Technical Guidelines for preparing assessment reports for the Medical Services Advisory Committee – Medical Service Type: Therapeutic
(Version 2.0)
March 2016
Medical Services Advisory Committee
Foreword
The Medical Services Advisory Committee (MSAC) is an independent committee that provides advice to the Minister for Health on the strength of the evidence relating to the comparative safety, clinical effectiveness and cost-effectiveness of any new or existing medical services or technology, and the circumstances under which public funding should be supported through listing on the Medical Benefits Schedule (MBS).
To achieve this, MSAC undertakes Health Technology Assessments (HTA) using the best available evidence to assess proposals for their comparative safety, clinical effectiveness, and cost effectiveness.
Applications for therapeutic services provide applicants with a number of challenges, requiring them to prove that both the proposed service provides accurate, meaningful information and also that the information improves the subsequent treatment (and health outcomes) of patients.
This document provides detailed advice to assist applicants with determining content and presentation of submissions of evidence for consideration by MSAC and the Evaluation Sub-committee (ESC).
This document provides detailed advice to assist applicants with determining content and presentation of submissions of evidence for consideration by the MSAC and the ESC.
Chair
Medical Services Advisory Committee
Foreword i
Contents
Foreword i
Contacts xi
Record of updates xii
Abbreviations xiv
PART I General information 1
1 Medical Services Advisory Committee 2
1.1 Purpose and roles of MSAC 2
1.2 Membership of MSAC 2
1.3 MSAC sub-committees 2
1.4 Overview of MSAC processes 3
1.4.1 Regulatory framework 3
1.4.2 The application and assessment process 3
1.4.3 Sources of advice 3
1.4.4 Publication of assessment report 3
1.4.5 Timing of MSAC processes 4
1.4.6 MSAC appraisal 4
2 Introduction to the Guidelines 5
2.1 Structure of these Guidelines 5
2.2 Associated documents 5
2.3 What is a therapeutic medical service? 6
2.4 Writing and style conventions used in these Guidelines 6
2.5 The future 7
3 Rationale and basis for the economic evaluation in the Australian context 8
3.1 Analysis of cost-effectiveness 8
3.2 Australian context 8
3.3 Relevant factors influencing MSAC decision making 8
3.4 Flexibility in interpretation of these Guidelines 9
4 Organisation of a standard assessment report 10
4.1 Choice of information 10
4.2 Overview of a standard assessment report 11
4.2.1 Sections of a standard assessment report 11
4.3 Presentation of the assessment report 11
4.3.1 Standard assessment report 11
4.3.2 Two stage approach to an assessment 13
4.3.3 Co-dependent/integrated assessment reports 13
5 Lodging an assessment report 14
5.1 Assessment report checklist 14
5.1.1 Information requirements 14
5.1.2 Information requests 14
5.2 Provision of information to allow independent verification of computer analyses 16
5.2.1 Information requirements 16
5.3 Provision of information after lodgement of the assessment report 16
5.3.1 Post-lodgement communication with MSAC 16
5.3.2 Provision of information sourced from the TGA after lodgement of the assessment report 16
Part II Preferred clinical and economic evidence for proposed medical services to be considered by MSAC 17
Section A Details of the proposed therapeutic medical service and its intended use on the Medical Benefits Schedule (or for other public funding) 18
A1 Address all items in the agreed PICO Confirmation 18
A2 Proposed medical service 18
A3 Proposed MBS listing or other public funding sought 18
A4 Proposed population 18
A5 Comparator details 18
A6 Clinical management algorithm(s) 19
A6.1 Algorithms for intended and current contexts 19
A7 Differences between the proposed medical service and the main comparator 20
A8 Clinical claim 20
A9 Summarise the primary elements of the decision analysis (PICO) 20
Section B Clinical evaluation for the proposed therapeutic medical service 21
B1 Description of search strategies 23
B2 Listing all direct randomised trials 24
B3 Assessment of the measures taken by investigators to minimise bias 30
B4 Characteristics of the trials 33
B5 Outcome measures and analysis 34
B6 Systematic overview of the results 38
B7 Extended assessment of comparative harms 46
B8 Interpretation of the clinical evidence 46
Section C Translation Issues 49
C1 Identification of issues to be addressed 51
C2 Focused analytical plan 56
C3 Results of pre-modelling studies 68
C4 Relationship of each pre-modelling study to the economic evaluation 69
Section D Economic evaluation for the main indication 71
D.1 Overview of the economic evaluation 73
D2 Population and circumstances of use reflected in the economic evaluation 80
D3 Structure and rationale of the economic evaluation 85
D4 Variables in the economic evaluation 93
D5 Results of the economic evaluation 100
D6 Sensitivity analyses 107
Section E Estimated utilisation and financial implications 110
E1 Justification of the selection of sources of data 112
