HTA was intended to be an independent and evidence-based process to promote optimal use of health technologies. However, it has become owned by payers who use it to limit use and costs. There are calls for HTA processes to be more transparent, include all stakeholders such as patients, and consider personal and societal costs and savings, not just the cost to the healthcare system. For HTA to be effective, cost-effective, and sustainable, it must earn patient trust through meaningful patient engagement in development and decision making.

1. HTA: What it was supposed to;
What it became; How can we
make it righ?”
Durhane Wong-Rieger, PhD
Consumer Advocare Network

2. HTA has Role in Access
2
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

3. ü Patients
ü Public
ü Industry
ü Regulator
Does HTA Set up Opponents?
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?

4. HTA:
“Ideal”
vs.
“Real”
IDEAL REAL
Independent, unbiased process
to promote optimal use
Owned by payers (government)
to limit use and costs
Evidence-based comparison with
standard or best alternative
Lack comparative data; select or
manipulate data
Cost-effectiveness includes all
health system, personal and
societal costs and savings
Limited to cost of therapy to
system, not personal or other
costs; nor other savings
All stakeholders engaged; timely
process; accountability & appeal
Payer-controlled process; delays,
limited input & transparency
Separate: budget impact,
affordability, competing interests
Budget constraint & opportunity
cost dominate HTA process
4
Definition of HTA: “The process of systematically reviewing
existing evidence and producing an evaluation of effectiveness,
cost-effectiveness, and impact, both on patient health and the
health care system of health technology and its use.”

5. Universal Health Coverage Needs
HTA to Assure Access
§ TREND: Universal Health Coverage and Access
👍 More people getting better access to care!
" More strain on health budgets!!
§ TREND: Innovative, personalized, and rare disease drugs
👍 Effective treatment for unmet needs!
" Too expensive, unaffordable; bankrupt public purse!!
§ TREND: Patients learn about new drugs thru social media
👍 We deserve access; unethical to put a price on someone’s life!
" Blame Industry; prices too high; drive up patient demand!!
§ TREND: HTA needs new methods to assess new therapies
👍 Pilot projects in managed access, risk sharing, CED
" Need infrastructure, registries, stakeholder buy-in and trust!!
5

6. How Can HTA Work…and Earn
Patient Trust?
1. Using available evidence, HTA determines appropriate
treatments for desired patient outcomes
2. Healthcare systems make available range of (cost-
effective) treatment options to meet range of patient
needs
3. Patients and clinicians choose best (effective and cost-
effective) alternative to achieve individually defined
patient outcomes
4. Patients provide feedback on treatment impact; evidence
used to re-assess individual treatment, value (price) of
therapies, and options available
6

7. HTA Needs Patient Trust to be
Effective
§ For Effective, Cost-effective, Sustainable Drug
Access, Patients Must be Accept HTA Guidance
§ Individual Level: To make appropriate choices and
adhere to optimal use
§ Services Level: To ensure patient-relevant outcomes,
real-world benefits and adverse effects are
addressed in healthcare delivery
§ Policy Level: To agree on values and rules for
equitable and sustainable resource allocation
§ No Voice => No Agreement => No Adherence
=> No Benefits = > No ROI
7

8. Patient Trust in HTA Requires
Patient Engagement
§ Knowledge: Patients and families have first-hand knowledge
about their condition and desired outcomes from treatment
§ Patient input improves likelihood of patient-appropriate
therapies and access criteria that are accepted by patients
§ Ethical: Patients have a right to be involved in decisions
that affect personal health and well-being
§ Patients who feel respected and treated fairly will more likely
respect and support societal values
§ Impact: Patient engagement improves quality of care,
quality of life and clinical outcomes
§ Trained knowledgeable patients make better informed choices
and are more likely to adhere to treatment
§ Empowered patients engage in self-care and responsible use of
health resources
8

9. May 2015
Harmonized Orphan Drugs/Rare Diseases Policy
-- Latin America 9
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

