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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

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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

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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

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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

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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

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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

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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.

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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,

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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.

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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.