Health Technology Assessment
Lecture 1 Introduction in HTA
Life expectancy/ health expenditures
Life expectancy at birth is increasing, but health expenditures as well. Most of the spending
on HC is publicly financed. This is bad news because
- Resources are scarce it is bad news.
- We can only spend money once.
- Money spent on A, cannot be spent on B.
- Money spent on HC cannot be spent on education, infrastructure etc.
➢ Choose what’s important
Example
Peruzumab (Perjeta)
- Breast cancer
- Incidence: 5.500 per year in UK
- Treatment costs: 2.000 a month, 24.000 a year.
- Treatment effect: +6-12 months
Ataluren
- Duchenne
- Incidence: 60-70 per year in the UK
- Treatment costs: 220.000 per year
- Treatment effect: delay the loss of walking for up to seven years
➢ Reimburse it? And which one?
o Use all information
o Alternative?
o Length of life/ quality of life: important (QALY)
o Rare diseases: often a higher threshold
o This is why HTA is important, not comparable with other medicine
o Political effect is also present: Denying treatment for children is harder than for
elderly
o HTA framework
o Total society cost: incidence * cost = budget impact. A lot of patients, but low costs
drug is still a high impact.
o Differences between countries in terms of focus: budget impact (NL, UK not so
much)/ cost effectiveness/
Why HTA?
- Health care expenditures increase
o Is this a problem?
▪ What happens is we reimburse every treatment?
• Positive + negative consequences
• Limiting the supply is also a way of making decision/rationing. It is
better to have a thorough costs and then make a decision.
- Choices in healthcare have to be made
, o It is better to make informed choices
➢ Health Technology Assessment (HTA)
o Important advisory tool > make transparent and unbiased decisions
o Compulsory for deciding on reimbursement in many countries
o Assure value for money
o In more and more countries there is a requirement of HTA.
Definition Health Technology Assessment
HTA is the systematic evaluation of properties, effects and/or impacts of health technologies and
interventions. It covers both the direct, intended consequences of technologies and interventions and
their indirect, unintended consequences. ~WHO
- Economic evaluation is usually the core
o compare two alternatives regarding costs and benefits.
- Organizational, patient aspects, equity also important
Economic evaluations
- Economic evaluations provide insight coss and effect of
o New interventions
o Compared with existing interventions
- Economic evaluations are basically everywhere e.g. choosing different types of cola.
Steps in performing an economic evaluation study
Step 1:
1.1: Perspective – Who is going to pay?
Two dominant perspectives
- Health care perspective
o Costs and effects falling on health care budget
o UK
- Societal perspective
o All relevant costs and effects
o Example: travel costs of a patient (not in hc perspective)
o NL
- But also patient or health insurer perspective
o Insurance: smoking programs
➢ Perspective determines which costs and effects to include in the assessment.
➢ Perspective is about answering the question who’s going to pay. The first step: from what
perspective do I judge this?
,1.2. Choice of comparator
- Most efficient alternative
o Comparing to a less inefficient alternative, the new treatment might look very
efficient
- Standard treatment
- Consider ‘no treatment’
o If there is something out there, very expensive, sometimes better to not compare
- Placebo is not preferred
o In trials there might be a placebo, but in medical practice placebo will not be used, so
placebo is not a preferred choice for a comparative treatment.
1.3. Type of Analysis
- Only costs
o Cost minimization analysis (CMA)
o effects are equal, focus on costs
o not common
- Cost and effects in monetary terms
o Cost benefit analysis (CBA)
o Value QALY to money
- Costs in monetary terms, effects in natural units
o Cost-effectiveness analysis (CEA)
o Natural units e.g. life years, Duchenne – minutes to walk
- Costs in monetary terms, effects in QALYs
o Cost-utility analysis (CUA)
o Life years that incorporate life duration and quality.
o Sometimes CUA is called a CEA. Just be aware.
