Documente Academic
Documente Profesional
Documente Cultură
Pharmaceuticals in Europe
Åsa KORNFELD
Mondher TOUMI
• Prof. Mondher Toumi is M.D. by training, M.Sc. in Biological Sciences and Ph.D. in Economic Sciences.
Mondher Toumi is Professor of Public Health at Aix-Marseille University.
• After working for 12 years as Research Manager in the laboratory of pharmacology at the University of
Marseille, he joined the Public Health Department in 1993. He worked from 1995 in the pharmaceutical
industry for 13 years.
• Mondher was appointed Global Vice President at Lundbeck A/S in charge of health economics, outcome research, pricing,
market access, epidemiology, risk management, governmental affairs and competitive intelligence.
• In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in
strategic decision-making.
• In February 2009 he was appointed Professor at Lyon I University in the Department of Decision Sciences and Health Policies.
The same year, he was appointed Director of the Chair of Public Health and Market Access. He conducted the first European
University Diploma of Market Access (EMAUD) in Paris, France. Additionally, he recently created the Market Access Society to
promote research and scientific activities around market access, public health and medico-economic assessment. Since 2009, he
also chairs the Annual Market Access Day, a purely academic event sponsored by EMAUD that has become a reference event in
the area.
• Since September 2014, he joined the research unit EA3279 of the public health department, at Aix-Marseille University (France)
as Professor. Mondher Toumi is also visiting Professor at two famous Chinese universities: Shenyang Pharmaceutical University
and Beijing University (Third Hospital).
• In addition to contributing as a reviewer on several editorial boards, he is Chief Editor at an online Journal of Market Access and
Health Policy. Furthermore, he did two mandates as Co-Chair of the Research Review Committee of ISPOR in 2012 and 2013.
• He is a recognized expert in drug development and an authority on market access and risk management. He has more than 200
scientific publications and oral communications, and has contributed to several books. He has just finished a book on Market
Access to become soon available.
3
Course Learning
Objectives
1. To provide an overview of the European reimbursement
policies
7
Determinants of Health
8
Pharmaceutical Spending
in Europe
Widening the Gap?
10
Current Situation
11
Pharmaceutical Spending
Total, % of health Spending (2012)
13
Global Pharmaceutical
Spending and Growth
Source:Vogler S, Zimmermann N, Habl C, Piessnegger J, Bucsics A. Discounts and rebates granted to public payers
for medicines in European countries. South Med Rev. 2012;5(1):38–46
17
Change in public spending on health as a share
of total public (government) spending,
2007–2011
1
0.8
0.6
0.4
0.2
-0.2
-0.4
-0.6
Sweden Germany Poland UK Italy Hungary France Spain
Source: Thomson S, Figueras J, Evetovits T, Jowett M,Mladovsky P, Maresso A et al., eds (2014). Economic crisis, health systems and health in Europe: impact and implications for policy.
18
Maidenhead: Open University Press.
Healthcare expenditure in 2013
(US$ per capita)
Sweden
Spain*
Germany
Italy
Hungary
Poland
21
All is About Affordability
22
The Oncology Tsunami
23
The Reverse Blockbuster
Pyramides
BLOCKBUSTER
GP Product 1 00 000
1 000
Specialist
100 000 10 000
product
X =
Orphan 10 000 100 000
Ultra-orphan 5 000
200 000
Orphan drug is the other pending tsunami with a couple of thousand of designated
orphan drugs
24
EBM used by HTA Will NOT Help
Containing the Cost
Co
“Some fear that evidence based medicine will be hijacked by
purchasers and managers to cut the costs of health care. This
would not only be a misuse of evidence based medicine, but
suggests a fundamental misunderstanding of its financial
consequences. Doctors practicing evidence based medicine will
identify and apply the most efficacious interventions to
maximize the quality and quantity of life for individual patients;
this may raise rather than lower the cost of their care.”
25
Is Incremental Cost
Effectiveness Ratio The
Solution?
26
Could Cost Effectiveness
Resources Allocation?
A 40,000
B 53,300
C 57,100
D 125,000
27
Could Cost Effectiveness Help
Allocating Resources?
A 250 40,000
B 300 53,300
C 70 57,100
D 80 125,000
Need for Budget Impact?
A 250 10 40,000
B 300 16 53,300
C 70 4 57,100
D 80 10 125,000
29
Affordability
Drive the Decision
A 250 10 40,000
B 300 16 53,300
C 70 4 57,100
D 80 10 125,000
Cost-
Effectiveness Budget Impact
31
From Price to Value and
Incremental Value
Innovation Pillars for
Pharmaceuticals
1. Prevent copy cat
– Patent
– Data protection
2. Value-based pricing
– Often unknown and source of multiple
confusion
33
Value-Based Pricing
“Value-Based Pricing or Value optimized pricing is a business
strategy. It sets selling prices on the perceived value to the
custumer, rather than on the actual cost of the product, the
market price, competitors prices, or the historical price.”
1
Consequences
34
What is Value?
