pcori interim progress report - biostatistics
TRANSCRIPT
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
1UpdatedJuly7,2015
PCORI INTERIM PROGRESS REPORT Use continuation pages as needed. Limit 20 pages, not including the Certification page.
Updated on Tuesday, July 7, 2015
Date:07/31/2015
TitleofProject:Sensitivity Analysis Tools for Clinical Trials with Missing Data
TypeofProgressReport: 6-month 12-month 18-month 24-month 30-monthOther(specifyintheboxbelow)
PeriodCoveredbythisReport:02/02/2015to07/31/2015
PrincipalInvestigator&InstitutionUpdatedContactInformation:
PIFirstName: Daniel
PILastName: Scharfstein
PIEmail: [email protected]
PIOfficePhone: 410-955-2420
AOFirstName: Marcy
AOLastName: DeSantis
AOEmail: [email protected]
AOOfficePhone: 410-614-2635
InstitutionLegalName: JohnsHopkinsUniversity
Address(street,city,state,zipcode):
615NorthWolfeStreet
Baltimore,MD21205
Telephone: 410-955-3067
KeyPatientandOtherStakeholderPartnerContactInformation(uptothree):N/A
Name:
Telephone:
Email:
Name:
Telephone:
Email:
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
2UpdatedJuly7,2015
Name:
Telephone:
Email:
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
3UpdatedJuly7,2015
MILESTONES UPDATE ExpandonmilestonestatusintheAccomplishmentsandChallengessection.Addadditionalrowsifnecessary.
Recordeachmilestonelabel,name,description,andprojectedcompletiondate,asshowninAttachmentB(MilestoneSchedule)ofyourContract.AllmilestonesinAttachmentBshouldbelistedinColumnsA–D.ColumnE:Checkappropriateboxindicatingmilestonecompletionstatusduringreportingperiod.ColumnF:Selectactualdateofmilestonecompletion.ColumnG:Selectappropriatereasonfordelay/non-completionofprojectedmilestoneduringthespecifiedreportingperiod.Note:Retaininformationregardingmilestonecompletionfromallpreviouslysubmittedreports(e.g.,MilestoneUpdatessubmittedonthe18-monthreportshouldcontaininformationfromthe12-monthreport,andsoon).
ColumnA ColumnB ColumnC ColumnD ColumnE ColumnF ColumnGMilestoneLabel
(e.g.,B-1,etc.)
MilestoneName DescriptionProjectedCompletion
Date
Completed?(Yes/No)
DateCompleted
IfNotCompleted,ReasonforDelay
B-1 Website Expandregistrationonwebsitetoinclude
PCOresearchers
7/31/2014 Yes No 10/31/2014 Chooseanitem.
B-2 AdvisoryBoard ConveneMeeting 7/31/2014 Yes No 7/21/2014 Chooseanitem. C SubmitInterimProgress
ReportInterimProgress
Report7/31/2014 Yes No 7/31/2014 Chooseanitem.
D-1 Casestudies/trainingmaterials
CreatePCO-centeredcasestudyand
trainingmaterials
1/31/2015 Yes No 10/30/2015 Chooseanitem.
D-2 Shortcourses Facilitatetwoshortcourses
1/31/2015 Yes No 1/12/2015 Chooseanitem.
D-3 Adobeconnectsession Adobeconnectsessionwithusers
1/31/2015 Yes No 1/12/2015 Chooseanitem.
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
4UpdatedJuly7,2015
D-4 Manuscriptformonotonemissingdata
SubmitcasestudytoPCORfocusedjournal
1/31/2015 Yes No 10/30/2015 Chooseanitem.
E SubmitInterimProgressReport
InterimProgressReport
1/31/2015 Yes No 2/2/2015 Chooseanitem.
F AdvisoryBoard ConveneMeeting 7/31/2015 Yes No 11/31/2015 Chooseanitem. G SubmitInterimProgress
ReportInterimProgress
Report7/31/2015 Yes No 8/21/2015 Chooseanitem.
Clickheretoenteradate.
Yes No Clickheretoenteradate.
Chooseanitem.
Clickheretoenteradate.
Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
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Yes No Clickheretoenteradate.
Chooseanitem.
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
5
RECRUITMENT UPDATE
Projectsenrollingparticipantsinatrialmustcompletethissection.LeaveblankifnotapplicableRecordthetargetrecruitmentnumberforyourproject:_______________CompleteTable1toassistinrecruitmenttrackingoverthedurationoftheproject.Retaininformationregardingrecruitmentfromallpreviouslysubmittedreports.Addadditionalrowsasnecessary.ColumnA:recordthedateofeachinterimreport.ColumnB:recordthenumberofparticipantsscreenedforenrollmenttodate.ColumnC:recordthenumberofeligibleparticipantsofthosescreened.ColumnD:recordthetotalnumberofparticipantsenrolledinthestudytodate.ColumnE:recordthenumberofparticipantsprojectedtobeenrolledtodate.ColumnF:recordthetotalnumberofparticipantsretainedtodate.(Ifthenumberofparticipantsretainedislessthanthenumberenrolledinthestudy,pleaseexplainintheAccomplishmentsandChallengessection.)Table1Recruitment
ColumnA ColumnB ColumnC ColumnD ColumnE ColumnF
ReportDate
Screened
Participants
(N)
EligibleParticipants
ofthoseScreened(N)
Recruited
Participantsto
Date(N)
ParticipantsProjected
tobeRecruitedto
Date,PerProject
MilestoneSchedule
(N)
Retained
Participants
toDate(N)
Example6/15/2014
1000 800 650 750 650
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
6
RECRUITMENT UPDATE (Continued)
CompleteTable2bylistingtheRacial/EthnicandGenderbreakdownoftheparticipantsenrolledinyourstudytodate.Ensuretotalsarecalculatedandappropriatelyrecorded.Ifyouhavenotcollectedthesedata,pleaseexplainwhy.
Table2Racial/EthnicandGenderEnrollmentTable*
Race Male(N) Female(N) Total(N)
AmericanIndian/AlaskaNative
Asian
Black/AfricanAmerican
Hawaiian/PacificIslander
White
Multi-race
Ethnicity Male(N) Female(N) Total(N)
Hispanic(Latino/Latina)
Non-Hispanic
*Ifmoredetailedinformationisavailableregardingracial/ethnicsubgroupsfortheparticipantsinyourstudy,pleaseprovidethatinformationinaseparatetable.
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
7
ENGAGEMENT REPORT (Annual report only)
Descriptiveinformationonengagementofpatientsand/orotherstakeholdersshouldbereportedineachannualreport(i.e.,atyear1,year2,etc.butnotat6monthsor18months).Thisreportisintendedtocapturetheperspectiveoftheresearcher(s)onyourteam.Youwillbegiventheopportunitytonominatepatientand/orstakeholderpartnerstoprovideadditionalengagementinformationtoPCORI.Usethelinkbelowtocompletethisreport.YourUsernameisyourPCORIcontractnumber(noletters,dashes,orspaces).Provideyourengagementreporthere: https://live.datstathost.com/PCORI-Collector/Survey.ashx?Name=Engagement_Report_Login Whenyouhavecompletedthequestions,recordyourconfirmationcode:__________
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
8UpdatedJuly7,2015
ACCOMPLISHMENTS AND CHALLENGES Use continuation pages as needed. Limit three pages.
Discussanddocumentstudyprogressandallsignificantevents.Inparticular,pleasediscuss:
• Anysignificantchangefromthefundedproposal,includingchangesinthestudyprotocol,engagementplan,endpoints,samplesize,etc.Includereasonsforthesechanges.PleasenotethatyoushoulddiscusschangeswithPCORIprogramstaffpriortoimplementation.Nosignificantchange.
• Progressandaccomplishmentsachievedduringthereportingperiod,withreferencetoplanned
projectactivities,milestones,andplanningfordissemination.
Solvedmethodologicalissuesregardingconfidenceintervalconstructionandintermittent
missingdata.PresentedposterattheFDAORSISymposiumandgaveshortcourseatthe
SocietyofClinicalTrialsAnnualMeetinginArlington,VA.PostedanewRversionofSAMON.
