PEDIATRICS (ISSN 0031 4005). Copyright © 1990 by the American Academy of Pediatrics.
COMMENTARIES
Opinions expressed in these commentaries are those of the authors and not necessarily those of the
American Academy of Pediatrics or its Committees.
Cost-Effectiveness
Analysis
in
Pediatric
Practice
The physician’s first responsibility is to the pa-tient: to apply appropriate current medical knowl-edge to treat and prevent illness and to assure that risks and harm from intervention are less than those of the untreated disease. Physicians have a stake in society, as well, however, and this implies prudent use of health care dollars. New medical technologies can sometimes simultaneously im-prove individual health status and reduce costs to society, but more often new technologies pose a
dilemma for physicians and society. Health benefits come with a price tag. The benefit to the individual patient results in more costs in an already
overbur-dened health care budget. When better health costs extra, how can we make rational choices?
In the past two decades, analytic methods have arrived in health services research to help us make these difficult choices of cost, risk, and benefit. Decision analysis”2 and cost effectiveness analysis3 have reached new levels of scientific maturity, and their products now appear frequently in clinical publications. For reasons unclear to us, these ana-lytic methods are relatively underrepresented in the pediatric literature compared with journals of in-ternal medicine, surgery, and radiology, but the article in this issue by Lieu et al.4 moves toward correcting that imbalance. It provides an excellent example of decision analysis applied to a common pediatric problem: the detection and management of streptococcal pharyngitis. The authors go beyond a simple risk-benefit analysis by studying cost-effectiveness ratios as well.
A clinical decision analyst explicity identifies a set of plausible alternatives in a medical decision, estimates relevant probabilities (such as test per-formance features and the likelihood of various outcomes), and quantifies the values or utilities of
each expected outcome. The outcomes of interest may be confined to medical risks and benefits (in a risk-benefit analysis) or may also include direct and indirect costs and savings (in a cost-effectiveness analysis). In a full cost-effectiveness analysis, the decision maker compares options to each other through ratios (which are much like prices) such as costs per case treated, cost per case prevented, or cost per additional year of life added. When re-sources are limited, it makes logical sense to invest first in options that buy desired outcomes at the lowest price available; that strategy assures that the greatest achievable good comes from the avail-able resources.5
One can study a decision analysis by following the same steps the authors trace to create the analysis.
WHAT ALTERNATIVES ARE CONSIDERED IN
THE DECISION
TREE?
All decision analyses are arbitrarily bounded by the selection of some initial set of options. Lieu et al.4 consider four alternative strategies for manag-ing pharyngitis but omit an infinite number of other possible strategies. Limiting analysis to a few op-tions is not an error; indeed, it is necessary. The reader must consider whether the authors have omitted some essential fifth or sixth option which,
if it had been included, might have turned out to be the best choice, however.
WHAT PROBABILITIES DO THE AUTHORS USE?
362 PEDIATRICS Vol. 85 No. 3 March 1990
best guesses. These authors have reviewed the lit-erature regarding pharyngitis extensively, but ulti-mately they must make their own best guess to continue the analysis. In so doing, they inevitably omit or oversimplify potentially important evi-dence, like that provided by Pichichero et al,6 who found a higher recurrence rate among patients treated early for Streptococcus (37%) than among those treated late (17%). Inclusion of such infor-mation would place additional weight in favor of using a throat culture. Estimates concerning pre-vention of rheumatic fever rely on a single, good study of military recruits in Wyoming during an epidemic of rheumatic fever in the late 1940s. The only large prospective randomized trial of preven-tion in an endemic situation had insufficient sample size to document the efficacy of penicillin.8 It is not unreasonable to generalize based on a single well-done study, but the notion that we can prevent rheumatic fever in an endemic situation is still an assumption, not a proven fact. The reader must always ask whether the authors of a decision analy-sis have incorporated the best available evidence in their probability estimates.
WHICH OUTCOMES ARE INCLUDED AND WHICH
ARE NOT?
