h i l i i i bPharmaceutical pricing, reimbursement, HTA and cost containment measures inHTA and cost containment measures in

Europe: an overview (II)

Elias Mossialos and Reinhard Busse

What decision-makers want

Safety and Efficacy are first steps to provide evidence for a d Eff ti d Effi i d t bnew drug; Effectiveness and Efficiency need to be proven

EffectivenessEfficacy EfficiencySafety

Measure of effect under

y

Measure of effect under

y

Relationships between

y

Measure of d “real life”

conditions and vs. other

d

effect under ideal

conditions

between costs and benefits

adverse effects

drugs

Licencing/Health TechnologyAssessment (HTA):Licencing/

market accessAssessment (HTA):

coverage?reimbursement price?reimbursement price?

Marketing Authorisation - Key Principlesg y p

– The EU is a Single Market for pharmaceuticals: g papprox. 0.5 billion people

I d t ll di i l d t i th EU– In order to sell a medicinal product in the EU, a company needs a Marketing Authorisation

– There are a number of ways ( ‘Procedures’ ) for a company to obtain a marketing authorisationa company to obtain a marketing authorisation.

– The main scientific principle used in theThe main scientific principle used in the evaluation of medicines is the benefit/risk balance, based mainly on quality, efficacy andbalance, based mainly on quality, efficacy and safety aspects

Since 2005: Three European Systemsp y

Centralised Procedure

Mutual Recognition Decentralised

Procedure(via EMA)g

procedure Procedure

EU Centralised Procedure

• Principle: single application / evaluation single authorisation ( direct access to all 28 single authorisation ( direct access to all 28 EU countries + Norway, Iceland and Liechtenstein)

• Scope:• Scope:Compulsory for:– Cancer, neurodegenerative disorders, diabetes, HIVCancer, neurodegenerative disorders, diabetes, HIV– Auto-immune diseases, viral diseases– All biotechnology productsgy p– Gene therapy– Monoclonal antibodies – Rare diseases (i.e. “orphan”)Optional for any other new active substance (instead of mutual recognition or decentralised procedure)

Health Technology Assessments:Health Technology Assessments: coverage, reimbursement price & usage

• Can lead to a refusal to reimburse or cover the licenced drug (or other technology) orthe licenced drug (or other technology) or

• Limits the price payers are reimbursing and/orand/or

• Restricts access to drug (e.g. 2nd or 3rd line use only for some patient groups etc )use, only for some patient groups, etc.)

• Some restrictions are harder to enforce than others and the implementation ofothers, and the implementation of recommendations is not automatic

• Sometimes the mere intention to conduct an• Sometimes the mere intention to conduct an HTA can impact on use of the product

What perspective to take into account?Different countries different scope (and data)Different countries – different scope (and data)

Complexity and breadth

Therapeutic Value Health System Value Societal Value

and breadth of data required to demonstrate valuee value

Pharmaceutical Budget

Health System Budget

Other Budgets (e.g. pensions, social security)

France UK Sweden

Representative Country

HTA in Europe simplified: direct after launchHTA in Europe simplified: direct after launch

New drug/ device/ intervention “single technology assessment”:Important input = structured information( dossier of manufacturer/ promoter

With i ( S d ) Without price ( F )

not reimburseable

Need (disease burden) & Effectiveness(also for patient sub groups and selected indications)

With price (e.g. Sweden) Without price (e.g. France) reimbursable only for selected indica-tions, selected pro-

viders, second line …(also for patient sub-groups and selected indications)

Additional benefit/ comparative effectiveness reimburseable

viders, second line …(“optimised“)

p(also for patient sub-groups and selected indications)

b fi

only in research(to generate

additional data)Cost-benefit

(comparative, sub-groups …)

)

• Free price-setting (if add‘l benefit large)• price negotiated (trend• price negotiated (trend

value-based pricing & volume-price)• price regulated © Reinhard Busse

HTA at later stage –comparing groups of technologies

„Old“ drug/ device/ inter-vention “real” HTA:

Important input =

New drug/ device/ intervention “single technology assessment”:

With i Without price systematic review

C i b fi

With price Without price

Comparative benefit(also for patient sub-groups

and selected indications)After a certain time, in case of new evidence,

Comparative cost-benefit

doubts about (cost-)effectiveness

© Reinhard Busse

T h l A i lTechnology Appraisals –the results 3/2000-1/2014the results 3/2000 1/2014

Decision Single TAs Multiple TAsTAs

Recommended 75 (54%) 242 (64%)

Optimised 25 (17%) 69 (18%)

Only in Research 4 (3%) 22 (6%)Only in Research 4 (3%) 22 (6%)

Not recommended 35 (25%) 44 (12%)

Total 139 (100%) 377 (100%)http://www.nice.org.uk/newsroom/nicestatistics/TADecisionsRecommendationSummary.jsp

Comparison of Final Recommendations in Canada & Scotland(110 drugs reviewed by both CDR and SMC as of March 2013)( g y )

51,6%46,7%45,1%50%

60%

28,7%24,6%

30%

40%

ListList with Conditions

3 3%

,

10%

20%

List with ConditionsDo not List

3,3%

0%Canada Scotland

• Scottish Medicine Consortium (SMC) is far more likely than the Canadian CDR to• Scottish Medicine Consortium (SMC) is far more likely than the Canadian CDR to recommend new drugs be publicly funded. 

• Analysis suggests that CDR is unconvinced that new products offer incremental value when older less expensive alternatives are availablevalue when older, less expensive alternatives are available. 

• These results are consistent with other studies that concluded that CDR is more restrictive than decisions made by other HTA agencies.

Source: Canadian Agency for Drugs and Technology in Health (CADTH), Scottish Medicines Consortium (SMC)

Segal, J. Introduction to Comparative Effectiveness Research. Johns Hopkins University.

Assessing CostAssessing Cost EffectivenessEffectiveness

1

Probability of rejectionof rejection

10 20 30 40 50

0

Cost per QALY (£’000)10 20 30 40 50

1 Wh t i th bj ti f l ti t thi1. What is the objective of regulation at this point or of the given actors in the pharmaceutical market?pharmaceutical market?

2. What forms do the regulatory intervention take?take?

3. What are the expectations of the regulatory interventions?interventions?

4. What evidence is there of the impact of the regulatory interventions with a particularregulatory interventions with a particular emphasis on cost-control, efficiency, quality and equity of access?and equity of access?

5. How can the regulatory environment be improved?p o ed

E N t k fEuropean Network for Health Technology Assessment

EUnetHTA | European network for Health Technology Assessment | www.eunethta.eu

Presentation available at:

www.mig.tu‐berlin.de

Introduction to the healthcare ecosystem