“Price
is what you pay
and value
is what you get”
35
VBP With No CAP Price
Price E
C
B
Benefit 36
VBP with CAP Price, Over Costs
Charged by Private Insurance
C
B
Benefit 37
VBP with CAP Price
Managed by NHS
Price
E CAP Price
D
C
A Benefit 38
There is No Solution and
No Wish to Find a Solution
• Today no country does a reasonable resource allocation
under budget constraint
• QALY is a wonderful universal measure instrument but
carry substantial limitations
• Excluding QALY means excluding the only way to allocate
fairly resource across heterogeneous conditions
• Relying primarily or exclusively on QALY is as wrong as
excluding QALY
• The solution is likely a multi-criteria decision, but we
still don’t know or don’t want to make it reproducible,
reliable and meaningful
• We operate under a deliberative process
39
Confusion Around
‘Paying for Innovation’
Payers buy health production measured through the proxy of
‘medicines’
• The payer does not buy ‘innovation’ for its own sake
• Innovation might not deliver improved outcomes
• Increased R&D cost of biologics might not translate into patient
benefits
• The innovative step is stimulated through national research policy
incentives and not through health insurance
What exactly constitutes ‘sufficient compensation’ for R&D, and whether each
country should contribute equally to R&D is heavily debated
40
Market Access Paradigm
Pharmaceutical Business
Environment
Drug Value Chain
42
Market Access is Different
From Regulatory
Regulatory Market Access
Fulfil the requirements
Negotiate with payers
of market authorisation
43
Market Access Is Different
From Marketing
Marketing Market Access
Perception based Evidence based
44
Market Access Is…
45
From Approval to Funding
National
Payers
agency
Safety
Funding, Price &
Efficacy
Reimbursement
Quality
46
From Approval to Funding
National
HTA Payers
agency
Efficacy, effectiveness
Safety
& ICER Funding, Price &
Efficacy
Evaluation for funding Reimbursement
Quality
or formulary listing
47
From Approval to Funding
National
EMA HTA Payers
agency
Efficacy,
Safety Transpose
effectiveness & ICER Funding, Price &
Efficacy decisions at a
Record for funding Reimbursement
Quality national level
or formulary listing
48
Payers are Heterogeneous
Who are the Payers? (1/3)
50
Who are the Payers? (2/3)
Member of
HTA
committees
Private health GPs in UK and
insurance Germany
Pharmacists Member of
in some
countries
Payers pricing
committees
Hospital
managers, Employers
Doctors
Patients
51
Who are the Payers? (3/3)
With diverse
Growing fast Heterogeneous
perspectives
52
What are the Payers Doing?
53
Cost-Containment Measures
54
General P&R Policies
Drug Reimbursement
Policies
Health
Technology • Conditional reimbursement on meeting specific clinical and/or
Assessment economic (cost-) effectiveness criteria
(HTA)
56
Reimbursement Policies in EU
Countries
Systematic literature review identified policy measures related to pharmaceutical
reimbursement in EU Member States (including Croatia) and the EEA countries (Iceland,
Lichtenstein, Norway) from 1995-2013
Co-payment
Reimbursement rates
Reference price systems
Positive list
Reimbursement process
Pharmaco-economic evaluation
Generic substitution
Reimbursement review
Tendering
INN prescribing
Negative list
HTA
Discounts/ Rebates/ Clawback/ Price negotiations
Managed entry agreements
Value based pricing
Policies linked to rational use
Pharmaceutical budgets
57
Source: Vogler S, Zimmermann N, Habimana K, Study of the policy mix for the reimbursement of medicinal products,january 2014, Vienna
Price Regulation Policies
International • Applied in 26 EU Member States (except Sweden and UK)
reference • Benchmarks product prices in one country against prices of the same
pricing product in a selected basket of other countries
58
Expenditure Control Policies
Discounts/ • Imposed upon manufacturers and pharmacists, such that they have to
rebates return a part of their revenue
Public • Currently, the Netherlands and Germany are well known examples for
tendering ample use of public tendering
59
A Matter of Culture Across
Countries
France
• Objective
– Secure all products gain access at the right price
• Process
– Driver: Public health relevance of benefit over the next
best alternative
– Method: Single double blind reference randomized clinical
trial
– Effect size
• Impact
– Gate-keeper for price and reimbursement
61
United Kingdom
• Objective
– Obtain rational allocation of resources
• Process
– Driver: Maximization of efficiency of the health care
output
– Method: Cost utility
– Threshold is £ 20,000/QALY
• Impact
– Recommendation for prescriber
– Formulary listing
62
Germany
• Objective
– Obtain savings on drug spending with no impact on
safety/efficacy
• Process
– Driver: Same effect same price (Jumbo group)
– Method:
– Meta-analysis
– Efficiency frontier
• Impact
– Reimbursement decreased
63
Other Countries Fall
in Between
• Sweden
– Between UK and Germany
• Canada
– Between France and UK
• Etc.
