Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee

350 Pages·2013·3.74 MB·English

Guidelines for preparing
submissions to the
Pharmaceutical Benefits
Advisory Committee
(Version 4.5)
July 2015
Pharmaceutical Benefits Advisory Committee
i

Guidelines for preparing
submissions to the
Pharmaceutical Benefits
Advisory Committee
(Version 4.5)
July 2015
Pharmaceutical Benefits Advisory Committee
iii
Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory
Committee version 4.5
Print ISBN: 978-1-74186-000-9
Online ISBN: 978-1-74186-001-6
Publications approval number: 10349
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this work in unaltered form for your own personal use or, if you are part of an organisation,
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reproduction for any commercial purpose and retain this copyright notice and all disclaimer
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to reproduce the whole or any part of this work in any way (electronic or otherwise) without first
being given the specific written permission from the Commonwealth to do so. Requests and
inquiries concerning reproduction and rights are to be sent to the Online, Services and External
Relations Branch, Department of Health, GPO Box 9848, Canberra ACT 2601, or via e-mail to
[email protected].
Technical writing assistance and informational design by Janet Salisbury and team,
Biotext, Canberra
Graphic design by Richard Stanford and Kirsten Duncan, Biotext, Canberra
Weblinks: Unless otherwise stated, URLs in this document were accessed on 30 June 2013
Contacts
Pharmaceutical Benefits Advisory Committee Secretariat
The Pharmaceutical Benefits Advisory Committee (PBAC) and its subcommittees have
secretariats within the Australian Government Department of Health:
PBAC Secretariat Section
Tel: [phone]
Fax: [phone]
Economics Sub-Committee (ESC) and Drug Utilisation Sub-Committee (DUSC)
Pharmaceutical Evaluation Section
Tel: [phone] (ESC Secretary)
[phone] (DUSC Secretary)
Fax: [phone]
Vaccines are considered by the Australian Technical Advisory Group on Immunisation
(ATAGI), which also has a secretariat within the Australian Government Department
of Health:
ATAGI: Immunisation Policy Section
Tel: [phone]
Fax: [phone]
The secretariats are available for discussion about proposed submissions or related
matters. They are also the first point of contact concerning the relevant committee’s
discussions and decisions.
Address for correspondence
The Secretary
Pharmaceutical Benefits Advisory Committee
GPO Box 9848
CANBERRA ACT 2601
Submissions should be delivered to:
Level 9
Sirius Building
23 Furzer Street
PHILLIP ACT 2606
Contacts v
Record of updates
Date Version Summary of changes
August 1990 1.0 Draft guidelines issued for comment
August 1992 2.0 Minor rearrangement, extension and clarification
November 1995 3.0 Clarification of technical aspects of measuring changes in costs and
outcomes
November 2002 3.1 Update to include minor changes endorsed since November 1995
November 2006 4.0 Major revision and reorganisation of text
December 2006 4.1 Version for co-publishing in HTML format, with excerpts in RTF
format, plus minor editorial corrections
December 2007 4.2 Version to provide clarification in Subsections A.1, A.5, B.7, C, D.5,
F.3, PT1 based on initial feedback on the major revision and to
update URLs
December 2008 4.3 Version to enable primary publication in pbs.gov.au website, update
URLs and correct a small number of typographical errors
July 2013 4.4 Version to make minor corrections and updates, and enable improved
information design and navigation, including development of a
designated website and online
July 2015 4.5 Version to make minor make minor updates to the requirements for
lodging submissions, including the requirement for submissions to be
lodged electronically on USB storage devices.
vi Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee
Contents
Contacts .......................................................................................................v
Record of updates ......................................................................................vi
Abbreviations .............................................................................................xv
About these guidelines .............................................................................................................xvii
PART I GENERAL INFORMATION ..............................................1
1 Role of the Pharmaceutical Benefits Advisory Committee ..............2
2 Types of submissions .......................................................................9
3 Rationale and basis for the economic evaluation ..........................34
4 Choice of information for inclusion in a major submission .............37
5 Lodging a major submission ..........................................................39
PART II Guidelines for preparing the main body of a
major submission .........................................................47
Submission executive summary ...............................................................48
Section A Context — Details of the proposed medicine and its main
comparator ..............................................................................................51
Introduction ...............................................................................................52
A.1 Requested PBS listing and pharmacological class and action ......54
A.2 Indications and requested restrictions ............................................56
A.3 Treatment details ............................................................................63
A.4 Main comparator .............................................................................65
A.5 Clinical management algorithms ....................................................68
A.6 Differences between the proposed medicine and the main
comparator .....................................................................................72
Section B Clinical evaluation — Systematic review of the clinical
evidence for the main indication ..........................................................73
Introduction ...............................................................................................74
B.1 Literature search (all submissions) ................................................76
Section B-DRT Guidance for preparing Section B based on direct randomised trials
(preferred) .................................................................................................79
B.2 Listing of all direct randomised trials ..............................................81
B.3 Assessment of the measures taken by investigators to
minimise bias in the direct randomised trials .................................89
B.4 Characteristics of the direct randomised trials ...............................93
B.5 Outcome measures and analysis of the direct randomised trials ..96
B.6 Systematic overview of the results of the direct randomised
trials ..............................................................................................101
B.7 Extended assessment of comparative harms ...............................110
Contents vii
B.8 Interpretation of the clinical evidence ............................................112
Section B-ICRT Guidance for preparing Section B based on an indirect comparison
of randomised trials .................................................................................115
B.2 Listing of all randomised trials considered for inclusion in an
indirect comparison .......................................................................117
B.3 Assessment of the measures taken by investigators to
minimise bias in the randomised trials included in the indirect
comparison ...................................................................................122
B.4 Characteristics of the randomised trials included in the
indirect comparison ......................................................................123
B.5 Outcome measures of the randomised trials included in the
indirect comparison ......................................................................124
B.6 Results of the indirect comparison ...............................................125
