Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
Intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text on the opening pages of each chapter.
Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.
Do not use for reproduction, copying, pasting, or reading; exclusively for search engines.
OCR for page 55
c
The Use of Quality-of-Life
Measures In the
Pnv ate Sector
Dreary R. Luce, Joan M. Weschier, and
Carol Underwood
This chapter explores indusoy's use of the quality-of-life concept, how
it is applied, and He expected outcomes of its use. Although we empha-
size Be private sector, most published accounts to date have been sup-
ported by Me public sector, usually fi~nded by government agencies
Bough universities. As discussed below, this trend may be changing.
As our references show, the published and fugitive literature indicates
wide-rang~ng interest in quality-of-life measures. Although only a few
studies funded by companies In Be private sector have been published,
most pharmaceutical companies are at least entertaining me idea of incor-
porating such measures into future clinical Bids. Some have made the
explicit decision to use Rem in aU cI~n~cal trials.
The belief In Be importance of quality-of-life measures in Be assess-
ment of palliative drugs appears to be well entrenched. The extent of We
use of these scales is not yet reflected in me literature because of Be time
lag between He conduct of clinical trials and me publication of results.
Our findings indicate that it is reasonable to anticipate an increase in me
number of companies Hat use such scales, an observation mat will soon
be manifested in He literature.
METHOD OF STUDY
To assess the use of quality-of-life measures by He private sector, we
devised a ~ree-part study. First, we conducted a literature review to
55
OCR for page 56
56
BE. LUCK, JM. WESCHLER, AND C. UNDERWOOD
provide background information on the field, as wed as to search for
pnvate-sector studies that incorporated these measures. Second, we de-
veloped and distributed a questionnaire to ask private pharmaceutical and
device composes about Weir current and prospective uses of quality-of-
life instruments in clinical teals. Third, we conducted interviews wad
officials at He Food and Drug Adm~s~ation (FDA), other government
agencies, and private companies to ascertain Heir respective positions on
the salience, validity, and usefulness of these measures. Our ultimate
objective was to identify groups that are using, or plan to use, quality-of-
life measures and to determine why Hey are using them.
Private-Sector Research in Quality of Life
In 1986, published research revealed for the first time that not only
were pnv ate companies interested in quality-of-life assessment, but also
that they were funding quality-of-life studies as part of their clinical trials.
an article published by the New England Journal of Medicine, Croog et
al. (1986) reported that, in a randomized double-blind clinical Dial, pa-
tients who took the oral antihypertensive pharmaceutical agent captopn]
enjoyed a higher quality of life Man Dose taking propranolo} or meth-
yidopa. Specifically, patients who took captor, as compared with
patients who took methyidopa, "scored significantly higher on measures
of general weB-being, had fewer side effects, and had better scores for
work performance, visual-motor functioning, and measures of life satis-
fac{ion.,' Patients who took propranolo] experienced intermediate well-
being compared Offs Hat when Hey took He two over agents.
A few months later, Bombardier et al. (1986) published In The Ameri-
can Journal of Medicine He results of a clinical trial Hat assessed He
effects of auranofin, a phannaceu~cical agent used to treat rheumatoid
arthritis, on patients' quality of life. In a double-blind study at 14 centers,
He effects of auranofin were compared win Lose of a placebo in tile
treatment of patients win classic or definite rheumatoid arthritis. The au-
ranofin group, as a whole, experienced relatively higher frequencies of
adverse effects, but such events were usually mild and transient More
importantly, *om He investigators' point of view, a greater proportion of
He auranofin-treated patients Han of the placebo-treated patients reported
a "marked improvement" In their mobility, including their ability to walk,
climb stairs, and raise unaided from a siding position.
These studies are important for several reasons. First, they indicate
Hat quality-of-life measures are considered an increasingly important part
OCR for page 57
MEASURES IN THE PRIVATE SECTOR
57
of clinical Dials, despite He lack of consensus on the meaning and opera-
tionalization of this concept. Second, Hey reinforce He argument favor-
ing an increased role for quality-of-life considerations In cI~n~cal deci-
sionmaking. Finally, Hey suggest that quali~r-of-life studies have poten-
tial marketing value.