64
Overview and Comparison of
Reimbursement Processes of
Pharmaceuticals in a Selection of
European Markets
Content
• Health care funding
• Decision Makers
• Pricing &
Reimbursement
SE
processes
UK • Pricing &
Reimbursement drivers
• Key specificities and
trends
PL
DE • Cost-containment tools
FR
HU
ES
IT
66
FRANCE
Health & Pharmaceutical
Expenditure
Total health expenditure, % of GDP Public health expenditure, % of GDP Pharmaceutical expenditure, % GDP
Transparency Committee
Health Technology French National Authority for (CT)
Opinion
Assessment Health (HAS)
Economic and Public Health
Assessment Committee
(CEESP)
Decision
Economic Committee for
Pricing Healthcare Products (CEPS)
ANSM, Agence Nationale de Sécurité du Médicament et des Produits de Santé; HAS, Haute Autorité de Santé ; CEESP, Commission Evaluation Economique et de Santé
70
Publique ; CT, Commission de la Transparence; CEPS, Comité Economique des Produits de Santé ; UNCAM, Union Nationale des Caisses d’Assurance Maladie
P&R Process
Marketing Authorisation
EU Level: European Medicines Agency (EMA)/European Commission
Timelines National Level: National Agency for the Safety of Medicine and Health Products (ANSM)
(Months)**
Reimbursement and pricing decisions are endorsed by the Ministry of Health and
published in the official journal
71
Medical Assessment by CT
Key Decision Drivers
• Disease severity
Actual benefit • Efficacy /safety
(AB) • Position in the therapeutic strategy Driver of
Service Médical • Impact on public health reimbursement rate
• Type of treatment (preventive, curative or
Rendu (SMR) symptomatic)
• Quantitative estimation of
Target population prevalence/incidence in France of the Driver of price-volume
population who might benefit from the
product in claimed indications
agreements
72
Medical Assessment by CT
AB and IAB
5 levels of AB 5 levels of reimbursement
Major 100%* or 65%
Actual benefit (AB) Important 65%
Service Médical Rendu Moderate 30%
(SMR)
Weak 15%
Insufficient 0%
* Can be 100% for specific drugs, such as drugs in oncology or transplantation
• Data submitted by the manufacturer to CEESP and CEPS, along with the request for
inclusion/renewal of inclusion of the product on the reimbursable drugs formulary
• No publication of CEESP opinions until the end of price negotiation
Expected to inform on the compliance of health economic evaluations with the HAS
guidelines, but not to inform on whether the intervention is cost-effective or not 74
Pricing & Reimbursement
per Channel
Channel Retail Hospital
Some retail medicines
used for hospital care
• Pharmaco-therapeutic class rebates: For each therapeutic class, the CEPS attributes
an annual sales growth target; Manufacturers are ‘taxed’ if sales exceed the target
• Rebates at product level (frequently used by CEPS): price-volume agreements, cost
clauses for daily dosages, dosage clauses: If the clauses are exceeded, a price
reduction or rebates will be applied
Risk-sharing agreements
• Each year, the Social Security funding law provides price cuts to generate savings (E.g.
For 2014, the target was €1 billion savings from price cuts of patented drugs and
generics)
• Drugs delisted following CT re-assessment
77
Key Cost-Containment
Measures (2/2)
New regulations to contain pharmaceutical budgets to
deal with growing development of innovative medicines
• Social security funding bill for 2015: Proposal regarding hepatitis C therapies to
contain potential impact on healthcare budget of costly medicines (Solvadi®)
• To create a progressive contribution mechanism for hepatitis C drug if annual
turnover exceeds a threshold set by law
• CEPS is responsible for creating reference price groups (including generic and
reference medicines) where generic penetration rates are below fixed levels at set
intervals following generic market launch Patient co-payment if product price is
over the reference price
• Financial incentives for physicians based on proportion of generic prescriptions
within five therapeutic classes (antibiotics, proton pump inhibitors, antidepressants,
statins, anti-hypertensives)
• Financial incentives for pharmacists to achieve substitution targetsFor 2015, this
target is fixed at 85%
78
GERMANY
Health & Pharmaceutical
Expenditure
Total health expenditure, % of GDP Public health expenditure, % of GDP Pharmaceutical expenditure, % GDP
Health insurance is compulsory since 1 January 2009 but patients are free to choose their
health insurance fund
Health Technology
Opinion
Institute for Quality and
Assessment Efficiency in Healthcare (IQWiG)
Decision
Federal Association of Health
Pricing Insurance Funds (GKV-SV)
BfArM, Bundesinstitut für Arzneimittel und Medizinprodukte; G-BA, Gemeinsamer Bundesausschuss; IQWiG, Institut für Qualität und Wirtschaftlichkeit im
Gesundheitswesen; GKV-SV, Gesetzliche Krankenversicherung-Spitzenverband
82
Market Access Process
Overview
83
P&R Process for
Timelines
New Drugs, EBA
(Months)
Marketing Authorisation
EU Level: European Medicines Agency (EMA)/European Commission
National Level: Federal Institute for Drugs and Medical Devices (BfArM)// Paul-Ehrlich-Institut (PEI)
Early Benefit Assessment
3
No additional
Additional benefit benefit Reference price
Not eligible possible or possibility to
Eligible for reference price create a reference group
for premium price
negotiation/arbitration
Manufacturer/GKV-SV
Manufacturer/GKV-SV
6
Reimbursement price
Reimbursement price negotiation Reference price
negotiation (Not exceeding the cost of comparative
Discount
No
No
