07.Health economics03.Individualized cost effectiveness analysis - sporedata/researchdesigneR GitHub Wiki

• Individualized CEA is used to generate patient-specific cost-effectiveness estimates for therapeutic and prophylactic interventions, as well as screening procedures.

• The pre-requisites for a CEA are that the new treatment be not only more expensive but also that this treatment be more effective. The reason is that if a treatment is more expensive but has the same effectiveness, the choice will always be to use the standard treatment. In other words, a CEA is not necessary.

A previous study provides a good overview of how we could evaluate the cost component of a study focusing on the cost-effectiveness analysis (CEA) section [19]. This can be done by;

1. Overview of the approach: the idea uses what is called a payer perspective and accounts for all costs in the hospital. This approach is in contrast with more demanding economic perspectives where we would have to capture patients' as well as societal costs (these are gigantic endeavors). This uses a sophisticated CEA method where we will be able to tell whether CEA is or not cost-effective for individual patients rather than for all patients -- the latter is the traditional approach.
2. Direct, itemized costs rather than charges. In practice, to get these data you might need to check with an institution administration regarding their financial database to get what they have in relation to itemized direct costs. If they offer to give the charge data (i.e., the amounts they are charging payers), insist on getting itemized direct costs since reviewers likely won't let us get by with charges. The difference between costs and charges is that costs represent how much hospitals pay for each item (drugs, devices, salaries, etc), while charges contain both costs and profit. Hospitals tend to be fairly protective of their cost data, and so you might need to persist and talk to upper administration.

When conducting a CEA, we will likely have to take what is called a "payer perspective," which is far less complex than a societal perspective. Assuming we will go for a payer perspective, reviewers will often ask for detailed information on costs over the course of treatment, and so it might be interesting to augment the registry with detailed information on direct costs over a one-year period, which would include in-hospital direct costs for the index procedure, readmissions, ED, and office visits.

When designing a cost-effectiveness analysis study, the time horizon defining the duration of time for outcomes assessment has to be long enough to capture the intended and unintended benefits and harms of the intervention(s). Using a longer time horizon than necessary would add unnecessary cost and complexity to the cost-effectiveness analysis model while adding too little would make the study less relevant.

Discounting is a technique commonly used in cost-effectiveness analysis to 'make fair' comparisons of interventions whose costs and outcomes occur at different times. In global health, such evaluations typically apply a discount rate of 3% for health outcomes and costs, mirroring guidance developed for high-income countries, notably the USA. However, this discount rate is inconsistent with rates of economic growth experienced outside the most advanced economies. For low- and lower-middle-income countries, a discount rate of at least 5% is more appropriate, and one around 4% for upper-middle-income countries.

1. Effectiveness interventions using observational or trial data. Observational data are often preferable since the cost component is more realistic, although trials tend to better control of bias. Effectiveness is often measured through QALY (quality-adjusted life year) since that metric can be generalized across clinical conditions (important for health economics comparisons).In addition, We can also calculate the QALY using the SF-12 [18].
2. Cost data

CEA models are estimated through Bayesian statistics since they allow for the estimating within subgroups containing small sample sizes.

The Second Panel on Cost-Effectiveness in Health and Medicine [1] recommends that, whenever possible, payor, as well as societal perspectives, should ideally be taken on individual projects [2] along with an impact inventory [3].

The following reporting guidelines are available:

1. Good research practices for cost-effectiveness analysis alongside clinical trials: the ISPOR RCT-CEA Task Force [4].
2. Recommendations for Conduct, Methodological Practices, and Reporting of Cost-effectiveness Analyses: Second Panel on Cost-Effectiveness in Health and Medicine [5].
3. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement [6].
4. Generalisability in Economic Evaluation Studies in Healthcare: A Review and Case Studies [7].
5. Cost-effectiveness of Leveraging Social Determinants of Health to Improve Breast, Cervical, and Colorectal Cancer Screening [8].
6. Choosing a time horizon in cost and cost-effectiveness analyses [9].

Organization and clarity in reporting cost-effectiveness analyses are important and there are expanded recommendations to improve and standardize reporting Recommendations for CEA.

1. Common packages
2. hesim
3. BCEA
4. rdecision

Possible companion includes:

• Chatbots to collect QALY and cost measurements.
• Causal models to evaluate effectiveness from observational data.
• Bayesian adaptive trials for effectiveness evaluation.
• Systematic reviews for effectiveness evaluations .

1. Books

   • Bayesian Cost-Effectiveness Analysis with the R package BCEA [10].
   • Cost-Effectiveness in Health and Medicine [11].

