should suboptimal clinical evidence be used to inform hta ... · guidance from 2016 pcodr metrics...
TRANSCRIPT
![Page 1: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/1.jpg)
Should Suboptimal Clinical
Evidence be Used to Inform
HTA Recommendations?
Alex Chambers
Director, pCODR
CADTH
![Page 2: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/2.jpg)
Disclosure
• The speaker has no financial or other conflict of interest to
report.
1
![Page 3: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/3.jpg)
Overview of Drug Review in Canada
Health Canada
Regulator (Effect & safety)
CADTH (CDR and pCODR)
INESSS (Quebec)
HTA (Assess value)
F/P/T Ministries of Health and Provincial Cancer Agencies
Decision maker/ funder
Pan Canadian Pharmaceutical Alliance (pCPA)
Price negotiator
2
![Page 4: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/4.jpg)
Does pCODR Review
Submissions with Suboptimal
Clinical Evidence?
![Page 5: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/5.jpg)
4
Source: Tiruneh et al, ASCO Quality, 2018
pERC Recommendations Based on
Non-Randomized Trials (Hematology-only)
![Page 6: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/6.jpg)
Cost of Drugs Without a Direct Comparison
to Standard of Care
5
$-
$5,000
$10,000
$15,000
$20,000
$25,000
$30,000
$35,000
$40,000
List
Pri
ce p
er 2
8 d
ays
2012 2015 2016 2017 2013 2014
![Page 7: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/7.jpg)
Guidance from 2016 pCODR Metrics
Report
• If non-comparative data are used, it is important for a
submitter to consider the following factors:
• If there is an important therapeutic need that a drug
under review may fill
• A randomized trial is not feasible due to various reasons
(e.g., the rarity of disease, unethical to randomize,
among others)
• Meaningful outcomes in a large sample of patients
when compared with historical data on current standard
of care.
6
![Page 8: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/8.jpg)
What are the Challenges of
Reviewing Suboptimal Clinical
Evidence?
![Page 9: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/9.jpg)
pCODR Expert Review Committee’s
(pERC) Deliberative Framework
8
Clinical Benefit • Effectiveness • Safety • Burden of Illness • Need
Patient Values • Alignment with values
Economic Evaluation • Cost effectiveness
Adoption and Feasibility • Economic feasibility (BIA) • Organizational feasibility
![Page 10: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/10.jpg)
Example 1: Avelumab for Merkel Cell
Carcinoma
9
![Page 11: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/11.jpg)
pERC Recommendation Avelumab
cont’d… • pERC concluded that there is a substantial unmet need
for alternative options
• Conducting a RCT in this setting with avelumab compared
with palliative chemotherapy would not be feasible given
the small population of patients with mMCC.
• pERC noted that a substantial proportion of patients
experienced an objective response rate (ORR), which is an
important and clinically meaningful outcome in mMCC.
pERC noted that the ORR was higher than historical
responses observed with chemotherapy treatments used
to treat mMCC.
10
![Page 12: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/12.jpg)
pERC’s Next Step for Avelumab
• Collecting Evidence to Reduce Uncertainty in the
Magnitude of Clinical Benefit and the Cost-
Effectiveness of Avelumab
• Additional prospective evidence should be collected to
decrease the uncertainty in the incremental effect and
provide a greater understanding of the true cost-
effectiveness of avelumab. Specific information on long-
term efficacy, safety, and QoL would be of particular value.
11
![Page 13: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/13.jpg)
Example 2: Nivolumab for
Hepatocellular Carcinoma
12
![Page 14: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/14.jpg)
pERC Recommendation on
Nivolumab cont’d…
• pERC concluded that there is a significant unmet need in
this setting
• pERC discussed the feasibility of conducting a phase III
RCT in this patient population. They considered that there
are RCTs investigating other therapies for this patient
population. pERC also considered that registered clinicians
noted that they wanted access to nivolumab, but recognized
the uncertainty of the non-comparative data.
• [pERC] noted that the ORR … did not reach the clinical
significance threshold
13
![Page 15: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/15.jpg)
Conclusions
• pCODR reviews all of the available evidence for drugs
under review
• pERC regularly makes positive, conditional, and negative
recommendations on non-comparative data
• Moving forward…
• Opportunities to re-review the evidence, especially in
circumstances were there is lower quality of evidence,
would be worthwhile to ensure that drugs are delivering
the value to the health system that was expected
14
![Page 16: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/16.jpg)
15
![Page 17: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/17.jpg)
© 2018 Evidera. All Rights Reserved.