agreement X Arbitration board X agreement
3
85
EBA: Methodology and
Decision Drivers (2/2)
4 Outcome Quality of Extent of Effect
Categories Available Evidence Size
Additional benefit
Mortality Proof (High) Major assessment based on
available evidence,
Morbidity Considerable
Indication assessment of quality of
Health-related (Moderate)
Minor available evidence and extent
quality of life
Hint (Low)
of effect size at outcome level
Adverse events Not quantifiable
Sustained and large improvement in outcome not previously achieved with the appropriate
1 Major comparator
2 Considerable Significant improvement in outcome not previously achieved with the appropriate comparator
3 Minor Moderate and not just small benefit not previously achieved with the appropriate comparator
There is evidence that additional benefit exists, however the scientific information is not sufficient
4 Not quantifiable to estimate the size of the additional benefit
5 None No additional benefit demonstrated
No additional
6 Inferior Less benefit than the appropriate comparator benefit 86
Specific Considerations
in EBA
Extent of benefit based on the 95% upper limit of the confidence
interval of the relative risk ratio
•Mortality:
• Major : UL CI95% < 0.85; Considerable : UL CI95% < 0.95; Minor : UL CI95% <1
•Morbidity/QoL
• Major : UL CI95%< 0.75; Considerable : UL CI95%< 0.90; Minor : UL CI95%< 1
•Adverse events or minor symptoms
• Major: Not possible; Considerable : UL CI95%< 0.80; Minor : UL CI95%< 0.90
Prescribing controls
• G-BA can set binding prescribing conditions for new drugs (e.g. indication, dosage,
patient population)
• GKV-SV and manufacturers can also agree on prescribing conditions following price
negotiation
Generic incentives
Central Technical-Scientific
Commission (CTS)
Decision
Italian Medicines Agency Pricing & Reimbursement
Pricing & Reimbursement (AIFA) Committee (CPR)
Opinion
Regional
Regional HTA Bodies
Decision
Regional authorities
AIFA, Italiana del Farmaco; CTS, Commissione Tecnico Scientifica;
94
CPR, Comitato Prezzi e Rimborso
P&R Process
Marketing Authorisation
Inclusion in National Pharmaceutical Formulary (PFN) and Publication in the Official Journal (Gazzetta
Ufficiale)
Reimbursement Reimbursement
Description
class rate
A 100% Essential pharmaceuticals
Prescription-only pharmaceuticals reimbursed only under specific
A with notes 100%
conditions
Prescription-only pharmaceuticals reimbursed only when used in
H 100%
hospitals under specialist supervision
C 0% Prescription-only pharmaceuticals which are not reimbursed
C bis 0% Over-the-counter pharmaceuticals (non-prescription drugs)
Temporary class for new drugs with marketing approval but not yet
C nn 0%
assessed by AIFA
Budget impact
Sales forecast
Cost-effectiveness
• Not a main driver in pricing decisions but can be provided by companies for innovative
products and be used for pricing negotiations
• For non-reimbursed medicines (Class C), prices are freely determined (with some limitations: price
declaration) by manufacturers and monitored by AIFA
• For hospital drugs, regional/local negotiations or tenders to set drug prices (max. price sets by AIFA)
97
Key Market Access
Specificities
Whole P&R process is not completely transparent
•No publication of assessments at national and few at regional level
•Uncertainty on impact of cost-effectiveness analyses in reimbursement decisions
•Level of innovation criteria unclear
• Set on case-by-case basis for innovative and expensive products to manage budget impact
and uncertainty about clinical benefit and/or cost-effectiveness
• Non-outcome-based (cost-sharing, budget cap, price volume agreements) or outcome-
based (risk-sharing and payment for performance)
99
Key Cost-Containment
Measures (2/2)
Mandatory discount and PFN review
• Annual 5% mandatory price discount on public prices of all drugs or payback system option
for equivalent savings (manufacturer choice) and 1% mandatory discount on retail drugs
• AIFA is reviewing the National Pharmaceutical Formulary (PFN) since Balduzzi Law
(189/2012): Expected to lead to price revision and reimbursement delisting
Prescribing restrictions
• Prescribing restrictions set by the CTS issuing prescribing guidelines (or AIFA nota) specifying
the conditions under which certain products can be reimbursed
100
SPAIN
Health & Pharmaceutical
Expenditure
Total health expenditure, % of GDP Public health expenditure, % of GDP Pharmaceutical expenditure, % GDP
103
Decision Making Bodies
European Medicines
Agency (EMA)/European European Level
Commission
Marketing authorization
Spanish Medicines
Agency (AEMPS) National Level
Central
Opinion
National health
Health Technology Technology Assessment
Assessment Agency (AETS)
General Subdirectorate of
Quality of Medicines and Health
Products (SGCMPS)
Decision
Ministry of Health
Pricing & Reimbursement (MSSSI) Interministerial Commission for
Pricing of Medicinal Products
(CIPM)
Opinion
Regional
Regional HTA Bodies
Drug regional access
Decision
Regional authorities
AEMPS, Agencia Española del Medicamento y Productos Sanitarios; CIPM, Comisión Interministerial de Precios de los Medicamentos ; MSSSI, Ministerio de Sanidad, Servicios
104
Sociales e Igualdad; SGCMPS, Subdirección General de Calidad de Medicamentos y Productos Sanitarios; AETS, Agencia de Evaluación de Tecnologías Sanitarias
P&R Process
Marketing Authorisation
(AETS)
Support for
Interministerial Commission for Pricing of Medicinal Products (CIPM)
P&R
Final pricing decision
decisions
Reimbursement conditions