Food and Drug Administration Perspective
Quality of life is a widely discussed concept Hat elicits a variety of
opinions. Its relative utility is debated by researchers in the field. Investi-
gators who use quality-of-life measures clearly believe they are a valuable
tool. Others, however, contend that they seem to be indistinguishable
from over measures routinely used ~ assess drug safely and efficacy.
Indeed, In the course of several interviews, officials at the FDA suggested
that quality-of-life instruments have as Heir focus aspects of tests already
in use to target side effects. In over words, Hey believe that these
measures are not particularly new but have merely been placed under a
new rubric.
The FDA has no specific quality-of-life regulatory requirements, In
large part because He agency believes that He research community Hat
has developed and refined quality-of-life scales has not been able to show
unequivocaBy Hat the ~nstrmnents are "sufficiently credible." As one
FDA official noted, highly refined measures are required to differentiate
the effects of a drug from He effects of He disease it is meant to treat.
Nevertheless, FDA of fiches express interest In better understanding qual-
ity of life, although Hey consider the state of the art too immature to
warrant mandatory inclusion in clinical teals.
This is not to suggest that He FDA has entirely dismissed quality of life
as a potentially import factor in clinical teals. One FDA official noted
Hat the usefulness of these measures lies in He attention given to He
"downside" of drugs. Although side effects have been recorded, He
broader notion of impact on a person's life has not been shriek. Qualit~r-
of-life scales could be useful, he continued, if they were refined to detect
subtle distinctions among pharmaceutical agents. CThis view can be con-
trasted wad the findings of the reports given as examples in Chapter 2.)
Thus, although the FDA seems to be interested in me concept, it
remains unconvinced of its ultimate validity. The results of our survey of
pharmaceutical companies (see discussion that follows) suggest, never-
theless, Hat there is a perceived advantage to incorporating quality-of-life
measurement in clinical drug chats; it is thought to increase the likelihood
OCR for page 58
58
BE. LUCK, ~M. W=CH~, ID C. UNDERWOOD
of FDA approval. Some workers in this field believe Hat Be FDA has
acmaDy mandated Me use of such series.*
The FDA is closely mon~tonng He use of quality-of-life measures In
clinical trials and He incorporation of quality~f-life claims in advertising
and comparative claims. Pharmaceutical companies often make such
chums to trier to show that their product has fewer adverse side effects than
those of their competitors. Although pharmaceutical companies are al-
lowed to incorporate these claims on their labels, they must present weB-
sllpported data. The FDA is particularly skeptical of vague claims and has
objected in the past to assertions mat a drug is "padent-fnendly."
Private Industry Perspective
Some spokespersons in the private sector were forthcoming in re-
sponding to questions about Heir use of quality~f-life instruments in
clinical Dials; others were reluctant, a result of He highly competitive
nature of pnv ate industry. Based on informal and formal conversations
with research scientists at several pharmaceutical companies, we deter-
m~ned that researchers in the private sector share a general interest in the
use of these measures in clinical Dials. The next four to five years are
expected to produce a proliferation of die use of quality-of-life instru-
ments to support claims that one drug is superior to another in this
important respect. We also detected a sense among individuals in private
industry that consumers as wed as physicians show a growing interest in,
and awareness of, the venous effects of medications on life quality. For
these reasons, many private pharmaceutical compares have made He
explicit decision to use quality-of-life measures in clinical trials.
~ an interview with one industry spokesperson, we reamed Hat their
research scientists are currently using quality-of-life instruments in cliIii-
cal trials of several drugs developed to palliate the symptoms of chronic
diseases. He reported furler that company research scientists have made
an explicit clinical policy decision to consider quality-of-life components
in ad clinical teals. He stated Hat He emphasis on quality of life comes
from cost-containment considerations, the need for ~ird-party cost justi-
ficabon, and from competition among similar agents. He believes that
* FOA officials have not indicated that qualigr~-life studies are required for premarket approval
Nevertheless, Battelle is conducting a quaky-of-life study and is about to begin another at the tone of
dais writing. Both are part of Phase m clinical teals. The FDA has reportedly requested that the
company submit quality-of-life data
OCR for page 59
MEASURES IN THE PRIVATE SECTOR
59
quality-of-life measures allow the company to demonstrate mat their
product is superior to another similar agent in the traditional market.
