and integration
integration
undergoing review review and and integration
integration