CompletedmanuscriptaboutthemethodologyunderlyingSAMON,whichwillbesubmittedto
theJournaloftheAmericanStatisticalAssociationby8/31/2015.RecordedlectureforPCORIonmethodsforanalyzingstudieswithmissingdata.
• Progressonyourengagementplanduringthereportingperiod,relativetotheactivitiesoutlinedinyourapplication.
Weinteractedinformallywithsomemembersofouradvisoryboardregardingtechnical
issues.
• Challengeswithprojectprogress(e.g.,delaysinIRBapproval,delaysinrecruitmentofsites,participantretentionissues).Haveyouovercomethesechallenges?Ifyes,pleasedescribe.Ifnot,whatresourcesorguidancedoyouneed?
Thegreatestchallengewassolvingmethodologicalissuesregardingconfidenceinterval
constructionandintermittentmissingdata.Sofar,thesesolutionsappearadequate.
o Describeanybarriersthatyouareexpectingtooccurinyourprojectinthenextsix
monthsandwaysyouplantoovercomethem.AccesstoPCO-orienteddatasets.Identificationofvenuesforshortcourses.
Disseminationofmethodsandsoftware.Weplantoworkwithouradvisoryboardto
addresstheseissues.
• SummarizeanysignificantactionstakenbytheIRB,DSMBorotherregulatoryoversightbody
duringthereportingperiod.
Notapplicable.
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
9UpdatedJuly7,2015
• Challengeswithpatientandstakeholderengagement.Haveyouovercomethesechallenges?Ifyes,pleasedescribe.Ifnot,whatresourcesorguidancedoyouneed?
WecouldusetheassistanceofPCORItohelpdisseminateourmethodsandsoftware,for
example,throughthePCORIlistserv,socialmedia,newsfeeds,andmethods
meetings/seminars.
• PCORIisinterestedinmonitoringprojectadherencetoPCORI’sMethodologyStandards.YourProgramOfficerwillreviewcompliancewithMethodologyStandardsandworkwithyoutodevelopaplanforfollow-uponallimportantmethodologicalissues.Aspartofthisreport,youmayprovidedocumentationofadherencetoStandardsthatapplytoyourongoingresearch.Youmayincludeadditionaldocumentswiththisreport,forexample,afinalstudyprotocol.
NotApplicable
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
10UpdatedJuly7,2015
PROGRESS STATEMENT FOR PUBLIC USE Limit 250 words.
Describenotableprogresstodate,preliminaryresults,orengagement/stakeholderexperiencesusingnontechnicallanguagethatisreadyforpublicuse.(Note:ThisinformationmaybepubliclydisseminatedbyPCORI.)WepostedanewversionoftheRsoftwarepackageSAMONonthewww.missingdatamatters.org
website.
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
11UpdatedJuly7,2015
FINANCIAL STATUS UPDATE Limit two pages.
Describeanysignificantdeviationsincostsandbudget,howthosedeviationsaffectedthestudyprogress(e.g.,staffingandcostestimates),andanyanticipatedneedforbudgetmodifications.Significantdeviationsareconsideredanyadjustmentthatexceeds25%ofabudgetcategory.Therehavenotbeenanysignificantdeviationsincostsandbudget.
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
12UpdatedJuly7,2015
KEY PERSONNEL EFFORT UPDATE Use continuation pages as needed. Limit one page.
KeyPersonnelchangesinexcessof25%ofcontractedlevelofeffort,mustbereportedannually.
Reporttheindividual’srole,changeinpercentageeffort,andanexplanationforchanges.Ifyouhavemorethanfivechangestoreport,pleaseincludeadditionalinformationunder“ExplanationofChanges.”SendPCORIabiosketchforallnewkeypersonnelworkingontheproject.
XNochangesinkeypersonnel
Name(First,Last) TitleContractedPercentage
EffortActualPercentageEffort
% % % % % % % % % %
ExplanationofChanges: Noteanyproposedchangestokeypersonnelinthenext6months,ifapplicable,andprovideanexplanationforchanges,below.Addrowsasnecessary.Ifyouhavemorethanfiveproposedchangestoreport,pleaseincludeadditionalinformationunder“ExplanationofChanges.”SendPCORIabiosketchforallnewkeypersonnelworkingontheproject.
