DuchenneConnect PPRN Holly L. Peay, PhD CGC RTI International - - PowerPoint PPT Presentation

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

12 Figure 1. Commu nity engagement process and outcomes for stated-preference attribute development !

Guiding Principles Stakeholders Committee Outputs Outcomes Impact Patients Caregivers Advocacy leadership Researchers Leadership committee [Scientific team] Guidance for entire project; conceptual survey design; initial attributes/levels Survey is ethical, acceptable and scientifically valid Patients Caregivers Clinicians Industry sponsor Stakeholder committee Inform eligibility criteria; refine attribute list Survey is meaningful to the community Patients Caregivers Review committee Cognitive interviews; determine acceptability; refine attributes Patient-centered benefit-risk data Incorporating patient perspectives into drug development and regulatory review is worthwh ile Patient and caregiver perceptions of meaningful benefits/risks may differ from researchers/clinicians Patient/caregiver community is actively engaged and willling to participate Study data belong to the patient/caregiver community All stakeholders deserve a voice in study development Regulators are receptive to preference data Identify patient/ caregiver priorities regarding therapuetic targets Enhanced regulatory review for patient- centered benefit-risk assessment

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

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

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Engagement process & outcomes

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Discrete choice experiment at PPMD 2016 Annual Conference

Results

Figure 1. Maximal acceptable risk, stratified

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.

Decision making about clinical trial participation

Addressing community needs using stakeholder engagement Case study 2

The problem.... Clinical trial decision influence

Influence Benefit item Mean* SD Scientists would learn important information about DBMD 6.09 1.47 …result in a better future for other children with DBMD 5.91 1.51 …result in finding a drug that will work 5.88 1.51 …help me to feel I am doing everything I can to help my child 5.66 1.66 …result in better quality of life for child 5.59 1.66 …make me feel like we are “doing our part” to advance science 5.33 1.93 …cause my child to live longer 5.24 1.97 …cause my child’s muscles to get stronger 5.17 1.98 Influence Harm Item Mean* SD My child would be bothered by side effects 3.54 1.82 …my child might miss out on another trial 3.26 1.98 My child might not like being in the trial 3.00 1.70 My child might not get any benefit from the trial 2.90 1.68 My child could be harmed during the trial 2.82 1.62 My child might not get to stay on the drug after the trial ends 2.66 1.87 My child could get the placebo 2.56 1.82 There would be too much travel for the trial 2.51 1.80

Contact

Holly Peay, PhD Co-PI, DuchenneConnect RTI International hpeay@rti.org Ann Martin, MS Co-PI, DuchenneConnect Parent Project Muscular Dystrophy alucas@parentprojectmd.org www.DuchenneConnect.org www.parentprojectmd.org