Table 52.4  Value gain conferred by health care interventions

*Data from the Center for Value-Based Medicine Pharmaceutical Value Index internal files.

QALY, AMD, age-related macular degeneration; SSRI, selective serotonin reuptake inhibitor.

than being cost-utilitarian. The cost utilities of various nonophthalmic, health care interventions and interventions for neovascular ARMD are shown in Table 52.5.28,,30

Discounting5

Both costs and value outcomes (QALYs) are discounted in a net present value (NPV) analysis to account for the time value of money and of good health. In essence a dollar is now worth more than a dollar in 10 years’ time, since that dollar can now be invested to yield more dollars. Good health can now also be used to create dollars that can be invested to yield more dollars.

Value-based medicine

Value-based medicine is the practice of medicine based upon the value conferred by health care interventions, especially drugs.5 Theoretically, it enables the practice of higher-quality care since it integrates patient utilities, thus incorporating patient preferences for health states and quality-of-life parameters often ignored in the outcomes of evidencebased medicine clinical trials.5 In addition, it incorporates all benefits associated with an intervention, as well as all adverse events using decision analysis (Figure 52.1).

A critical aspect of value-based medicine is the fact that it standardizes the input criteria and outcomes used in pharmacoeconomic analysis.

Standardization

Unfortunately, most of the cost–utility studies in the current literature are not comparable since they use different utility methodologies, different utility respondents, as well as different cost perspectives and cost bases.5 Value-based medicine cost–utility analyses use the stand­ ards listed in Table 52.6 to allow a comparison of studies across all specialties.

Table 52.5  Cost–utility of various health care interventions8

QALY, quality-adjusted life year; ROP, retinopathy of prematurity; SSRI, selective serotonin reuptake inhibitor; PPV, pars plana vitrectomy, DME, diabetic macular edema; CNVM, choroidal neo-vascular membrane; AMD, age-related macular degeneration; Rx, treatment; HIV, human immunodeficiency virus; PVR, proliferative vitreoretinopathy; C3F8, perfluoropropane; CRAO, central retinal artery obstruction; AC, anteriorchamber; CO2, carbon dioxide; O2, oxygen.

Patient respondents

Value-based medicine specifically uses patient utilities since community and expert (physician) surrogate values often differ considerably. For AMD, physicians who treated the disease underestimated its burden upon the quality of life of patients with the condition by anywhere from 96% to 750%.31

COST PERSPECTIVE

The third-party insurer perspective includes only those costs the insurer has to pay, or the direct medical costs. The societal perspective includes the third-party insurer costs, but also includes direct nonmedical costs such as caregiver costs and travel costs, as well as indirect costs such as loss of wages and disability payments obviated.

Thus, the third-party perspective and the societal perspective are quite different and difficult to compare without a thorough breakdown. A thorough cost–utility analysis is more complete if it offers ratios utilizing both the third-party insurer cost perspective and the societal cost perspective. Typically the societal cost perspective yields a more favorable cost–utility ratio versus the third-party insurer cost perspective. One note of caution, however, is that societal costs are not as standardized and readily available as the direct medical costs.

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and medicine based• 52-Valuechapter

pharmacoeconomics

Table 52.6  Cost–utility analyses to allow comparability across studies: summary of recommended cost–utility variable standards

Perspective – third-party insurer (using direct health care costs) + societal (direct medical, direct non-medical and indirect costs)

Population analysis – reference case

Utilities

Methodology: time tradeoff Source: patients

Comorbidities: should not be used to discriminate against those who are disabled

Costs: direct health care costs

Annual discount rate – 3% for quality-adjusted life-years (QALYs) and costs

Sensitivity analysis – should be performed at least one way, and often two way. Those input variables in which there is the least confidence should be analyzed.

Adapted from Brown MM, Brown GC, Sharma S. Evidence-Based to Value-Based Medicine. Chicago: AMA Press, 2005: pp. 1–324.

IMPLICATIONS FOR RETINAL

PHARMACOTHERAPY

Pharmacoeconomics has already had a huge impact on the use of retinal pharmacologic agents,35,36 a phenomenon that will only become more pronounced in the near future as the USA adopts cost–utility principles.37 Pharmacoeconomic principles are currently used in public policy in the UK, Canada, Australia, Estonia, Latvia, and Lithuania, and will be adopted in other countries as well.

The National Institute for Health and Clinical Excellence (NICE), the agency which recommends whether new therapies should be adopted by the National Health Service in the UK, originally recommended the use of ranibizumab for only second eyes with classic subfoveal neovascular AMD, or approximately 10% of eyes which might benefit.35 When the conferred value and cost–utility of ranibizumab for the treatment of all forms of subfoveal choroidal neovascularization became apparent in NICE analyses and through cost–utility analyses in the peer-reviewed literature,30 the policy was changed to allow 14 injections of ranibizumab per eye for cases of subfoveal choroidal neovascularization.36

It is very likely that combination therapies for neovascular AMD will be undertaken in the near future.38 When this is the case, value-based

medicine will objectively assess the value and cost-effectiveness of these interventions so the clinician will be able to provide the highest-quality care to patients for the most reasonable cost.

SUMMARY AND KEY POINTS

Value-based medicine pharmacoeconomics, as applied to vitreoretinal interventions and across all of medicine, signals a new era of quality in the health care arena.

The advantages of value-based pharmacoeconomic analyses include the facts that they:

1.  integrate all benefits and adverse events associated with a drug to demonstrate its overall value

2.  incorporate patient quality-of-life preferences often ignored in clinical trials

3.  allow physicians to appreciate better what patients consider most relevant so they can provide higher-quality care

4.  identify drugs with superior value, an aspect especially important for drug companies that often have done poorly in demonstrating the value of their drugs

5.  demonstrate the often underappreciated benefits of drugs, such as the facts that they prevent disability and allow patients to continue to work

6.  allow a head-to-head comparison of the value conferred by drugs and other interventions

7.  assess the cost of comparator drugs of similar value so the least expensive can be identified.

In essence, value-based medicine pharmacoeconomics provides an information system which allows all stakeholders the ability to appreciate the value conferred by drugs, as well as the costs expended for that value. It will bring a new era of quality care to medicine as it takes evidence-based medicine outcomes to a higher level, and is a methodology to facilitate the most efficient use of scarce health care resources.

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