1.4. Time horizon
- All consequences must be taken into account
o Lifetime horizon
- How to do so?
o Randomised trial (RCT)
o Observational study
o Model (cohort or individual patient model)
o Combination
➢ Time horizon must be sufficiently long to include all consequences. Treatment that increases
life expectancy, it should be long enough to see all the years. For example, increase from 10
to 12, at least 12 years and preferably longer. Sometimes with a short life-expectancy left, 5
years can be enough, but often it is longer. This is important in economic evaluation. Usually
there’s evidence from RCT’s with a follow-up of 1 à 2 years.
➢ Sometimes observational studies, but often a model with limited follow-up with a time
horizon that is sufficiently long for the model, but this brings uncertainty.
Case: obesity and overweight.
- Factsheet WHO:
o Worldwide obesity has more than doubled since 1980.
o In 2014, more than 1.9 billion adults (39%), were overweight; 600 million (13%) were obese.
o 41 million children under the age of 5 were overweight or obese in 2014.
o Obesity is preventable.
, Treatment options for obesity
1. Life style program
2. Pharmacotherapy
3. Bariactric surgery
Assignment 1: perform the first step for obesity treatments
- Perspective:
- Comparator:
- Type of analysis:
- Horizon: life time
Step 2: measuring and valuing cost
- Identify all relevant cost items (all costs included in the analysis, e.g. complications, costs by
professionals relevant to the treatment)
o Depends on perspective
- Measure resource use (how many .. in both groups)
- Value resource use (attach a certain value, how much does .. cost)
- Costs are caused by…
Example obesity
Assignment 2:
- Casus obesity treatment
o Lifestyle, pharmacotherapy, surgery
- Give three examples for costs that you would include
o Hospital days, treatment costs (health sector costs), productivity costs (societal perspective),
Step 3: Measuring and valuing effects
- Identify, measure and value effects of interventions (how measure the effectiveness of the
treatment (obesity – weight loss (more intermediate), prevention of diabetes (long time to
measure), blood pressure, life years, quality of life).
- Disease specific measures
- Generic measures
Lecture 1 Introduction in HTA
Life expectancy/ health expenditures
Life expectancy at birth is increasing, but health expenditures as well. Most of the spending
on HC is publicly financed. This is bad news because
- Resources are scarce it is bad news.
- We can only spend money once.
- Money spent on A, cannot be spent on B.
- Money spent on HC cannot be spent on education, infrastructure etc.
➢ Choose what’s important
Example
Peruzumab (Perjeta)
- Breast cancer
- Incidence: 5.500 per year in UK
- Treatment costs: 2.000 a month, 24.000 a year.
- Treatment effect: +6-12 months
Ataluren
- Duchenne
- Incidence: 60-70 per year in the UK
- Treatment costs: 220.000 per year
- Treatment effect: delay the loss of walking for up to seven years
➢ Reimburse it? And which one?
o Use all information
o Alternative?
o Length of life/ quality of life: important (QALY)
o Rare diseases: often a higher threshold
o This is why HTA is important, not comparable with other medicine
o Political effect is also present: Denying treatment for children is harder than for
elderly
o HTA framework
o Total society cost: incidence * cost = budget impact. A lot of patients, but low costs
drug is still a high impact.
o Differences between countries in terms of focus: budget impact (NL, UK not so
much)/ cost effectiveness/
Why HTA?
- Health care expenditures increase
o Is this a problem?
▪ What happens is we reimburse every treatment?
• Positive + negative consequences
• Limiting the supply is also a way of making decision/rationing. It is
better to have a thorough costs and then make a decision.
- Choices in healthcare have to be made
, o It is better to make informed choices
➢ Health Technology Assessment (HTA)
o Important advisory tool > make transparent and unbiased decisions
o Compulsory for deciding on reimbursement in many countries
o Assure value for money
o In more and more countries there is a requirement of HTA.
