Cost-Benefit Analysis of The Newborn Screening Program of The Philippines
Cost-Benefit Analysis of The Newborn Screening Program of The Philippines
Department of Pediatrics, College of Medicine and Philippine General Hospital, University of the Philippines Manila;
1
2
Institute of Human Genetics, National Institutes of Health, University of the Philippines Manila;
3
Department of Clinical Epidemiology, College of Medicine, University of the Philippines Manila;
4
Department of Anesthesiology, College of Medicine and Philippine General Hospital, University of the Philippines Manila
ABSTRACT Conclusion: This study demonstrates that the benefits of an NBS program
Background. Newborn Screening (NBS) is a public health activity in the Philippines far outweigh the societal costs of a “do-nothing”
aimed at the early identification of infants who are affected by certain alternative. The benefit:cost ratio for the 5-disorder program is 1.4 and
genetic/metabolic/infectious conditions. A cost analysis is critical for the net benefit for the 5-disorder screening program is US$ 11.42 M.
national implementation for integration as a public health program.
Objectives. 1) To determine the incidence rates of congenital Key Words: cost benefit analysis, newborn screening
hypothyroidism (CH), congenital adrenal hyperplasia (CAH),
galactosemia (GAL), phenylketonuria (PKU) and glucose-6-phosphate Introduction
dehydrogenase (G6PD) deficiency; and 2) To determine whether NBS Newborn Screening (NBS) is a public health activity
is cost-beneficial for each disorder individually or in combination, from aimed at the early identification of infants who are affected
a societal perspective. by certain genetic/metabolic/infectious conditions. Early
Study Design. Cross sectional survey and cost-benefit analysis. identification of these conditions is particularly crucial,
Subjects and Methods. The study was conducted through a screening
since timely intervention can lead to a significant reduction
survey of the original 24 Metro Manila hospitals. Newborns were
screened for CH, CAH, GAL, PKU and G6PD deficiency after the 24th hour
of morbidity, mortalities, and associated disabilities in
of life. Those who screened positive underwent serum confirmatory affected infants. Since its inception in the 1960’s, at least
testing. Using incidence rates from the screening survey, a population 20,000 affected patients worldwide are now leading normal
of 1.5 million, and different screening combinations, the costs for the lives. NBS in other settings currently includes as many as
detection and treatment of the five disorders were compared to the 50 different conditions, including metabolic and infectious
benefits projected from preventing the corresponding complications diseases.1-3 NBS has been universally accepted for almost
and consequent productivity losses. For economic evaluation, we five decades and yet has remained a challenge for the
compared sequential analysis of doing tandem/multiple testing for the Philippines. It was introduced in the Philippines by the
different disorders vs a “do-nothing” alternative. Sensitivity analyses ‘Newborn Screening Study Group (NSSG) in 1996. The
for different incidence and discount rates were conducted to test the recommended panel of disorders consisted of congenital
strength of the conclusions.
hypothyroidism (CH), congenital adrenal hyperplasia
Results. The incidences of the disorders with 95% confidence intervals
are: CH is 1:3 235 (1:2 219 - 1:5 946); CAH is 1:7 455 (1:4 046 - 1: 14245);
(CAH), galactosemia (GAL), phenylketonuria (PKU) and
GAL is 1: 106 006 (1: 44 218-1:266 796); and G6PD deficiency is 1:167 glucose-6-phosphate dehydrogenase (G6PD) deficiency.4
(1:151 - 1: 186). Screened individually, CH and G6PD deficiency had The economic benefits of newborn screening have been
net benefits of US$ 5.29 M and US$ 15.44 M, respectively. The other well described.5-8 A Philippine study by Dans et al. reported
conditions yielded net costs when screened individually - CAH (US$ that a newborn screening program (NBSP) for CH is cost-
2.61 M), GAL (US$ 0.90 M) and PKU (US$ 6.74 M). Pairing the disorders beneficial when the blood collection occurs after the first day of
with CH showed the following benefit:cost ratios - CH + CAH, 1.3; CH life. Currently, the NBSP has adapted this recommendation.9
+ GAL, 2.0; CH + G6PD deficiency, 3.4; and CH + PKU, 0.9. Combining Our study aims to evaluate the efficiency of establishing a
disorders resulted in the following benefit:cost ratios - CH + CAH + national NBSP. The specific objectives are: 1) to establish the
GAL, 1.2; CH + CAH + GAL + PKU, 0.8; and CH + CAH + GAL + G6PD incidence rate of CH, CAH, GAL, PKU and G6PD deficiency
deficiency, 2.1. Screening for the 5 disorders in tandem resulted in a
in the Philippine newborn population; 2) to determine
benefit:cost ratio of 1.4 and a net benefit of US$ 11.42 M.
whether a Philippine NBSP is cost-beneficial compared to
a “do-nothing” alternative from the societal perspective; 3)
to determine whether newborn screening is cost-beneficial
for each disorder taken individually; 4) to determine if
combinations of screened conditions yield benefits greater
Corresponding author: Carmencita David - Padilla, MD, MAHPS than costs; and 5) to determine if it is cost-beneficial to
Institute of Human Genetics, National Institutes of Health - Philippines include all 5 disorders in a national NBS panel.
