1. Introduction to HTA
Let’s start with the good news..
- Life expectancy increasing
Healthcare costs increase
- Health expenditures per inhabitant increases
Most is publicly financed
- Different for different countries
- Overall most countries large part of healthcare expenditures are publicly financed
Health care expenditures in Netherlands
Estimates for future costs
More bad news
- Resources are scarce
- We can only spend money once
- Money spent on technology A cannot be spent on technology B
- Money spent on health care cannot be spent on education, infrastructure etc.
Pertuzumab (perjeta)
- Breast cancer
- Incidence 5500 per year in UK > 20% HER2+
Treatment costs 2000 per month, 24000 per year (pond)
Treatment effect: +6-12 months
→ Would you reimburse the drug? > depends on other/alternative drugs
Ataluren (Translarna)
- Duchenne muscular dystrophy
- Incidence 60-70 per year in UK > 6-9 mutation relevant for the drug
Treatment costs 220000 per year
Treatment effect: delay the loss of walking for up to seven years
→ Which drug would you reimburse if you can only reimburse one?
> Looking at population: more effects focusing on breast cancer
To reimburse or not reimburse?
> Resources in health are limited
> Decision regarding reimbursement have far reaching consequences
Why HTA?
- Health care expenditures increase
> Is this a problem?
>> What happens if we reimburse every treatment?
>>> Positive + negative consequences
>>> Other options?
1
,If choices in health care have to be made…
It is better to make informed choices!
- Health Technology Assessment (HTA)
> Important advisory tool - make transparent and unbiased decisions
> Compulsory for deciding on reimbursement in many countries
> Assure value for money
Definition
Health Technology Assessment (HTA)
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 definition
> Economic evaluation is usually the core
→ Compare two alternative regarding costs and benefits
Economic evaluations
> Economic evaluations provide insight costs and effects of
(new) interventions compared with existing interventions
> Economic evaluations are everywhere
The 6 steps of performing an EE in health care
Step 1.
A- Perspective - Who is going to pay?
^ Societal perspective but not in health care perspective: productivity (at work) for example
^ Patiënt costs to travel to hospital: societal and health care perspective
→ Includes all costs irrespective under what budget they fall
B- Choice of comparator
> Most efficiënt alternative
> Standard treatment
> Consider ‘no treatment’ → Comparison to no treatment
> Placebo not preferred → Because placebo is not plausible treatment
alternative in daily practice
2
,C- Type of analyses
> Only costs
(if treatments are universal; no differences in outcomes = difficult tho)
→ Cost minimization analysis (CMA)
→ Effects are equal, focus on costs
> Cost and effects in monetary terms
(immediately sign monetary value to all effects = rarely done)
→ Cost benefit analysis (CBA)
> Cost in monetary terms, effects in natural units
→ Cost-effectiveness analysis (CEA)
> Costs in monetary terms, effects in QALYs
→ Cost-utility analysis (CUA)
(cost-utility analysis often referred to as cost-effectiveness analysis)
^ Utility is QALY
D- Time Horizon
> All consequences must be taken into account
(takes time)
→ Lifetime horizon (need to be taken if you want to account properly)
> How to do so?
→ Randomised trial (RCT)
→ Observational study
→ Model (cohort or individual patiënt model)
→ Combination
Casus: radiotherapy
- Cancer leading cause of death
- 55% of cancer patients receive radiation therapy (60% curative intent)
- Radiotherapy can also be added to other treatment options
- Radiotherapy is associated with toxicities
> Short-term vs long-term
> Impact on quality of life, life expectancy, medical resource use
Radiotherapy development
- Traditional radiotherapy (photon radiotherapy)
- Proton radiotherapy > Investment costs
Radiotherapy Exercise 1
- Traditional radiotherapy vs photon radiotherapy > Perform the first step for the comparison
Step 1 > Perspective - who is going to pay?
^ societal perspective (about children; long term consequences taken into account)
> Comparator
^
> Type of analysis
^ Cost-utility analysis; QALY important and about comparison
^ Cost-effectiveness also possible (looking at differences of (cost) outcomes)
> Time horizon
^ Usually to fully account effects on health care costs
^ Useful to see what happens when you apply short time horizon (and be critical)
3
, Step 2. Measuring and valuing costs
- Identify all relevant cost items
- Measure resource use
- Value resource use
Which costs to include depends on the perspective!
Exercise 2
- Casus radiotherapy treatment
- Give three examples for costs that you would include
^ Medication cost should be taken into account
^ Future medical cost (risk of other diseases cause patiënt is surviving other disease)
^ Hospital days (related to for example visits to hospital)
Other societal costs examples
^ Costs of travelling to that facility (to one facility could be higher than other)
^ Time it takes to get to the facility
→ Opportunity costs of treating other patients (comes after evaluation
Step 3. Measuring and valuing effects
- Identify, measure and value effects of interventions (different ways to do this)
- Disease specific measures
- Generic measures
Exercise 3: measuring and valuing effects
- Casus radiotherapy treatment
^ interesting to measure QALY because differences are expected
- Identify, measure and value effects of interventions
Step 4. Discounting
- We have time preference for both costs and health effects
> We want positive health effects now
> We want to postpone costs
- Costs later in time weight less
> Effects later in time have less value
→ Want to take into account; if we gain QALY’s, we prefer them now than later
^ Because of time preference
^ Because of uncertainty
4
Let’s start with the good news..
