Session 6: Choosing and using HRQoL measures vs Multi-Attribute Utility Instruments QLU-C10D and EQ-5D as examples - Madeleine King & Richard De Abreu Lourenco-
Overview Learning Objectives To discuss the principles guiding the choice of HRQOL and utility measures for a clinical study To gain experience using the QLU-C10D and the EQ-5D Topics covered Choosing HRQOL measures for a clinical study Choosing a utility measure for a clinical study How to score the QLU-C10D and EQ-5D
CHOOSING HRQOL MEASURES FOR A CLINICAL STUDY
Types of HRQoL and other PRO measures Generic QOL e.g. SF-36 Cancer-specific QOL E.g. applicable to all cancers e.g. QLQ-C30, FACT-G Tailored for specific cancers (e.g. ovarian QLQ-OV28, FACT-O) Domain/symptom-specific e.g. PROMIS suite, HADS (anxiety & depression), Brief Pain Inventory (BPI), EORTC Fatigue, symptom indexes (e.g. FOSI + more in FACIT suite; MDASI suite) Treatment-specific QOL Tailored for specific treatments (e.g. Neurotoxicty + more in FACIT suite) Utility measures; e.g. EQ-5D, SF-6D, QLU-C10D
MANY questionnaires to choose from! H E L P!!! Too many!
PROs what s in the toolkit? Choose the right tool for the job
Q: What is the best HRQoL measure for your study? A: Which patient-reported outcomes matters most to you and your patients? In any particular trial, that will depend on will the specific effects of the treatments in the trial arms and expected differences between them The right questionnaire is the one that measures everything that matters, in a psychometrically sound way, especially responsive to change in health/hrql Do you want to conduct an economic evaluation as part of your study? DO NOT choose a measure because someone you know used it, or everyone uses it, or it s the only one you know DO think and read about what measures are available, get specimen copies (e.g. PROQOLID), understand how they score items into outcome scales DO understand the difference between HRQoL Profile v Utility measures Ask the QOL Office and CREST for advice
Approaches to measuring and valuing QOL for economic evaluation Options Use a purpose built MAUI (eg EQ-5D-5L) Use a generic QOL instrument that can be used to convert to a MAUI (eg SF-36 to SF-6D) Use a disease specific instrument that can be used to convert to a MAUI (eg QLQ-C30 to QLU-C10D)
So You Have Used a MAUI Patients completed MAUI (eg within the trial) Multiple completions at different time points Data reasonably complete (in an ideal world) Are there more steps? Patients have rated their own health. Now apply the preference algorithm to produce their utility values. The algorithm already exists YOU DON T NEED TO DO THE VALUATION YOURSELF.
The (5-level) EQ-5D Dimension Level Description Mobility 1 2 3 4 5 Self-care 1 2 3 4 5 Usual Activities (e.g. work, study, housework, family or leisure activities) Pain/Discomfort 1 2 3 4 5 Anxiety/Depression 1 2 3 4 5 1 2 3 4 5 I have no problems in walking about I have slight problems in walking about I have moderate problems in walking about I have severe problems in walking about I am unable to walk about I have no problems washing or dressing myself I have slight problems washing or dressing myself I have moderate problems washing or dressing myself I have severe problems washing or dressing myself I am unable to wash or dress myself I have no problems doing my usual activities I have slight problems doing my usual activities I have moderate problems doing my usual activities I have severe problems doing my usual activities I am unable to do my usual activities I have no pain or discomfort I have slight pain or discomfort I have moderate pain or discomfort I have severe pain or discomfort I have extreme pain or discomfort I am not anxious or depressed I am slightly anxious or depressed I am moderately anxious or depressed I am severely anxious or depressed I am extremely anxious or depressed
Scoring Algorithm (UK) EQ-5D-5L value set for England Full health (11111) 1.000 Mobility slight 0.057 moderate 0.075 severe 0.208 unable 0.255 Self-care slight 0.058 moderate 0.083 severe 0.176 unable 0.208 Usual activities slight 0.048 moderate 0.067 severe 0.165 unable 0.165 Pain/discomfort slight 0.059 moderate 0.080 severe 0.245 extreme 0.298 Anxiety/depression slight 0.073 moderate 0.099 severe 0.282 extreme 0.282 Estimate utility as 1- the score for each level of each dimension (in order presented). Level 1 s have score of zero. Eg. 11111=1-0-0-0-0-0 = 1 12331=1-0-0.058-0.067-0.080-0 = 0.795 Shifts between states can have large impacts on utility
Using the EQ-5D-5L Levels Utility Decrements Utility Calculation 1 2 3 4 5 0 0.057 0.075 0.208 0.255 Profile Score: 1 2 3 4 5 0 0.058 0.083 0.176 0.208 23222 1 2 3 4 5 0 0.048 0.067 0.165 0.165 Calculation: 1 0.057 0.083 0.048 0.059 0.073 = 1 2 3 4 5 0 0.059 0.080 0.245 0.298 0.68 1 2 3 4 5 0 0.073 0.099 0.282 0.282 Decrement values: Devlin et al (2014)
Exercise: Using the EQ-5D-5L Calculate your EQ-5D-5L utility score using the Your Health-Related Quality of Life scores and Your Utility Scores Handout
Exercise: Using the QLU-C10D We will now show you how to calculate your QLU-C10D utility score using the Your Health-Related Quality of Life scores and Your Utility Scores Handout
13/30 items for QLU-C10D 1 Do you have any trouble doing strenuous activities, like carrying a heavy shopping bag or a suitcase? 2 Do you have any trouble taking a long walk? 3 Do you have any trouble taking a short walk outside of the house? 4 Do you need to stay in bed or a chair during the day? 5 Do you need help with eating, dressing, washing yourself or using the toilet? 6 Were you limited in doing either your work or other daily activities? 7 Were you limited in pursuing your hobbies or other leisure time activities? 8 Were you short of breath? 9 Have you had pain? 10 Did you need to rest? 11 Have you had trouble sleeping? 12 Have you felt weak? 13 Have you lacked appetite? 14 Have you felt nauseated? 15 Have you vomited? 16 Have you been constipated? 17 Have you had diarrhea? 18 Were you tired? 19 Did pain interfere with your daily activities? 20 Have you had difficulty in concentrating on things, like reading a newspaper or watching television? 21 Did you feel tense? 22 Did you worry? 23 Did you feel irritable? 24 Did you feel depressed? 25 Have you had difficulty remembering things? 26 Has your physical condition or medical treatment interfered with your family life? 27 Has your physical condition or medical treatment interfered with your social activities? 28 Has your physical condition or medical treatment caused you financial difficulties? 29 How would you rate your overall health during the past week? 30 How would you rate your overall quality of life during the past week?
