This document discusses using personalized outcomes and goal attainment scaling to evaluate treatment effectiveness at the individual patient level. It proposes developing personally meaningful outcomes (POSI) through patient narratives and collaborative goal setting, then tracking progress over time using goal attainment scaling. This provides a structured way to measure outcomes that matter most to each patient, complementing standardized measures. The approach is illustrated with examples from rare disease trials and has potential applications in mental health and activity therapies by personalizing treatment evaluation.

1. INDIVIDUALIZED PATIENT
OUTCOMES:
GOALATTAINMENT SCALING
William McKellin, PhD
Department of Anthropology, UBC
Sravan Jaggumantri, MSc, PhD Candidate
Department of Experimental Medicine and Pediatrics, UBC
Christopher Condin, PhD
Department of Anthropology, UBC
Mojgan Gitimoghaddam, MD
Department of Experimental Medicine and Pediatrics, UBC
Jean Paul Collet, MD, PhD
Department of Pediatrics, UBC
Child Family Research Centre, BC Children’s Hospital

2. Questions
• What is personalized medicine?
• What is the role of the patient in setting treatment goals?
• How can patients play a significant role in assessing the effectiveness of
treatments?
• How can narrow concepts of personalized medicine be expanded to include
personalized mental health and therapeutic activity?

3. • “Genomics-based knowledge
promises the ability to
approach each patient as the
biological individual he or
she is, thereby radically
changing our paradigms and
improving [how we assess]
efficacy.”
• -Guttmacher et al. (2007)​

4. Making medicine personal
• Personalized Medicine
• Targeted therapies based on an
individual's genetic mutation / genome
• Patient Centered Outcomes
• Therapies targeted to patient’s diagnosis,
demographics, and patient group’s
preferences
• Patient Reported Outcomes
• Patient provided information vs
physiological measurements
• Patient Outcomes of Specific Interest
• Individual genome-based targeted
therapies with individual’s preferred
outcomes assessed

5. Patient-reported outcomes (PRO) for comparative
effectiveness
• Regulatory bodies (FDA, EMA) require Patient Reported Outcomes
In prospective studies, investigators could include lay representatives (patient or care-giver) in study
steering groups or consult patient associations for guiding research questions. This approach will
enhance validity and relevance of research.
Acaster S, Cimms T, Lloyd A. The design and selection of patient-reported outcome measures (PROMS) for use in patient-centered outcomes research. Washington, D.C.:
Patient Centered Outcomes Research Institute (PCORI) 2012.Pp. 12
• PRO scales and tools use standard assessments
• Normed to general population
• Normed to specific common conditions
• General domains and items
• Do not reflect the outcomes of personal importance to a particular individual
• Physicians in clinical practice rarely use these tools.

6. Personal Goals – Duchenne Muscular Dystrophy
• I think the 6 minute walk test for
measuring outcomes of a
treatment ignores all of the
possible treatments for the boys
who just want to preserve some
dignity.
Ataluren trial parent
Condin CJ. Families’ experiences with medical research for pediatric rare
diseases : a qualitative ethnographic study of parents and children participating
in clinical trials for Duchenne muscular dystrophy (DMD). Vancouver, B.C.:
University of British Columbia; 2014. p. 206

7. Personal Goals – DMD Ataluren trial parents
“He’s carrying things that he wouldn’t normally try
to pick up, like a couple of plates and cups, with
no other hand to grab a wall, he walked them into
the kitchen.
… [This makes him] feel better about himself,
more independent, more empowered … He’s less
angry, he cries less, he’s less frustrated, because
he can do things himself.”
Condin CJ. Families’ experiences with medical research for pediatric rare diseases : a qualitative
ethnographic study of parents and children participating in clinical trials for Duchenne muscular
dystrophy (DMD). Vancouver, B.C.: University of British Columbia; 2014

8. POSI
Personal Outcomes of Specific Importance
Why?
• Rare Diseases are genetically and phenotypically heterogeneous
• Personal treatment goals are not standardized
What is needed?
• Personalized outcomes most relevant to the patient’s everyday needs
and perception of wellness
• Developed from patients’ understandings of their conditions expressed
as narratives
• Collaborative therapeutic targets that can be set and tracked
Solution?
• POSI identified, articulated and tracked through Goal Attainment Scaling
(GAS)