RESULTS OF THE QUALITY-OF-LIFE SURVEY
In cooperation with me Pharmaceutical Manufacturers Association
(PMA) and the Heath Industry Manufacturers Association (HIMA), Bat-
teDe conducted a survey of pharmaceutical and medical companies to
determine how widely quality-of-life instruments are being used in the
private sector. Bow He PMA and HIMA agreed to send BatteBe's survey
questionnaire to a subset of Heir respective memberships.
The queshonna~re was designed to produce an estimate of the number
of companies that have used or are currently using quality-of-life instru-
ments In the conduct of their clinical teals of drugs and devices and to
team whether they plan to continue using them. The questionnaire also
probes the reasons companies are or are not using these instruments and
asks what types of specific insurgents are being used.
Pharmaceutical Industry
The Pharmaceutical Manufacturers Association sent the BaueDe ques-
tionnaire to a total of 61 pharmaceutical companies, representing approxi-
mately two-~irds of its membership. Thirty-four companies (56 percent)
responded to He questionnaire. Highlights of He results are presented in
Table 5-~.
Of He 34 companies responding, 21 (62 percent) reported they have
used some type of quality-of-life instrument in Heir clinical trials of
drugs. All but one reported they are currently using such instruments.
~ this survey, He two most frequently cited reasons for using quality-
of-life ~nstnaments In cI=cal Ends are marketing considerations and
internal management or clinical decisionmaking. One company pointed
out Hat quality-of-life measurement is one way to help determine a drug's
efficacy when a complicated disease state is present. About one-half of
He comparues believe mat He likelihood of FDA approval will be in-
creased if such measures are used. Some report Hat quality-of-life studies
are required for FDA approval, although this may be a misperception.
Somewhat less Man one-half of the companies consider having publica-
tions in scientific journals an important reason to conduct these studies.
The pharmaceutical companies represented in this sample are using
several other instruments in addition to me genera, standardized research
OCR for page 60
60
BE. WOE, Jet. WESCH~:R, AND C. UNDERWOOD
TABLE 5-1 Highlights of Survey Results on Q;uality~f-Life Measure-
ment by Pharmaceutical Fames
Company
Actrvity
Total
Number of Number
Con~parues Reporting Percent
Companies Using
Qualib~of-Life
Instrument
Have used 34 21 62
Are currency using 34 20 59
Reasons for use 21
1. Marketing considerations
2. Internal managementlclinical decisic~nmaldug
3. Creased likelihood of FDA approval
- 4. Publications in scientific journals
5. FDA requirements for approval
Standardized instrument used
1. General Heals Rating Index (GHRI)
2. Quality of Well-Being (QWB) Index
3. General Well-Being (GWB) Index
4. Nottingham Beady Profile (NHP)
5. Sickness hnpact Profile (Sip)
6. McMasterHeal~dex
21
Developed own quality~f-life instrument(s) 21
Specific to drug 14
Bow general and specific
Criteria used in selecting quality~f-]ife ins~ument(s) 21
Validity
Reliability
Sensitivity
Specificity
Length
Comprehensiveness
Cost
Will continue to use
15
13
10
8
4
4
3
3
3
2
2
14
11
18
16
15
12
13
11
9
71
62
48
38
19
19
14
14
14
10
10
67
79
36
86
76
71
57
62
52
43
quality-of-life instruments 21 100
Companies Not Using
Quality~f-Life Instruments
Have never used
Reasons for nonuse
1. Not relevant
2. Too expensive
3. Not aware of ins~nents
34 13 38
13
6 46
8
8
Table 5-1 continues
OCR for page 61
MEASURES nv TlIE PRIVATE SECTOR
TABLE 5-1 (Continue
61
Total
Company Number of Number
Active Compazues Reporting Percent
l
Will use in future 13 9 69
Reasons 9
1. Marketing considerations 9 100
2. Creased likelihood of
FDA approval 9 100
3. Publications in scientific journals 7 78
4. ~t~nalmanagement/
clinical decisiomnalcing 5 56
5. FDA requirements for approval 2 22
NOTE: Surveys wem strut to 61 companies; 34 responded.
tools we listed In our survey (see Table 5-~). Although each of these all
also use other quality-of-life scales. Among Hose listed are the Beck
Depression Inventory, Dupuy Life Satisfaction, Wechsler Memos Scale,
Fleming Self-Esteem Hospice Anxiety and Depression Scale, and Women's
Heath Questionnaire.