Linking QLU-C10D dimensions to QLQ-C30 items QLU-C10D QLQ-C30, 13 items included in QLU-C10D 10 DIMENSIONS Item # and Content 1 Physical 2 Do you have any trouble taking a long walk? Functioning 3 DITTO short walk outside of the house? 2 Role Functioning 6 Were you limited in doing either your work or other daily activities? 3 Emotional 24 Did you feel depressed? Functioning 4 Social Functioning 26 Has your physical condition or medical treatment interfered with your family life? 27 DITTO social activities? 5 Fatigue 18 Were you tired? 6 Pain 9 Have you had pain? 7 Sleep 11 Have you had trouble sleeping? 8 Appetite 13 Have you lacked appetite? 9 Nausea 14 Have you felt nauseated? 10 Bowel 16 Have you been constipated? disturbance 17 Have you had diarrhea?
Calculating your QLU-C10D score Your utility score = 1 minus each the utility decrement for each level down from perfect health, i.e. no problems on any dimension Utility = 1 Σ (your utility decrements)
What is your utility decrement for Physical Functioning? QLU-C10D Level 1 Level 2 Level 3 Level 4 Utility decrement 0-0.110-0.170-0.215 Recall: empirical results Session 5 Long walk QLQ-C30 Item 2 Short walk QLQ-C30 Item 3 Not at all A little Quite a bit Very much Total Not at all 802 7 0 0 809 A little 568 128 3 0 699 Quite a bit 176 198 99 2 475 Very much 50 180 177 226 633 Total 1596 513 279 228 2616
What is your utility decrement for Social Functioning? QLU-C10D Level 1 Level 2 Level 3 Level 4 Utility decrement 0-0.053-0.099-0.106 Recall: empirical results Session 5 Family life QLQ-C30 Item 26 Social activities QLQ-C30 Item 27 Not at all A little Quite a bit Very much Total Not at all 883 195 73 41 1192 A little 104 492 134 46 776 Quite a bit 16 51 259 74 400 Very much 10 8 32 198 248 Total 1013 746 498 359 2616
What is your utility decrement for Bowel Disturbance? QLU-C10D Level 1 Level 2 Level 3 Level 4 Utility decrement 0-0.068-0.088-0.11 Recall: empirical results Session 5 Constipation QLQ-C30 Item 16 Diarrhoea - QLQ-C30 Item 17 Not at all A little Quite a bit Very much Total Not at all 1048 277 90 45 1460 A little 395 193 25 14 627 Quite a bit 202 48 65 4 319 Very much 113 14 8 75 210 Total 1758 532 188 138 2616
Remaining QLU-C10D dimensions: Utility decrements for single items QLU-C10D dimension Level 1 Level 2 Level 3 Level 4 (QLQ-C30 Item) Not at A little Quite a bit Very much all Role (6) 0-0.055-0.107-0.124 Emotional (24) 0-0.055-0.095-0.127 Fatigue (18) 0-0.047-0.059-0.059 Pain (9) 0-0.078-0.124-0.141 Sleep (11) 0-0.052-0.061-0.061 Appetite (13) 0-0.05-0.078-0.078 Nausea (14) 0-0.076-0.099-0.113
Discussion How do your scores for the QLQ-C30 scales compare with your QLU-C10D utility score? How does your EQ-5D utility score compare with your QLU-C10D utility score? What are the pros and cons of the HRQoL Profile Measure approach (QLQ-C30) versus the utility approach?
Choosing a MAUI
Choosing a MAUI What is impacting on patient QoL? Which instrument is likely to be most sensitive to that effect? What have others used in this space? Do you want a generic ors disease specific instrument? Are there special considerations for your patient group eg. paediatrics, palliative care? Are there regulatory considerations? Are there budget considerations?
A MAUI is not a MAUI is not a MAUI. They all try to measure the same thing, but differ Choice of attributes and levels Valuation techniques Khan MA, Richardson J (2011)
Sensitive to Different Effects Based on dimensions in SF-36, MAUI sensitivity: Richardson et al (2012)
No One MAUI to Bind them All NICE UK (2008) The value of changes in patients HRQL (that is, utilities) should be based on public preferences using a choice-based method. The EQ-5D is the preferred measure of HRQL in adults PBAC (2008) To maximise comparability across submissions, it would be ideal to request that a single off-the-shelf MAUI be used in randomised trials However, in practice, no single MAUI has demonstrated unequivocal superiority against all the others and no single MAUI has been universally accepted.
Get the right tool for the job! Questions? Thank you