National Institute for Clinical Excellence and its value judgments
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《英国医生杂志》
1 Wolfson Unit of Clinical Pharmacology, Medical School, University of Newcastle upon Tyne, Newcastle NE2 4HH, 2 Department of Economics and Related Studies, University of York, York YO10 5DD
NICE has to make both scientific and social value judgments when appraising health technologies and developing clinical guidelines for the NHS. Here, its chair and previous vice chair explain the rationale behind the decisions
Introduction
Social value judgments have a critical role if resources are to be distributed with efficiency and equity. NICE and its advisory bodies, however, have no particular legitimacy to determine the social values of those served by the NHS. To ensure that these values resonate broadly with the public, NICE has formed a Citizens Council.11 12
Efficiency
A fundamental value judgment is that efficiency in health care involves maximising the health of the population subject to the resources available. The main social value judgments regarding efficiency relate to the measure of health used and to the scope of costs and benefits. NICE uses the QALY as the principal measure of health outcome. This measure embodies the important social value judgment that to count only gains in life expectancy, without considering the quality of the additional life years, omits important dimensions of human welfare.11 The QALY has the advantage of having been extensively validated in experimental conditions.13-17 The main value judgments embodied in QALYs are that health related quality of life can reasonably be captured in terms of physical mobility, ability to self care, ability to carry out activities of daily living, absence of pain and discomfort, and absence of anxiety and depression.
NICE believes that, while differential productivity at work should be considered, it ought not be used to disadvantage people who are not in regular paid employment, including children and those who are retired.11 It needs to explore how best to reflect productivity effects without causing inequity in the ways in which services are allocated.
It is sometimes held that NICE ought to give a higher priority to novel treatments for conditions for which no alternative specific forms of therapy are currently available, or to conditions associated with social stigma such as mental illness or sexually transmitted diseases.11 These, too, are social value judgments that need to be considered in more detail in the future.
Equity
Equity lies at the heart of the NHS. Lack of equity (in the form of so called postcode prescribing) was one of the reasons why NICE was established. Much of the philosophical literature on equity is far from being applicable to the real world.18 19 NICE has therefore had to make its own judgments. For NICE, equity also refers to fairness in the ways in which the costs and benefits of available care are distributed among all those who use the NHS.19-21 NICE's recommendations are intended to apply across the whole of England and Wales, regardless of where people live or work. Thus, NICE has made the social value judgment that local variations in cost ought not to result in variations in availability of health care.11
Value judgements about equity are often implicit within both clinical and cost effectiveness analyses. An assumption that underlies most of NICE's technology appraisals has been that "a QALY is a QALY is a QALY." By this NICE means that a QALY gained or lost in respect of one disease is equivalent to a QALY gained or lost in respect of another. It also means that the weight given to the gain of a QALY is the same, regardless of how many QALYs have already been enjoyed, how many are in prospect, the age or sex of the beneficiaries, their deservedness, and the extent to which the recipients are deprived in other respects than health. The decision to give no differential weight is the result of a social value judgment that an additional adjusted life year is of equal importance for each person.12
Summary points
NICE exists to give health professionals advice on providing their NHS patients with the highest clinical standards of care
It undertakes its economic assessments using a cost utility approach (cost per quality adjusted life year)
Decisions about cost effectiveness are made on a case by case basis
Judgment is needed to balance the tensions between efficiency and equity
The Citizens Council has also considered how NICE might take account of age in its considerations of clinical and cost effectiveness.12 The council recommends that age should be taken into account when it is an indicator of either risk or benefit. It does not recommend, though, that NICE should be more generous in its judgments of cost effectiveness merely because of individuals' social roles or age.
Conclusions
National Institute for Clinical Excellence. A guide to NICE. London: NICE, 2003.
National Institute for Clinical Excellence. Scientific and social value judgements. London: NICE, 2004. www.nice.org.uk/Pdf/boardmeeting/brdmay04item6.pdf (accessed 15 Jun 2004)
National Institute for Clinical Excellence. Guidance on the use of fludarabine for B-cell chronic lymphatic leukaemia. London: NICE, 2002. (Technology appraisal No 29.) www.nice.org.uk/page.aspx?o=22179 (accessed 25 Jun 2004).
Weinstein ML. From cost effectiveness ratios to resource allocation: where to draw the line. In: Sloan FA, ed. Valuing health. Cambridge: Cambridge University Press, 1995.
Towse A, Pritchard C, Devlin N, eds. Cost effectiveness thresholds: economic and ethical issues. London: Office of Health Economics, Kings Fund, 2002.
Laupacis A, Feeny D, Detsky AS, Tugwell PX. How attractive does a new technology have to be to warrant adoption and utilisation? Tentative guidelines for using clinical and economic evaluations. CMAJ 1992;146: 473-81.
