Ethical issues and HTA
-
Upload
htai-bilbao-2012 -
Category
Health & Medicine
-
view
594 -
download
0
description
Transcript of Ethical issues and HTA
Improving the Value of Patient
Submissions on Drug Review Processes
Durhane Wong-Rieger, PhDDevidas Menon, PhD
Tania Stafinski, PhDGabriel Wong-Rieger, BCom
Purpose: How to Promote the Value of Patient Submissions
Challenge that HTA (value of therapy) did not sufficiently account for patient impact Concern: HTA (as applied to the public drug review
processes in Canada) do not adequately reflect the value of the therapy to patients
HTA theoretically includes societal values but evident
Need for direct patient input Beyond Quality of Life scales (does not sufficiently
capture all of the value to patients; quantitative summary does not convey patient perspective)
Patient is best positioned to provide experience of disease, previous therapy, new therapy
Introduction of Patient Submissions
Introduced by CADTH, Ontario, and BC (process in Quebec)
Opportunity for patients and patient representatives to provide direct input, in their own worlds
In most cases, will be presented by the patient/pubic member on the HTA committee (accurately reflect, interpret and advocate for the patient perspective)
Challenge: How does committee integrate patient input in HTA process?
Purpose of Workshop Sessions
Purpose: Provide Insight on Patient Submissions and Influence on HTA process
Questions AddressedGiven all of the data available to HTA, what
is perceived value of direct information from patients on impact of disease and therapy
What are ways in which patient data could be meaningfully considered within HTA processes?
Does the process (group deliberation) for considering patient data affect the outcome (decision on value of therapy)
Objectives of Sessions
2 workshops (Ontario and BC) with patients and other stakeholders
Objectives Learn about patient submission process: what, how. why
Learn from patient/public members of HTA committees on perceived value and process for considering patient input
Gain insights on HTA/drug review process through simulation
Compare methods for considering patient input as part of HTA/drug review process
Learn factors that potentially influence drug review
Participants
Two SessionsOntario: 90 (50% or 45 patients)BC: 75 (65% or 49 patients)
Patient representativesPatients, patient advocates, board, and staffPrevious participation in HTA training
Some experienced in making submissions to CADTH or CED
Session Agenda (Intro)
Pre-assessment: knowledge and trust of agencies
Introduction to HTA and Committees Overview of HTA Overview of CADTH (Common Drug Review) and
Patient Submission Process
Overview of provincial drug review process
Perspectives of patient/public members CADTH (CEDAC) Ontario Committee to Evaluate Drugs Patient organizations
Small Group Simulation of Drug Review Committee
Each group reviews 3 of 4 drugs for different conditions Disease (health condition) and drug information, including
safety and efficacy data, size of patient population Cost-effectiveness data, including alternative treatment
May only recommend 2 of 3 drugs Without patient input, make Initial group decision on
whether to recommend drug for funding Question: what additional patient information do you
want? Provided “patient submission” on impact of condition,
current therapy, proposed drug impact Provided with 3 of 4 formats for considering the patient
submission along with rest of data
Drugs and Condition Information
A: Progressive debilitating; affects 2 in 1000 Current: injection every 4 weeks; reduces relapses,
slows progression, mild to severe side effects New: Injection every 4 weeks, reduces relapses by
half; 1% severe disability or death; $20K/year; $68K/QALY
B: Genetic, auto-inflammatory; affects 1 in 100,000; debilitating, mental retardation, vision loss, deformities No approved therapy but RA therapy used off-label,
daily injection. New drug injected every 6 weeks; nonlife-threatening
side effects, $96K/year; $110K/QALY
Drugs and Condition Information
C: Blood cancer, affects 1 in 20,000 Current: IV chemotherapy, 2-year survival at 25% New: oral; 3 single-arm CTs = 2-year survival at 93%;
1% risk liver toxicity; life-long therapy $30K/year; $20K/QALY
D: Obesity, affects 25% Canadians, linked to diabetes, CVD, cancers New drug: oral, twice daily. RCT loss of 8kg more than