The decision analyst makes choices about the domains of outcome to explore. In the simplest cases, only one outcome, such as death, is analyzed, but many conditions raise other salient issues of quality of life, psychological wellbeing, functional
status, and level of pain. As outcomes become
sev-eral, utilities may compete with each other. Perhaps the risk of death from surgery must be weighed against the potential relief of pain: that is, there is a comfort-survival trade-off. These problems of multiattribute utility are among the most interest-ing and difficult in modern decision analysis re-search and in many ways are the most meaningful to the real world practitioner who knows well how often medical practice involves the balancing of competing goals for the patient.#{176} Lieu et al focus on at least four different clinical outcomes: rheu-matic fever, rheumatic heart disease, suppurative complications of pharyngitis, and allergic reactions to pencillin. They omit study of other outcomes of plausible interest: sick days, pain, and anxiety, for example. These omissions may be unwise; it was suggested in several studies, for example, that early treatment with antibiotics results in earlier symp-tom resolution,’#{176}” although other evidence sug-gests that the key to early symptom resolution rests with analgesics instead of antibiotics.12 The reader
must judge whether the outcomes analyzed are
in-deed those that the reader and the patient truly care about.
WHAT COSTS ARE INCLUDED? HOW ARE THEY
ASSESSED?
When decision analysis enters the terrain of cost-effectiveness analysis, as in the article by Lieu et al,4 choices are made about how to assess dollar costs and benefits. In their article, direct medical costs are included, but indirect costs such as patient time, work lost, and some forms of institutional overhead are not. These choices are subtle and important and become even more complex when one attempts to move from medical charges (which may not reflect true costs) to more accurate cost estimates. For example, many of the conclusions about costs in the study by Lieu et al4 are contingent on the antigen test being only 60% as costly as the throat culture.
IS SENSITIVITY
ANALYSIS
THOROUGH?
Because decision analyses depend on many esti-mates that do not have firm bases, it is customary to repeat the calculations of cost, risk, and benefit many times, letting the uncertain variables change through their range of uncertainty. This procedure is called sensitivity analysis, because it allows one to explore the sensitivity of the preferred choice within the range of current uncertainty. Sometimes the preferred options are shown to be “robust” with sensitivity analysis, that is, unlikely to change even if the base-case estimates are far from the truth. At
other times, particular variables are shown by sen-sitivity analysis to be especially crucial to the de-cision, and these variables become good candidates for further study or research to refine the estimates. In the article by Lieu et al,4 for example, the prey-alence of streptococcal disease in sensitivity analy-sis has little impact on the preferred treatment strategy, whereas the sensitivity of the antigen test and compliance with medication matters greatly. Consequently physicians should carefully monitor the sensitivity of new antigen tests. The informa-tion about compliance also has important clinical implications. In the article it is clearly indicated that as antigen test sensitivity decreases or patient compliance increases, a throat culture becomes the preferred strategy. For individual patients known
by their physicians to be reliable in taking pre-scribed medicines, a throat culture is an excellent choice. Although physicians can do little to improve a test’s sensitivity, they do have the power to at-tempt to improve compliance. Spending a few sec-onds discussing the importance of adhering to pre-scribed regimens may have additional benefits in
at Viet Nam:AAP Sponsored on September 7, 2020
www.aappublications.org/news
teaching appropriate health behavior to parents and children and demonstrating concerns that can enhance the therapeutic alliance.
The authors conclude that the preferred strategy for management of streptococcal pharyngitis is an-tigen test alone if the antigen test sensitivity is greater than 0.71 and is antigen test + culture if the antigen test sensitivity is 0.71 or less. The strength of this conclusion for any particular reader serving any particular patient depends not just on the formal methods of the authors, which are in this case excellent, but also on the degree to which the many hidden assumptions of decision analysis are also credible and applicable to that patient. Were the correct options considered? Were the probability estimates well founded given the state of the medical literature? Were the appropriate outcomes considered and are they representative of this patient’s utilities? Were appropriate costs and savings correctly estimated? Did sensitivity analy-sis show the preferred choices to be robust under the current level of uncertainty? Only when the reader has asked and satisfactorily answered these questions is it prudent to take the published con-clusion-that strategy “A” is preferred to strategy “B”-and bring it into the office in service to the patient and to society at large.
The dilemma is that it may be unrealistic to expect that a physician’s principal motivation and a patient’s primary concerns both coincide with an analytic model in which all benefits and costs are altruistically distributed equally in determining what is best for society. It can be argued that in the management of sore throats, although the amount of uncertainty about options, assumptions, and probabilities is considerable, the actual cost differ-ence between the options is small. For the price of a fast food hamburger, we can actually offer an individual patient the technically ideal combination of an antigen test and culture instead of the slightly less accurate procedure of an antigen test alone. However, there is considerable aggregate social im-pact of using even a slightly costlier procedure at higher frequency: if we do spend the extra two dollars for every patient with a sore throat, we will without doubt be deciding not to buy something else.