Incorporating evidence quality into pCODR decisions using an MCDA
Kevin Marsh Executive Director, Patent Centered Research 2019 CADTH Symposium
![Page 18: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/18.jpg)
Objectives
Premise: Society is willing to provide early access, based on sub-optimal evidence for patients with unmet need
Objective:
1. Is there any evidence that this is the case?
2. How might we more formally operationalize such preference in decision making?
![Page 19: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/19.jpg)
What factors influence pCODR decisions?
18
![Page 20: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/20.jpg)
What factors influence pCODR decisions? Methods: Data extraction
19
Identify variables (56)
Test extraction Extract (22
items, decisions 7/10-4/18)
Analysis
Data were extracted on the variables focusing on four main areas:
Clinical Benefit Economic Evaluation
Patient Inputs Adoption Feasibility
Quality of clinical evidence ● Number of trials ● Trial design
Clinical outcomes ● PFS/OS/RR ● Safety ● QoL
Quality of economic evaluation ● Submission quality ● Limitations
Economic evaluation result ● Manufacturer- submitted
ICER ● HTA re-analyzed ICER ● Conclusion of CE
Alignment with patients’ value in terms of
● Disease control / symptom relief
● Drug admin type ● Access to new treatment ● Side effect ● Patient QoL
Quality of budget impact analysis
Magnitude of budget impact
Abbreviations: admin = administration; HTA = health technology assessment; ICER = incremental cost-effectiveness ratio; OS = overall survival; pCODR = pan-Canadian Oncology Drug Review; PFS = progression-free survival; QoL = quality of life; RR = response rate
![Page 21: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/21.jpg)
What factors influence pCODR decisions? Statistical Analyses: Overview
Of the 111 recommendation decisions published, 12 were full list, 74 conditional list, and 25 do not list.
Our predictive analyses assumed that the pCODR decisions were conducted in a sequential manner:
Two methodologies were explored to model each stage of the decision process:
● Logit binary regression model using Cordeiro and McCullagh bias correction1
● Classification and Regression Trees (CART)
Decision
Conditional List Fully List
Rejection Approval
Source: 1. Cordeiro, G. M. and P. McCullagh (1991). Bias correction in generalized linear models. Journal of the Royal Statistical Society, Series B: Methodological 53(3), 629–643.
20
![Page 22: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/22.jpg)
Results: Predictive Model - List vs. Do Not List
21
![Page 23: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/23.jpg)
Results: Predictive Model - Full List vs. Conditional List
22
![Page 24: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/24.jpg)
Can MCDA support pCODR to make decisions?
23
![Page 25: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/25.jpg)
What is MCDA?
24
A collection of analytical methods used to support decision
making and value communication in the context of:
• Complex problems involving multiple, potentially conflicting,
objectives
• Involving potentially multiple stakeholders
![Page 26: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/26.jpg)
What is MCDA? Common Steps
25
Problem structuring
Measure treatment
performance
Elicit stakeholder preferences
Evaluate and compare
alternatives
Overall Objective
Benefit Risk Convenience
Treatment A
Treatment C
Treatment B
𝑤𝑏 𝑤𝑐 𝑤𝑟
• Identify treatments
• Stakeholders
• Criteria
• Performance
Overall performance
score
Treatment A
Treatment C
Treatment B
Analysis contributes here
![Page 27: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/27.jpg)
Benefits of the MCDA Process
26
Ensures all relevant factors (including intangibles) are
considered
A transparent link between performance, judgments and value
Quantify priorities and preferences Fosters a shared understanding of the decision problem and identifies
areas of important disagreement
Better decisions (consistent, transparent)
![Page 28: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/28.jpg)
How is MCDA used to support HTA? There is precedent for the adoption of MCDA
27
IQWiG: 2 types of MCDA “can contribute to determining the most important outcomes for patients as part of economic evaluation”
Colombia: Developing an MCDA for HTA
Thailand: MCDA used to inform coverage decisions for HIV/AIDS interventions
Russia: MCDA for orphan drugs
* Illustrative examples; not comprehensive, IQWiG: The Institute for Quality and Efficiency in Healthcare
Hungary: MCDA has been used to evaluate new hospital medical technologies since 2010
Indonesia
Ghana
Cote d’Ivoire
South Africa
Italy: The Lombardy region introduced MCDA in 2008 to decide on the introduction and delisting of health technologies
New Zealand: MCDA approach to ‘points system’ for elective surgery
NHS England MCDA used in commissioning specialised services
![Page 29: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/29.jpg)
Implemented in 2010
Evaluation of 14 applications for medical technology
Criteria and weights established by a committee comprising of:
● The Health Financing Agency
● The Ministry of Health
● Clinical experts
● Health economists
How is MCDA used to support HTA? Case Study: Reimbursing Medical Technology in Hungary
Source: Endrei, et al. Value Health 2014
28
![Page 30: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/30.jpg)
Criteria Levels Weight SOC Treatment
High quality evidence? Clinical benefit
Yes, Improved 4
No, Improved 3
No improvement 0
Side effects Aligned 2
Not aligned 0
QoL
Improved 2
Similar 1
Deteriorated 0
Unmet need Yes 2
No 0
8 9
$X $Y
29
What might an MCDA look like?
