Risk Tolerance Study Pat Furlong, BSN, MS Holly Peay, MS CGC Vice - - PowerPoint PPT Presentation

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Treatment Preferences and Risk Tolerance Study

Pat Furlong, BSN, MS Holly Peay, MS CGC Vice President, Education and Outreach

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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

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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

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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)

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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

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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
• nline

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

A Child focused My child missing out on new treatments B Child focused My child getting weaker C Child focused Getting the right care for my child over time D Child focused My child feeling happy E Child focused My child having good friends F Child focused My child feeling like a burden on the family G Child focused My child becoming independent from me over time H Child focused My child not being able to express deep worries J External focused Being a good enough parent for my child K External focused Me handling the emotional demands of Duchenne L External focused Managing my uncertainty about my child’s future M External focused Having time for myself N External focused Feeling isolated from other families P External focused Affording care my child needs within the family budget Q External focused Effects of Duchenne on my closest relationships R External focused The wellbeing of my other children

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Treatment Attributes

LABEL EXPERIMENT DESCRIPTION A1 Muscle function Stops the progression of weakness A2 Muscle function Slows the progression of weakness A3 Muscle function Does not change progression of weakness B1 Lifespan 5 year gain in expected lifespan B2 Lifespan 2 year gain to expected lifespan B3 Lifespan No extra gain to expected lifespan C1 Drug knowledge 2 years of post-approval drug information available C2 Drug knowledge 1 year of post-approval drug information available C3 Drug knowledge No post-approval drug information available

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Treatment Attributes Con’t

Label Experiment description D1 Nausea No increased chance of nausea D2 Nausea Causes loss of appetite D3 Nausea Causes loss of appetite with occasional vomiting E1 Bleeds No increased risk of bleeds E2 Bleeds Increased risk of bleeding gums and increased bruising E3 Bleeds Increased risk of hemorrhagic stroke and lifelong disability F1 Arrhythmia No increased risk of heart arrhythmia F2 Arrhythmia Increased risk of harmless heart arrhythmia F3 Arrhythmia Increased risk of dangerous heart arrhythmia and sudden death

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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

• f age, and able to complete an online

survey in English

• Study determined to be exempt by the

Western Institutional Review Board

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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

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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

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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.

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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

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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

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• 6. What is your annual household income?

Frequency % Valid % Less than $25,000 7 5.9 6.0 $25,000-$50,000 10 8.4 8.5 $50,000-$75,000 22 18.5 18.8 $75,000-$100,000 22 18.5 18.8 More than $100,000 56 47.1 47.9 Total 117 98.3 100.0 System 2 1.7 Total 119 100.0

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• 5. What is the highest level of education you have completed?

Freq % Valid % High school or GED 5 4.2 4.2 Some college but no degree 17 14.3 14.4 Technical school 6 5.0 5.1 Associate’s degree (2-year college degree) 9 7.6 7.6 4-year college degree (e.g., BA, BS) 44 37.0 37.3 Some graduate school but no degree 7 5.9 5.9 Graduate or professional degree (e.g., MBA, MS, MD, PhD) 30 25.2 25.4 Total 118 99.2 100.0 System 1 .8 Total 119 100.0

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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

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• 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 5 4.2 Walks independently for long distances outdoors (more than ½ mile) 24 20.2 Walks less than ½ mile, but more than short distances 35 29.4 Walks independently outdoors for short distances (such as to the car) 12 10.1 Walks outdoors with help from a person 1 .8 Walks independently indoors but needs a wheelchair for outdoors 2 1.7 Walks indoors with help from a person and requires wheelchair

• utdoors

4 3.4 Uses wheelchair and can go indoors and outdoors 30 25.2 Uses wheelchair but unable to go outdoors in some situations (such as cold weather) 3 2.5 Unable to control wheelchair without help 3 2.5 Total 119 100.0

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Attribute description Rank Utility Score Std Dev Std Error T-test P-Value Stops progression of weakness 1 0.88 0.337 0.01 69.44 0.0000 Slows progression of weakness 2 0.80 0.400 0.02 53.36 0.0000 Does not change progression of weakness 11

• 0.08

0.514 0.02

• 4.15 0.0000

5 year gain in expected lifespan 3 0.46 0.545 0.02 22.74 0.0000 2 year gain in expected lifespan 4 0.41 0.514 0.02 21.19 0.0000 No extra gain in expected lifespan 12

• 0.11

0.367 0.01

• 8.27 0.0000

2 years of post-approval drug info available 5 0.06 0.496 0.02 3.02 0.0013 1 years of post-approval drug info available 6 0.02 0.182 0.01 3.29 0.0005 No post-approval drug info available 9

• 0.02

0.368 0.01

• 1.52 0.0637

No increased chance of nausea 7

• 0.01

0.251 0.01

• 1.04 0.1483

Causes loss of apetite 13

• 0.13

0.342 0.01

• 10.27 0.0000

Causes loss of appetite with occasional vomiting 16

• 0.28

0.500 0.02

• 14.98 0.0000

No increased risk of bleeds 8

• 0.01

0.140 0.01

• 2.14 0.0160

Increased risk of bleeding gums and increased bruising 15

• 0.27

0.442 0.02

• 16.08 0.0000

Increased risk of hemorrhagic stroke and lifelong disability 17

• 0.72

0.449 0.02

• 42.81 0.0000

No increased risk of heart arrhythmia 10

• 0.04

0.225 0.01

• 4.50 0.0000

Increased risk of harmless heart arrhythmia 14

• 0.17

0.379 0.01

• 11.941 0.0000

Increased risk of dangerous heart arrhythmia and sudden death 18

• 0.79

0.41 0.02

• 51.13 0.0000

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Weakness Lifespan Drug info Nausea Bleeding Arrhythmia

Attribute Importance Statistic

• Weakness= 0.29
• Lifespan= 0.17
• Drug info= 0.02
• Nausea= 0.081
• Bleeding= 0.21
• Arrhythmia= 0.22

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WORRY PRELIMINARY RESULTS

Utility score

My child getting weaker

• 0.637

Getting the right care for my child over time

• 0.254

My child missing out on new treatments

• 0.245

My child feeling happy

• 0.161

Managing my uncertainty about my child’s future

• 0.127

Affording care my child needs within the family budget

• 0.065

My child having good friends

• 0.038

My child not being able to express deep worries

• 0.025

Being a good enough parent for my child

• 0.012

The wellbeing of my other children 0.038 Me handling the emotional demands of Duchenne 0.049 My child feeling like a burden on the family 0.179 Effects of Duchenne on my closest relationships 0.217 My child becoming independent from me over time 0.232 Feeling isolated from other families 0.300 Having time for myself 0.557

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Preliminary Conclusions

Participants prioritized protection of muscle function over any other attribute, including longer lifespan and each of two serious risks. Participants’ most significant worries were related to the child’s illness progression and care. The study suggests a parent population that is highly concerned about DMD’s effect on their child’s strength, and is willing to accept risk and uncertainty for a treatment that would slow or stop muscle weakness.

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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

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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