• Negative list for products excluded from reimbursement
• Hospital drugs reimbursed at 100%
• Co-payment for retail drugs:
• Based on patient’s income for drugs indicated for non chronic/severe diseases:
o Workers: 0% co-payment for long-term unenmployed, 40%, 50% or 60% co-payment based on
income with no maximum co-payment
o Pensioners: 0% co-payment for underpriviledged pensioners, 10% or 60% co-payment based on
income with a maximum co-payment
• Fixed co-payment system for drugs indicated for chronic/severe diseases:
o 10% co-payment with a maximum co-payment per prescription
106
CIPM Pricing Negotiation
Criteria
Degree of therapeutic innovation
Budget impact
Company profit
•Disparities in drug access and cost-containment policies across regions with disparities in
terms of:
•Formulary listings
•Prescribing guidelines/incentives
•Use of market access agreements
•Drug assessment performed by numerous healthcare department (7regional HTA agencies
and drud evaluation committees in each region)
•To date, no formal health economic assessment BUT expected since Decree-Laws 9/2011
and 16/2012
•A new committee composed by health economics experts would be responsible for cost-
effectiveness and budget impact evaluation
•Its recommendations would support the CIPM pricing decisions
•At this time, no details on the implementation have been released
Payback system
•Payback based on percentage of quarterly turnover of reimbursed drugs (1.5% if turnover
<€3 million and 2% if turnover >€3 million)
110
Key Cost-Containment
Measures (2/2)
Price cuts & delisting
•In 2012, price cut of 20% for drugs to treat urinary incontinence
•In 2012, 417 drug presentations belonging to 19 drug classes were delisted from the public
funding (reimbursable for specific chronic conditions)
•In 2012, co-payment rate for the working population has been based on annual incomes
with no maximum threshold, and has been extended to pensioners
111
United Kingdom
Health & Pharmaceutical
Expenditure
Total health expenditure, % of GDP Public health expenditure, % of GDP Pharmaceutical expenditure, % GDP
114
Decision Making Bodies
European Medicines Agency European Level
(EMA)/European Commission
Marketing authorization
Medicines and Healthcare
Products Regulatory National Level
Agency (MHRA)
Opinion
Scottish Medicines Consortium
Assessment (SMC)
Decision
Funding Regional authorities
115
P&R Process
Marketing Authorisation
EU Level: European Medicines Agency (EMA)/European Commission
National Level: Medicines and Healthcare Products Regulatory Agency (MHRA)
ICER < £20,000 £20,000 < ICER < £30,000 ICER > £30,000
Recommendation likely to Recommendation not Recommendation likely to
be positive predictable be negative
• NICE adopts a more flexible approach for life-extending treatment at the end of life
Short life expectancy<24 months
Life extension with drugs>3 months vs current NHS treatment
Small patient populations
• Drugs which meet end-of-life criteria can potentially be recommended at higher ICER
threshold (usually between £30,000 and £50,000)
117
NICE & SMC Process
NICE in England SMC in Scotland
Scope • Binding guidance in England and Wales • Binding guidance in Scotland
119
Key Market Access
Specificities
Cancer Drugs Fund in England
•£21million fund launched in 2013 for one year to cover the cost of medicines not available
for routine prescription for rare diseases (not recommended by SMC) and extended until
2016
•Hospital drugs in England are funded by the CCGs through Diagnosis-Related Group (DRG)
system called Payment by Results (PbR) (do not apply to Scotland, Wales or Northern
Ireland)
•Some high-cost medicines may be excluded from PbR and directly funded by the CCGs 120
120
Key Cost-Containment
Measures (1/2)
Payback
• PPRS Scheme members have to payback the Department of Health for expenditure on
branded medicines above allowed growth rate (0% in 2014 and 2015, 1.8% in 2016 and
2017, 1.9% in 2018)
Prescribing controls
• Delisting by NHS England of drugs eligible for funding from Cancer Drugs Fund budget
• In November 2015, 16 therapies will be delisted
Generic policies
122
POLAND
Health & Pharmaceutical
Expenditure
Total health expenditure, % of GDP Public health expenditure, % of GDP Pharmaceutical expenditure, % of GDP
Supplementary Private • Increasing since recent years to cover procedures and treatments not
Insurance covered by NFZ plan
125
Decision Making Bodies
European Medicines Agency
European Level
(EMA)/European Commission
Opinion
Assessment and Tariff System
Assessment (AOTMiT)
Economic Committee
Reimbursement & Pricing (representatives of Ministry of
Health and NFZ)
Decision
Final Decision Ministry of Health
126
AOTMiT, Agencji Oceny Technologii Medycznych i Taryfikacji ; URPL, Urząd Rejestracji Produktów Leczniczych, Wyrobów Medycznych i Produktów Biobójczych
P&R Process
Marketing Authorisation
Agency for Health Technology Assessment and Tariff
EU Level: European Medicines Agency (EMA)/European System (AOTMiT)
Commission Assessment of pricing & reimbursement applications
when no reimbursed therapeutic alternatives
National Level: Office for Registration of Medicinal Products,
available in Poland
Medical Devices and Biocidal Products (URPL)
Timelines AOTMiT
(Months) Initial verification and methodological
assessment of application
Pricing & Reimbursement Decision
Ministry of Health
Transparency Council
Appraisal of the application
Opinion
Economic Committee ( representatives of Ministry of Health
AOTMiT President
and NFZ)
Reimbursement recommendation
Pricing negotiations & reimbursement recommendations