ICER: (Y-X)/(9-8)
![Page 31: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/31.jpg)
How might decision makers respond to the MCDA proposal? Consultations have identified some challenges
30
1. Consultation on the Development of 2013 Guide to the Methods of Technology Appraisal.
o Benefits of MCDA (consistency) outweighed by ‘advantages of deliberation’ flexibility to undertake context specific decisions
2. Consultation on the Implementation of Value Based Assessment (1)
o Allow conditions with a higher burden and societal impact to obtain a higher threshold of up to £50,000/QALY?
(1) https://www.ispor.org/ValueOutcomesSpotlightResources/health-policy_value-based-assessment-UK.pdf
“too formulaic with not enough flexibility”
“lack of standard approach to elicit weights”
“difficult to find weights that satisfied all members of society”
![Page 32: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/32.jpg)
31
The pERC Deliberative Framework [9] identifies four main criteria that need to be considered during pERC’s review process
Clinical Benefit Economic Evaluation
Patient Inputs Adoption Feasibility
Quality of clinical evidence ● Number of trials
submitted/considered by pCODR
● Trial design Clinical outcomes
● PFS/OS/RR ● Safety ● QoL
Quality of economic evaluation ● Submission quality ● Limitations
Economic evaluation result ● Manufacturer- submitted
ICER ● HTA re-analyzed ICER ● Conclusion of cost-
effectiveness
Alignment with patients’ value in terms of
● Disease control / symptom relief
● Drug admin type ● Access to new treatment ● Side effect ● Patient QoL
Quality of budget impact analysis
Magnitude of budget impact
Abbreviations: admin = administration; HTA = health technology assessment; ICER = incremental cost-effectiveness ratio; OS = overall survival; pCODR = pan-Canadian Oncology Drug Review; PFS = progression-free survival; QoL = quality of life; RR = response rate
How might decision makers respond to the MCDA proposal? A complete framework?
![Page 33: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/33.jpg)
How might decision makers respond to the MCDA proposal? Are attribute levels sufficiently nuanced?
32
Value
Clinical outcomes/trial quality
Clinical benefit by trial quality
Clinical efficacy
(clinical benefit vs. no benefit)
PFS
OS
RR
Trial quality (high vs. low)
Phase
Phase III
Phase II or I
Randomization
Randomized
Non-randomized
Control arm
Active/placebo controlled
Single arm
QoL
Deteriorated/ uncertain/
limited
Improved
Similar/ maintain/stable
Patient value-side effect
No (not aligned)
Unknown
Yes (aligned)
Unmet need
Yes
No
Criteria Levels
Trial Quality
High – RCT, blinded
Low
![Page 34: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/34.jpg)
Are HTA value frameworks additive? E.g. The value of health gain is a function of baseline severity?
33
Nord E, Daniels N, Kamlet M. QALYs: some challenges. Value in Health 2009; 12: S10–5.
Rowen et al http://journals.sagepub.com/doi/full/10.1177/0272989X15619389
![Page 35: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/35.jpg)
Are HTA value frameworks additive? Additive frameworks can produce erroneous results
34
Overall Objective
Health gain
Severity # Patients
40% × 40 + 40% × 70 + 20% × 40 = 52
𝑤𝑏 = 40% 𝑤𝑝=20% 𝑤𝑠 = 40%
100
0
40
70
40
![Page 36: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/36.jpg)
Are HTA value frameworks additive? Additive frameworks can produce erroneous results
35
Overall Objective
Health gain
Severity # Patients
40% × 40 + 40% × 70 + 20% × 40 = 52
𝑤𝑏 = 40% 𝑤𝑝=20% 𝑤𝑠 = 40%
100
0
40
70
40
40% × 0 + 40% × 100 + 20% × 100 = 60!
