ParentProjectMD.org

Treatment Preferences and Risk Tolerance Study

Pat Furlong, BSN, MSHolly Peay, MS CGC

Vice President, Education and Outreach

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

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

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)

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

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

ParentProjectMD.org

Worry List: In the past 7 days, most/least worried…A My child missing out on new treatmentsB My child getting weakerC Getting the right care for my child over timeD My child feeling happyE My child having good friendsF My child feeling like a burden on the familyG My child becoming independent from me over timeH My child not being able to express deep worriesJ Being a good enough parent for my child K Me handling the emotional demands of Duchenne L Managing my uncertainty about my child’s futureM Having time for myselfN Feeling isolated from other familiesP Affording care my child needs within the family budget Q Effects of Duchenne on my closest relationshipsR The wellbeing of my other children

ParentProjectMD.org

Treatment AttributesLABEL 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

ParentProjectMD.org

Treatment Attributes Con’tLabel 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

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

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

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

ParentProjectMD.org

Experiment ExampleChoose 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.

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

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

ParentProjectMD.org

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 98.3 100.0System 2 1.7Total 119 100.0

ParentProjectMD.org

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

Freq % Valid %High school or GED 5 4.2 4.2Some college but no degree 17 14.3 14.4Technical school 6 5.0 5.1Associate’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

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

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 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 outdoors 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

ParentProjectMD.org

Preliminary ConclusionsWithin 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.

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

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