625 Pedro Gil St., Ermita, Manila 1000, Philippines
Tel/ Fax Number: +632 525 7459
Email: cpadilla@upm.edu.ph
Conditions screened Total Costs* Total Benefits* Net Benefits* Benefit:Cost Ratio
CH + CAH 9.98 12.91 2.93 1.3
CH + GAL 4.80 9.37 4.58 2.0
CH + G6PD deficiency 8.83 29.83 20.98 3.4
CH + PKU 16.92 15.72 (1.20) 0.9
*
in million US$
Conditions screened Total Costs* Total Benefits* Net Benefits* Benefit:Cost Ratio
CH 3.87 9.16 5.29 2.4
CH + CAH 9.98 12.91 2.93 1.3
CH + CAH + GAL 10.91 13.12 2.22 1.2
CH + CAH + GAL + PKU 23.96 19.68 (4.27) 0.8
CH + CAH + GAL + G6PD deficiency 15.89 33.80 17.91 2.1
CH + CAH + GAL + PKU + G6PD deficiency 28.94 40.36 11.42 1.4
*
in million US$
CH was used as a common element for all disorder 7% during the follow-up years. A 7% discount rate is higher
combinations. than in developed countries like the United States. This can
Discounting was crucial in the evaluation since the costs be rationalized by considering that developing countries give
were spent “now” while the benefits were projected into the more importance to the present because of more immediate
future. All costs of treatment and benefits were discounted at needs.
Results
Phase 1
From June 1996 to December 2000, a total of 384 985
newborns were screened from the 24 study hospitals. There
were 4,773 newborns who screened positive for at least one
of the disorders, and 1,029 newborns were confirmed to have
one of the five disorders included in the panel. Screening Figure 1. Comparison of net benefits according to incidence and
discount rates for screening for 5 disorders.
for G6PD deficiency started only in 1998 and screening for
homocystinuria (HCY) was eventually dropped in 2000
since no cases were detected from the population screened.
Table 1 shows the incidence rates for the disorders
screened and the various incidence rates computed from Discussion
the study. The point estimates of true incidence rates were A cost-benefit analysis of the NBSP of the Philippines
used for the baseline analysis to compute the total costs was conducted to determine whether screening for various
and projected benefits of the NBSP. The lowest and highest disorders would be beneficial despite the inherent costs of
estimates of the 95% confidence intervals were used for the setting up a nationwide program. The disorders were chosen
sensitivity analysis in the cost benefit analysis. based on whether there are reliable and efficient NBS tests,
the disorder results in high morbidity and mortality if left
Phase 2 untreated, there is effective treatment that reduces negative
Table 2 shows the component screening costs per disorder outcomes, and there is a relatively high incidence.4
used in computations for each patient. A societal perspective was employed to include not only
Table 3 presents the component costs of a nationwide the monetary benefits of preventing serious complications,
NBSP compared to the itemized costs of a “do-nothing” thus saving on medical fees, but also the projected losses
alternative and the benefits to be gained from a functioning in productivity of the individuals who would have been
NBSP. functional in society had they been screened and their
Table 4 shows the cost benefit analysis for screening in possible disabilities prevented.
tandem with CH. Certain costs (i.e. staff time, filter paper) A previous study by Dans et al. on the cost-benefit
were eliminated to avoid overlapping of costs. analysis of the screening program for CH showed net
As shown in Table 5, the total cost of the screening benefits of US$ 5 M, hence concluding that NBS for CH
program to include the 5 disorders in tandem is projected was cost-beneficial from a societal perspective.9 Dans’
to be US$ 28.94 while the cost of a do-nothing alternative or study served as the model on which the current study is
the benefits gained is projected at US$ 40.36 M. Therefore, based, since it was conducted using the same Philippine
a net benefit of US$ 11.42 M can be gained from the NBSP. population and setting.
The benefit:cost ratio is 1.4. The initial phase of this study involved establishing
Figure 1 shows the effect of different discount rates with the incidence rates of the five disorders included in the
varying incidence rates on the net benefits of screening for screening program. Data from the 24 study hospitals
all 5 disorders. originally included in the pilot study of the program
were used since these centers have gathered the most
The estimates for sensitivities of the 5 disorders are information from the target population for entire duration
generally considered to be very good. Therefore, missed of screening. A weighted incidence for each disorder was
cases were only computed for the two most commonly calculated, taking into consideration the contribution of
detected disorders (CH and G6PD deficiency), since the each participating hospital to the sample population. The
number of missed cases for the other three disorders would incidence was considered the more appropriate estimate of
be almost insignificant due to the high sensitivity rate of the the incidence of the disorders since it reflects the stratified
NBS procedure and their low incidences. The sensitivity rate sampling procedure that was employed. It will be noted that
of the screening test for CH is 99.5% while the sensitivity rate there is a slight discrepancy between the crude incidence
of the screening test for G6PD deficiency is 94%.16 and the weighted incidence. The confidence intervals of the
weighted incidence were also computed at 95% level. It is
assumed that the true incidence of each disorder should fall
Incidence
Incidence
and 5). However, increasing the incidence rate produced The sensitivity analysis in Figure 1 showed the effect of
lesser net costs, suggesting that a nationally implemented varying incidence and discount rates on the overall net
screening program may eventually yield some benefits. benefits of screening for all 5 disorders. First, net benefits were
Interestingly, the pattern is not the same for PKU (Figure 6). realized only at discounts rates of 3% up to 7%, regardless of
The actual identification and management of PKU patients the incidence. Increasing the discount rate to 12% yielded net
cost far more than the no screen and no treatment scenario. costs. Second, with several disorders in place, a disorder like
An increased incidence, in fact, leads to greater net costs. PKU, with high treatment and management costs and low
The rarity of disorders like PKU translates into high net incidence, tends to decrease the overall benefit such that an
costs for case detection. This raises the question whether increased incidence could not provide the expected increase
such disorders should be included in a national screening in benefit.
program, a question that can only be answered in a larger Children directly benefit from screening since they are
societal context. spared from possible mental retardation and death. The
The next step involved determining whether any families of these children also benefit since they do not
combination of disorder screening would yield greater bear the costs of caring for a mentally retarded individual
benefits than costs. Since screening for CH was already or having a non-productive family member. Society
determined to be cost-beneficial, it was used as the benefits indirectly through prevention of productivity loss
foundation for assessing combinations of other disorders. of a mentally handicapped individual or the death of an
All combinations of disorders considered were added to individual. Researchers point out that for PKU, the cost
ongoing CH screening to see if the benefits still outweighed of screening and treating PKU patients is greater than the
the costs (Tables 4 and 5). The ultimate goal of this study expenses of a no screen scenario. Realizing the responsibility
was to determine if it is cost-beneficial to include all 5 of providing available and attainable treatment when
disorders in a national screening panel. Note that the costs proposing a screening program, proponents of the Philippine
for multiple disorders are not just a simple addition of the NBSP are laying the foundation for future assistance to PKU
costs for single disease screening because some additional patients in obtaining and affording treatment.
savings can be realized when screening is done in tandem. Nevertheless, when translated into monetary terms, the
As shown in Table 4, pairing disorders with CH resulted collective benefits gained when screening for all 5 disorders
in mostly benefit:cost ratios greater than 1, meaning greater more than offset the costs incurred by the family through
benefits than costs. The most cost-beneficial pair was CH + screening. As much as US$ 11.42 M in potential benefits
G6PD deficiency and the least cost-beneficial pair was CH + can be gained from a nationally implemented NBS for all 5
PKU. This was due to the fact that the cost of screening for disorders.
PKU was far greater than the potential benefits. Treatment
for PKU, once detected by screening, is expensive and the Conclusions and Recommendations
incidence in our population is comparatively low versus The incidences of the disorders with 95% confidence
the other disorders. intervals are: CH is 1:3 235 (1:2 219 - 1:5 946); CAH is 1:7 455
In Table 5, CH was again used as the common element (1:4 046 - 1: 14245); GAL is 1: 106 066 (1: 44218 - 1: 266 796);
in all screening combinations. This order of additions to CH and G6PD deficiency is 1:167 (1:151 - 1:186). Screening for all
was conceived based on the severity of the consequences of 5 disorders in tandem resulted in a net benefit of US$ 11.42
each disorder (CAH > GAL > PKU > G6PD). CAH was the M and a benefit:cost ratio of 1.4, clearly demonstrating that
first considered since its complications are life-threatening the benefits of NBSP of the Philippines versus a do-nothing
and they occur shortly after birth. GAL was next since it can alternative far outweighs the costs. Only screening for CH
also result in fatal consequences, although not as common and G6PD deficiency are cost-beneficial when screening is
as in CAH. The severity of the resulting disabilities in PKU done individually.
is more significant than in G6PD deficiency. For this reason,
G6PD deficiency was the last disorder to be added to the Epilogue
panel despite having an individually favorable benefit:cost This paper was used as one of the supporting documents
ratio. for the enactment of Republic Act No. 9288 or the Newborn
As shown in Table 5, adding multiple disorders to a CH Screening Act of 2004.17 The law provides the mandate to
screening program results in benefit:cost ratios that are offer every newborn the opportunity to undergo newborn
greater than 1, except for PKU where the cost of screening screening. The current panel of disorders includes CH, CAH,
was far greater than the potential benefits. However, when GAL, PKU and G6PD deficiency.
G6PD deficiency was added to the panel, the program
became cost-beneficial once again. G6PD has a high
incidence and a relatively inexpensive form of treatment
(e.g. avoidance of certain food and drugs). We conclude that
a 5-disorder NBS panel would result in greater net benefits
than costs.