Welcome

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Cost benefit issues in clinical development

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synopsis

Clinical development in clinical trials benefit issues of cost

Economics Cost

Types

Strategies

in clinical development and Outcomes Research

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synopsis
Strategy Loops

in multicentric trials of Cost Evaluation Analysis

in cost n benefit issues

Principles

Studies
Cost-Benefit Cost Cost

Effectiveness Analysis utility analysis

Practical

Considerations in Cost 4/21/12 Evaluation Studies

Clinical development
Clinical development is a blanket term used to define the entire process of bringing a new drug or device to the market. It includes drug discovery / product development, pre-clinical research (microorganisms/animals) and clinical trials (on humans). ...

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economics as a king in clinical research

the identification, measure, and comparison of the costs (i.e. resources consumed) and outcomes (clinical, economic, and humanistic) of interventions (pharmaceuticals, non-drug therapies, public health programs)
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When a cost play role in CT

1. Define objective: which regimen should be of prime choice 2. Identify ways to achieve the objective 3. Identify and measure drug cost of each option 4. Identify and measure benefits (clinical outcome) of each option 5. Calculate and interpret the benefits 6. Perform sensitivity analysis : check 4/21/12

When a economics comes into a play

Three basic questions arise: What goods and services shall we produce? How shall we produce them? Who shall receive them

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Answers
economics addresses these questions primarily from the perspective of efficiency maximising the benefits from available resources (or ensuring benefits gained exceed benefits forgone).

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outline

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Cost-benefits issues
Costs Research complex therapies Manufacturing molecule therapy Quality control of molecule quality Preclinical evaluation the risks Application for IND Clinical evaluation the risks phases-I Phase-II
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benef break

replac

assuri

analy

quality

low

Types of costs
Costs vs. Charges Direct costs
?

medical vs. nonmedical Intangible costs

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Direct costs
In the developing a molecule In pre clinical evaluation of a molecule IND (investigational New Drug) application Clinical development Protocol designing CRO selection patents
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Direct medical
Acquisition cost of drug ( price) Supplies to administer drug Supply management cost Professional services cost Other direct costs ADR treatment In and out patient treatment of poor response Emergency room use Hospital over head costs Laboratory services
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Direct non-medical
Patient time cost for treatment or intervention Formal and informal caregiver time Transportation

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Intangible cost
the cost of pain and suffering resulting from a disease, condition, or intervention.

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Strategies in clinical development and Outcomes Research Cost

Higher Lower Higher Evaluate Accept Same

Accept Benefits Accept

Same

Reject

Indifferent

Lower Evaluate

Reject
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Reject

Loops in cost n benefit issues

Benefits may some time be broken into Value if healthy time gained Saving in treatment cost Other savings or benefits

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There has been growing concern that the pooled (i.e., average) economic results from multinational trials may not be reflective of the results that would be observed in individual countries that participated in the trial
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Strategy in multicentric trials

Transnational differences in morbidity/mortality patterns; practice patterns (i.e., medical service use); and absolute and relative prices for this service use (i.e., price weights) Thus decision makers may find it difficult to draw conclusions about the value for the cost of the
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Strategy in multicentric trials contd.,

Types of economic analysis in clinical trials

Cost - efficiency Studies - What should it cost? Cost-of-illness Studies (COI) Economic burden of illness Cost Evaluation Studies
Cost Benefit Analysis (CBA) Cost Effectiveness Analysis (CEA) Cost Utility Analysis (CUA)
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Principles of Cost Evaluation Studies

All

relevant costs and benefits should be counted of evaluation is to compare alternative used of resources is incremental

Purpose

Measurement
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Cost-Benefit Analysis
Evaluates the value of all resources consumed in implementing a program or intervention against the value of the outcome in terms of dollars

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Cost Benefit Analysis

All

costs and health effects are expressed in monetary terms (i.e., must put a $ value on a year of life) Benefit -> all benefits minus all costs, sometimes call Social Return on Investment Benefit Ratio -> All benefits 4/21/12 divided by all costs, sometimes

Cost

Cost

Cost Effectiveness Analysis

CEA

is a method to determine

which program or treatment accomplishes a given objective at the least cost.

Cost-effectiveness

analysis is a

method of comparing alternative treatments in which the costs and

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Cost Effectiveness Analysis

Costs

terms

are expressed in monetary

Benefits

are expressed in natural units, e.g., life-years Effectiveness Ratio -> Cost divided by life-years (or other measure of benefit)
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Cost

Cost utility analysis

Evaluates

the value of an

intervention or a program against the value of the outcome in terms of quality-adjusted life years

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Cost Utility Analysis

Costs

terms

are expressed in monetary

Benefits

are expressed in qualityadjusted natural units, e.g., quality adjusted life-years Utility Ratio -> Cost divided by Quality Adjusted Life Years
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Cost

Practical Considerations in Cost Evaluation Studies

Sources

of Cost Data

Technical

Economic Considerations Considerations

Statistical

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Potential Sources of Economic Data

Clinical

trial forms/medical record abstraction Hospital bills Health system cost-accounting systems (e.g. HMOs) Administrative claims data (e.g. Medicare, Medstat) Patient/provider survey (e.g. MEPS) 4/21/12 Cost scenario

Some Technical Economic Issues

Adjusting

for price (unit cost)

differences

For different years For different settings/locations For different countries (currencies)
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example

Cost-Benefit Analysis of Folic Acid Fortifiction

Cost

Domains Included

Medical care Developmental services CORRECTING DEFECTS Spina bifida: $349,133 Anencephaly: $485,016

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Cost

of low level folic acid fortification = $27.94 million per yr folic acid fortification can mask vitamin B12 deficiency of surveillance of those with undiagnosed vitamin B12 deficiency$5 million per year
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Note:

Cost

Benefits of Folic Acid Fortification

Proportion

of target population with inadequate folate intake - 66% of birth defects averted: 191 spina bifida, 113 anencephaly

Cases

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Folic Acid Fortification: Cost-Benefit Analysis

Economic

benefit of birth defects averted: $121.5 million benefit of fortification program = $93.6 million Ratio = 4.3

Net

Benefit/Cost

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conclusion
for

Clinical trials may provide the best opportunity developing information about a medical therapys value for the cost early in its product life When appropriate types of data are collected and when they are analyzed appropriately, these evaluations can provide data about uncertainties related to the assessment of the value for the cost of new therapies that may be used by policy 4/21/12

eferences
pharmacoeconomics05.pd
clinicaltrials.gov/ct2/show/NCT0

www.uphs.upenn.edu/dgimhsr/prese

1140958
Novel

approaches to incorporating pharmacoeconomic studies into... 0922340

www.ncbi.nlm.nih.gov/pubmed/2 Quality

of life and

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Any questions

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