9. Medical histories and Illness narratives
Medical histories
• Goal to interpret symptoms to
produce a diagnosis
• Reported symptoms elicited by clinician
• Symptoms used to exclude possible
diagnoses
• Symptoms matched with the natural
history of probable diagnosis
• Histories support clinical
explanatory models of the condition
Illness narratives
• Goal to make changes in physical
conditions sensible in everyday life
• Events selected by the patient as
relevant for the audience
• Based on assumptions about cause-
effect relations
• Events selected to form a coherent
narrative
• Illness narratives represent the
patient’s explanatory model of the
condition

10. POSI using Goal Attainment Scaling
Personal Narrative
Collaborative
Therapeutic Scenario
Medical history,
diagnosis
Identify POSI
Integrate with Standardized outcome measures
Transform POSI to Goals and scaling
GAS Scaling analysis
Re-assess treatment decisions

11. Patient 1
• Patient is a 9 year old girl
• Glucose transporter deficiency
• Standard ketogenic diet treatment over 2 years but still keeps having seizures.
• She was started on an experimental treatment (Triheptanoin oil)
• In an interview she said that an ideal treatment will be where she can stop the diet
and take some kind of a pill.
• She understands that treatments are not there yet.
• With Triheptanoin she expects to have fewer/no seizures because it hampers her
daily functioning
• She also feels tired most of the time and is not able to participate in most activities at
school.
• She wants to reduce her seizures improve her energy levels and also be able to
improve her motor skills.
Sravan Jaggumantri, A prospective patient centered decision making model to evaluate patient specific outcomes of interest and establish treatment
effectiveness at individual level. (PhD Project)

12. POSI 1
• Goal
• Decrease seizures
• Indicator
• Seizure frequency
• Baseline
• Levels of attainment
-2 much less than goal
-1 somewhat less than goal
0 expected level
+1 somewhat more than goal
+2 much more than goal
• Score at follow up
• Treatment decision
POSI – 01:
(Eg: Sleep)
Decrease in Seizures
Indicator
(Eg: Sleep Duration,
Frequency of waking up
etc.)
Seizure frequency
How is it measured? Number of seizure free days in a week
Present Status/ Baseline She has seizures everyday
Levels of predicted attainment / Expected target for the outcome
Much less than the
expected level of
outcome (-2)
Zero seizure free days in a week
Somewhat less than the
expected level of
outcome (-1)
Only 1 seizure free day in a week
Expected level of
outcome (0)
Two seizure free days in a week
Somewhat more than the
expected level of
outcome (+1)
Three to four seizure free days in a week
Much more than the
expected level of
outcome (+2)
Five to seven seizure free days in a week
Date for
reevaluation:
01/Oct/2014 Score at Follow
up 1:
-1
Date for
reevaluation:
06/Jan/2015 Score at Follow
up:
0
Outcome Extend Follow up /Continue Treatment/Stop Treatment/ Revise POSI

13. POSI 2
POSI – 02:
(Eg: Sleep)
Improvement in Energy
Indicator
(Eg: Sleep Duration,
Frequency of waking up
etc.)
Play time
How is it measured? Time spend playing hard activities and still not feeling tired
Present Status/ Baseline After 30- 40 min of physical activity gets tired and sits out at
school for some activities during gym class
Levels of predicted attainment / Expected target for the outcome
Much less than the
expected level of
outcome (-2)
Gets tired in less than 30 min while playing everyday
Somewhat less than the
expected level of
outcome (-1)
Gets tired after 30 min while playing everyday
Expected level of
outcome (0)
Can play for an hour every day and still not feel tired
Somewhat more than the
expected level of
outcome (+1)
Can play for at least 1.5 hours every day and still not feel
tired
Much more than the
expected level of
outcome (+2)
Can play for at least 2 hours every day and still not feel tired
Date for
reevaluation: 01/Oct/2014
Score at Follow
up:
0
Date for
reevaluation:
06/Jan/2015 Score at Follow
up:
+2
Outcome Extend Follow up /Continue Treatment/Stop Treatment/ Revise POSI
• Goal
• Improve energy
• Indicator
• Time spent playing hard activities and not
feeling tired
• Baseline
• Levels of attainment
-2 much less than goal
-1 somewhat less than goal
0 expected level
+1 somewhat more than goal
+2 much more than goal
• Score at follow up
• Treatment decision