Fourteen (67 percent) companies have developed Heir own quality-of-
life instruments, and He majority of these have been specific to He drug
or disease state under consideration. In our survey, He greatest number of
instruments developed by He companies themselves pertained to heart
disease, hypertension, and congestive heart failure. Companies also men-
tioned that Hey had designed scares related to sexual dysfunction, gastro-
~ntestina] disorders, and cancer.
Enters used to select or develop a quality-of-life measure including
validity, reliability, sensitivity, specificity, and leng~are cited by at
least half of the companies surveyed that have used such measures. Less
than half (43 percent) of the comparues acknowledged cost as a cntenon.
Also listed as important considerations were ease of adrnin~stration and
scoring, simplicity and time of admin~suabon, and the need to evaluate
the padent's cognitive state.
Of the 34 comparues responding to the questionnaire, 13 have never
used a quality-of-life instrument In Heir cI~rucal trials. Nine (69 percent)
of these companies, however, report that Hey plan to use diem in the
future; four (31 percent) do not. The most *equendy cited reason for not
using these instruments is Hat they are not relevant to He particular thug
or disease state.
OCR for page 62
62
BE. LUCK, JM. WESCHLER, AND C. UNDERWOOD
Several respondents commented ~at, until recently, quality-of-life stud-
ies have simply not been an issue in certain therapeutic areas or have not
been considered necessary to confien efficacy and safety. Only one
respondent cited cost as a reason for not sponsoring such studies. Most of
We companies In this group cited marketing considerations and increased
likelihood of FDA approval as reasons for using such instruments In me
future. Over half included publications in scientific journals as a reason.
A few respondents anticipate increased attention to quality-of-life studies
from He FDA forte company mentioned oncology specifically).
Medical Device Companies
The Hearth Industry Manufacturers Association (HIMA) sent Bat-
tede's questionnaire to a sample of 25 member companies. This sample
was selected by HIMA's Health Care Financing Committee and is consid-
ered to be representative of Heir membership as a whole.
Only six medical device companies responded to the questionnaire,
and only one reported using qualin,r-of-life instruments in their clinical
trials of devices. Two companies said that Hey plan to use them in He
future, and Tree do not Reasons cited for not using such instnunents are
that they are not relevant or the company has not been aware of ~em.
A second mailing conducted by HIMA yielded no additions responses
from He sample of medical device companies. That ~ree-qua~ters of the
medical device companies did not respond to He questionnaire suggests
low salience and sparse usage of quality~f-life instruments in the device
sector, especially compared win the drug sector.
CONCLUSIONS
Quality-of-life instruments are being more widely used and more thor-
oughly debated Can ever before. The industry-we trend to use these
measures in clinical trials has been noticeable dung the past Bee years.
Researchers in He field expect this trend to continue to be strong and ~at,
ultimately, usage win become routine. These studies can be expected to
continue to gain importance in He coming years, bow in the public and
private sectors and in assessing the comparative effects of different medi-
cal interventions on padents. Therefore, instruments designed to measure
quality of life win be subjected to increasingly sophisticated refinement
and elaboration, even as the theoredca] debate about He meaning of
quality of life persists. The continued emphasis on, and development of,
OCR for page 63
MEASURE ~ THE PRWA~ SPOOR
63
quality-of-life instruments can be expected to have significant marketing
value for He private sector and to contribute to more humane hemp care
services.
Nevertheless, because it Is difficult to grasp a complex concept and
even more challenging to capture it In a measurement instrument, dis-
agreements win persist about quality of life and its quantification. This
ongoing struggle Ural Be concept of quality of life and its ramifications
should continue to prove fruitful.
