Example of Health Technology Assessment (HTA)

of a therapy for the reduction of alcohol

consumption

David TyasGlobal HEOR - Lundbeck

Contents• Introduction into a HTA process

• Use SMC as an example but equally could be from many other countries

• Summary of our submission• Main argument• Types of analysis• Clarification question stage

• Summary of questions (what sort)• Final recommendation

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Economic Evaluations in EuropeNorway: Pharmacoeconomic data required for reimbursement; official guidelines in operation.

Finland:Pharmacoeconomic evidence mandatory for evaluating newtherapies for reimbursement and may also be requested for existing therapies.

Sweden:Cost-effectiveness data required for reimbursement.

Denmark:Cost-effectiveness data may be requested for reimbursement decisions.

UK:NICE, SMC, and AWMSG evaluates the cost effectiveness of medicines.

Germany:Guidelines prepared. Institute for Quality and Efficiency in the Health Service established in 2004.

France:Not a formal requirement but increasingly used in reimbursement decisions. Guidelines prepared.

Spain:Health technology assessment at a regional level.

Portugal:Cost-effectiveness data incorporated into reimbursement decisions.

Italy:Cost-effectiveness considered in pricing and reimbursement decisions.

Greece: Guidelines for pharmacoeconomic studies prepared; cost-effectiveness data may be requested.

Belgium: Formal requirement for economic evaluation.

Netherlands:Pharmacoeconomic evidence explicitly required for reimbursement of new products.

Ireland: Guidelines for pharmacoeconomic studies prepared; cost-effectiveness data may be requested.

Poland: C/E and BIA may be requested. HTA agency.

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Manufacturers submission

Clarification questions

Draft advice

Final recommendati

on

SMC process

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Example of a HTA submission dossier- SMC requirements

Chapter 1 Registration DetailsChapter 2 Overview and Positioning Chapter 3 Comparative EfficacyChapter 4 Comparative SafetyChapter 5 Clinical EffectivenessChapter 6 Pharmaco-Economic EvaluationChapter 7 Resource ImplicationsTotal ~ 100 pages

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NalmefeneMain arguments and data

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Indication

Nalmefene is indicated for the reduction of alcohol consumption in adult patients with alcohol dependence who have a high drinking risk level (DRL), without physical withdrawal symptoms and who do not require immediate detoxification.

WHO categoryTotal Alcohol Consumption

(g/day)Women Men

Very high-risk consumption > 60 > 100High-risk consumption 40–60 60–100Medium-risk consumption 20–40 40–60Low-risk consumption 1–20 1–40

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Relative risk for all-cause mortality by average daily intake of alcohol

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Place in therapy

Stages of alcohol abuse/ dependence

Early Middle Late

Brief intervention

Reduction

Abstinence

Trea

tmen

t int

ensi

ty

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Clinical efficacy

Study name Study duration Patients enrolled High drinking risk

ESENSE 1(Mann 2013; Wim van den Brink 2013)

24-week604

(306+298)350

180+170

ESENSE 2(Gual 2013; Wim van den Brink 2013)

24-week718

(358+360)317

(155+162)

SENSE(Wim van den Brink 2014)

52-week675

(509+166)187

3 RCTs in patients with alcohol dependence

Mann et al. 2013. Biol Psychiatry 73(8) 706-713Gual et al. 2013. Eur Neuropsychopharmacol 23(11) 1432-42Wim van den Brink et al. 2014. J PsychopharmacolWim van den Brink et al. 2013. Alcohol and Alcoholism. 1–9 11

Pharmaco Economic analysis

Objective:• To show nalmefene is cost-

effective

Treatment alternatives:• Nalmefene + psychosocial

support• Psychosocial support alone

Perspective:• Healthcare system

Time horizon:• 1 year: period covered by RCTs• 5 years

Population:• nalmefene indication as informed

by phase III clinical programme

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General concept of the model

Decreasecosts

IncreaseQALYs

Reduction of alcohol intake

Quality-Adjusted Life Year (QALY)

QALY=patient quality of life * patient survival

Reduction of alcohol-attributable harms and mortality

Severe morbidities and injuries considered:

• Transport injuries• Injuries other than

transport• Ischaemic heart

disease• Ischaemic stroke• Liver cirrhosis• Pancreatitis• lower respiratory

infections13

Clarification questions

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1. Patient discontinuation2. Calculation of number of days taking therapy3. Application of utility in the model4. Proportion who receive care at a specialist level5. Real world discussion of relapse rate

Final recommendation

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Questions….