E2 Estimation of use and costs of the proposed medical service 114
E3 Estimation of changes in use and cost of other medical services 117
E4 Estimated financial implications for the MBS 118
E5 Identification, estimation and reduction of uncertainty 119
Section F Options to present additional relevant information 122
F1 Other relevant factors 122
Part III Alternative clinical evidence for proposed medical services to be considered by MSAC 125
Section B(i) Clinical evaluation for the main indication: indirect comparison of randomised trials 126
B(i)1 Description of search strategies 128
B(i)2 Listing all randomised trials considered for inclusion in indirect comparisons 128
B(i)3 Assessment of the measures taken by investigators to minimise bias 132
B(i)4 Characteristics of the trials 132
B(i)5 Outcome measures 133
B(i)6 Results 133
B(i)7 Extended assessment of comparative harms 134
B(i)8 Interpretation of the clinical evidence 135
Section B(ii) Clinical evaluation for the main indication: Presenting non-randomised studies 136
B(ii)1 Description of search strategies 138
B(ii)2 Listing all non-randomised studies 138
B(ii)3 Assessment of the measures taken by investigators to minimise bias 140
B(ii)4 Characteristics of the studies 142
B(ii)5 Outcome measures 142
B(ii)6 Results of the comparison involving non-randomised studies 142
B(ii)7 Extended assessment of comparative harms 142
B(ii)8 Interpretation of the clinical evidence 143
Section C(i) Translating the clinical evaluation: indirect comparisons of randomised trials or non-randomised studies 144
C(i)1 Relevant pre-modelling studies 145
Section D(i) Economic evaluation for the main indication: presenting a cost-minimisation approach 147
D(i)1 Cost-minimisation analysis 147
D(i)2 Presentation of a cost-minimisation analysis or a cost analysis 149
Appendices 151
Appendix 1 Relevant factors influencing provision of advice by MSAC 152
Appendix 2 Expert opinion 154
A2.1 Uses of expert opinion 154
A2.2 Presenting expert opinion 155
A2.3 Describing the collection and collation of expert opinion 155
Appendix 3 Assessment of non-inferiority 158
A3.1 Introduction 158
A3.2 Service delivery information 158
A3.3 Non-inferiority threshold 158
A3.4 Method of analysis 159
A3.5 Presenting an assessment of non-inferiority 159
A3.6 Assessing comparative harms in the context of non-inferiority 160
A3.7 Interpretation of the clinical evidence 160
Appendix 4 Utility valuation of health outcomes 161
A4.1 Use of health-related QALYs gained and cost-utility analysis 161
A4.2 Obtaining utility weights 162
A4.3 Trial-based utility valuation of health outcomes 163
A4.4 Scenario-based utility valuation of health outcomes 165
A4.5 Other methods for obtaining utilities 172
Appendix 5 Monetary valuation of healthoutcomes 175
A5.1 Preference for cost-utility analyses over cost-benefit analyses 175
A5.2 Scenario-based monetary valuation of health outcomes 176
Appendix 6 Including non-health care resources and non-health outcomes in a supplementary analysis 181
A6.1 Identifying, measuring and valuing non-health care resources 181
A6.2 Identifying, measuring and valuing non-health outcomes 181
A6.3 Resources and outcomes to be excluded 184
Appendix 7 Measures taken by the investigators to minimise bias in non-randomised studies 185
A7.1 Classical observational designs 185
A7.2 Quasi-experimental designs 187
Tables and Figures
Figure 4.1 Sections of a standard assessment report 12
Table 5.1 Checklist of information to be included in a therapeutic assessment report 15
FigureB1 Key information requests for assessment report Section B of an assessment for MSAC 22
Table B2.1 Summary of identification of direct randomised trials from the search of the published literature 25
Table B2.2 Summary of identification of applicants’a direct randomised trials and information from the manual search of retrieved citations 26
Table B2.3 Trials (and associated reports) presented in the assessment report 27
Table B2.4 Reasons to exclude each trial from further detailed assessment 28
Table B2.5 Comparative summary of characteristics of each direct randomised trial 28
Table B2.6 Comparative summary of results of each direct randomised trial 29
BoxB3.1 Checklist for assessing the quality (internal validity) of randomised trials 30
Table B3.1 Summary of the measures undertaken to minimise bias in the direct randomised trials 32
Table B3.2 Flow of participants through the direct randomised trials 32
Table B4.1 Eligibility criteria 33
Table B4.2 Characteristics of participants across randomised groups 33
Table B4.3 Therapeutic medical services compared in trials 34
Table B5.1 Primary outcomes and statistical analyses of the direct randomised trials 35
Table B5.2 Patient-relevant secondary outcomes and analyses 36
Table B6.1 Results of [patient-relevant outcome] (available as dichotomous data) across the direct randomised trials (relative risk) 41
Table B6.2 Results of [patient-relevant outcome] (available as dichotomous data) across the direct randomised trials (risk difference) 41
Table B6.3 Results of [patient-relevant outcome] (available as continuous data) across the direct randomised trials (end point) 42