6
Ministry of Health
Final pricing & reimbursement decision
Reimbursement Levels
100% Proven efficacy in the treatment of malignant cancers, psychotic disorder, mental
impairment, developmental disorder or infectious disease that constitutes serious threat to
the population
100%+fixed co- Use >30 days + high monthly cost with 30% co-payment (exceeds 5% of minimum wage)
payment (PLN Use ≤30 days + high monthly cost with 50% co-payment (exceeds 30% of minimum wage)
3.20)
70% Use >30 days but do not meet criteria for 100% reimbursement
50% Use ≤30 days but do not meet criteria for 100% reimbursement
128
*Rationalization analysis (if budget impact demonstrates increase in reimbursement expenditure)
Pricing Criteria
Pricing decision drivers
The following criteria are considered:
• AOTMiT recommendations
• Drug prices and any price agreements in other EU/EFTA countries where the drug is reimbursed
• Treatment cost of the new drug versus therapies already available
• Budget impact
• Cost-effectiveness (cost/QALY or cost/life-year gained)
No reimbursed
therapeutic •Price negotiation between the manufacturer and the Economic Committee
alternatives
Reimbursed •One alternative: maximum ex-factory price≤75% ex-factory price of
therapeutic alternative
alternatives •More than one alternative: maximum ex-factory price≤reference price
• “Ex-officio” pricing procedure allow the Ministry of Health to set the drug price without the
manufacturer (notification and request for information about drug) for generally highly
expensive medicines under specific conditions
• Free prices for non-reimbursed drugs
• For hospital drugs, maximum prices set as defined above and actual prices negotiated with
hospitals 129
Key Market Access
Specificities (1/2)
Drug programmes
•To cover high-cost medicines not reimbursed through the standard reimbursement process
•Named-patient basis for specific conditions
•Application through the standard P&R process with additional requirements (proposed
programme, inclusion criteria, drug use within the programme and monitoring)
•List of programmes updated every 2 months
Chemotherapy programmes
•Outcome cannot easily be broken down into simple associations or easily predicted
•Decision more driven by budget impact than cost-effectiveness
Paybacks
• Payback shared by pharmaceutical companies if total annual prescription drug
reimbursement budget exceeds 17% of the total annual healthcare budget
135
Decision Making Bodies
European Medicines Agency
European Level
(EMA)/European Commission
Marketing authorization
National Institute of Pharmacy National Level
and Nutrition (OGYÉI)
Opinion
OGYÉI Health Technology
Assessment Assessment Office (TÉI)
Medical Professionals
Group
Health Technology
Assessment Committee
National Health Insurance (TÉB)
Reimbursement & Pricing Fund (OEP)
Director-General of the
Decision
National Health
Insurance Fund (OEP)
Ministry of Human Resources Ministry of Human
Final Decision with consent from Ministry of Resources, Secretariat
Economy (NGM) for Health Care
136
NGM, Nemzetgazdasági Minisztérium; OEP , Országos Egészségbiztosítási Pénztár; OGYÉI, Országos Gyógyszerészeti és Élelmezés-egészségügyi Intézet;
TÉI , Technológia Értékelő Iroda; TÉB , Technológia Értékelő Bizottság
P&R Process
Marketing Authorisation
Opinion
Health Economics Group
Ministry of Human Resources with consent from Ministry of Assessment of health economic
Economy (NGM) data
Final ratification of the decision for first-in-class products/drugs
approved in specific indications (issue of ministerial decree)
* Branded drugs: pricing and reimbursement
process takes 90 days for medicines in already
reimbursed ATC4-groups and without indication
Inclusion in OEP formulary limitation. For all other medicines, it takes much 137
longer because of ministry involvement
Reimbursement Process
& Categories
Pre-condition for reimbursement:
Drug must be reimbursed in at least 3 other EU countries
Standard reimbursement procedure Simplified reimbursement procedure
• New active substances, new presentation, • Bioequivalent products (mostly generics)
new indication, new combination • New strength, new pack size
• Price increase
Reimbursement categories
Normative reimbursement: Special reimbursement categories:
• Unrestricted prescription rights for all physicians in • Drugs approved in specific indications (e.g. cancer,
all authorised indications diabetes, epilepsy, asthma) and prescribed by a
• 3 rates: 80%, 55% and 25% depending on drug specialist or on recommendation of a specialist:
effectiveness, disease severity/burden, public • 4 rates: 100%, 90%, 70%, 50% depending on
health impact drug effectiveness, disease severity/burden
Full reimbursement for low income patients (up to HUF 12,000 per month) 138
TÉI Cost-Effectiveness
Assessment
Methodology
• Payer perspective required
• CEA, CUA, CMA acceptable
• All health economics models should be adapted using Hungarian data
• No explicit QALY threshold
• Financially-driven evaluation
• No public hearing or publicly available reports
139
Key Market Access
Specificities
Centralised hospital drug procurement
•Introduced in 2014 for some drugs considered as “essential medicines” to avoid drug
withdrawal due to low prices/volumes
•Specific exemptions from administrative fees for marketing/distribution and from 20%
clawback on drug sales
• Informal negotiations
• Price-volume agreements and outcome-based agreements between manufacturers
and OEP required for new active substances (and some extensions of indications of