REFERENCES
Bombardier, C., Ware, J., RusselE, I.J., Larson, M., Chalmers, A., and
Read, J.L. Aurarlofin therapy and quality of life in padents win
rheumatoid arthritis. Results of a muldcenter Dial. The American
Joumal of Medicine 81~4~:565-578, 1986.
Croog, S.H., ~vine, S., Testa, M.A., Brown, B., Bulpiu, C.J., Jenkins,
C.D., Klennan, G.~., and Williams, G.H. The effects of andhy-
pertensive therapy on me quality of life. New England Journal of
Medicine 314(26):1657-1664, 1986.
SELECTED FURTHER READING
Anderson, J.P., Bush, J.W., and Betty, C.C. Classifying function for
health outcome and quality of life evaluation. Medical Care 24~5~:454-
469, 1986.
Bergman, L.F., and Syme, SO Social networks, host resistance, and
moronity: A n~ne-year foBow-up study of Alameda County Resi-
dents. American Journal of Epidemiology 109~2~:186-204, 1979.
de Haes, I.C., and Welvaart, K. Quality of life after breast cancer surgery.
Joumal of Surgical Oncology 28~2~:123-125, 1985.
Evans, R.W., Manninen, D.I-., Garrison, L.P., Jr., Hart, L.G., Blagg, C.R.,
Gunman, R.A., Hull, A.R., arid L`ownie, E.G. The quality of life of
patients wad end-stage renal disease. New England Joumal of Medi-
cine 312~9~:553-559, 198S.
Kutner, N.G., Brogan, D., and Kutner, M.H. End-stage renal disease
trea~anent modality and patients' quality of life. American Joumal of
Nephrology 6(53:396402, 1986.
Lastly, J.C., Margolese, R.G., Poisson, R., Shibata, H., FIeischer, D.,
Lafleur, D., Legault, S., and Taillefer, S. Depression arid body image
following mastectomy and lumpectomy. Journal of Chronic Diseases
40~6~:529-534, 1987.
OCR for page 64
64
BE. WCE, JM. WESCHLER, AND C. UNDERWOOD
Levine, S., and Croog, S.H. What conshmms quality of life? A concepm-
alizabon of me dimensions of life quality In hearty populations and
patients win cardiovascular disease. In Wenger, N.K., Mattson,
M.E., Furberg, C.D., and Elinson' I., eds. Assessment of Quality of
Life in Clinical Tnals of Cardiovascular Therapies. New York, Le
lacq Publishing, ~c., 1984.
Moms, J.N., and Sherwood, S. Quality of life of cancer padents at
different stages In me disease ~ajecto~y. Journal of Chronic Diseases
40~61:545-556, 1987.
Priesanan, T.J., and Baum, M. Evaluation of quality of life in padents
receiving treatment for advanced breast cancer. Lancer 1~7965~:899-
900, 1976.
Schipper, H., and Levitt, M. Measuring quality of life: Risks and
benefits. Cancer Treatment Reports 69(10):1116, 1985.
Schuessler, K.F., and Fisher, G.A. Quality of life research and sociology.
AnnuaIReviewof Sociology 11:129-149, 1985.
Siegnst, I. Impaired quality of life as a risk factor in cardiovascular
disease. loumal of Chic Diseases 40~6~:571-578, 1987.
Spitzer, W.O., Dobson, A.~., Hall, I., Chesterman, E., Levi, I., Shepherd,
R., Banista, R.N., and Catchlove, B.R. Measuring the quality of life
of cancer patients: A concise QL-index for use by physicians. Jour-
na1 of Chronic Diseases 34~12~:585-597, 1981.
Troidl, H., Kusche, I., Vestweber, K.H., Eypasch, E., Koeppen, Lo., and
Bouillon, B. Quality of life: An important endpoint both in surgical
practice and research. journal of Chronic Diseases 40~6~:523-528,
1987.
WoAd Heath Orgaruzadon. Constitution of He World Heath Organ~za-
tion. In: Basic Documents. Geneva, WHO' 1947.
Representative terms from entire chapter:
pharmaceutical companies
