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HTA: What every Patient Needs to Know
Durhane Wong-Rieger, PhD Consumer Advocare Network
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?
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
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
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 Other Therapy
Life Saving
Stop Progression
Fewer Side
Effects
Easier to
Manage
Return to
Work
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
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?
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
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
CEA: Cost Minimization
10
Patient Population
Submitted Drug
Comparator Drug
Similar clinical effects
Consider costs - Drug costs - Total cost (drug & health care)
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 > ?
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.)
CO
ST $
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
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?
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
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
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 Patient Representativeness
Patient Representativeness
Patient Representativeness
Par
tici
pat
ion in S
yste
m
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”
Contact:
Durhane Wong-Rieger
Consumer Advocare Network
www.consumeradvocare.org
416-969-7435