drugs included in special reimbursement categories) (see cost-containment
section)
• For hospital drugs, maximum prices cannot exceed the IRP price as defined above
• Free prices for non-reimbursed drugs
141
Key Cost-Containment
Measures (1/2)
National reference pricing
• Active ingredient and therapeutic reference pricing system (ATC 4 and 5)
• Reference pricing for originators (patented/off-patent) and generics/biosimilars
• Reference price set at the lowest daily treatment cost
• New reference price groups set by OEP on a quaterly basis
• Preferred pricing policy with specific rules to ensure preferred reimbursement conditions
for drugs priced at the reference price (lower patient co-payments)
•Reimbursement at the level of reference product for drugs priced up to 15%
•Reduced reimbursement rate of 15% for drugs priced between 15% and 100% (50% for
biologics) above the reference price
•No reimbursement for drugs priced more than 100% (50% for biologics) above the
reference price
• Financial incentives for pharmacists to dispense products from the « preferred » list
• Higher patient co-payments for drugs which are not on the « preferred » list
• Physicians must prescribe drugs by INN for statins only
Delistings
• If drugs in reference pricing systems are priced above defined threshold (see above)
• If ex-factory price of a drug exceed by more than 20% the average ex-factory price of the
same drug in 3 EU reference countries with the lowest price (only in theory, not applied)
142
Key Cost-Containment
Measures (2/2)
Reduction in the level of reimbursement
• Since 2012, dietary habits monitoring for patients treated with insulin analogues: potential
reduction in the level of reimbursement if no compliance with physician’s recommendations
(assessed by blood sugar level)
Price cuts
• In 2013, average price cut of 13.5% for more than 680 drugs
• In 2014, average price cut of 7% for more than 30 drugs
Paybacks
• Paybacks in case OEP’s drug budget excess
• 20% payback on reimbursed drugs sales (+10% for single-source substances reimbursed for more
than 6 years)
•Exemptions for drugs priced below the reference price and products with more than 50% price cuts, R&D
cost rebate
2009
Sweden: 1.1% GDP
OECD: 1.4 % GDP
2013 2013
Sweden: 11% GDP Sweden: 9.2% GDP
OECD: 8.9% GDP OECD: 6.5% GDP
146
Decision Making Bodies
Health Technology
Decision
Dental and Pharmaceutical
Assessment & Benefits Agency (TLV)
Reimbursement Decisions
20 County councils
Opinion
Regional Access Pharmaceutical Committées
147
TLV,Tandvårds- och läkemedelsförmånsverk
P&R Process Retail drugs
Marketing Authorisation
EU Level: European Medicines Agency (EMA)/European Commission
National Level: Medical Products Agency (MPA)
Timelines
(Months)
SBU
- Swedish Agency for
Health Technology Assessment Health Technology
Assessment
Dental and Pharmaceutical Benefits Agency (TLV)
• Do not directly
Department for Value based Pricing Scientific committee influence P&R
Evaluation of the submission to be included in Can contribute to the evaluation with clinical decisions
the pharmaceutical benefit scheme expertise • Source of knowledge
for decision-making
Pharmaceutical Benefits Group for County Councils Company bodies in general
Requested to comment on the evaluation Possibility to comment (but not submit new • Evaluate medical
before decision data) if decision is not general reimbursement products without
3-6
manufacturers’
submission
Pricing & Reimbursement Decision
Dental and Pharmaceutical Benefits Agency (TLV)
Pharmaceutical Benefits Board
Decides if the pharmaceutical is to be included in the pharmaceutical benefit scheme, and if any
restrictions or conditions should be applied Pricing of
hospital drugs is
free
Publication of decision on TLV website
148
TLV Reimbursement
Criteria
The TLV’s Pharmaceutical Benefits Board meets once every month to make
decisions about inclusion of drugs in the pharmaceutical benefit scheme
There are no price negotiations and the board does not suggest any price level
If the submission is rejected the company can resumbit with more data or lower price 149
Reimbursement System
• The annual spending for products included in the pharmaceutical benefit
scheme is limited for the patient
• During a 12 month period a patient can pay maximum 2200 SEK
• The level of co-payment decreases with increasing overall spending.
•Be performed from a societal perspective and should use Swedish data where
possible
•Cover the entire patient population for which reimbursement is being sought
•Use quality-adjusted life-years (QALY) as well as other metrics where appropriate
•Include data on benefits and cost versus the most appropriate comparator
(typically the moste widely used treatment in Sweden)
•Set out costs in terms of the drug’s proposed pharmacy sales price (AUP)
151
Future P&R Trends
152
Key Cost-Containment
Measures (1/2)
Generic policies
• Automatic substitution by pharmacies to the cheapest generic
• Patient out-of-pocket payment if the patient takes the branded medicine instead of the
generic
• Severe price competition, prices updated every month
Prescribing control
• Both TLV and County councils may impose restrictions and/or conditions on the use of
reimbursed drugs
• Prescribing budgets decentralised to individual healthcare centres
• County drug committees set prescribing targets and incentives to help ensure compliance
with the prescribing budget
• Electronic prescribing with support systems to assist physicians with their prescribing
decisions.