How do physicians decide how to balance their responsibilities to an individual patient with their social responsibility? Is there a subtle breach in patient-physician contract if a physician makes a cost-saving choice knowing fully well that he or she is increasing the likeihood that the patient will acquire a preventable disease? Should it be the physician, the patient, or society at large who is the principal decision maker in whether the benefit
accrued from an additional two-dollar cost is worth-while? Is good societal cost containment always the best way for physicians to execute their responsi-bilities toward an individual patient? How do we account for the cost as well as physician and patient chagrin and regret if we miss an opportunity to prevent a serious disease?’3 These are questions that go beyond the answers provided by decision analysis.
Decision analysis is a tool to support decision, not a replacement for judgment. In the Lieu et al article, it is the authors’ best guess that their pre-ferred strategy, antigen test alone, can prevent rheumatic fever at a cost of $52 208 per case pre-vented and severe rheumatic heart disease at more than $1 million per case prevented. The wisdom of these investments in prevention is an issue that goes far beyond formal analytic techniques. Such choices raise questions of ethics, social justice, and competing societal values that no decision analysis can resolve. In the end, decision analysis and cost-effectiveness analysis are windows through which the physician, the patient, and society can more clearly see the choices available to them. They offer little escape from the challenge and pain of choice itself.
REFERENCES
ROBERT H. PANTELL, MD Division of General Pediatrics University of California San Francisco
DONALD M. BERWICK, MD Harvard Community Health Plan Brookline, Massachusetts
1. Weinstein, MC, Fineberg HV, Elstein AS, et al. Clinical Decision Analysis. Philadelphia, PA: WB Saunders Corn-pany; 1980
2. Pauker SG, Kassirer, JP. Decision analysis. N EngI J Med.
1987;316:250-257
3. Weinstein MC, Stason WB. Foundations of cost-effective-ness analysis for health and medical practices. N EngI J
Med. 1977;296:716-721
4. Lieu JA, Fleisher GR, Schwartz JS. Cost-effectiveness of rapid latex agglutination testing and throat culture for strep-tococcal pharyngitis. Pediatrics 1989;85:246-256
5. Doubilet P, Weinstein MC, McNeil BJ. Use and misuse of the term “cost-effective” in medicine. N EngI J Med. 1986;314:253-256
6. Pichicerco ME, Disney FA, Talpey WB, et al. Adverse and beneficial effects of immediate treatment of group A beta-hernolytic streptococcal pharyngitis with penicillin. Pediatr Infect Dis J. 1987;6:635-643. Pantell 3
7. Denny FW, Wannamaker LW, Brink WR, et al. Prevention of rheumatic fever. JAMA. 1950;143:151-160
8. Siegel AC, Johnson EE, Stollerman EG. Controlled studies of streptococcal pharyngitis-a pediatric population. N Erigl J Med. 1961;265:559-564
PEDIATRICS (ISSN 0031 4005). Copyright © 1990 by the American Academy of Pediatrics.
364 PEDIATRICS Vol. 85 No. 3 March 1990
laryngeal cancer. N EngI J Med. 1981;305:982-987
10. Krober MS, Bass JW, Michels GN. Streptococcal pharyn-gitis: placebo-controlled double-blind evaluation of clinical response to penicillin therapy. JAMA. 1985;235:1271-1274
11. Randolph MF, Gerber MA, DeMeo KK, et al. Effect of antibiotic therapy on the clinical course of streptococcal pharyngitis. J Pediatr. 1985;106:870-875
12. Middleton DB, D’Amico F, Merenstein JH. Standardized symptomatic treatment versus penicillin as initial therapy for streptococcal pharyngitis. J Pediatr. 1988;1 13:1089-1094 13. Feinstein AR. The “chagrin factor” and qualitative decision
analysis. Arch Intern Med. 1985;145:1257-1259
The Pediatrician
as a Role
Model
in the Career
Choice
of
Medical
Students
The results of the recent residency matching program revealed a decline in the number of US medical school graduates applying for positions in pediatric training programs. In 1987, 1366 gradu-ating seniors, 10% of the graduating class, applied for 2009 PL 1 positions. In 1989, 1256, 9.3% of those graduating, applied for 2068 positions. Sixty-eight percent of PL 1 positions were filled by grad-uates of US medical schools in 1987, and an addi-tional 14% (284) by foreign medical graduates. In
1989, 61% of the available positions were filled by
US graduates and 15% (317) by foreign medical graduates.1
In part, the lower percentage of positions filled is the result of an increase in the number of avail-able positions. There has also been a slight decline in the total number of US medical school graduates
entering the matching program. Nevertheless, the figures indicate a downward trend in the number of students applying for PL 1 positions. This has had a significant effect on pediatric training pro-grams in many parts of the country.