Table B6.4 Results of [patient-relevant outcome] (available as continuous data) across the direct randomised trials (change) 42
Table B6.5 Results of [patient-relevant outcome] across the direct randomised trials (available as time-to-event data) 44
TableB8.1 Classification of the comparative effectiveness and safety of the proposed therapeutic medical service compared with its main comparator and guide to the suitable type of economic evaluation 47
Table C2.1 Assessment of treatment effect variation across subgroups 60
Table C2.2 Assessment of relative treatment effect variation across subgroups 61
Table C2.3 Assessment of absolute treatment effect variation across subgroups 62
Table C2.4 Summary of assessment of treatment effect variation across subgroups 63
Table C2.5 Reasons to exclude each direct randomised trial 67
Table C4.1 Summary of results of pre-modelling studies and their uses in the economic evaluation 70
Figure D1 Key information requests for assessment report Section D of a standard assessment for MSAC 72
TableD1.1 Classification of a service under MSAC consideration/Classification of the effectiveness of the proposed medical service over its main comparator and guide to the suitable type of economic evaluation 75
TableD1.2 Type of economic evaluation that should be presented for various classifications of a service under MSAC consideration 77
Table D2.1 Definitions for populations and circumstance of use that should be taken into account in the evaluation 80
Table D2.2 Comparison of characteristics of trial and requested populations and circumstances of use 82
TableD2.3 Example of a comparison of the characteristics of target, study and wider populations and settings 84
FigureD3.1 Example of the structure of a basic economic evaluation 87
TableD3.1 Key elements of the base-case economic evaluation 89
Table D4.1 List of health care resource items and unit costs included in the economic evaluation 97
Table D5.1 List of health care resource items and summary of cost impacts in the economic evaluation 102
Table D5.2 List of health care resource items and summary of cost impacts for each health state in a state transition model 102
Table D5.3 List of health states and summary of cost impacts included in the economic evaluation 102
Table D5.4 List of health states and summary of health outcomes included in the economic evaluation 103
Table D5.5 Assessment of the implications for the economic evaluation of applying the clinical evaluation (Step1 then Step2) 106
Table D5.6 Assessment of the implications for the economic evaluation of extrapolating and transforming the clinical evaluation (Step3) 107
Table D6.1 Analyses of the implications for the economic evaluation of usage beyond the requested populations and circumstances of use 109
Figure E1 Key information requests for assessment report Section E of a standard assessment for MSAC 111
TableE1.1 Categories of data sources 113
Figure B(i)1 Key information requests for assessment report Section B of a standard assessment for MSAC with clinical data from an indirect comparison of randomised trials 127
Table B(i)2.1 Trials (and associated reports) presented in the assessment report 130
Table B(i)2.2 Reasons to exclude each trial from the indirect comparison 131
Table B(i)2.3 Summary of randomised trials used to conduct the indirect comparison 132
Table B(i)6.1 Summary of results of the indirect comparison of randomised trials 134
Figure B(ii)1 Key information requests for assessment report SectionBof a standard assessment for MSAC with clinical data from non-randomised studies 137
Table B(ii)2.1 Studies (and associated reports) presented in the assessment report 139
Table B(ii)2.2 Reasons to exclude each study 140
Figure C(i)1 Key information requests for assessment report Section C of a standard assessment for MSAC with clinical data from an indirect comparison of randomised trials (SectionB(i)) or from non-randomised studies (Section B(ii)) 144
Figure D(i)1 Key information requests for assessment report Section D of a standard assessment for MSAC in which the therapeutic conclusion from assessment report Section B is non-inferior 148
TableA1.1 Factors that are more readily quantified 152
TableA1.2 Examples of factors that are less readily quantified 153
Table A2.1 Methods to collect and collate expert opinion 156
Contents iii
Contacts
The Medical Services Advisory Committee (MSAC) and its two sub-committees have secretariats within the Australian Government Department of Health.
Departmental Staff are available through the Health Technology Assessment (HTA) Team on the contact numbers and email below to discuss proposals for MSAC consideration or related matters. Any correspondence or assessment reports should also be lodged at via the address below. Staff within the HTA Team are also the first point of contact concerning the relevant committee or sub-committee’s discussions and decisions.
HTA Team
Australian Government Department of Health
MDP 851
GPO Box 9848
CANBERRA ACT 2601
Or courier
Sirius Building
23 Furzer Street