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Presentation on theme: " Measuring the priorities and treatment preferences of caregivers of a child with Duchenne Muscular Dystrophy using best-worst scaling."— Presentation transcript:

1 Measuring the priorities and treatment preferences of caregivers of a child with Duchenne Muscular Dystrophy using best-worst scaling Holly Peay, M.S. Parent Project Muscular Dystrophy

2 “I understand the need for caution and care, but I also know that our children are dying. Parents should be able to decide the risk/benefit of a drug that has gone through and passed preliminary testing. I would rather my son die trying and fighting than waiting and wondering and wishing….I am one parent willing to take an educated risk!” PPMD “Share Your BRAVE Story”

3 FDA FDASIA and PDUFA legislation (2012) Committed the FDA to initiate the Patient- Focused Drug Development Program Aims to inform the FDA’s benefit/risk assessments and includes goals of systematically obtaining the patient’s perspective on disease impact and treatment benefits ( /ucm326192.htm)

4 A time of unique opportunity How do we influence regulatory bodies? –Who represents patient/family preferences? What do regulators want? –Less “emotion”? –More “patient stories”? –Quantifiable, generalized data? What does the community want? –What is a “good enough” benefit? –Is any risk too high for approval? –What information needed for decision making?

5 Engagement models Direct engagement via representation, consultation and/or testimony Formal study of the preferences and views of patients and the public “When asking the public to assist in determining health priorities, we should use techniques that allow people to reveal their true preferences. If not, why bother asking them at all?” ( Gafni,1995)

6 PPMD’s benefit/risk study Advocacy-led, community-engaged study to: 1.Quantify treatment preferences of caregivers for benefits and risk of potential treatments (BWS case 2) 2.Prioritize Duchenne-related worries of caregivers to describe impact (BWS case 1) –Compare worry priorities of parents of ambulatory and non-ambulatory children

7 A BWS benefit: Offers alternative to complex algorithms: score a level given the times a level was rated as best or worst. –Level receives +1 if rated as best –Level received -1 if rated as worst –Level receives 0 otherwise An approximation to the utility of the level is given by averaging the score, or as –Score = (B – W)/N –where N is the number of times a level appears across all choice sets.

8 Treatment preferences Hypothetical treatment features (attributes) identified and refined in consultation with parents, clinicians, researchers and industry In-depth pilot testing with 7 parents Parents/guardians identify what parts of each treatment were best and worst Experimental design identified 18 potential treatments that systematically varied across the six chosen attributes

9 Attributes and levels Effect on muscle function (none, slows, stops) Gain in expected lifespan (none, 2, 5 years) Post-approval information (none, 1, 2 years) Nausea (none, loss of appetite, loss of appetite and occasional vomiting) Risk of bleeds (none, risk of bleeding gums and increased bruising, risk of hemorrhagic stroke) Risk of heart arrhythmia (none, risk of harmless heart arrhythmia, risk of dangerous heart arrhythmia and sudden death)

10 Example BWS choice task Choose the best thing in this treatment 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 worst thing to move on. Remember that a computer chose the combinations to make the experiment work, and some of them seem bad. Even so, please pick the best and worst thing. BestTreatmentWorst ○ Slows the progression of weakness ○ ○ 2 year gain in expected lifespan ○ ○ 1 year of post- approval drug information available ○ ○ Causes loss of appetite ○ ○ Increased risk of bleeding gums and increased bruising ○ ○ Increased risk of harmless heart arrhythmia ○

11 DMD worries: BWS case 1 Responsive to the FDA’s request to understand disease impact Aim: prioritize “qualitative” DMD-related worries using quantitative methods 5 parents contributed worries, ultimately resulting in list of 16 DMD-related worry statements after stakeholder engagement, categorized under 4 domains Pilot study conducted with 7 parents

12 Worries and domains DomainAttribute description Child affectMy child feeling happy My child having good friends My child not being able to express deep worries My child feeling like a burden on the family Family and socialThe wellbeing of my other children Effect of Duchenne on my closest relationships My child becoming independent from me over time Feeling isolated from other families Child medical concernsMy child getting weaker Getting the right care for my child over time My child missing out on new treatments Affording care my child needs within the family budget Parent well-beingManaging my uncertainty about my child's future Being a good enough parent for my child Me handling the emotional demands of Duchenne Having time for myself

13 BWS worry choice task In the past 7 days, choose which of the following concerns you have been most worried about and which you have been least worried about. Most worriedConcernsLeast worried ○ My child having good friends ○ ○ The wellbeing of my other children ○ ○ My child not being able to express deep worries ○ ○Being a good enough parent for my child○ ○My child feeling like a burden on the family○ ○ My child feeling happy ○

14 Recruitment Recruitment: –Using PPMD, DuchenneConnect Registry, and snowball recruiting Inclusion/exclusion: –At least one living child with DMD, living in the US, over 18 years of age, and able to complete an online survey in English Ethics: –Study determined to be exempt by the Western Institutional Review Board

