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Treatment Preferences and Risk Tolerance Study

Treatment Preferences and Risk Tolerance Study . Pat Furlong, BSN, MS Holly Peay, MS CGC Vice President, Education and Outreach . Study Goals. Objective: explore how parents/guardians of individuals with DMD prioritize risk and benefit in the context of new therapies Specific Aims:

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Treatment Preferences and Risk Tolerance Study

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  1. Treatment Preferences and Risk Tolerance Study Pat Furlong, BSN, MS Holly Peay, MS CGC Vice President, Education and Outreach

  2. Study Goals • Objective: explore how parents/guardians of individuals with DMD prioritize risk and benefit in the context of new therapies • Specific Aims: • Describe risk tolerance, health-related QoL, and numeracy • Explore treatment preferences, risk tolerance and benefit priorities • Evaluate the effect of child’s disorder progression on treatment preferences • Explore Duchenne-related worries

  3. Methodology • Developed in consultation with health economist • Best/worst scaling type 2 • “BWS is a theory about how people make best and worst (most and least, smallest and largest, two extremes, etc.) choices from choice sets consisting of three or more ‘things’.” (Louviere and Flynn 2010) • Based on random utility theory- value respondent derives from an object compared with a comparator is proportional to how often he/she chooses it in preference to comparator

  4. Attribute Development: Worries • 5 parents contributed a broad list of DMD-related worry items • Researchers refined the items and grouped under domains • Final worry domains • child-focused (health, QoL, and social support) • external to the child (parent/guardian QoL, social support, family effects)

  5. Attribute Development: Treatments • Large pool of hypothetical attributes and levels identified in consultation with parents, providers, and biotech/pharma • Items reduced and refined during the stakeholder consultation • Identified plausible attributes that are sufficiently balanced to allow a successful experiment

  6. Refinement and Piloting • Draft instruments refined in consultation with social scientists with expertise in clinical research and rare disorder populations • Survey instrument piloted by 7 parents who provided extensive feedback on the items and instrument as a whole • Final survey developed and implemented online

  7. Worry List: In the past 7 days, most/least worried…

  8. Treatment Attributes

  9. Treatment Attributes Con’t

  10. Inclusion Criteria & Recruitment • Recruited from PPMD, DuchenneConnect Registry, and snowball recruiting • Parents or guardians of at least one living child with Duchenne muscular dystrophy, living in the United States, over 18 years of age, and able to complete an online survey in English • Study determined to be exempt by the Western Institutional Review Board

  11. Survey Components • Treatment experiment: 18 treatment scenarios • Worries experiment: 16 worries lists • Risk Taking Measure (Pearson et al.,1995): 6 items from the Jackson Personality Index • Numeracy (Fagerlin et al., 2007) 3 items adapted from Subjective Numeracy Scale • SF-12 Health-Related QoL • Child DMD status (mobility and self-care PROM) • Care/support items • Demographics

  12. Design • Detailed description of attributes and levels; example task • 18 treatment choice tasks generated from Youden design assessing the best and worst attribute • Each treatment scenario followed by acceptability question (“If this treatment were real, would you use it for your child?”) • 16 worry choice tasks assessing the most and least significant worries over the past 7 days

  13. Experiment Example Choose the best thing by clicking the circle under “best” and choose the worst thing by clicking the circle under “worst.” You have to choose a best thing and a worst thing to move on. Remember that a computer chose combinations to make the experiment work, and some of them seem bad. Even so, please pick the best and worst thing.

  14. Preliminary Analysis • Level utility scores (across all choice sets and respondents) • # of times attribute level chosen worst - # of times chosen best/# times attribute appears in experiment*# participants • Attribute importance scores • Max level mean - min level mean/total of all attribute max-min means • Multinomial analysis ongoing

  15. Preliminary Results • 119 parents completed entire survey • Mean participant age 43.7 (SD 7.7) • Mean affected child age 12.1 (SD 6.4) • 80 (67%) biological mothers, 34 (29%) biological fathers, 5 (4%) adoptive parents • 109 (92%) Caucasian • 107 (90%) married, 11 (9%) divorced/separated, and 1 (1%) widowed

  16. Affected Children • 110 (92%) have one affected child; 9 (8%) have two or more affected children • 101 (85%) have private insurance; 40 (34%) have a state/government program • 68 (58%) participated in clinical research and 40 (34%) participated in a clinical trial • 22 (19%) child has experienced a life-threatening emergency that caused parent to worry that the child would die

  17. Preliminary Conclusions Within the context our experiment: • Worries related to child’s illness progression and care accounted for the largest proportion of the variance in the worries attributes. • Stopping or slowing the progression of muscle weakness accounted for the largest proportion of the variation in treatment attributes. Our evidence suggests that the presence of side effects/risks could be compensated for by a treatment that stops progression to muscle function.

  18. Next Steps • Further analysis ongoing • Seeking input from FDA about acceptability and interest • Possible refinement and second survey • Focus groups/community input • “Tell Your Story” open-ended data collection and analysis ongoing

  19. Collaborators • John Bridges, PhD, Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health • Ilene Hollin, Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health • Sharon Hesterlee, PhD, PPMD • Hadar Sheffer, MPH, PPMD

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