Definition Health Technology Assessment
HTA is the systematic evaluation of properties, effects and/or impacts of health technologies and
interventions. It covers both the direct, intended consequences of technologies and interventions and
their indirect, unintended consequences. ~WHO
- Economic evaluation is usually the core
o compare two alternatives regarding costs and benefits.
- Organizational, patient aspects, equity also important
Economic evaluations
- Economic evaluations provide insight coss and effect of
o New interventions
o Compared with existing interventions
- Economic evaluations are basically everywhere e.g. choosing different types of cola.
Steps in performing an economic evaluation study
Step 1:
1.1: Perspective – Who is going to pay?
Two dominant perspectives
- Health care perspective
o Costs and effects falling on health care budget
o UK
- Societal perspective
o All relevant costs and effects
o Example: travel costs of a patient (not in hc perspective)
o NL
- But also patient or health insurer perspective
o Insurance: smoking programs
➢ Perspective determines which costs and effects to include in the assessment.
➢ Perspective is about answering the question who’s going to pay. The first step: from what
perspective do I judge this?
,1.2. Choice of comparator
- Most efficient alternative
o Comparing to a less inefficient alternative, the new treatment might look very
efficient
- Standard treatment
- Consider ‘no treatment’
o If there is something out there, very expensive, sometimes better to not compare
- Placebo is not preferred
o In trials there might be a placebo, but in medical practice placebo will not be used, so
placebo is not a preferred choice for a comparative treatment.
1.3. Type of Analysis
- Only costs
o Cost minimization analysis (CMA)
o effects are equal, focus on costs
o not common
- Cost and effects in monetary terms
o Cost benefit analysis (CBA)
o Value QALY to money
- Costs in monetary terms, effects in natural units
o Cost-effectiveness analysis (CEA)
o Natural units e.g. life years, Duchenne – minutes to walk
- Costs in monetary terms, effects in QALYs
o Cost-utility analysis (CUA)
o Life years that incorporate life duration and quality.
o Sometimes CUA is called a CEA. Just be aware.
1.4. Time horizon
- All consequences must be taken into account
o Lifetime horizon
- How to do so?
o Randomised trial (RCT)
o Observational study
o Model (cohort or individual patient model)
o Combination
➢ Time horizon must be sufficiently long to include all consequences. Treatment that increases
life expectancy, it should be long enough to see all the years. For example, increase from 10
to 12, at least 12 years and preferably longer. Sometimes with a short life-expectancy left, 5
years can be enough, but often it is longer. This is important in economic evaluation. Usually
there’s evidence from RCT’s with a follow-up of 1 à 2 years.
➢ Sometimes observational studies, but often a model with limited follow-up with a time
horizon that is sufficiently long for the model, but this brings uncertainty.
Case: obesity and overweight.
- Factsheet WHO:
o Worldwide obesity has more than doubled since 1980.
o In 2014, more than 1.9 billion adults (39%), were overweight; 600 million (13%) were obese.
o 41 million children under the age of 5 were overweight or obese in 2014.
o Obesity is preventable.
, Treatment options for obesity
1. Life style program
2. Pharmacotherapy
3. Bariactric surgery
Assignment 1: perform the first step for obesity treatments
- Perspective:
- Comparator:
- Type of analysis:
- Horizon: life time
Step 2: measuring and valuing cost
- Identify all relevant cost items (all costs included in the analysis, e.g. complications, costs by
professionals relevant to the treatment)
o Depends on perspective
- Measure resource use (how many .. in both groups)
- Value resource use (attach a certain value, how much does .. cost)
- Costs are caused by…
Example obesity
Assignment 2:
- Casus obesity treatment
o Lifestyle, pharmacotherapy, surgery
- Give three examples for costs that you would include
o Hospital days, treatment costs (health sector costs), productivity costs (societal perspective),
Step 3: Measuring and valuing effects
- Identify, measure and value effects of interventions (how measure the effectiveness of the
treatment (obesity – weight loss (more intermediate), prevention of diabetes (long time to
measure), blood pressure, life years, quality of life).
- Disease specific measures
- Generic measures