15 Sample (119) Participant characteristicsMean (SD) Caregiver age in years43.7 (SD=7.7) Relationship to child(ren) Biological mother67.2% Biological father28.6% Adoptive mother3.4% Adoptive father0.8% Marital status Married/long-term relationship89.9% Divorced/Separated9.2% Widowed0.8% Race Caucasian91.6% Education High school/GED4.2% Some college14.3% Technical school5.0% Associated degree7.6% Four-year college degree42.9% Graduate/professional degree25.2% Income <$25,0005.9% $25,000-$50,0008.4% $50,000-$75,00018.5% $75,000-$100,00018.5% >$100,00047.1% Child characteristicsPercent Child age in years21.1 (SD=6.4) Number of affected children One child92.4% Two or more children7.6% Living arrangements In caregiver's home98.3% Independent0.8% Other0.8% Ambulation status Ambulatory63.9% Non-ambulatory36.0% Research participation Clinical research58.0% Clinical trial34.0% Had life-threatening emergency Yes18.5% No81.5%

16 Relative attribute importance 28.66%17.29%2.31%8.10%21.23%22.41% Effect on muscle functionLifespan Knowledge about drug NauseaRisk of bleedsRisk of heart arrhythmia Figure 2. Relative best-worst scores for attribute levels and relative attribute importance


18 Conclusions Preference experiment: –Stopping/slowing the progression of muscle weakness accounted for the largest proportion of the variation. –The presence of side effects/risks could be compensated for by a treatment that stops/slows progression to muscle function. Worries experiment: –Child’s disease management was ranked as of greatest concern, followed by child’s affect. –Impacts of DMD on the family and parents’ wellbeing were less prioritized.

19 Some benefits of community- engaged approach Identify the “right” attributes and levels –Meaningful to patients/families –Acceptable to other stakeholders Set up the experiment “properly” for the community Bring together stakeholders in a mutually- beneficial process Give the community a productive action

20 Engaging the FDA On July 9, 2013, PPMD met with Dr. Janet Woodcock and senior staff from FDA; one objective: review BR results On December 12, 2013 PPMD held an FDA/community engagement meeting that also covered the BR study Anecdotally, FDA senior staff have encouraged other advocacy organizations to use it as a model

21 Acknowledgements Co-authors: –John Bridges, JHSPH –Ilene Hollin, JHSPH –Hadar Sheffer, PPMD –Ryan Fischer, PPMD This research was supported through funding from Parent Project Muscular Dystrophy (PPMD).

22 Results: Treatment preferences Treatment benefits and risksBestWorstUtilitySET-test P- Value Stops progression of weakness62820.8770.0169.4<0.001 Slows progression of weakness57100.8000.0253.4<0.001 Does not change progression of weakness68125-0.0800.02-4.1<0.001 5 year gain in expected lifespan348170.4640.0222.7<0.001 2 year gain in expected lifespan29980.4080.0221.2<0.001 No extra gain in expected lifespan1293-0.1130.01-8.3<0.001 2 years of post-approval drug info available109690.0560.023.00.001 1 years of post-approval drug info available2040.0220.013.30.001 No post-approval drug info available4156-0.0210.01-1.50.064 No increased chance of nausea1926-0.0100.010.148 Causes loss of appetite195-0.1320.01-10.3<0.001 Causes loss of appetite with occasional vomiting17217-0.2800.02-15.0<0.001 No increased risk of bleeds311-0.0110.01-2.10.016 Increased risk of bleeding gums and increased bruising0190-0.2660.02-16.1<0.001 Increased risk of hemorrhagic stroke and lifelong disability0514-0.7200.02-42.8<0.001 No increased risk of heart arrhythmia532-0.0380.01-4.5<0.001 Increased risk of harmless heart arrhythmia1122-0.1690.01-11.9<0.001 Increased risk of dangerous heart arrhythmia and sudden death0561-0.7860.02-51.1<0.001

23 Results: Worry prioritization WorryDomainEstimateStd ErrorZ scoreP-value Child missing new treatmentsMC 0.930.07312.73p<0.001 Child getting weakerMC2.300.07431.09p<0.001 Getting the right care over timeMC1.000.07313.64p<0.001 Child feeling happyCA0.670.0768.84p<0.001 Child having good friendsCA0.100.0761.340.18 Child feeling like burdenCA-0.670.073-9.22p<0.001 Child becoming independentFS-0.850.071-11.92p<0.001 Child unable to express worriesCA0.090.0751.270.21 Good enough parentPWB-0.050.083-0.610.54 Handling emotional demandsPWB-0.200.076-2.650.01 Managing my uncertaintyPWB0.490.0766.43p<0.001 Having time for myselfPWB-1.970.071-27.59p<0.001 Isolated from other familiesFS-1.100.071-15.5p<0.001 Affording care my childMC0.270.0743.67p<0.001 Effect on closest relationshipsFS-0.850.072-11.77p<0.001 Wellbeing of other childrenFS-0.160.078-2.120.03

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