14. POSI 3
• Goal
• Improve in fine and gross motor
function
• Indicator
• Ability to do patterns in Sun
Hang Do class
• Baseline
• Levels of attainment
-2 much less than goal
-1 somewhat less than goal
0 expected level
+1 somewhat more than goal
+2 much more than goal
• Score at follow up
• Treatment decision
POSI – 03:
(Eg: Sleep)
Improvement in Fine and Gross Motor function
Indicator
(Eg: Sleep Duration,
Frequency of waking up
etc.)
Sun Hang Do – Martial art
How is it measured? Ability to do patterns in Sun Hang Do class
Present Status/ Baseline Cannot do any patterns and loses balance
Levels of predicted attainment / Expected target for the outcome
Much less than the
expected level of
outcome (-2)
Not able to go to next level of basic moves and unable to
maintain balance
Somewhat less than the
expected level of
outcome (-1)
Not able to do patterns but can maintain balance with basic
moves
Expected level of
outcome (0)
Able to do at least 1 pattern without any balance issues
Somewhat more than the
expected level of
outcome (+1)
Able to do at 2 patterns without any balance issues
Much more than the
expected level of
outcome (+2)
Able to do at 3 patterns without any balance issues
Date for
reevaluation: 01/Oct/2014
Score at Follow
up:
-1
Date for
reevaluation: 06/Jan/2015
Score at Follow
up:
0
Outcome Extend Follow up /Continue Treatment/Stop Treatment/ Revise POSI
Adverse Event?

15. GAS
Wellness Tracker
• Goal Attainment Scaling implemented on (http://wellnesstracker.org)

16. Effectiveness
POSI and GAS
• Contextually meaningful and embedded in the personal and family narrative of
illness and understanding
• Desired treatment outcomes are articulated as goals in GAS
• Treatment effectiveness is measurable
• Makes decision making made explicit
• Identifies undesired / adverse events
• Documents therapeutic decision making
• Complements information from standardized outcome measures

17. POSI and GAS in Mental Health
• Design
• Prospective cohort study in 60 youth admitted to the Mental Health Division (MHD) of BC
Children’s Hospital (BCCH) for a short period (range is 4 to 6 weeks)
• Goal
• Determine the effects of drugs via the POSI approach and other standardized outcomes
assessments
• Setting
• Inpatient setting in order for measurements and to evaluate possible occurrences of POSI
changes over time, in relation to the drug(s)’ effects
• Treatments and Assessment
• Personal outcomes will be identified using the POSI approach.
• Standardized PROM for comparative validity and reliability, and responsiveness of POSI
• The feasibility of integrating POSI into clinical practice

18. POSI and GAS in Mental Health
• Study Criteria & Population:
• Male and female patients aged 12-18 years
• Common conditions: mood disorders, anxiety disorder, disruptive behavior disorder
• Drug treatments include antidepressants (e.g. SSRI), anti-psychotics (e.g. risperidone),
mood stabilizers (lithium), and stimulant medications (e.g. methylphenidate).
• Clinical context
• Medications take effect rapidly and have known safety profiles
• Standard scales are already used for clinical evaluation
• Scores on Standard scales will be used for assessing the validity of POSI
• Reflective of real life clinical situations
• Cohort with different diseases and different drugs - enables assessment of POSI validity
across multiple diseases and drugs

19. GAS in Activity Therapies
• GAS as Scaffolding / Coaching
• Psychological development occurs though social interaction
Empowering Steps Movement Therapy
• non-categorical
• holistic
• child centered
• family and community based

20. Conclusions
Personal Outcomes of Specific Importance and
Goal Attainment Scaling
• Collaboration in developing a Therapeutic Scenario, POSI, and
Goals
• Structured and measurable evaluation process (indicators, timeline,
scaling)
• Clear documentation of the decision-making process
• Complements information from standardized outcome measures
• Not disease specific – well suited for rare / undiagnosed diseases
• Provides a personal assessment of real-life effectiveness

21. Thanks
• Rare Disease Foundation
• NeuroDevNet
• TIDE-BC
• BC Children’s Hospital
• Child and Family Research Institute
• University of British Columbia
• (http://wellnesstracker.org)