- Life expectancy increasing
Healthcare costs increase
- Health expenditures per inhabitant increases
Most is publicly financed
- Different for different countries
- Overall most countries large part of healthcare expenditures are publicly financed
Health care expenditures in Netherlands
Estimates for future costs
More bad news
- Resources are scarce
- We can only spend money once
- Money spent on technology A cannot be spent on technology B
- Money spent on health care cannot be spent on education, infrastructure etc.
Pertuzumab (perjeta)
- Breast cancer
- Incidence 5500 per year in UK > 20% HER2+
Treatment costs 2000 per month, 24000 per year (pond)
Treatment effect: +6-12 months
→ Would you reimburse the drug? > depends on other/alternative drugs
Ataluren (Translarna)
- Duchenne muscular dystrophy
- Incidence 60-70 per year in UK > 6-9 mutation relevant for the drug
Treatment costs 220000 per year
Treatment effect: delay the loss of walking for up to seven years
→ Which drug would you reimburse if you can only reimburse one?
> Looking at population: more effects focusing on breast cancer
To reimburse or not reimburse?
> Resources in health are limited
> Decision regarding reimbursement have far reaching consequences
Why HTA?
- Health care expenditures increase
> Is this a problem?
>> What happens if we reimburse every treatment?
>>> Positive + negative consequences
>>> Other options?
1
,If choices in health care have to be made…
It is better to make informed choices!
- Health Technology Assessment (HTA)
> Important advisory tool - make transparent and unbiased decisions
> Compulsory for deciding on reimbursement in many countries
> Assure value for money
Definition
Health Technology Assessment (HTA)
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 definition
> Economic evaluation is usually the core
→ Compare two alternative regarding costs and benefits
Economic evaluations
> Economic evaluations provide insight costs and effects of
(new) interventions compared with existing interventions
> Economic evaluations are everywhere
The 6 steps of performing an EE in health care
Step 1.
A- Perspective - Who is going to pay?
^ Societal perspective but not in health care perspective: productivity (at work) for example
^ Patiënt costs to travel to hospital: societal and health care perspective
→ Includes all costs irrespective under what budget they fall
B- Choice of comparator
> Most efficiënt alternative
> Standard treatment
> Consider ‘no treatment’ → Comparison to no treatment
> Placebo not preferred → Because placebo is not plausible treatment
alternative in daily practice
2
,C- Type of analyses
> Only costs
(if treatments are universal; no differences in outcomes = difficult tho)
→ Cost minimization analysis (CMA)
→ Effects are equal, focus on costs
> Cost and effects in monetary terms
(immediately sign monetary value to all effects = rarely done)
→ Cost benefit analysis (CBA)
> Cost in monetary terms, effects in natural units
→ Cost-effectiveness analysis (CEA)
> Costs in monetary terms, effects in QALYs
→ Cost-utility analysis (CUA)
(cost-utility analysis often referred to as cost-effectiveness analysis)
^ Utility is QALY
D- Time Horizon
> All consequences must be taken into account
(takes time)
→ Lifetime horizon (need to be taken if you want to account properly)
> How to do so?
→ Randomised trial (RCT)
→ Observational study
→ Model (cohort or individual patiënt model)
→ Combination
Casus: radiotherapy
- Cancer leading cause of death
- 55% of cancer patients receive radiation therapy (60% curative intent)
- Radiotherapy can also be added to other treatment options
- Radiotherapy is associated with toxicities
> Short-term vs long-term
> Impact on quality of life, life expectancy, medical resource use
Radiotherapy development
- Traditional radiotherapy (photon radiotherapy)
- Proton radiotherapy > Investment costs
Radiotherapy Exercise 1
- Traditional radiotherapy vs photon radiotherapy > Perform the first step for the comparison
Step 1 > Perspective - who is going to pay?
^ societal perspective (about children; long term consequences taken into account)
> Comparator
^
> Type of analysis
^ Cost-utility analysis; QALY important and about comparison
^ Cost-effectiveness also possible (looking at differences of (cost) outcomes)
> Time horizon
^ Usually to fully account effects on health care costs
^ Useful to see what happens when you apply short time horizon (and be critical)
3
, Step 2. Measuring and valuing costs
- Identify all relevant cost items
- Measure resource use
- Value resource use
Which costs to include depends on the perspective!
Exercise 2
- Casus radiotherapy treatment
- Give three examples for costs that you would include
^ Medication cost should be taken into account
^ Future medical cost (risk of other diseases cause patiënt is surviving other disease)
^ Hospital days (related to for example visits to hospital)
Other societal costs examples
^ Costs of travelling to that facility (to one facility could be higher than other)
^ Time it takes to get to the facility
→ Opportunity costs of treating other patients (comes after evaluation
Step 3. Measuring and valuing effects
- Identify, measure and value effects of interventions (different ways to do this)
- Disease specific measures
- Generic measures
Exercise 3: measuring and valuing effects
- Casus radiotherapy treatment
^ interesting to measure QALY because differences are expected
- Identify, measure and value effects of interventions
Step 4. Discounting
- We have time preference for both costs and health effects
> We want positive health effects now
> We want to postpone costs
- Costs later in time weight less
> Effects later in time have less value
→ Want to take into account; if we gain QALY’s, we prefer them now than later
^ Because of time preference
^ Because of uncertainty
4