placebo; reduced metabolic CDV risks; 25% psychiatric events with 0.02% suicides; on treatment until healthy weight; $3K/year, $60K/QALY
Group Decision-Making Formats
Each group assigned Open Discussion + 2 other formats
Open Discussion: Individuals freely share opinions, beliefs, and preferences to arrive at a group decision. Pro’s: Equal voice, relevant attributes specific to current case,
unrestricted conversation
Con’s: Leader/champion influence, lack of prioritization or systematic consideration of all attributes
Nominal Group Technique: Individuals formulate and express own opinions prior to influence from others Pro’s: Initial personal opinion shared; al options considered; identify
and discuss personal biases
Con’s: Support own “uninformed” opinion; polarization of views
Group Decision-Making Formats
Each group assigned Open Discussion + 2 other formats
Dialectical Inquiry/Deliberative Dialogue: All aspects of “funding or not funding” explored prior to making decision. Pro’s: Attributes relevant to key stakeholders strongly positioned
Con’s: Arriving at consensus; need for representative views
Simple Multi-attribute Rating Technique: Systematically analyze the attributes of the drug and arrive at a decision based on a “weighted” assessment of the criteria Pro’s: Consistent and systematic approach across drugs
Con’s: Restricted discussion of perspectives; too reductionist
Patient Submission Summaries
Drug A for Progressive neuromuscular Impact: Devastating, severe attacks, relapses Treatment outcomes desired: reduce severity,
frequency of attacks; less severe side effects Drug characteristics: small fatal risk acceptable
Drug B for Rare Genetic Disorder Impact: Fever, pain, joint and tissue deterioration Treatment outcomes desired: short-term reduction of
flare-ups, fever, pain; long-term damage reduction; Drug characteristics: fewer injections
Patient Submission Summaries
Drug C for Blood Cancer Impact: devastating, low survival, helpless, hopeless Treatment outcomes desired: survival, reduced blood
counts Drug characteristics: oral, nausea manageable
Drug D for Obesity Impact: Physical and psychological, low self-esteem Treatment outcomes desired: sustainable weight loss
which is very difficult to attain Drug characteristics: most effective, least risky
options; immediate and steady weight loss
Results: Group Decisions
Total 19 groups and 57 decisions Each group: may approve maximum 2 of 3 drugs Groups segregated as patients and non-patients
Small difference in recommendations from patients and non-patients Patients: approved 67% drugs (2 out of 3) Non-patients approved 59% (fewer than allowed)
Small difference between Ontario and BC groups BC: 71% approval (1 group approved all 3 drugs) Ontario: 58% (2 groups approved only 1 drug)
Results by Condition and Drug
Approvals (all groups combined)
Drug C (Blood Cancer) approved by 100% of groups
Drug B (Rare Genetic) approved by 86% of groups
Drug A (Progressive) approved by 67% of groups
Drug D (Obesity) approved by 0% of groups
Rationale for Drug Decisions
Important Rationale
Drug C: Increased chances for survival; everyone relates to cancer; acceptable $/QALY
Drug B: No other treatments; no proof of long-term benefit; small numbers; approve despite very high $/QALY
Drug A: Reduced relapses but no impact on progression; severe disease; high $/QALY
Drug D: Not significant benefit; no direct disease impact; should not be public funding
Approvals by Discussion Format
No difference in Approvals by Discussion Format
Multi-Attribute Rating: 67% Approvals
Open Discussion: 63% Approvals
Nominal Group: 64% Approvals
Dialectic Dialogue: 58% Approvals
Approvals by Drug and Discussion Format
Progressive
Debilitating
Rare Geneti
c
Blood Cancer
Obesity Overall
Open Discussion
60% 80% 100% 0% 63%
Nominal Group 50% 100% 100% 0% 64%
Mutli-Attribute Rating
75% 75% 100% 0% 67%
Dialectic Dialogue 75% 100% 100% 0% 58%
OVERALL 67% 86% 100% 0% 63%
Frequency of Decision Reasons
For positive decisions, frequently cited: Safety (lack of or manageable side effects) Cost, cost-effectiveness, or cost mitigated by other
benefits Meeting patient needs and survival benefits Quality of life; lack of alternatives
For negative decisions, frequently cited: Lack of comparator (placebo only) Lack of adequate information Risk of side effects Lack of sustainability and long-term outcomes Lack of cost-effectives (cited by only 1/3)