• Prescription monitoring and counties may send prescribing advisors
• Patient co-pay increased in 2012 and overall patient funding increased from 26 to 29%
Price cuts
• The prices of reimbursed off-patent brands subject to generic competition are cut by 65%
once the pharmacy purchase price of the lowest priced generic version is at least 70% below
the pre-patent expiry price of the off-patent original
• From 2015, certain older drugs (ie those that have held marketing authorisation for 15 years
or more but that have not had their prices cut under the ceiling price system [see above])
will be subject to a 7.5% price cuts
• The county councils can individually (or collectively via the New Drug Therapies group) enter
into discount agreements (eg risk-sharing, outcome guarantees, price-volume) with
manufacturers for high-cost hospital drugs
154
Cross-Country Comparison of
MA Pathways
SE
UK
DE PL
FR
HU
ES
IT
155
Formal vs Informal HTA
• A term and mission are set
• Transparent decision framework
process
Informal • Do not meet formal HTA criteria
Formal HTA • Meeting agenda available
HTA • No decision report is published
• Decisions are publicly available and
argued based on evidence submitted
by manufacturer
Formal Informal
France
Germany
Hungary*
Italy *Available decision
framework but not
Poland transparent (no
publication of
Spain assessments)
Sweden
156
UK
HTA Key Decision
Criteria
Absolute Relative Budget Cost-
therapeutic therapeutic impact effectiveness
value* value**
France (innovative
products)
Germany
Hungary
Italy
Poland
Spain
Sweden
UK
*Disease severity and burden, unmet needs, efficacy/safety of the product 157
**Incremental efficacy/safety versus available comparators
Pricing Rules for Reimbursed
Prescription Drugs
Free pricing International National reference Price Managed
reference pricing pricing negotiations entry
agreements
France (main criteria for (by active substance)
innovative drugs)
Sweden (acceptance of
rejection)
Germany
Hungary
Poland
Spain
Sweden
UK
159
Conclusion
Unprecedent
increase
demand Affordability?
Decrease
funding
Need of
adoption of cost-containment measures, to reduce
expenditure growth for public health
Rapid Pace of Therapeutic
Innovation
Dramatic advances in technology
162
New Challenges in Drug
Development
Development of companion diagnostic
Large benefit in small trials leading to early approval with limited evidence
New types of clinical trials emerging , known as clinical trials using genomic
profiling
• Imply new methodologies such as integrated protocols (several phases in only one trial), use and
comparisons of several treatments without marketing authorisation, new endpoints and adaptive
designs 163
Targeted Therapies
• New developments involved highly targeted therapies
– Highly sliced population
– Benefits in small populations
– Request for crossover for ethical reasons
• Payers impact
– Considerable potential social value
– Low level of payers evidence
– Difficulty to quantify the benefit
Genomics is a Moving Target
Traditional view 1987 2004 2009 PI3KCA
AKT1
MAP2K1
MET
BRAF
EGFR HER2
Large-cell KRAS EML4-ALK
KRAS Unknown
Adenocar EGFR
cinoma
Squamous Unknown
Unknown
KRAS
Prescribers
medical
?
Uncertainty
Patient
behaviour
practice
Study design
166
Shift in Payer Model
• The promised benefit must be evidenced in real life clinical
practice
• Outcomes must be shown in well designed real world studies
with limited or no intervention on the field or within databases
• The internal validity will be the door entry outcome
• The external validity will be the value acquired by payers
167
US-EU Closer Models
• Access to uncovered products through the National Public Insurance via the private
insurance
• Depending on one’s revenue, various options are accessible
Key Trends and Future Perspectives
in P&R of Drugs in Europe
Greater pan-European coordination of HTA, and greater dialogue
between regulatory and HTA bodies
• To meet outstanding regulator and HTA body requirements for relative effectiveness
evidence
172
http://www.ispor.org/HTARoadMaps/Default.asp
ISPOR Global Health
Care Systems Road Map-France
173
http://www.ispor.org/HTARoadMaps/France.asp
ISPOR Global Health
Care Systems Road Map-Germany
174
http://www.ispor.org/HTARoadMaps/Germany.asp
ISPOR Global Health
Care Systems Road Map-Hungary
175
http://www.ispor.org/HTARoadMaps/Hungary_Pharm.asp
ISPOR Global Health
Care Systems Road Map-Italy
176
http://www.ispor.org/HTARoadMaps/Italy.asp
ISPOR Global Health
Care Systems Road Map-Poland
177
http://www.ispor.org/HTARoadMaps/Poland.asp
ISPOR Global Health
Care Systems Road Map-Spain
178
http://www.ispor.org/HTARoadMaps/Spain.asp
ISPOR Global Health
Care Systems Road Map-Sweden
179
http://www.ispor.org/HTARoadMaps/Sweden.asp
ISPOR Global Health
Care Systems Road Map-UK
http://www.ispor.org/HTARoadMaps/Scotland.asp
http://www.ispor.org/HTARoadMaps/UK.asp 180
Thank you
Prof. Mondher Toumi, MD, PhD, MSc
Chairman Public Health & Market Access
+33 6 86 66 35 50
Åsa Kornfeld
Director, Pricing Reimbursement Market
Access, Creativ-Ceutical
www.emaud.org