Patient-CenteredApproachestoResearchPathways

SharronClose,PhD,MS,CPNP-PC,FAANEmoryUniversity

SchoolofNursingandDepartmentofHumanGeneticssharron.m.close@emory.edu

WhyCareaboutPatient-CenteredResearch?

• Outcomesthatpatientscanusesoonerratherthanlater• Comparativeeffectivenessofinterventionsmostlikelytohelppatients

• Researchthatinformsclinicaldecision-makinginreasonabletimeframes.

• Patientsandfamiliesoftenoffersolutions(interventions)thatcanbestudied

LessonsLearned:ThePCORIPerspective

• ThoughtIunderstoodgoingin:

• WhatIlearnedintheprocessofwritingagrant

• WhatIlearnedaboutpatient-centerednessintheprocess

Inputs Outputs Outcomes/ImpactThe Problem

• Gaps in Knowledge• Gaps in Research• Lack of Treatment or

Interventions that are Useful to Patients or Families

• Inadequate Communication with Clinicians & Scientists

• Powerlessness to Influence Symptom Management

• Scientists study problems that have limited interest or utility to patients and families

The Investment

• AXYS• Emory University• PCORI• Staff• Volunteers• Time• Money• Research• Technology

ActivitiesHow & What

We do

• Brainstorm Mtg. AXYS/Emory

• Stakeholder Selection

• Online Stakeholder Planning Mtg

• Family Conference 2015

• Scientific Conference 2016

ParticipantsWho We Reach

• Patients• Families• Clinicians• Scientists• Payers• Pharma• Clinical

Administration• AXYS• Emory

University• PCORI

Short Term Results

• AXYS• Emory

University• PCORI• Staff• Volunteers• Time• Money• Research• Technology

Medium TermResults

• AXYS• Emory

University• PCORI• Staff• Volunteers• Time• Money• Research• Technology

Long Term Results

• AXYS• Emory

University• PCORI• Staff• Volunteers• Time• Money• Research• Technology

Assumptions:FamiliesInformingScienceScienceInformingFamilies

ExternalFactors:Time,Location,IndividualCommitment,Conflicts,UnexpectedEvents

BidirectionalTranslationEngagementProject

MonthlyEngagementProcess

• PImeteverymonthwithgeneticsignaturerepresentatives(theywishedtobecalled”ambassadors”toconductastepwiseexplorationofpersonalandclinicalneeds

• Systematiccollaborationensuedtoward“Bidirectionality”ofcommunication

• Goal-drivenplansinitiatedtoinitiatemeaningfulcommunicationbetweenpatients,families,cliniciansandscientitsts

• Buildingtowardtheultimategoalofchangingtheway“science”considersX&Yparticipants.

Science&FamilyAmbassadorEngagement

CallSeptember3,2015

SharronClose,PhD,MS,CPNP-PC

Science&FamilyAmbassadorEngagement

CallOctober5,2015

SharronClose,PhD,MS,CPNP-PCKristyMartyn,PhD,CPNP-PCFAAN

Science&FamilyAmbassadorEngagement

CallDecember10,2015

SharronClose,PhD,MS,CPNP-PCKristyMartyn,PhD,CPNP-PCFAAN

JimMoore,BS

Science&FamilyAmbassadorEngagement

CallApril,2016

SharronClose,PhD,MS,CPNP-PCKristyMartyn,PhD,CPNP-PCFAAN

JimMoore,BS

Science&FamilyAmbassadorEngagement

CallJune28,2016

SharronClose,PhD,MS,CPNP-PCKristyMartyn,PhD,CPNP-PCFAAN

JimMoore,BS

ArrivingatOutcomesYear1

• 2FocusGroups:Patients,FamilyMembers,CliniciansandScientists(Conference)

• UsingQualitativeMethodology:InterpretiveDescription• Results

• MindfulnessofPerson• ClinicalConfusion• YouCan’tStudyThat• Invisibility• SystemsLetdown

FramingtheStructureofFutureResearch

Over24monthsofEngagement

• ProbingclinicalandallX&YChromosome-relatedneeds• Teachingpatientsandfamiliesabouthowresearchisdone• TakingtheoutcomesfromYear2andturningthemintoresearchquestionsthatmattertothem

• Plantore-engagepatients,families,cliniciansandscientists(Conference)toform

• Newresearchquestions• Determinebestmethodologies• Formnewcollaborations

FramingthePATHWAYofFutureResearch

Productivity-DrivenOutcomesYear2

• ResearchProjectOutcomes(Emory)• TrisomyX(HealthandReproductiveConcernsofWomenwithTriX)• XXYY(VoicesofPatientsandCaregiverswithXXYY)• NIPTStudy(AccessibleGeneticCounselingwithNIPT)• Managementofpubertyinadolescents

• Autism• X&YVariations

• HealthcareTransitions• Autism• X&YVariations

UnexpectedDividendsYear2

• CreationofSoutheastRegionalSupportGroup• AttentionofEmoryforeXtraordinarYClinic• StatelegislativeAwareness• DocumentaryFilm

PCORIBlogPost:http://bit.ly/2sklmOV

Patient-DerivedResearchQuestions

“Iwanttoknowwhattodowhenhecomeshomeat3o’clock”• What’sbehindthisstatement?• Executivefunctionandbehavior• Formingaresearchquestion:

• Inboyswith47XXYdoesasystematicprogramwithinatechnology-basedcompanionimproveexecutivefunctiontasksandundesirablebehaviorcomparedtoboyswith47XXYnotreceivingtheintervention?

• Homework,same-daytaskcompletion• Advancedreadinessfornextday• Self-efficacy• Self-esteem• Riskfordepression• QOLchildandparent

InitiatingCollaborations

•KnownCenters&PeoplewithExpertise•AcademicSources

• Universities• Clinics• NIH/PCORI

• SpecificAreasofSpecialty•CreationofNetworks•Co-PI’ing andsubcontractstoco-promoteproductivity

ThinkingaboutComparisonsAcrossGroups

• Severaldiagnosticgroupssharingdisabilityconcerns• X&YVariationswithAutism,FragileX,othergroups• X&YVariationsacrossnationalandinternationalgeographiclocations

Needed:TransparencyofEffortandCo-potentiationofresearch

• Whowantstoplayinthesandbox?• Differentapproachtooutcomes• Patient-centerednessfocusesonuseableoutcomes• Finessingthecurrentfundingstructurestofeaturepatientoutcomesandnotacademiccentersoracademicians

• ReconcilingIMPACToverCLAIMEDOWNERSHIP;Differentandshiftingapproach.

Questions?