This document discusses the importance of patient involvement in health technology assessment (HTA). It notes that HTA evaluates new drugs and medical technologies but typically does not incorporate patient perspectives on factors like quality of life. The document advocates that patients should play a role in HTA processes by providing input on treatment outcomes, tolerability, and other real-world considerations to ensure funding decisions align with patient values and needs. It provides examples of how patients could be engaged as representatives, through surveys or public input, and highlights the need for HTA to consider individual patient impacts rather than just average population effects.

1. HTA: What every Patient
Needs to Know
Durhane Wong-Rieger, PhD
Consumer Advocare Network

2. Scenario
1:
Regulator
has
approved
a
new
drug
for
your
condition.
There
are
already
four
other
effective
therapies
available.
§ As
a
pa&ent,
what
ques&ons
would
you
ask
before
switching
to
this
new
drug?
§ Benefits
vs.
Risks
§ Is
it
safe?
Is
it
effec&ve?
§ What
are
the
side
effects?
§ Comparison
to
current
§ How
does
it
compare
to
old
therapies?
§ Is
it
easier
or
more
difficult
to
use?
§ How
happy
am
I
with
my
current
therapy?

3. Scenario
1:
Regulator
has
approved
a
new
drug
for
your
condition.
There
are
already
four
other
effective
therapies
available.
§ As
a
drug
plan
manager
(funder),
what
ques&ons
would
you
ask
before
making
the
drug
available?
§ What
are
added
benefits
of
new
therapy?
§ What
are
new
risks
of
therapy?
§ How
does
“cost”
affect
the
balance?
Benefits Risks

4. Scenario
2:
Regulator
has
approved
three
new
drugs
for
three
different
conditions.
§ As
the
drug
plan
manager,
you
recognize
each
is
likely
to
have
a
significant
impact
on
your
drug
budget,
so
you
could
not
afford
all
three.
Total
budget
impact
of
each
is
about
the
same.
What
do
you
choose
to
do?
Which One?
Hi$,
Few
Patients
Low$,
Many
Patients
Very Hi
$, Life
Saving

5. Scenario
2:
Role
of
patient
preference
As
a
pa&ent,
what
factors
would
you
suggest
the
drug
plan
consider?
What
if
you
had
one
of
the
condi&ons?
Quality of
Life
Disease
Impact
No OtherTherapy
Life Saving
StopProgression
Fewer Side
Effects
Easier to
Manage
Return to
Work

6. HTA has Role in Access
6
Researcher:
Does drug
work in lab
tests? Is it
safe?
Company:
Does drug
work in
patients?
What are
adverse
effects?
Regulator: Do
benefits
outweigh
harms? What
follow-up
monitoring
required?
HTA: How does
drug compare
to existing
therapies? Is it
worth the cost?
Payer: What is
budget
impact?
Affordable, or
spend on
something
else?
Patient Impact

7. ü Patients
ü Public
ü Industry
ü Regulator
IS THERE A PROBLEM?
Healthcare Demand vs. Fiscal Constraint
USER vs. PAYER
² HTA
² Government
² Other payers
² Other services
We need better,
faster, more
tolerable therapies
We need to
manage rising
costs, demands,
expectations
Healthcare
Provider?

8. Roles
of
Regulator
and
HTA
Regulator HTA
Conducted by government
authority, has legal implications
and grants right to sell drug
Conducted by a variour entities,
recommends what “should” be
funded and not legally binding
Evidence: From clinical trials;
controlled conditions; compared
to placebo or standard of care
Evidence: Compared to best
alternative; real-world usage,
costs and savings of intervention
Evaluate based on benefits
outweigh risks (potential harms)
Assess benefits-risks compared
to alternatives and relative costs
Process: Researcher develops,
industry collects CTs and
submits, regulator reviews
Process: Company submits CTs
and cost-effectiveness data; HTA
comparison to alternatives
Approve based on benefits-risks;
post-market monitoring to deal
with uncertainty
Recommend based on cost to
benefits, added cost for added
benefit, or value of alternatives
8

9. CEA: Cost Comparison
9
Patient
Population
Submitted Drug
Comparator Drug
• Current standard of care
• Drugs in a similar group
• Nothing/no treatment
• Previous standard of care

10. CEA: Cost Minimization
10
Patient
Population
Submitted
Drug
Comparator
Drug
Similar clinical effects
Consider costs
- Drug costs
- Total cost (drug & health care)

11. CEA: Cost Effectiveness
11
Patient
Population
Submitted
Drug (1)
Comparator
Drug (2)
Clinical effects (1)
Costs (1)
COMPARE:
Clinical effects (2)
Costs (2)
Effect Drug 1
Cost Drug 1
Effect Drug 2
Cost Drug 2
> ?