2. Articles combining theory and scripts

   • Individualized cost-effectiveness analysis (iCEA).
   • Introduction to hesim.
   • Practical guide to cost-effectiveness analysis [12].
   • Practical guide to comparative effectiveness research using observational data [13].
   • Overview of cost-effectiveness analysis [14].
   • The "Utility" in composite outcome measures [15].
   • Explaining health state utility assessment [16].
   • Choice Defines QALYs: A US Valuation of the EQ-5D-5L

• Two standard outcome metrics for cost-effectiveness analyses are commonly applied: Quality-adjusted life-years (QALYs) and disability-adjusted life-years (DALYs), for example, in cost-utility analyses. QALYs refer to measures of years lived in full health. In contrast, DALYs refer to measurements of years in health loss (quality and length of life). The first is commonly used to help make decisions regarding current practice by comparing the cost-effectiveness of competitor medical options. In contrast, the latter is frequently used in low- and middle-income countries and helps understand the economic burden of death, diseases, and disabilities [20]. These metrics are used to determine resource allocation and priority-setting.

[1] Second Panel on Cost-Effectiveness in Health and Medicine

[2] Should Value Frameworks Take A 'Societal Perspective'?

[3] impact inventory.

[4] Ramsey S, Willke R, Briggs A, Brown R, Buxton M, Chawla A, Cook J, Glick H, Liljas B, Petitti D, Reed S. Good research practices for cost‐effectiveness analysis alongside clinical trials: the ISPOR RCT‐CEA Task Force report. Value in health. 2005 Sep;8(5):521-33.

[5] Sanders GD, Neumann PJ, Basu A, Brock DW, Feeny D, Krahn M, Kuntz KM, Meltzer DO, Owens DK, Prosser LA, Salomon JA. Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine. Jama. 2016 Sep 13;316(10):1093-103.

[6] Husereau D, Drummond M, Petrou S, Carswell C, Moher D, Greenberg D, Augustovski F, Briggs AH, Mauskopf J, Loder E, CHEERS Task Force. Consolidated health economic evaluation reporting standards (CHEERS) statement. Cost Effectiveness and Resource Allocation. 2013 Dec 1;11(1):6.

[7] Sculpher MJ, Pang FS, Manca A, Drummond MF, Golder S, Urdahl H, Davies LM, Eastwood A. Generalisability in economic evaluation studies in healthcare: a review and case studies. Health technology assessment (Winchester, England). 2004 Dec 1;8(49):iii-v.

[8] Mohan G, Chattopadhyay S. Cost-effectiveness of Leveraging Social Determinants of Health to Improve Breast, Cervical, and Colorectal Cancer Screening: A Systematic Review. JAMA oncology. 2020 Jun 18.

[9] Basu A, Maciejewski ML. Choosing a time horizon in cost and cost-effectiveness analyses. Jama. 2019 Mar 19;321(11):1096-7.

[10] Baio G, Berardi A, Heath A. Bayesian cost-effectiveness analysis with the R package BCEA. New York: Springer; 2017 May 25.

[11] Neumann PJ, Sanders GD, Russell LB, Siegel JE, Ganiats TG, editors. Cost-effectiveness in health and medicine. Oxford University Press; 2016 Oct 3.

[12] Brooke BS, Kaji AH, Itani KM. Practical Guide to Cost-effectiveness Analysis. JAMA surgery. 2020 Mar 1;155(3):250-1.

[13] Merkow RP, Schwartz TA, Nathens AB. Practical Guide to Comparative Effectiveness Research Using Observational Data. JAMA surgery. 2020 Apr 1;155(4):349-50.

[14] Sanders GD, Maciejewski ML, Basu A. Overview of cost-effectiveness analysis. Jama. 2019 Apr 9;321(14):1400-1.

[15] Irony TZ. The “utility” in composite outcome measures: measuring what is important to patients. Jama. 2017 Nov 14;318(18):1820-1.

[16] Chang EM, Saigal CS, Raldow AC. Explaining Health State Utility Assessment. Jama. 2020 Mar 17;323(11):1085-6.

[17] Devlin N, Parkin D, Janssen B. Methods for analysing and reporting EQ-5D data. Springer Nature; 2020.

[18] John E B, Jennifer R. The estimation of a preference-based measure of health from the SF-12. Med Care; 2004.

[19] Cynthia GB et al. Cost-effectiveness of Antenatal Corticosteroid Therapy vs No Therapy in Women at Risk of Late Preterm Delivery. JAMA Pediatr; 2019.

[20] Larson, B A. Calculating disability-adjusted-life-years lost (DALYs) in discrete-time. Cost Effectiveness and Resource Allocation. 2013, 11:18.