Culyer AJ. The rationing debate: maximising the health of the whole community. BMJ 1997;314: 667-9.
National Institute for Clinical Excellence. Anakinra for rheumatoid arthritis. London: NICE, 2003. (Technology appraisal No 72.) www.nice.org.uk/page.aspx?o=94670 (accessed 25 Jun 2004).
National Institute for Clinical Excellence. Beta interferon and glatiramer acetate for the treatment of multiple sclerosis. London: NICE, 2002. (Technology appraisal No 32.) www.nice.org.uk/page.aspx?o=27588 (accessed 25 Jun 2004).
National Institute for Clinical Excellence. Guidance on the use of imatinib for chronic myeloid leukaemia. London: NICE, 2002. (Technology appraisal No 50.) www.nice.org.uk/page.aspx?o=37604 (accessed 25 Jun 2004).
NICE Citizens Council. Report of the first meeting: Determining clinical need. London: NICE, 2003. www.nice.org.uk/pdf/FINALNICEFirstMeeting_FINALReport.pdf (accessed 25 Jun 2004).
NICE Citizens Council. Report on age. London: NICE, 2003. www.nice.org.uk/pdf/Citizenscouncil_report_age.pdf (accessed 25 Jun 2004).
Brazier JN, Jones N, Kind P. Testing the validity of the Euroqol and comparing it with the SF-36 health survey questionnaire. Qual Life Res 1993;2: 169-80.
Anderson RT, Aaronson NK, Bullinger M, McBee WL. A review of the progress towards developing health-related quality-of-life instruments for international clinical studies and outcomes research. Pharmacoeconomics 1996;10: 336-55.
Brooks R. EuroQol: the current state of play. Health Policy 1996;37: 53-72.
Roset M, Badia X, Mayo NE. Sample size calculations in studies using the EuroQol 5D. Qual Life Res 1999;8: 539-49.
Kind P, Hardman G, Macran S. UK population norms for EQ-5D. York: Centre for Health Economics, 1999 (Discussion paper No 172).
Culyer AJ, Wagstaff A. Equity and equality in health and health care. J Health Econ 1993;12: 431-57.
Culyer AJ. Economics and ethics in healthcare. J Med Ethics 2001;27: 217-22.
Culyer AJ. Equity—some theory and its policy implications. J Med Ethics 2001;27: 275-83.
Culyer AJ. Need: the idea won't do—but we still need it. Soc Sci Med 1995;40: 727-30.(Michael D Rawlins, profes)
NICE has to make both scientific and social value judgments when appraising health technologies and developing clinical guidelines for the NHS. Here, its chair and previous vice chair explain the rationale behind the decisions
Introduction
Social value judgments have a critical role if resources are to be distributed with efficiency and equity. NICE and its advisory bodies, however, have no particular legitimacy to determine the social values of those served by the NHS. To ensure that these values resonate broadly with the public, NICE has formed a Citizens Council.11 12
Efficiency
A fundamental value judgment is that efficiency in health care involves maximising the health of the population subject to the resources available. The main social value judgments regarding efficiency relate to the measure of health used and to the scope of costs and benefits. NICE uses the QALY as the principal measure of health outcome. This measure embodies the important social value judgment that to count only gains in life expectancy, without considering the quality of the additional life years, omits important dimensions of human welfare.11 The QALY has the advantage of having been extensively validated in experimental conditions.13-17 The main value judgments embodied in QALYs are that health related quality of life can reasonably be captured in terms of physical mobility, ability to self care, ability to carry out activities of daily living, absence of pain and discomfort, and absence of anxiety and depression.
NICE believes that, while differential productivity at work should be considered, it ought not be used to disadvantage people who are not in regular paid employment, including children and those who are retired.11 It needs to explore how best to reflect productivity effects without causing inequity in the ways in which services are allocated.
It is sometimes held that NICE ought to give a higher priority to novel treatments for conditions for which no alternative specific forms of therapy are currently available, or to conditions associated with social stigma such as mental illness or sexually transmitted diseases.11 These, too, are social value judgments that need to be considered in more detail in the future.