12. Decision Matrix
Drug A Costs more and is
less effective than Drug B
(Choose B)
Drug A Costs more and is
more effective than Drug
B
(Do Cost-Effectiveness)
Drug A Costs less and is
less effective than Drug B
(Do Cost-Effectiveness)
Drug A Costs less and is
more effective than Drug
B
(Choose A)
12
Effectiveness (life years, QALY’s, etc.)
COST$

13. Cost-utility Analysis
Same principle as cost-effectiveness
Compare increase in cost with increased “Quality of
life” benefits
Cost A - Cost B = Cost-Utility Ratio
QALYA - QALYB
§ Use units of Quality of Life in denominator as “utility”
§ Represents Measure of “satisfaction” with state of
health
13

14. HTA Cost-Effectiveness Measures
§ QALY = Years of additional life, adjusted for quality
§ “Quality of life” from questionnaire asking physical and
cognitive abilities, well being, work and social activities
§ QOL for specific disease health status is “score” based on sum
of capabilities and sense of well being
§ Assumes all life years are equivalent, regardless of age, health
status, disease prognosis
§ QALY -= Quality-Adjusted life year based on QOL score, where
“1” = perfect health and “0” = death
§ ICER = Incremental cost effectiveness ratio
§ Cost for additional QALY (how much is a year of life worth?)
§ Opportunity cost = Other benefits for the same $
§ Funds are limited and can’t spend the same $ twice
§ Could you get more health benefits if spent on another therapy
or disease?

15. May 2015
Harmonized Orphan Drugs/Rare Diseases Policy
-- Latin America 15
Patient Engagement => Optimal Use
Patient
defines
treatment
outcomes,
quality of
life,
tolerability
Patient has
legal, ethical,
moral right to
informed
choice
Patient helps
define start/
stop criteria;
uses drug
appropriately
Patient
collaborates to
monitor
outcomes;
provide
feedback; adjust
therapy
Patient
partners to
improve
access criteria
and support
optimal use

16. Lifecycle Approach with Patient
Input & Real-World Data
16
Researcher/
Clinician:
Disease
Knowledge; Drug
Discovery;
Treatment
Guidelines
Company:
Clinical Trials &
Outcome
Measures;
biomedical,
clinical, PROs,
Real-World
Impact
Regulator:
Approval on
Benefits-Risks-
Uncertainties;
Use & Real-
World
Monitoring
HTA:
Comparison
Benefits, Risks,
Cost w/
Alternatives;
Place in Therapy
Payer: Budget
Impact; Access
Criteria; R-W
Data Collection
Patient Input

17. How Could Patients Engage in HTA
Consultee, Informant
Input thru Council, Task Force,
Collect Info: Survey, Poll, Focus Group
Form of Info: Answers, Opinions,
Deliberation
Impact: Advise, Discretionary
Examples: NICE Citizens Council,
IQWiG, Ontario Citizens Council
Patient Representative
Input thru Committee, Board, Council
Collect Info: Experts, Deliberation
Form of Info: Analytical, Guidelines
Impact: Varied, Based on Guidelines
Examples: NICE, AU MASC, CEDAC,
pERC Ontario CED
Individual Patients
Input thru Clinical Trials, Testimony
Collect Info: QoL, PROs, Impact
Statement
Form of Info: Ratings, Qualitative
Impact: Varied, Emotional Suasion
Examples: SMC, IQWiG
Quebec INESSS, BC Pharmacare,
Patient Groups
Input: Submission
Collect Info: Written, Oral, Meetings
Form of Info: Qualitative Statement
Degree of Impact: Response
Examples: NICE, SMC AU MASC,
CADTH, pCODR, Ontario CED
Patient Representativeness
ParticipationinSystem

18. Need Patient Values As
Alternative to System Values
System Values Patient Values
“Faint hope” “Chance for life”
Convenience Quality of Life
Standards of care for
average patient
Personalized treatment
protocol
Delayed access based on
long-term outcomes (real-
world data)
Timely access based on
sufficient data (coverage with
evidence development)
Restrictive practice guidelines
to avoid inappropriate use
Facilitative guidelines to allow
optimal prescribing
Prefer: Small benefits to many Allow: Large benefit to “1”

19. Contact:
Durhane Wong-Rieger
Consumer Advocare Network
www.consumeradvocare.org
416-969-7435
durhane@sympatico.ca