Transcript Intro QALY & need assessment

Temporary reimbursement:
VoI going Dutch;
Jan J.V. Busschbach
Erasmus MC
Department of Medical Psychology and Psychotherapy
De Viersprong
Viersprong Institute for Studies on Personality Disorders (VISPD)
1
Temporary reimbursement


Start reimbursement at T0
Evaluate at T3

Since 2006
T3 is ‘temporary’ T4
 First T4 at December 2010
 39 application between T0 and T3 (T4)

When applicable?

Expensive hospital drugs
 > 0.5% of total national budget
 > € 2.500.000 per year

Orphan drug in academic hospital setting
 > 5% of academic hospital medication budget
• € 600.000
 < prevalence 5 / 10.000
 < 8000 patients in the Netherlands

A ‘cost effectiveness indication’ must be
provided
 ?? ….. An cost effectiveness estimate
Why?

Officially problem:
 Medication comes late on the market
 As research is to expensive given the budget
• Medication does not come on the market

Unofficial problem:
 Difficulty in denying health care when:
• High burden of disease
• Highly exposed diseases
 Lack of arguments
• As no trials are running
• Although expensive, low budget impact
Because of low prevalence in orphan drugs
Paradox


Officially argument ‘no evidence’
But many applications are clearly not cost
effective
 > € 80.000 per year

Why no rejecting: no threshold value in NL
 Cost effectiveness research is necessary
 Not cost effectiveness…
 Clear roles for assessment
 No clear roles for appraisal

Anyway:… no effect data as well
Who wants it?

We all love temporary reimbursement…
 Manufacturers
 Patient
 Clinicians
 Policy makers
• It looks humane
• It looks like policy
 Scientist
• The promises of research in between
There scientist who object…

Why are we going to do it, if we know the out
come at forehand….
 € 80.000 per year will never be cost effective
 It is extremely difficult to get drug of the market


If you think research is to expensive: VoI
Well a cost ‘effectiveness indication’ must be
given at T0
 What is a cost effectiveness indication
A cost effectiveness ‘indication’

1. Definition of research question
 Literature investigation
 Rational
 Plan for ‘outcome investigation’

2. An estimate of the CE-ratio
 Model, a description of a model, only a description
 An indication of the ‘critical parameters’


3. According to national CE guidelines
4. rational of additional data collection
 ‘outcome research’

(Budget estimates)
Funds available

Fund for evaluation research
 Not for the cost effectiveness indication
 Only for period between T0 – T3

Aimed at:
 hospitals,
 health care professional organizations and
 patient organizations


Matched funding
Not really clear if industry may benefit
 Through third party?
Temporary…

After three years (T3 …. Ehh T4)
 Budget impact
 Effectiveness
 Cost effectiveness
• Along normal guidelines
 Rightful (target) population

If not, reimbursement stops
The scientist who where
hoping on new research…

Clinical investigators
 Setting up registers and database investigation
 Effectiveness
• Seen as substitute for CE
 Rightful (target) population
• Seen as substitute for CE

Health economist
 Full model at T3
 Value of information at TO
Scientist had difficulty
understanding each other…

Health economists
 What is the point in collection data is you know that it not
going to be cost effective in the first place
 What else (should) you do than a VoI

Clinical scientist
 Cost per QALY are bad thing anyway…
 What is VOI?
Guideline for outcome research
Guideline

Pragmatic
 Unclear quality for the research…
 Understandable is funding is limited

A strange mix of
 clinical outcome
• Data base building
• Data base analysis
 Health economics
Changes



Start with a VoI
Get funding
Get your medication reimbursed
 For at least 3 years

Not cost effective?
 Will they be able to stop reimbursement?

Why not start all over again for another period
of three years…