XNotApplicable
Name(First,Last) TitleContractedPercentage
Effort
ActualPercentage
Effort
% % % % % % % % % %
Explanationofchanges:
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
13UpdatedJuly7,2015
PUBLICATIONS UPDATE
REMINDER:Pleasemakesurethatallpublications/communication/mediapiecescontainthefollowingacknowledgementofPCORIfunding:“Researchreportedinthis[work,publication,article,report,presentation,etc.]was[partially]fundedthroughaPatient-CenteredOutcomesResearchInstitute(PCORI)Award(##-###-####).”
Pleasemakesureyourpublicationsthatpresentresearchfindings,conclusionsorothereditorialcontentalsoincludethisdisclaimer:“The[views,statements,opinions]inthis[work,publication,article,report]aresolelytheresponsibilityoftheauthorsanddonotnecessarilyrepresenttheviewsofthePatient-CenteredOutcomesResearchInstitute(PCORI),itsBoardofGovernorsorMethodologyCommittee.”
Inthetablesbelow,recordinformationregardingpublicationsandpresentations(scientificandnon-scientific)relatedtoyourPCORI-fundedresearchthatoccurredasofthereportingdate.Retaininformationsubmittedinpreviousreports.Publicationsand/orpresentationsbyanymemberoftheresearchteam,includingpatientandstakeholderpartners,shouldincludethose:
• Inpreparationtobesubmitted.• Thathavebeensubmittedtoapublication.• Thathavebeenacceptedtoapublication.• Thatarein-press.• Thathavebeenpublished.
a) PeerandNonPeer-ReviewedPublications(e.g.,scientificpublicationsandnon-scientificpublicationssuchas,organizationaljournals,
magazines,newsletters,newspapers,blogs,otherlaypress)
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
14UpdatedJuly7,2015
Author(s) Date Title
Type(e.g.,manuscript,abstract,poster,
newspaperormagazine
article,blog,other)
Nameofpublication
URL,ifapplicable
Status(e.g.,inpreparation,submitted,accepted,in
press,published)
PEERREVIEWED
Scharfstein,Rotnitzky,Abraham,McDermott,Chaisson,Geiter
7/26/2015
OntheAnalysisofTuberculosisStudieswithIntermittentMissingData
Manuscript
AnnalsofAppliedStatistics
www.missingdatamatters.org
InPress
Wang,Scharfstein,Colantuoni,Girard,Yan
6/25/2015
InferenceinRandomizedTrialswithDeathandMissingness
Manuscript
Biometrics www.missingdatamatters.org
UnderRevision
Scharfstein,McDermott,Diaz,Carone,Lunardon,Turkoz
8/31/2015
GlobalSensitivityAnalysisofRepeatedMeasuresStudieswithInformativeDrop-out:ASemi-
Manuscript
JournaloftheAmericanStatisticalAssociation
Submitted
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
15UpdatedJuly7,2015
ParametricApproach
Colantuoni,Scharfstein,Wang,Needham,Girard
8/21/2015
AccountingforMortalityandMissingDatawhenComparingOutcomesAcrossTreatmentGroupsinRandomizedTrials
Manuscript
AnnalsofInternalMedicine
InPreparation
Scharfstein,McDermott
8/21/2015
GlobalSensitivityAnalysisofClinicalTrialswithMissingPatientReportedOutcomes
Manuscript
ClinicalTrials InPreparation
NON-PEERREVIEWED
Kharazzi,Wang,Scharfstein
4/18/2014
ProspectiveHER-BasedClinicalTrials:TheChallengeofMissingData
Editorial JournalofGeneralInternalMedicine
www.missingdatamatters.org
Published
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
16UpdatedJuly7,2015
b) Presentations(e.g.,invitedtalk,localprovidermeeting,webinar,YouTubevideo)
Presenter(s) Date TitleType(e.g.,oral,poster,
YouTubevideo,webinar,other)
NameandLocation(e.g.,conference/meetingnameandlocation,
recordedwebinarwithURL,localmeetingname
andlocation)
