David Tyas Global HEOR - Lundbeck

1. Example of Health Technology Assessment (HTA) of a therapy for the reduction of alcohol consumption David Tyas Global HEOR - Lundbeck

2. 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

3. Economic Evaluations in Europe Norway:Pharmacoeconomic datarequired for reimbursement; official guidelines in operation. Finland: Pharmacoeconomic evidence mandatory for evaluating new therapies for reimbursement and may also be requested for existing therapies. UK: NICE, SMC, and AWMSG evaluatesthe cost effectiveness of medicines. Sweden: Cost-effectiveness data required for reimbursement. Ireland:Guidelines for pharmacoeconomic studies prepared; cost-effectiveness data may be requested. Denmark: Cost-effectiveness data may be requested for reimbursement decisions. Netherlands: Pharmacoeconomic evidence explicitly required for reimbursement of new products. France: Not a formal requirement but increasingly used in reimbursement decisions. Guidelines prepared. Poland: C/E and BIA may be requested. HTA agency. Belgium: Formal requirement for economic evaluation. Spain: Health technology assessment at a regional level. Germany: Guidelines prepared. Institute for Quality and Efficiency in the Health Service established in 2004. Italy: Cost-effectiveness considered in pricing and reimbursement decisions. Greece: Guidelines for pharmacoeconomic studies prepared; cost-effectiveness data may be requested. Portugal: Cost-effectiveness data incorporated into reimbursement decisions.

4. SMC process Manufacturers submission Clarification questions Draft advice Final recommendation

5. 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

6. Nalmefene Main arguments and data

7. 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.

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

10. Place in therapy Abstinence Reduction Treatment intensity Brief intervention Early Middle Late Stages of alcohol abuse/ dependence

11. Clinical efficacy 3 RCTs in patients with alcohol dependence Mann et al. 2013. Biol Psychiatry 73(8) 706-713 Gual et al. 2013. EurNeuropsychopharmacol 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

12. 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

13. General concept of the model Decrease costs Reduction of alcohol intake Reduction of alcohol-attributable harms and mortality 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

14. Clarification questions

15. Patient discontinuation • Calculation of number of days taking therapy • Application of utility in the model • Proportion who receive care at a specialist level • Real world discussion of relapse rate

16. Final recommendation

18. Questions….