There are those who would argue that there are too many training programs, and undeniably there are programs of marginal quality. There are others who believe that there is a surplus of pediatricians and that a decrease in number is desirable. The Graduate Medical Education National Advisory Committee report published in 1982 projected a surplus of 7500 pediatricians by 1990.2 Subse-quently an Academy critique pointed out flaws in
the study and challenged the conclusions.3 At a recent hearing of the newly appointed Committee on Graduate Medical Education, members of the American Academy of Pediatrics and the pediatric
research societies testified “that because of the
uncertainties surrounding pediatric manpower needs, pediatrics is not an oversupplied specialty.” The statement concluded with the recommendation “that the current number of pediatric residents be maintained until data are found to substantiate the need for either an increase or a decrease in the numbers of residents based upon changes in chil-then’s health care needs.”4
Although there may be differences of opinion about the number of pediatricians required to meet this country’s child health needs, there is general agreement that efforts should be made to attract the most highly qualified students into pediatrics.
How can this be accomplished? The American Academy of Pediatrics provides up-to-date infor-mation concerning the specialty of pediatrics at the annual American Medical Student Association meeting. The American Board of Medical Special-ties and the Council on Medical Specialty Societies, in collaboration with the American Academy of Pediatrics, publish information concerning pediat-nc training programs and career opportunities within the specialty.
These efforts should continue, but I submit that mentors have the greatest influence on the career choices of medical students. Beginning in the fresh-man year, pediatric faculty members can be role models for medical students. Full-time faculty members engaged in pediatric research reveal the excitement of discovery. Those whose interests are primarily in teaching reflect the personal satisfac-tion that comes from interactions with students, housestaff, and other physicians. Full-time and clinical faculty members providing care in ambu-latory settings are role models for students who have selected medicine because of its tradition of service.
The service role model should be expanded into pediatric offices where first, second, and third year medical students can observe the interaction be-tween physicians and patients on the physician’s “home ground” and come to a better understanding of the rewards of clinical practice.
Pediatrics is an exciting blend of psychosocial, developmental, and biomedical sciences unique among medical specialties. Assisting children, ado-lescents, and families in times of stress is an im-portant part of pediatric practice. At the same time, practice offers an opportunity to apply basic science principles essential to the delivery of quality child health care. Students interacting with practicing pediatricians who are not only satisfied with their careers, but also enthusiastic and excited about what they are doing can significantly influence the career choice of medical students. Practicing pedia-tricians should volunteer as advisors/mentors in
at Viet Nam:AAP Sponsored on September 7, 2020
www.aappublications.org/news
1990;85;361
Pediatrics
ROBERT H. PANTELL and DONALD M. BERWICK
Cost-Effectiveness Analysis in Pediatric Practice
Services
Updated Information &
http://pediatrics.aappublications.org/content/85/3/361
including high resolution figures, can be found at:
Permissions & Licensing
http://www.aappublications.org/site/misc/Permissions.xhtml
entirety can be found online at:
Information about reproducing this article in parts (figures, tables) or in its
Reprints
1990;85;361
Pediatrics
ROBERT H. PANTELL and DONALD M. BERWICK
Cost-Effectiveness Analysis in Pediatric Practice
http://pediatrics.aappublications.org/content/85/3/361
the World Wide Web at:
The online version of this article, along with updated information and services, is located on
American Academy of Pediatrics. All rights reserved. Print ISSN: 1073-0397.
American Academy of Pediatrics, 345 Park Avenue, Itasca, Illinois, 60143. Copyright © 1990 by the
been published continuously since 1948. Pediatrics is owned, published, and trademarked by the
Pediatrics is the official journal of the American Academy of Pediatrics. A monthly publication, it has
at Viet Nam:AAP Sponsored on September 7, 2020
www.aappublications.org/news
