# Presentation: 333830

## DEFINING  QUALITY OF LIFE FOR ONCOLOGIC DRUG APPROVAL

**Carol M. Moinpour, PhD**
**Southwest Oncology Group Statistical Center**
**Fred Hutchinson Cancer Research Center**
**Seattle, Washington/USA**
**ODAC QUALITY OF LFE SUBCOMMITTEE**
**February 10, 2000**

## What is Quality of Life?

**Subjective**
**Patient perception is critical **
**Health-related**
**Yes, we are primarily measuring health status**
**Multidimensional**
**Which dimensions are relevant?**
**Usually not driven by psychosocial theories**

## Proposal: Data Source

**  ****Expert re: “patient benefit” = patient**
**  ****Best equipped to evaluate claims about   ****	****impact of treatment **

## Quality of Life Vs Health-Related QOL (HRQL)

**Not feasible to measure the myriad of non-medical influences**
**Restrict QOL domains likely to be affected by medical intervention (HRQL)**
**Attribution problem**
**Randomization addresses unmeasured factors**
**Combine quality with quantity of  life?****	**

## Proposal: QOL vs HRQL

**Restrict quality of life assessments to HRQL**
**Patients asked to report current status**
**Address known covariates **
**HRQL can include duration of life **
**A complicated question deserving our attention**

## HRQL Domains

## Examples of Quality of Life Domains

**Physical Functioning****	****Spirituality**
**Occupational/Role Functioning****	****Future Orientation**
**Social Functioning****	****Sexuality/Intimacy**
**Emotional Functioning****	****Health Concerns**
**Symptom Status****	****Family Well-Being**
**Financial Concerns****	****Satisfaction w/Care**
**Global/Overall Perception of Quality of Life**

## HRQL Domains

**Inclusion of symptoms important because**
**Corroborate physician-rated toxicities**
**Document palliation in advanced stage disease**
**Need to examine the “reach” of better/worse symptoms to general functioning **

## Assessment of Multiple HRQL Dimensions

**Specifies how treatments affect patients**
**Complements physician-related toxicity information**
**Informs patients and physicians about risks/benefits tradeoffs**
**Identifies ways to improve cancer treatments**
**Identifies survivors rehabilitation needs**

## Proposal: HRQL Domains

**Three basic domains**
**Psychological**
**Physical**
**Social**
**[Global HRQL vs summary score?]**
**Measure symptoms but also document their effect on basic domains**

## Proposal: HRQL Domains

**Symptom status is not a manifestation of patient HRQL**
_**Sx outcomes alone should not be called HRQL**_
**Sx outcomes alone may be appropriate in Phase II, single institution, or supplemental submissions **
**Sx outcomes can be designated primary **
**Clinical issues should drive content of sx measures**

## Proposal: Role of Theory

**Psychological or social science theories usually not driving HRQL assessment design**
**Psychometric theory has done so**
**First obligation:  identify HRQL issues critical to evaluating treatments**
**Can suggest broad impacts on the patient **
**Tx rationale usually doesn’t address HRQL**

## Additional Slides

## HRQL Research Issues

**Independence of HRQL dimensions**
**Incorporation of importance ratings?**
**Impact of substantial individual variation conclusions re: patient benefit**
**Integration of symptom and basic domain data **
**To document scope of patient benefit/harm**
**Validity of total scores with symptom component**
**Role of methodological and theory-driven research in defining HRQL for drug claims?**

## Problem with Single Item Measures

**Only 1 chance to capture concept or dimension**
**OK for detecting moderate to large differences**
**Attractive for clinical trials (burden issue)**
**Likely to miss differences at individual ****patient level**
**Compromise - brief, multi-item scales**

## World Health Organization Definition

**“****Health is not only the absence of infirmity and disease but also a state of physical, mental and **
**social well-being.”**