Clinical Development

8/2/2019 Clinical Development

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Welcome


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Cost benefit issues in clinica

development


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synopsis

Clinical development

Economics in clinical trials

Cost benefit issuesTypes of cost

Strategies in clinical

development and OutcomesResearch


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synopsis

Strategy in multicentric trials

Loops in cost n benefit issues

Principles of Cost EvaluationStudies

Cost-Benefit Analysis

Cost Effectiveness AnalysisCost utility analysis

Practical Considerations in CostEvaluation Studies


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Clinical development

Clinical development is a blanket termused to define the entire process ofbringing a new drug or device to themarket. It includes drug discovery /product development, pre-clinicalresearch (microorganisms/animals)

and clinical trials (on humans). ...


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economics as a king inclinical research

the identification, measure,and comparison of the costs

(i.e. resources consumed)and outcomes (clinical,economic, and humanistic)

of interventions(pharmaceuticals, non-drugtherapies, public healthprograms)


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When a cost play role inCT

1. Define objective: which regimenshould be of prime choice

2. Identify ways to achieve theobjective

3. Identify and measure drug cost ofeach option

4. Identify and measure benefits(clinical outcome) of each option

5. Calculate and interpret the benefits

6. Perform sensitivity analysis : check


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When a economics comesinto a play

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


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Answers

economics addresses thesequestions primarily from theperspective of efficiency

maximising the benefits fromavailable resources (or ensuringbenefits gained exceed benefits

forgone).


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outline


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Cost-benefits issuesCosts bene

Research complex break

therapies

Manufacturing molecule repla

therapy

Quality control of molecule assurquality

Preclinical evaluation anal

the risks

Application for IND qualit

Clinical evaluation lo

the risks

phases-I

Phase-II


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Types of costs

Costs vs. Charges

Direct costs

? medical vs. nonmedical

Intangible costs


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Direct costs

In the developing amoleculeIn pre clinical evaluation of

a moleculeIND (investigational NewDrug) application

Clinical developmentProtocol designingCRO selectionpatents


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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 orinterventionFormal and informal caregiver

timeTransportation


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Intangible cost

the cost of pain and sufferingresulting from a disease,condition, or intervention.

S i i li i l d l d


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Strategies in clinical development andOutcomes Research

Cost

Higher SameLower

Higher Evaluate AcceptAccept

Benefits

Same Reject Indifferent

Accept

Lower Reject RejectEvaluate


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Loops in cost n benefitissues

Benefits may some time be broken

into

Value if healthy time gained

Saving in treatment cost

Other savings or benefits


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Strategy in multicentrictrials

There has been growing concern that the

pooled (i.e., average) economic results

from multinational trials may not bereflective of the results that would be

observed in individual countries that

participated in the trial


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Transnational differences in morbidity/mortalitypatterns; 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

Strategy in multicentrictrials contd.,


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Types of economicanalysis in clinical trials

Cost - efficiency Studies - Whatshould it cost?

Cost-of-illness Studies (COI) -Economic burden of illness

Cost Evaluation StudiesCost Benefit Analysis (CBA)Cost Effectiveness Analysis (CEA)Cost Utility Analysis (CUA)


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Principles of CostEvaluation StudiesAll relevant costs and benefits

should be counted

Purpose of evaluation is tocompare alternative used ofresources

Measurement is incremental


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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 areexpressed in monetary terms(i.e., must put a $ value on a yearof life)

Cost Benefit -> all benefits minusall costs, sometimes call Social

Return on Investment

Cost Benefit Ratio -> All benefits

divided by all costs, sometimes


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Cost EffectivenessAnalysisCEA is a method to determine

which program or treatment

accomplishes a given objective atthe least cost.

Cost-effectiveness analysis is amethod of comparing alternative

treatments in which the costs and


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Cost EffectivenessAnalysisCosts are expressed in monetary

terms

Benefits are expressed innatural units, e.g., life-years

Cost Effectiveness Ratio -> Costdivided by life-years (or othermeasure of benefit)


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Cost utility analysis

Evaluates the value of an

intervention or a program against

the value of the outcome in termsof quality-adjusted life years


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Cost Utility Analysis

Costs are expressed in monetaryterms

Benefits are expressed in quality-adjusted natural units, e.g.,quality adjusted life-years

Cost Utility Ratio -> Cost dividedby Quality Adjusted Life Years


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Practical Considerations inCost Evaluation StudiesSources of Cost Data

Technical Economic

Considerations

Statistical Considerations


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Potential Sources ofEconomic DataClinical trial forms/medical record

abstraction

Hospital bills

Health system cost-accountingsystems (e.g. HMOs)

Administrative claims data (e.g.

Medicare, Medstat)Patient/provider survey (e.g.

MEPS)

Cost scenario


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Some Technical EconomicIssuesAdjusting for price (unit cost)

differences

For different years

For different settings/locations

For different countries

(currencies)

(e.g. DCPP)


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example

Cost-Benefit Analysis of Folic AcidFortifiction

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

Note: folic acid fortification can

mask vitamin B12 deficiency

Cost of surveillance of those with

undiagnosed vitamin B12 deficiency-$5 million per year

Bene ts o Fo c Ac


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Bene ts o Fo c AcFortification

Proportion of targetpopulation with inadequatefolate intake - 66%

Cases of birth defectsaverted: 191 spina bifida,113 anencephaly


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Folic Acid Fortification:Cost-Benefit AnalysisEconomic benefit of birth defects

averted: $121.5 million

Net benefit of fortificationprogram = $93.6 million

Benefit/Cost Ratio = 4.3

conclusion


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conclusionClinical trials may provide the best opportunity

for

developing information about a medical therapys

value

for the cost early in its product lifeWhen 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


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ferences

www.uphs.upenn.edu/dgimhsr/prespharmacoeconomics05.pd

clinicaltrials.gov/ct2/show/NCT0

1140958

Novel approaches to incorporatingpharmacoeconomic

studies into...

www.ncbi.nlm.nih.gov/pubmed/20922340

lit f lif n
http://books.google.co.in/books?id=8wlpQgAACAAJ&dq=pharmacoeconomics+in+clinical+trials&hl=en&sa=X&ei=bB-OT8KiJ8XYrQfQ8c2SCQ&ved=0CDsQ6AEwAAhttp://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pdhttp://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pdhttp://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pdhttp://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://books.google.co.in/books?id=8wlpQgAACAAJ&dq=pharmacoeconomics+in+clinical+trials&hl=en&sa=X&ei=bB-OT8KiJ8XYrQfQ8c2SCQ&ved=0CDsQ6AEwAAhttp://books.google.co.in/books?id=8wlpQgAACAAJ&dq=pharmacoeconomics+in+clinical+trials&hl=en&sa=X&ei=bB-OT8KiJ8XYrQfQ8c2SCQ&ved=0CDsQ6AEwAAhttp://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.ncbi.nlm.nih.gov/pubmed/20922340http://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pdhttp://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pdhttp://www.uphs.upenn.edu/dgimhsr/presenta/.../pharmacoeconomics05.pd


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Any questions


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Clinical Development