B.7 Extended assessment of comparative harms ..............................127
B.8 Interpretation of the clinical evidence ...........................................128
Section B-NRS Guidance for preparing Section B based on nonrandomised studies ...129
B.2 Listing of all nonrandomised studies ............................................131
B.3 Assessment of the measures taken by investigators to
minimise bias in the nonrandomised studies ...............................134
B.4 Characteristics of the nonrandomised studies .............................136
B.5 Outcome measures of the nonrandomised studies .....................137
B.6 Results of the comparison involving nonrandomised studies ......138
B.7 Extended assessment of comparative harms ..............................139
B.8 Interpretation of the clinical evidence ...........................................140
Section C Translation — Adapting the clinical evaluation to the listing
requested for inclusion in the economic evaluation ........................141
Introduction .............................................................................................142
C.1 Identification of issues to be addressed .......................................145
C.2 Focused analytical plan ................................................................151
C.3 Results of premodelling studies ...................................................164
C.4 Relationship of each premodelling study to the economic
evaluation .....................................................................................166
Section D Economic evaluation — Economic analysis of substituting the
proposed medicine for the main comparator ...................................169
Introduction .............................................................................................170
Section D-CEA Guidance for preparing Section D based on a cost-utility (preferred)
or other cost-effectiveness analysis ......................................................173
D.1 Overview of the economic evaluation ..........................................175
D.2 Population and circumstances of use reflected in the
economic evaluation .....................................................................179
D.3 Structure and rationale of the economic evaluation .....................181
viii Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee
D.4 Variables in the economic evaluation ...........................................186
D.5 Results of the economic evaluation .............................................194
D.6 Sensitivity analyses ......................................................................203
Section D-CMA Guidance for preparing Section D based on a cost-minimisation
approach ................................................................................................206
D.1 Estimation of the equi-effective doses .........................................208
D.2 Presentation of a cost-minimisation analysis or a cost analysis ...211
Section E Budgetary implications — Estimated extent of use and
financial estimates ...............................................................................213
Introduction .............................................................................................214
Section E-Epi Guidance for preparing Section E based on an epidemiological
approach .................................................................................................216
E.1 Justification of the selection of sources of data ...........................218
E.2 Estimation of use and costs of the proposed medicine ...............221
E.3 Estimation of changes in use and cost of other medicines ..........226
E.4 Estimated financial implications for the PBS/RPBS or the NIP ...229
E.5 Estimated financial implications for government health budgets .230
E.6 Identification, estimation and reduction of uncertainty .................234
Section E-MS Guidance for preparing Section E based on a market-share approach 238
E.1 Justification of the selection of sources of data ...........................241
E.2 Estimation of use and costs of the proposed medicine ...............242
E.3 Estimation of changes in use and cost of other medicines ..........244
E.4 Estimated financial implications for the PBS/RPBS or the NIP ...245
E.5 Estimated financial implications for government health budgets .246
E.6 Identification, estimation and reduction of uncertainty .................247
Section F Options to present additional relevant information .........................249
Introduction .............................................................................................250
F.1 Quality use of medicines ..............................................................252
F.2 Risk-sharing arrangements ..........................................................254
F.3 Other relevant factors ...................................................................257
PART III Information requests for specific product types .....261
Product type 1 Fixed combination products ...............................................................263
Introduction .............................................................................................264
PT1.1 Additional information requests to support listing of fixed
combination products .............................................................265
PT1.2 Additional information requests to support advice for
combination items under Subsection 101(4AC) ....................268
Contents iixx
Product type 2 Nutritional products .............................................................................271
Introduction .............................................................................................272
PT2.1 General requests for additional information ...........................273
PT2.2 Additional information for specific medical conditions ............277
Product type 3 Vaccine products ..................................................................................279
Introduction .............................................................................................280
PT3.1 Details of the proposed vaccine and its comparator
(Section A) ...............................................................................281
PT3.2 Clinical evaluation (Section B) ................................................286
PT3.3 Translation (Section C) ............................................................288
PT3.4 Economic evaluation (Section D) ............................................289
PT3.5 Budgetary implications (Section E) .........................................292
APPENDIXES .....................................................................................295
Appendix 1 Development of the guidelines ...............................................................296
Appendix 2 Relevant factors influencing decision making by PBAC ........................298
Appendix 3 Improving the alignment between TGA registrations and PBS
restrictions...............................................................................................301
Appendix 4 Grandfathering ........................................................................................304
Appendix 5 Expert opinion .........................................................................................306
Appendix 6 Assessment of noninferiority ..................................................................310
Appendix 7 Utility valuation of health outcomes ........................................................313
Appendix 8 Monetary valuation of health outcomes .................................................328
Appendix 9 Including nonhealth care resources and nonhealth outcomes in a
supplementary analysis ..........................................................................334
Appendix 10 Developing utilisation and financial estimates........................................338
Appendix 11 Measures taken by the investigators to minimise bias in
nonrandomised studies ..........................................................................339
xx Guidelines for preparing submissions to the Pharmaceutical Benefits Advisory Committee