Example of Health Technology
Assessment (HTA)
of a therapy for the
reduction of alcohol consumption
David Tyas
Global 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
2
Economic Evaluations in Europe
UK:
NICE, SMC, and AWMSG
evaluates the cost
effectiveness of medicines.
Ireland: Guidelines for
pharmacoeconomic
studies prepared; costeffectiveness data may
be requested.
France:
Not a formal requirement but
increasingly used in
reimbursement decisions.
Guidelines prepared.
Spain:
Health technology
assessment at a
regional level.
Norway:
Pharmacoeconomic data
required for reimbursement;
official guidelines in
operation.
Finland:
Pharmacoeconomic evidence mandatory for evaluating new
therapies 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.
Netherlands:
Pharmacoeconomic evidence explicitly
required for reimbursement of new
products.
Poland:
C/E and BIA may be requested.
HTA agency.
Belgium:
Formal requirement for economic
evaluation.
Italy:
Cost-effectiveness considered in Greece: Guidelines for
Portugal:
pharmacoeconomic studies
pricing and reimbursement
Cost-effectiveness data
prepared; cost-effectiveness data
decisions.
incorporated
may be requested.
into reimbursement decisions.
Germany:
Guidelines prepared.
Institute for Quality and
Efficiency in the Health
Service established in
2004.
3
SMC process
Manufacturers
submission
Clarification
questions
Draft advice
Final
recommendation
4
Example of a HTA submission dossier
- SMC requirements
Chapter 1
Registration Details
Chapter 2
Overview and Positioning
Chapter 3
Comparative Efficacy
Chapter 4
Comparative Safety
Chapter 5
Clinical Effectiveness
Chapter 6
Pharmaco-Economic Evaluation
Chapter 7
Resource Implications
Total ~ 100 pages
5
Nalmefene
Main arguments and data
6
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 category
Total Alcohol Consumption (g/day)
Women
Men
> 60
> 100
High-risk consumption
40–60
60–100
Medium-risk consumption
20–40
40–60
Low-risk consumption
1–20
1–40
Very high-risk consumption
7
Relative risk for all-cause mortality by
average daily intake of alcohol
8
9
Place in therapy
Treatment intensity
Abstinence
Reduction
Brief intervention
Early
Middle
Late
Stages of alcohol abuse/ dependence
10
Clinical efficacy
3 RCTs in patients with alcohol dependence
Study name
Study duration
Patients enrolled
High drinking risk
24-week
604
(306+298)
350
180+170
24-week
718
(358+360)
317
(155+162)
187
52-week
675
(509+166)
ESENSE 1
(Mann 2013; Wim van
den Brink 2013)
ESENSE 2
(Gual 2013; Wim van
den Brink 2013)
SENSE
(Wim van den Brink
2014)
Mann et al. 2013. Biol Psychiatry 73(8) 706-713
Gual et al. 2013. Eur Neuropsychopharmacol 23(11) 1432-42
Wim van den Brink et al. 2014. J Psychopharmacol
Wim 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
12
General concept of the model
Reduction of
alcohol intake
Reduction of
alcohol-attributable
harms and mortality
Decrease
costs
Increase
QALYs
Severe morbidities and
injuries considered:
•Transport injuries
•Injuries other than transport
•Ischaemic heart disease
•Ischaemic stroke
•Liver cirrhosis
•Pancreatitis
•lower respiratory infections
Quality-Adjusted Life Year (QALY)
QALY=patient quality of life * patient survival
13
Clarification questions
14
1.
Patient discontinuation
2.
Calculation of number of days taking therapy
3.
Application of utility in the model
4.
Proportion who receive care at a specialist level
5.
Real world discussion of relapse rate
15
Final recommendation
16
17
Questions….