100 100
E.g. Morton A (2017) Treacle and Smallpox: Two Tests for Multicriteria Decision Analysis Models in Health Technology Assessment. Value in Health 512 – 515.
![Page 37: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/37.jpg)
How might decision makers respond to the MCDA proposal? Value concepts are not additive?
36
Value
Clinical outcomes/trial quality
Clinical benefit
Trial quality (high vs. low)
QoL
Patient value-side effect
Unmet need
Value interactions/ overlaps
![Page 38: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/38.jpg)
A proposal ‘MCDA with (multiplicative) decision rules’
37
Unmet need Trial quality ICER Threshold
High High A
Low B
Medium High C
Low D
Low High E
Low F
Step 1: ‘Rules’
Step 2: Deliberation
Factors for which there is some consensus on their relative importance
A. Validate / refine framework with pCODR
B. Stated preference
List of other factors considered important, but for which it is not possible to agree weights
![Page 39: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/39.jpg)
Should MCDA be used to support HTA decision making?
Christopher McCabe PhD
April 15 2019
![Page 40: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/40.jpg)
Conflicts of Interest
The Institute of Health Economics is a Not-for-Profit founded by the Government of Alberta, the Universities of Alberta and Calgary, and the Canadian Pharmaceutical Industry. It receives core funding from the Government of Alberta, the Universities of Alberta and Calgary and Canadian pharmaceutical companies who are members of the Institute
In addition, the Institute receives project specific funding from public and private sector organizations.
Dr. McCabe receives research funding from CIHR, Genome Canada, Alberta Innovates and the UK National Institute for Health Research
The views expressed in this presentation do not necessarily represent the views of our Board Member Organizations or funders
![Page 41: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/41.jpg)
Overview
• Multi-factorial decision making
– The challenge of preference independence
– Appropriate use of evidence
• Factor weights in MCDA
• Values and MCDA for Health Technology Appraisal
![Page 42: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/42.jpg)
Multi-factorial Decision Making
Mutual Independence of Preferences: 1. The value attached to side effects are
unlikely to be independent of the value attached to changes in quality of life.
2. The value attached to any given clinical benefit is unlikely to be independent of the value attached to addressing unmet need.
3. The value attached to a given clinical benefit is unlikely to be independent of the value attached to a given change in quality of life – although the value attached to an improvement in quality of life may well be independent of the value attached to the clinical benefit that produced it.
![Page 43: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/43.jpg)
Multi-factorial Decision Making
Inte
rnal
val
idit
y External validity
Trial Quality
![Page 44: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/44.jpg)
Multi-factorial Decision Making
![Page 45: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/45.jpg)
Factor weights in MCDA
Life threatening acute disease gets twice the weight of non-life threatening chronic disease.
The total budget impact is only as important as the reputational impact.
Cost Effectiveness is twice as important as any other health policy priority
Are these values the right values?
![Page 46: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/46.jpg)
Values and health care resource allocation
![Page 47: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/47.jpg)
Values and health care resource allocation
1. What is to be valued?
2. How is it to be described?
3. How is it to be valued
4. Who is to value it?
5. How are values for all possible combinations to be generated?
6. How are the valuations to be aggregated?
![Page 48: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/48.jpg)
MCDA in HTA: some challenges
• Should weights represent values/preferences? – If not, what should they represent and why?
• Does the wider scope of MCDA obviate the need to choose: – How values are elicited – Whose values are used for the decision – Methods for combining and extrapolating from sample value data
• Does the wider scope of MCDA capture opportunity cost any more convincingly than standard HTA?
• Are MCDA models any more accurate at predicting actual preferences?
![Page 49: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/49.jpg)
![Page 50: Should Suboptimal Clinical Evidence be Used to Inform HTA ... · Guidance from 2016 pCODR Metrics Report • If non-comparative data are used, it is important for a submitter to consider](https://reader034.vdocuments.mx/reader034/viewer/2022042805/5f5f87adc18d236388420eba/html5/thumbnails/50.jpg)
THANK YOU