Audience,ifknown(e.g.,researchers,patients,clinicians,policymakers,
students,communityorganizations)
Status(e.g.,inpreparation,submitted,accepted,completed)
PEERREVIEWED
NON-PEERREVIEWED
McDermott 8/3/2014 GlobalSensitivityAnalysisofRepeatedMeasuresStudieswithInformativeDropout:ASemi-ParametricApproach
Oral JointStatisticalMeetings,Boston,MA
Researchers Completed
Scharfstein 9/18/2014 GlobalSensitivityAnalysisofRepeatedMeasuresStudieswithInformativeDropout:ASemi-ParametricApproach
Oral AndreiYakovlevColloquium,UniversityofRochester,Rochester,NY
Researchers Completed
Wang 9/24/2014 InferenceinRandomizedTrialswithDeathandMissingness
Oral ASABiopharmaceuticalSectionFDA-IndustryWorkshop,Rockville,MD
Researchers,Practitioners
Completed
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
17UpdatedJuly7,2015
Scharfstein 12/8/2014 GlobalSensitivityAnalysisofRandomizedTrialswithMissingData:RecentAdvances
Oral ShortcourseatDemingConference,AtlanticCity,NJ
Researchers,Practitioners
Completed
Scharfstein,Wang,McDermott
1/12/2015 AnalysisofRandomizedTrialswithMissingData
Oral,AdobeConnect ShortcourseatJohnsHopkinsUniversity,Baltimore,MD
Researchers,Practitioners
Completed
Scharfstein 4/27/2015 GlobalSensitivityAnalysisofRandomizedTrialswithMissingData
Poster FDAORSISymposium Researchers,Practitioners,PolicyMakers
Completed
Scharfstein 5/17/2015 GlobalSensitivityAnalysisofRandomizedTrialswithMissingData
Oral ShortcourseatSocietyforClinicalTrials,Arlington,VA
Researchers,Practitioners
Completed
Scharfstein 7/31/15 AnalysisofProspectiveStudieswithMissingData
OnlineLecture JohnsHopkinsUniversity,Baltimore,MD
Researchers,Practitioners,PolicyMakers
UnderEditorialReview
AwardeeInstitution/Contract#:JohnsHopkinsUniversity/ME-1303-6016
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
18UpdatedJuly7,2015
PUBLICATIONS UPDATE (Continued)
c)PeerReviewRejectionNotificationsIfapplicable,provideinformationregardingscientificarticle(s)submittedforpublicationandrejected,duringthereportingperiod.
Author(s) DateRejected TitleType(e.g.,
manuscript,abstract,poster,other)
Nameofpublication URL,ifapplicable
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
19
ADDITIONAL DOCUMENTS All attachments should be combined with this document and submitted to PCORI as one PDF.
Additionally, please complete the “Other Publications” section below, if necessary.
Anydocumentsthatyoufeelarerelevantandnoteworthycanbeshared,suchas:• Copiesofdraftsofinstruments,datadictionaries,educationalmaterials,manuals,orother
projectdeliverables,ifnotalreadydelivered• Abstractsfrompresentationsmadetoprofessionalgroupsorassociations• Summariesofpreliminarydata• Bibliographies• SummariesfromDSMBmeetings• Finalstudyprotocol• Othercommunicationsefforts• Copiesofreportsfromanyconsultantsoradvisors,whereapplicable• Otherdocumentsormaterials,asappropriate
PrincipalInvestigator(Scharfstein,Daniel,Oscar)
20
CERTIFICATION
This document must be certified by the Principal Investigator and the designated Administrative Official (AO).
PrincipalInvestigator:
IcertifythatI,asthePrincipalInvestigator,havereviewedandapprovedthisdocument(andanyassociatedattachments,ifapplicable).PIFirstName:DanielPILastName:ScharfsteinDate:8/21/2015AdministrativeOfficial:
IcertifythatI,asthedesignatedAdministrativeOfficial,havereviewedandapprovedthisdocument(andanyassociatedattachments,ifapplicable).AOFirstName: AOLastName: Date: