Health Care Systems: How to Resolve the Dilemma Between Clinical Needs and Limited Resources?

Clinical Needs and Limited Resources?

M. B

, S. N

Magnitude of the Problem

Are the appropriate indications for ICDs (based on clinical evidence) limited by health care system resources? An overview of the number of ICD (includ- ing CRT-D) implants and their costs in USA, Europe, and Italy in 2003 and 2004 is provided in Tables 1 and 2. ICD therapy accounts for only a small percentage of the total expenditure of health care systems, and there are large numbers of appropriate patients who do not receive ICDs, although the annual incidence of implants is increasing. The number of patients receiving ICDs (or CRT-Ds) has increased ten-fold over the last 10 years in Europe and in the USA [1]. In Italy, the implantation rate in 2003 increased by 45%

compared to the rate in 2002 and by a further 23% in 2004 compared to 2003 (Tables 3, 4). Nevertheless, the total expenditure for ICDs (which includes device costs plus implantation and follow-up) still remains a modest per- centage of total health expenditures. Furthermore, the number of patients who could benefit from this therapy is miniscule compared to the general population. For example, in Europe, in 2004, about 40 000 patients received ICDs, and the associated in-patient expenditures, amounted to € 0.6 billion, accounting for 0.2% of total in-patient expenditures. Similarly in Italy, in 2004, about 8000 patients received ICDs/CRT-Ds, and the in-patient expendi- tures for these were € 0.13 billion, accounting for 0.3% of total in-patient expenditures. Figure 1 compares the expenditure for ICDs with those of other accepted therapies in the USA in 2000. The cost of ICD was four-fold lower than that for PTCA or CABG and 15-times lower than that for anti- biotics.

Centro Aritmologico, Dipartimento di Cardiologia, Ospedali del Tigullio, Lavagna

(Genua), Italy; Guidant Corporation, Brussels, Belgium

Table 1. Number of ICDs (and CRT-Ds) and their in-patient costs in 2003 (source:

Guidant)

ICDs/CRT-Ds ICDs/CRT-Ds Estimated costs

(implants/million (total number (Euros) inhabitants) implanted)

USA 280 70 000 2.3 billion

Europe 60 22 500 0.6 billion

Italy 110 6 400 0.1 billion

ICD Implantable Cardioverter defibrillator, CRT-D cardiac resynchronisation therapy plus defibrillator

Table 2. Number of ICDs (and CRT-Ds) and their in-patient costs in 2004 (source:

Guidant)

ICDs/CRT-Ds ICDs/CRT-Ds Estimated costs

(implants/million (total number (Euros) inhabitants) implanted)

USA 320 80 000 2.6 billion

Europe 100 36 000 1.0 billion

Italy 146 8163 0.13 billion

ICD Implantable Cardioverter defibrillator, CRT-D cardiac resynchronisation therapy plus defibrillator

Table 3. Number of ICDs/CRT-Ds and revenues in Italy in 2003 (source: personal com- munication)

ICDs: Number Revenues Number Revenue

(Euros) difference (%) difference (%) compared compared to 2002 to 2002 Single chamber (VR-VVI) 2584 32 583 699 42.3% 34.1%

Dual-chamber 1947 29 900 243 16.4% 12.6%

CRT-D 1846 29 761 657 90.7% 85.0%

Total ICDs 6377 92 245 599 43.1% 37.8%

Leads 6605 9 566 921 28.0% 20.6%

Total ICD/CRT-D - 101 812 520 - 36.0%

revenues

ICD Implantable Cardioverter defibrillator, CRT-D cardiac resynchronisation therapy

plus defibrillator

Thus, by considering the global health care system, it is clear that the impact of ICD therapy on total health care expenditures is modest. The main reason that so much attention has been given to the costs of ICD therapy, compared to other established therapies, is that the upfront costs of the device (and implantation) are high. Whether these initial costs are reason- able when considered over the lifetime of the patient is the important issue that is addressed in this article.

Table 4. Number of ICDs/CRT-Ds and revenues in Italy in 2004 (source: personal com- munication)

ICDs: Number Revenues Number Revenue

(Euros) difference (%) difference (%) compared compared to 2002 to 2002 Single chamber (VR-VVI) 2795 32 640 921 8.2% 0.2%

Dual-chamber 2523 36 402 528 29.6% 21.7%

CRT-D 2845 44 137 047 54.1% 48.3%

Total ICDs 8163 113 180 496 28% 22.7%

Leads 8806 11 375 480 33.3% 18.9%

Total ICD/CRT-D - 124 555 976 22.3%

revenues

ICD Implantable Cardioverter defibrillator, CRT-D cardiac resynchronisation therapy plus defibrillator

Fig. 1. Comparison between some therapies

Different Perspectives

While it cannot satisfy all the individual demands made upon it, the primary objective of a health care system is to provide a homogeneous level of assis- tance based on the priorities of the patient population. Correct evaluation of those priorities requires consideration of the scientific, objective, and repro- ducible criteria for the best allocation of available economic resources.

Because of its ethical implications (i.e., ‘how to improve the health status of the citizens’), the decision cannot be left exclusively to the prescribing spe- cialist but must involve all stakeholders of the national health system.

Although their perspectives differ (Table 5), the common goal is to improve citizens’ health.

Table 5. Different perspectives

Stakeholder Direct objective Notes

Doctor of the national No profit, but budgeting Possible conflict between the

health care system obligation to provide the best

therapy and that of limiting the consumption of public resources (efficiency/efficacy ratio); possi- ble conflict with profit issues Doctor outside of the No profit, no budget Obligation to provide the best national health care system therapy; possible conflict with

profit issues

ICD companies For profit Interest in producing high-quali- ty devices and providing techni- cal support, and in re-reinvesting profits in research & develop- ment, clinical support, trials, etc.

Administrator of the health For budget Obligation to provide efficient

care system, professional health care

Administrator of the health For consent Obligation to fulfil the perceived care system, elected member needs of patients

Patient (and family) For him-/herself Interest in maintaining his/her

own health

The Perspective of the Clinical Cardiologist: Evidence-Based Medicine and Guidelines The solution appears simple and easy to apply in clinical practice: ‘Follow the recommendations of the guidelines.’ Indeed, a major objective of the guidelines is to enunciate clearly which patients are appropriate for which therapies according to the rules of evidence-based medicine. Guidelines mean nothing, if clinicians do not adhere to them, so much of the guidelines process also aims at increasing the appropriate implementation of particular therapies – on the basis of evidence-based-medicine – for appropriately selected patients. Further objectives of the guidelines include improving the quality of interventions, the clinical outcome of the patients, and the cost/efficacy ratio, and should help authorities in the allocation of resources.

With the advent of evidence-based medicine, doctors have been induced to abandon empirical medicine, based on ‘logical’ or ‘common belief,’ and to move towards ‘evidence of efficacy’ [2]. Also, the use of ICDs must be based on the guidelines established by the leading international medical societies, following their careful review of all available data. Adoption of the standards defined by the scientific community is the best method to avoid both

‘overuse’ (‘inappropriateness’) and ‘underuse’ (‘malpractice’). A doctor has the ethical obligation to adopt a confirmed therapy in the interest of his/her patients and, conversely, to be aware of the legal implications of not doing so.

Whether the recommendations of the guidelines are affordable and with- in the limitations of available resources is an obviously critical decision, but it cannot be made by the clinical cardiologist alone. There are other deci- sion-makers, starting with the cardiologist-manager, who have the responsi- bility to put guidelines in the proper economic context within the health sys- tem and the clinical governance of health. Going from these principles to the specific case of ICD therapy, we have provided evidence that, in Italy and western Europe, resources are sufficient to permit the guidelines to be imple- mented. It is the right of the clinical cardiologist to make use of these resources; it is an obligation not to abuse them.

From the Perspective of the Cardiologist-Manager: Evidence-Based Health Care and Guidelines

The objective of a health system is to establish scientific, objective and repro- ducible criteria for optimal allocation of the available economic resources [3].

To accomplish this, we need objective methods of measurement in order to

define priorities. All of the critical factors can be measured and compared, and

the decision on the cost-effectiveness of a therapy must be based on the evi-

dence of such measurements. The most useful methods are the number need-

ed to treat (NNT) and the cost-effectiveness. Regarding the use of ICDs, the

results of ICD therapy vs other therapies must be analysed.

The NNT is a normalised measurement of clinical efficacy and efficiency that allows comparison of different treatments. In the case of ICDs, the NNT is the number of patients that must receive an ICD implant in order to demonstrate prolonged survival by 1 year in one patient. The importance of the NNT is that it is easy to understand in clinical practice and is unrelated to the difference in costs of therapy among different countries [4]. Some examples of NNTs are listed in the Tables 6 and 7. Comparison of the data in these tables shows that ICD therapy, when used according to the recommen- dations of the guidelines, is more effective than many other established ther- apies. For example, ICD therapy for patients fitting the MADIT II criteria has an NNT of 11 (at 3 years of follow-up), which compares quite favorably with Captropril (SAVE), which has an NNT of 20, also at 3 years of follow-up. The NNT for ICD therapy remains favourable even when it is compared with therapies in which a softer end-point, namely, morbidity instead of mortali- ty, is used (Table 7). As explained below, the NNT for ICDs improves ‘dramat- ically with time of follow-up,’ which illustrates the importance of evaluating ICD cost-effectiveness after several years of follow-up, not simply at the mean time elapsed when a particular study has ended [5]. This is precisely the reason why studies with shorter duration of follow-up give misleadingly higher NNT values; for example, in the AVID/CIDS/CASH meta-analysis, the 1-year follow-up time is far too short, and as demonstrated by Salukhe et al., longer follow-up in those trials would have shown a much lower NNT [5].

Moreover, NNT has no meaning for patient cohorts considered contra-indi- cated for ICDs on the basis of negative trials, such as CABG-Patch, DINAMIT and BEST_ICD. These trials simply showed that patients requiring revascu- larisation and those within 40 days of myocardial infarction are considered not appropriate candidates for ICDs.

‘Cost-effectiveness’ is the ratio of the difference in cost of ICD therapy

compared to an alternative (e.g., amiodarone) divided by the prolongation of

life achieved by ICD vs the alternative therapy. The cost-effectiveness ratio is

expressed as cost/year life saved [4]. Some examples are listed in Tables 8

and 9. ICD therapy, especially when used for primary prevention, has a more

favourable cost-effectiveness than many other commonly established thera-

pies, for example compared to hospital dialysis or 2-vessel CABG surgery

(Table 8) [6, 7]. In general, the duration of follow-up of the ICD studies has

been shorter than that of others, which were interrupted, mainly for ethical

reasons, at the time when the benefit became manifest. A recent study [5], in

which the results of eight randomised trials were pooled together and the

predicted effect was calculated at 1, 2, and 3 years of follow-up, showed that

the benefit of ICD therapy increases with the duration of follow-up, at least

for the first 3 years, without an increase of costs. For example, the NNT to

save a year of life is seven-fold higher after 1 year than after 3 years of fol-

Table 6. Number needed to treat (NNT) to save one life for some established therapies based on the results of randomised controlled trials. Negative studies have resulted in ICDs (and other active therapies that have been tested) contraindicated or found to be not useful for patients (see text for further explanation)

Trial Therapy Duration NNT

MUSTT ICD: primary prevention 5 3

MADIT ICD: primary prevention 2.4 4

MADIT 2 ICD: primary prevention 3 11

SCD-HeFT ICD: primary prevention 5 14

DEFINITE ICD: primary prevention, DCM 2 29

AVID ICD: secondary prevention 3 9

AVID, CASH, CIDS ICD vs amiodarone 1 29

meta-analysis

CABG-PATCH ICD post by-pass 4 NA

DINAMIT ICD post-AMI 2.5 NA

BEST-ICD ICD post-MI 1.5 NA

COMPANION ICD + CRT 1 14

CARE-HF CRT 2.5 10

CASS CABG, left main artery 5 6

CASS CABG, 3 vessels, EF < 50% 5 11

CASS CABG, 3 vessels, EF > 50% 5 NA

Zwolle RCT Primary PTCA vs thrombolysis 7 10

Primary PCI Primary PTCA vs thrombolysis 1 25

meta-analysis

FRISC 2 PTCA-CABG, unstable angina, ACS 0.5 100

TIME PTCA-CABG, stable angina 0.5 NA

ACME PTCA, stable angina 0.5 NA

Amiodarone Amiodarone 2 37

meta-analysis

SAVE Captopril, heart failure 3 20

SOLVD Enalapril, heart failure 4 22

CIBIS 2 Bisoprolol, heart failure 1 23

MERIT Carvedilol, heart failure 1 26

ISIS 2 Streptokinase, AMI 2 24

Hypertension Antihypertensive drugs (various) 5 NA meta-analysis

4S Simvastatin (secondary prevention) 6 28

HPS Simvastatin (primary and secondary 5 59

prevention)

NA Not applicable

Table 8. Costs per year of life saved for some established therapies (adapted from [6])

Intervention Cost/year

life saved (1999) (Euro)

Pacemaker implant 1860

Beta-blockers in survivors of high risk acute myocardial infarction 3400 Simvastatin in a 70-year-old man with cholesterol blood level of 309 mg/dl 4300 Pravastatin for secondary prevention of chronic coronary artery

disease in a man with 2 risk factors 9770

CABG (3-vessels disease) vs medical therapy 13 700 ICD in asymptomatic chronic coronary artery disease, depressed EF

and non-sustained ventricular tachycardia 14 200

Streptokinase therapy for acute myocardial infarction 24 000

CABG 3-vessel disease vs PTCA 26 500

Home dialysis 26 700

tPA vs streptokinase for AMI at 1 year 37 000

In-hospital haemodialysis 61 700

CABG (2-vessels disease) vs medical therapy 85 300 Table 7. NNT needed to avoid some composite end-points for some established thera- pies based on the results of some randomised controlled trials

Trial Therapy End-point Duration NNT

(years) FRISC 2 PTCA-CABG in ACS Death, non-fatal AMI 0.5 37

TACTICS PTCA-CABG Death, non-fatal AMI, 0.5 11

in ACS Tn+ hospitalisation

TIME PTCA-CABG, Death, non-fatal AMI, 0.5 3

stable angina hospitalisation

ACME PTCA, stable angina Angina 0.5 6

HPS Simvastatin Coronary artery disease, 5 18

stroke, revascularisation

Hypertension Antihypertensive Stroke, coronary 5 125

meta-analysis drugs (various) events

low-up. The cost-effectiveness ratio remains acceptable also when compared with therapies that have a combined end-point of mortality and morbidity.

For example, in several studies on the effects of statins, the cost per patient per year free from death, non-fatal myocardial infarction, or stroke was

€ 51 400 for the study AFCAPS/TexCAPS, € 26 013 for WORSCOPS, € 9970 for CARE, € 8028 for LIPID and € 6695 for 4S [8].

In addition to the need to be aware of the effectiveness of a particular therapy, the optimal use of economic resources (efficiency) is a major objec- tive of any health care manager. For any therapy, the rule must be to provide the appropriate therapy at the lowest price. The price of an ICD varies con- siderably, up to two-fold or three-fold, from one model to another. This means, for example, that relatively lower priced, simple models can be used for many (most) patients, reserving the more expensive ICDs with more sophisticated features for those patients needing such features. This approach allows more efficient use of available resources.

Other Issues Outside the Competency of the Cardiologist That Need To Be Considered

There are several other issues that are outside the strict professional compe- tency of the cardiologist that, nonetheless, need to be explored. These are political and ethical issues that are at the base of a global health care strate- gy. For example:

1. Who has the right to decide or control what is the ‘right’ price of an ICD or any other therapy?

2. In the health care system, do open markets and competition exist?

3. Or, conversely, is some kind of political control of prices justified?

4. Who should make the decision regarding the maximum affordable cost to save 1 year of life?

Table 9. Costs per year of life saved of some established therapies (adapted from [7])

Intervention Cost/year life saved

(1999) (US dollars) PTCA (chronic coronary artery disease, 1 vessel) 88 944

Cardiac transplant 43 087

Hypertension (diastolic 95–104 mmHg) 40 753

Primary PTCA 31 244

ICD (no pre-implant EPS) 16 555

5. How should the latent conflicts of interest between industry and scientific societies be resolved [2]?

6. How should the latent conflicts of interest between industry and the authors of clinical practice guidelines be resolved [9]?

Discussion and Perspectives

ICDs, largely due to their proven efficacy as a life-saving therapy, have creat- ed quite a dilemma for health care providers. On the one hand, following years of prospective, controlled, clinical trials, the evidence base (number of positive studies) in support of ICD therapy is stronger than for any other medical therapy [10, 11]. On the other hand, the up-front costs are high, and–precisely due to the proven clinical efficacy of ICDs–the numbers of patients receiving this therapy is increasing rapidly. There could be as much as a ten-fold increase in the number of implants during the next 10–15 years (corresponding to a prudent epidemiological estimate of 400 000 patients needing an ICD according to the present indications, as recently updated by the European Society of Cardiology [12]). Even were this to occur, the corre- sponding expenditures would account for only 2% of total in-patient expen- ditures (since the cost of ICD therapy currently is 0.2% of global in-patient expenditures).

Looked upon in another way, even with this increase in patients, the number receiving ICDs would represent 0.1% of the European population (source: OECD Health Database 2004, Guidant estimates). In Italy, in 2004, ICDs were prescribed for about 8000 patients, about 0.01% of the overall Italian population; even with a five-fold increase in the number of implants during the next 5–10 years (which corresponds to a prudent epidemiological estimate of 40 000 patients needing an ICD according to the present indica- tions), the expenditure would account for 1.5% of total in-patient expendi- tures and would be prescribed to 0.05% of the Italian population.

In conclusion, at least for Italy and Western countries, the dilemma between clinical needs and limited resources can reasonably be solved.

Therefore, the use of ICDs should not be decided upon based on economic reasoning, but only by the appropriateness of the indications.

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