Stakeholder Engagement: DuchenneConnect PPRN Holly L. Peay, PhD CGC RTI International
DuchenneConnect PPRN Engagement Identifying and addressing needs in the Duchenne and Becker muscular dystrophy communities
Identifying community needs Real-time engagement through PPMD and DCN forums Leadership and advisory committees Asynchronous focus groups Innovative approaches to needs prioritization
Community engagement activities
BWS Case 1 prioritization
Patient-focused drug development Addressing community needs using stakeholder engagement Case study 1
Extending the reach of testimony
Patient focused drug development engagement process Figure 1. Commu nity engagement process and outcomes for stated-preference attribute development Guiding Principles Stakeholders Committee Outputs Outcomes Impact Patients Guidance for entire Leadership Survey is ethical, Identify patient/ Incorporating patient perspectives into drug Caregivers project; conceptual committee acceptable and caregiver priorities development and regulatory review is worthwh ile Advocacy leadership survey design; initial [Scientific team] scientifically valid regarding therapuetic Researchers attributes/levels targets Patient and caregiver perceptions of meaningful benefits/risks may differ from researchers/clinicians Patients Inform eligibility Patient/caregiver community is actively engaged and Caregivers Stakeholder Survey is meaningful criteria; willling to participate Clinicians committee to the community refine attribute list Industry sponsor Study data belong to the patient/caregiver community Enhanced regulatory Cognitive All stakeholders deserve a voice in study development review for patient- interviews; Patients Patient-centered Review committee centered benefit-risk determine Regulators are receptive to preference data Caregivers benefit-risk data assessment acceptability; refine attributes ! Hollin, I. L., Young, C., Hanson, C., Bridges, J. F. P., & Peay, H. (2016). Developing a patient-centered benefit-risk survey: A community-engaged process. Value in Health, 19 (6), 751-757. doi: 10.1016/j.jval.2016.02.014 12
Engagement guiding principles Incorporating patient perspectives into drug development and regulatory review is worthwhile Patient and caregiver perceptions of meaningful benefits/risks may differ from researchers/clinicians Patient/caregiver community is actively engaged and willing to participate Regulators are receptive to preference data All stakeholders deserve a voice in study development Study data belong to the patient/caregiver community 13
Engagement process & outcomes 14
Discrete choice experiment at PPMD 2016 Annual Conference
Results If muscle benefit increased from small to medium improvement, the caregivers are willing to accept a 30.64% higher chance of developing fracture as a side effect of the drug. Figure 1. Maximal acceptable risk, stratified
Decision making about clinical trial participation Addressing community needs using stakeholder engagement Case study 2
The problem.... Clinical trial decision influence Influence Influence Mean* SD Mean* SD Benefit item Harm Item Scientists would learn important My child would be bothered by side effects 3.54 1.82 information about DBMD 6.09 1.47 …my child might miss out on another trial 3.26 1.98 …result in a better future for other children 5.91 1.51 with DBMD My child might not like being in the trial 3.00 1.70 …result in finding a drug that will work 5.88 1.51 …help me to feel I am doing everything I My child might not get any benefit from the 2.90 1.68 trial can to help my child 5.66 1.66 My child could be harmed during the trial 2.82 1.62 …result in better quality of life for child 5.59 1.66 My child might not get to stay on the drug 2.66 1.87 …make me feel like we are “doing our after the trial ends part” to advance science 5.33 1.93 My child could get the placebo 2.56 1.82 …cause my child to live longer 5.24 1.97 There would be too much travel for the trial 2.51 1.80 …cause my child’s muscles to get stronger 5.17 1.98
Contact Holly Peay, PhD Co-PI, DuchenneConnect www.DuchenneConnect.org RTI International hpeay@rti.org www.parentprojectmd.org Ann Martin, MS Co-PI, DuchenneConnect Parent Project Muscular Dystrophy alucas@parentprojectmd.org
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