Equity
Equity lies at the heart of the NHS. Lack of equity (in the form of so called postcode prescribing) was one of the reasons why NICE was established. Much of the philosophical literature on equity is far from being applicable to the real world.18 19 NICE has therefore had to make its own judgments. For NICE, equity also refers to fairness in the ways in which the costs and benefits of available care are distributed among all those who use the NHS.19-21 NICE's recommendations are intended to apply across the whole of England and Wales, regardless of where people live or work. Thus, NICE has made the social value judgment that local variations in cost ought not to result in variations in availability of health care.11
Value judgements about equity are often implicit within both clinical and cost effectiveness analyses. An assumption that underlies most of NICE's technology appraisals has been that "a QALY is a QALY is a QALY." By this NICE means that a QALY gained or lost in respect of one disease is equivalent to a QALY gained or lost in respect of another. It also means that the weight given to the gain of a QALY is the same, regardless of how many QALYs have already been enjoyed, how many are in prospect, the age or sex of the beneficiaries, their deservedness, and the extent to which the recipients are deprived in other respects than health. The decision to give no differential weight is the result of a social value judgment that an additional adjusted life year is of equal importance for each person.12
Summary points
NICE exists to give health professionals advice on providing their NHS patients with the highest clinical standards of care
It undertakes its economic assessments using a cost utility approach (cost per quality adjusted life year)
Decisions about cost effectiveness are made on a case by case basis
Judgment is needed to balance the tensions between efficiency and equity
The Citizens Council has also considered how NICE might take account of age in its considerations of clinical and cost effectiveness.12 The council recommends that age should be taken into account when it is an indicator of either risk or benefit. It does not recommend, though, that NICE should be more generous in its judgments of cost effectiveness merely because of individuals' social roles or age.
Conclusions
National Institute for Clinical Excellence. A guide to NICE. London: NICE, 2003.
National Institute for Clinical Excellence. Scientific and social value judgements. London: NICE, 2004. www.nice.org.uk/Pdf/boardmeeting/brdmay04item6.pdf (accessed 15 Jun 2004)
National Institute for Clinical Excellence. Guidance on the use of fludarabine for B-cell chronic lymphatic leukaemia. London: NICE, 2002. (Technology appraisal No 29.) www.nice.org.uk/page.aspx?o=22179 (accessed 25 Jun 2004).
Weinstein ML. From cost effectiveness ratios to resource allocation: where to draw the line. In: Sloan FA, ed. Valuing health. Cambridge: Cambridge University Press, 1995.
Towse A, Pritchard C, Devlin N, eds. Cost effectiveness thresholds: economic and ethical issues. London: Office of Health Economics, Kings Fund, 2002.
Laupacis A, Feeny D, Detsky AS, Tugwell PX. How attractive does a new technology have to be to warrant adoption and utilisation? Tentative guidelines for using clinical and economic evaluations. CMAJ 1992;146: 473-81.
Culyer AJ. The rationing debate: maximising the health of the whole community. BMJ 1997;314: 667-9.
National Institute for Clinical Excellence. Anakinra for rheumatoid arthritis. London: NICE, 2003. (Technology appraisal No 72.) www.nice.org.uk/page.aspx?o=94670 (accessed 25 Jun 2004).
National Institute for Clinical Excellence. Beta interferon and glatiramer acetate for the treatment of multiple sclerosis. London: NICE, 2002. (Technology appraisal No 32.) www.nice.org.uk/page.aspx?o=27588 (accessed 25 Jun 2004).
National Institute for Clinical Excellence. Guidance on the use of imatinib for chronic myeloid leukaemia. London: NICE, 2002. (Technology appraisal No 50.) www.nice.org.uk/page.aspx?o=37604 (accessed 25 Jun 2004).
NICE Citizens Council. Report of the first meeting: Determining clinical need. London: NICE, 2003. www.nice.org.uk/pdf/FINALNICEFirstMeeting_FINALReport.pdf (accessed 25 Jun 2004).
NICE Citizens Council. Report on age. London: NICE, 2003. www.nice.org.uk/pdf/Citizenscouncil_report_age.pdf (accessed 25 Jun 2004).
Brazier JN, Jones N, Kind P. Testing the validity of the Euroqol and comparing it with the SF-36 health survey questionnaire. Qual Life Res 1993;2: 169-80.
Anderson RT, Aaronson NK, Bullinger M, McBee WL. A review of the progress towards developing health-related quality-of-life instruments for international clinical studies and outcomes research. Pharmacoeconomics 1996;10: 336-55.
Brooks R. EuroQol: the current state of play. Health Policy 1996;37: 53-72.
Roset M, Badia X, Mayo NE. Sample size calculations in studies using the EuroQol 5D. Qual Life Res 1999;8: 539-49.
Kind P, Hardman G, Macran S. UK population norms for EQ-5D. York: Centre for Health Economics, 1999 (Discussion paper No 172).
Culyer AJ, Wagstaff A. Equity and equality in health and health care. J Health Econ 1993;12: 431-57.
Culyer AJ. Economics and ethics in healthcare. J Med Ethics 2001;27: 217-22.
Culyer AJ. Equity—some theory and its policy implications. J Med Ethics 2001;27: 275-83.
Culyer AJ. Need: the idea won't do—but we still need it. Soc Sci Med 1995;40: 727-30.(Michael D Rawlins, profes)