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

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Presentation on theme: "ParentProjectMD.org Treatment Preferences and Risk Tolerance Study Pat Furlong, BSN, MS Holly Peay, MS CGC Vice President, Education and Outreach."— Presentation transcript:

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

2 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org Worry List: In the past 7 days, most/least worried… AMy child missing out on new treatments BMy child getting weaker CGetting the right care for my child over time DMy child feeling happy EMy child having good friends FMy child feeling like a burden on the family GMy child becoming independent from me over time HMy child not being able to express deep worries JBeing a good enough parent for my child KMe handling the emotional demands of Duchenne LManaging my uncertainty about my child’s future MHaving time for myself NFeeling isolated from other families PAffording care my child needs within the family budget QEffects of Duchenne on my closest relationships RThe wellbeing of my other children

8 ParentProjectMD.org Treatment Attributes LABELEXPERIMENT DESCRIPTION A1 Muscle functionStops the progression of weakness A2 Muscle functionSlows the progression of weakness A3 Muscle functionDoes not change progression of weakness B1 Lifespan5 year gain in expected lifespan B2 Lifespan2 year gain to expected lifespan B3 LifespanNo extra gain to expected lifespan C1 Drug knowledge2 years of post-approval drug information available C2 Drug knowledge1 year of post-approval drug information available C3 Drug knowledgeNo post-approval drug information available

9 ParentProjectMD.org Treatment Attributes Con’t LabelExperiment description D1 NauseaNo increased chance of nausea D2 NauseaCauses loss of appetite D3 NauseaCauses loss of appetite with occasional vomiting E1 BleedsNo increased risk of bleeds E2 BleedsIncreased risk of bleeding gums and increased bruising E3 BleedsIncreased risk of hemorrhagic stroke and lifelong disability F1 ArrhythmiaNo increased risk of heart arrhythmia F2 ArrhythmiaIncreased risk of harmless heart arrhythmia F3 ArrhythmiaIncreased risk of dangerous heart arrhythmia and sudden death

10 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 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 ParentProjectMD.org 6. What is your annual household income? Frequency%Valid % Less than $25, $25,000-$50, $50,000-$75, $75,000-$100, More than $100, Total System21.7 Total

17 ParentProjectMD.org 5. What is the highest level of education you have completed? Freq%Valid % High school or GED54.2 Some college but no degree Technical school Associate’s degree (2-year college degree) year college degree (e.g., BA, BS) Some graduate school but no degree75.9 Graduate or professional degree (e.g., MBA, MS, MD, PhD) Total System1.8 Total

18 ParentProjectMD.org 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

19 ParentProjectMD.org 4. Chose the option that best describes your child’s physical abilities today. Frequency% Is a baby, a toddler, or a very young child who is too young to walk far yet 54.2 Walks independently for long distances outdoors (more than ½ mile) Walks less than ½ mile, but more than short distances Walks independently outdoors for short distances (such as to the car) Walks outdoors with help from a person1.8 Walks independently indoors but needs a wheelchair for outdoors21.7 Walks indoors with help from a person and requires wheelchair outdoors 43.4 Uses wheelchair and can go indoors and outdoors Uses wheelchair but unable to go outdoors in some situations (such as cold weather) 32.5 Unable to control wheelchair without help32.5 Total

20 ParentProjectMD.org 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.

21 ParentProjectMD.org 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

22 ParentProjectMD.org 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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