HTA - lecture 8 - HTA and policy making

Question 1

policy goal in health care

Answer 1

ensuring affordable and equitable access for (all) patients to effective therapies in a sustainable manner

Question 2

HTA definition

Answer 2

“A multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in it’s lifecycle.
The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system.”

Question 3

reimbursement, based on what criteria?

Answer 3

• Disease burden: what patients and society think of it
  o End of life drugs can costs the most
• Budget impact
  o Costs to the drug (and additional costs)
  o Number of patients

Question 4

revealed preferences

Answer 4

looking at what people actually do, rather than what they say they prefer or intend to do

Question 5

stated preferences

Answer 5

refer to what people express or say about their preferences when it comes to healthcare treatments or interventions.

Question 6

provisional conclusion criteria

Answer 6

• Disease severity, cost-effectiveness, health gain crucial
• Budget impact relevant, esp. at 50 million euros and more
• Productivity gain not important (societal perspective?)

Preference building process part utilistic (more health, at lower costs, part egalitarian (disease severity prominent))
 the priority view: benefits to worse off count for more, in terms of overall utility, than comparable benefits to better off

Conclusions in line with research abroad.

Question 7

market access

Answer 7

Research and development  European medicines agency (EMA)  European commission  registration in EU  reimbursement

Question 8

policy dilemma in drug reimbursement

Answer 8

New expensive drug, probably effective, hardly cost-effective
(limited evidence from RCT drug vs placebo, short FU etc. )

Reimburse at proposed monopoly price?
+ fast access, maybe health gain for those in need
- Risk of no/limited health gain, waste of money

No reimbursement?
+ no risk of inefficient spending
- Missed opportunity of health gain

Question 9

uncertainty in policy making

Answer 9

• DCE -> uncertainty relevant for policymakers.
• Clinical uncertainty:
  o Effectiveness (endpoints vs surrogate outcomes)
  o Safety (number/seriousness adverse events): we know a lot about short term but nota bout long term
  o Quality of life
• Cost-effectiveness (CE in USA -> CE abroad?)
• Budget impact (no of patients, price per patient)
• Technical uncertainty (model disease & treatment)

Question 10

uncertainty: no cure no pay. pro’s and con’s

Answer 10

• Advantages:
  o “no cure, no pay” => value for money
  o application on best patient sub groups
  o after contract new decision possible
• Disadvantages:
  o transaction costs contract
  o clear outcome indicator crucial
  o cost of monitoring/registration
  o Budget impact may be very high