Average Number Reasons Cited by Variable
Variable Avg. # Reasons
Discussion Format Open 2.2
Nominal Group 2.5
Multi-Attribute Rating 1.6
Deliberate Dialogue 1.4
Drug Progressive Disease 2.1
Rare Genetic 1.4
Cancer 2.1
Obesity 2.3
Decision Yes 1.9
No 2.1
All 2.0
Summary—Number of Reasons
By decision, more reasons cited for “no” (2.1) than for “yes” (1.9)
By deliberation format, fewer reasons with more structured group format (multi-attribute (1.6) and deliberate dialogue (1.4)) than with more open discussions (open format (2.3) and nominal group technique 2.5))
By type of drug, fewest reasons given for rare genetic drug (1.4) than for all others (2.1 – 2.3)
Preferences for Discussion Format
Preferences for Discussion Formats
MA Rating: best liked; easy to do; sorted options for easy decision making
Open: Usual process; best when high consensus; leader may set direction
Nominal: Assured all opinions heard; allowed differences to surface
Dialectic: Difficult to “role play”; helpful in hearing patient perspective
Advocare Simulation: Patient Input Shifts Discussion
Groups felt patient information was very important for conditions that were unfamiliar to them, specifically the progressive debilitating disease and the rare genetic condition.
Information about the patient experience of the challenges of the conditions were cited as important, as was information about the current therapies, or lack thereof.
Prior to receiving patient information, groups said they were more focused on cost, cost per QALY, and affordability.
Introduction of patient submission focused discussion, regardless of the format, on the patient’s perception of the burden of the condition, the challenges of current therapy, and the potential benefit on hope, manageability, and quality of life.
Advocare Simulation: How Does Patient Input Impact Discussion?
Groups reported that the patient submission helped them to visualize the patient, see him/her as a person rather than statistic
The “lack of concern” about potentially fatal (but very rare) side effect was important
Risk of imminent death (blood cancer) was a significant factor and had greater weight than debilitating condition. Drug C for blood cancer always chosen regardless of alternatives.
Slowing progression was not as important as reducing risk of developing organ, joint, tissue damage. Drug A for progressive condition NOT chosen if alternatives were B (genetic disorder) AND cancer.
Advocare Simulation: Findings of Patient Submission Workshops
Direct patient input (submissions) enhance discussion of patient impact and consideration of patient values and preferences
Cost factors (individual cost/patient and $/QALY) are important factors but can be superseded by patient impact (especially if rare disease or not other treatments)
Survival (even for limited time) given more weight than reducing risk of future serious organ/joint damage and reducing progression of debilitating disease
Groups liked rating tool; easy way to integrate variety of factors and rank options.
Mutli-attribute Rating technique also resulted in the highest percentage of approvals overall
Drug fo common condition (obesity) not given priority over serious diseases.
Patient Trust in Drug Review Agencies Pre-and Post-Workshop
Trust knowledgeable and fair process by… (agree somewhat and stronglY
Ontario Pre-
OntarioPost
BCPre
BCPost
Health Canada approving drug 90% 100% 95% 92%
CADTH (Common Drug Review) recommending 65% 84% 43% 65%
Provincial Drug Program funding drug 55% 68% 45% 425
Physician recommending drug 90% 74% 95% 92%
Patient Group informing re: drug and options 90% 100% 100% 92%
28
Recommendations for Patient Engagement with HTA
Dialogue with patient groups to define submission: what information, how to collect, how to present
Engage patients in CT design to ensure patient values included in measures
Train potential HTA patient-public members on technical processes of HTA and decision making
Provide means for patient-public members of HTA committees to dialogue with patient representatives
Train all HTA committee members on methods for integrating qualitative information
Promote transparent decision-making (records of deliberation as well as outcomes); open meetings.
COMING: Patient Experts in Health Technology!
Contact:
Durhane Wong-Rieger
Consumer Advocare Network
www.consumeradvocare.org
416-969-7435