10. Patient Input Essential in
Emerging HTA Systems
§ As emerging healthcare systems move
toward more systematic health technology
assessment, it will be important to ensure
the patient voice is included.
• Policy makers and HTA agencies must define
processes for legitimate patient input (who,
what, when, and how
• Patient advocates must receive training and
support to provide an informed voice

11. What Patient Advocates Can Do
to Engage in HTA
§ Become an “expert patient” in HTA
§ Ask HTA agencies in your country to present to your
patient organization
§ Take part or offer HTA training to your patient advocates
§ Ask for and read HTA summary reports; get academic
support to interpret; ask for “patient friendly” summaries
from your HTA agency
§ Make HTA process transparent
§ Publish HTA summaries on your website or FACEBOOK
§ Publicly critique decisions, timeliness, appropriateness
§ Ask for advance notice of HTA reviews and timelines
11

12. What Patient Advocates Can Do
to Engage in HTA (2)
§ Provide input to HTA reviews
§ Ask for schedule or notification of review prior to start
§ Prior to review, provide summary of patient experience and
perspectives, using “standardized” form
§ Consult with company, clinicians, academics; seek feedback
§ Declare “conflict of interest” in terms of information,
financial, research, or preparation support receive but don’t
apologize
§ Form patient-led, cross-disease patient organization
§ Share knowledge, experience, materials
§ Engage experts; conduct training
§ Participate in meetings: ISPOR, HTAi, IAPO, ABRALE, PAHO
12

13. HTA:
Not
Just
Economics!
§ Quasi-­‐scien*fic
(Evidence-­‐based)
Approach
“The
process
of
systema*cally
reviewing
exis*ng
evidence
and
producing
an
evalua*on
of
the
effec*veness,
cost-­‐effec*veness,
and
impact,
both
on
pa*ent
health
and
the
health
care
system
of
health
technology
and
its
use.”
§ Evidence-­‐Based
Quan*ta*ve
(Cost)
Assessment
§ Effec*veness
(compared
to
alterna*ves)
§ Cost-­‐
(comparison,
to-­‐benefits,
per
QALY)
§ Savings
(replacement,
long-­‐term
healthcare,
societal
benefit)
§ Appraisal
Includes
“Other”
Criteria,
ESP
Pa*ent
Impact
§ Appropriate
use
(adherence,
management)
§ Pa*ent
psychological
and
social
Impact
(lifestyle,
ability
to
work,
tolerance,
family,
last
chance,
hope)
§ Societal
Values
13

14. 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”

15. Strategies to Improving Impact of
Patient Submission in HTA
Barriers to Patient Input Suggested Best Practices
QOL Scales not capture patient
experience; no long-term impact
Patient perceived outcomes built
into clinical trials (QOL, PROs)
No process to integrate patient
submissions into quantitative
approach; ignore, discount
Patient submissions to HTA give
qualitative experience; own
words; collective group input
Patient/public member roles
poorly defined; lack technical
expertise; no active patient voice
Patient/public members are
patient advocate; summary
validated by patient group
Most public/patients unaware of
processes; too technical or
academic
Patient consultations, training,
input templates, feedback
improve expertise and trust
Patients want full access;
don’t trust unless present:
want right to appeal
Plain language reports increase
patient understanding and
acceptance of HTA outcomes
15

16. 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

17. Different Roles of Patients in HTA
Charles River Associates, 2013

18. Best Practice: Patient Input on
Managed Access Programs
§ Ask patients about outcomes that clinically meaningful and
relevant; customize MAPs based on patient disease status
and lifestyle preference
§ Prescribe drug based on individual response and lifestyle;
maximize adherence for best outcomes
§ Engage patient in self-management; provide option for oral
therapy with commitment to adherence
§ Set up individualized start-stop plan; ensure patient and
family understanding criteria and rationale prior to gaining
commitment; monitor with on-going feedback and patient
engagement
§ Example: Ontario Oncology Evidence Building Program
NIHTA November 2013 18

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