Preparing Health Technology Submissions for Pharmaceutical Products : Market Research Report

Preparing Health Technology Submissions for Pharmaceutical Products

Published: Apr. 1, 2006 - 146 Pages

Executive Summary
Background
Overview
Chapter 1: Global Formulary Submission Requirements: 1.1 Introduction; 1.2 Current formulary submission standards; 1.3 Hierarchy of clinical evidence; 1.4 Formulary recommendations and assignments; 1.5 The role of guidelines; 1.6 Linking cost-effectiveness and budget-impact claims; 1.7 Overview: managing patient populations; Notes
Chapter 2: Guidelines from a Global Perspective: 2.1 A global guideline overview; 2.2 Formulary submission guidelines: documentation and process; 2.3 Health technology assessments (HTAs) and the life cycle of a drug; 2.4 Disease area and therapeutic class reviews; 2.5 Bias and compliance; 2.6 Technology scoping; 2.7 The global dossier: meeting evidentiary and analytical standards; 2.2: Proposed outline for a global dossier; Notes
Chapter 3: Uncertainty - Net Benefits, Product Ranking and the Reference Case: Chapter outline; 3.1 Uncertainty in cost-effectiveness claims; 3.2 Ranking therapy interventions; 3.3 ICERs and net benefit measures; 3.4 Defining net benefits; 3.5 Interpreting ICERs; 3.6 Net monetary benefit; 3.7 Probabilistic sensitivity analysis; 3.8 Estimating cost-effectiveness acceptability curves; 3.9 Interpreting, monitoring and validating claims; 3.10 The NICE reference case; 3.11 Implications of the reference case requirements; 3.12 Overview: thresholds and evidentiary standards; Notes
Chapter 4: The Clinical Outcomes Case: 4.1 Literature searches; 4.1.1 Key databases; 4.1.2 Reference inclusion/exclusion criteria; 4.2 Bias and systematic reviews; 4.3 Hierarchies of clinical evidence; 4.4 Summarising clinical studies; 4.5 Quality-scoring clinical studies; 4.6 Pooled clinical data and meta-analyses; 4.7 Adverse events and side-effect profiles; 4.8 Defining comparator products 4; 4.9 Epidemiology; 4.10 Place of product in therapy; 4.11 Product profile; 4.12 Therapy intervention strategies; 4.13 Linking meta-analyses to modelled claims; 4.14 Monitoring and validating clinical claims; Notes
Chapter 5: The Health Economics Case I - Generating Modelled Cost-effectiveness Claims: 5.1 Types of modelled claim; 5.2 Decision-model frameworks; 5.3 Resource units and direct costs; 5.4 Valuing resource units; 5.5 Indirect costs; 5.6 Measuring outcomes; 5.7 Modelling, sensitivity and simulation analyses; 5.8 Spreadsheet models; 5.9 Monitoring and validating cost-outcome claims; 5.10 Meta-models; Notes
Chapter 6: The Health Economics Case II - Estimating System Impacts: Chapter outline; 6.1 Defining terms; 6.2 Forecasting product uptake; 6.3 Patient switching and target populations; 6.4 Budget-impact claims; 6.5 Estimated pharmacy budget impact; 6.6 Estimated medical budget impact; 6.7 Estimated total budget impact; Note
Chapter 7: Responding to Disease Area and Therapeutic Class Reviews: 7.1 Life-cycle product assessment; 7.2 Assessing claims; 7.3 Contractual requirements; 7.4 Experimental approaches: naturalistic trial designs; 7.5 Non-experimental designs; 7.6 Practice pattern variations; Notes
Chapter 8: Summary and Conclusions: 8.1 The future of technology appraisals; 8.2 Technology appraisals in the short term; 8.3 Technology appraisals in the longer term; Glossary
List of Figures: Figure 3.1 Benefit and willingness to pay; Figure 3.2 Cost-effectiveness plane; Figure 3.3 Net monetary benefit; Figure 3.4 Ranking net monetary benefits; Figure 3.5 Cost-effectiveness acceptability curve; Figure 3.6 Decision model: Therapy A versus Therapy B; Figure 3.7 Simulated distribution of differences in costs; Figure 3.8 Simulated distribution of differences in outcomes; Figure 3.9 Distribution of cost and outcome difference coordinates in the cost-effectiveness plane; Figure 3.10 Simulated cost-effectiveness acceptability curve
List of Tables: Table 2.1 Key formulary submission guidelines: documentation and process; Table 3.1 Parameter values: Therapies A, B and C; Table 3.2 Simulation pairs of cost and outcome differences; Table 3.3 Simulated proportion of coordinate cost and outcome difference by willingness-to-pay threshold; Table 4.1 Grading of clinical studies

Abstract

This essential report will help you:

Prepare a global dossier to ensure successful formulary listing
Understand formulary submission guidelines in Australia, UK and USA
Respond to emerging evidentiary and analytical standards
Structure your organisation to make a convincing case to the regulators

The rapid uptake of formulary submission guidelines in the last decade is forcing pharmaceutical manufacturers and biotechnology companies to comply with more rigorous evidentiary and analytical standards in clinical and cost-effectiveness evaluations.

Two developments in 2004 further transformed the situation:

The introduction of a revised health technology assessment guideline by the National Institute for Clinical Excellence (NICE) in the UK; and
The introduction of revised technology assessment guidelines for new products and for the re-evaluation of products by WellPoint Pharmacy Management (WPM) in the USA.

Where the NICE and WPM guidelines represent a major change in requirements with the need: (i) to accommodate adequately uncertainty in modelled cost-effectiveness claims and the requirement for a reference case (NICE); and (ii) the requirement for naturalistic, active comparator trials together with ongoing monitoring and validation of claims for cost-effectiveness and systems impact (WPM).

Put together, the NICE and WPM guidelines present formidable challenges to manufacturers in both the UK market and in the US managed care sector. If other health technology assessment and reimbursement gatekeepers adopt these new evidentiary and analytical standards, then manufacturers will have to rethink seriously not only how they adapt their clinical development programs to accommodate the reference case and active comparator requirements in Phase III trials, but the ways in which they present cost-effectiveness and system impact claims to meet monitoring and validation standards.

Preparing health technology submissions for pharmaceutical products - Meeting Formulary Submission Requirements for New Product Assessments and Disease Area and Therapeutic Class Reviews, considers how manufacturers should respond to emerging evidentiary and analytical standards as exemplified by the NICE and WPM guidelines for formulary submissions.

The report considers, in particular, the implications of the standards required in the NICE and WPM guidelines for manufacturers preparing reimbursement submissions and it goes beyond being simply a review of evidentiary and analytical standards required by reimbursement and pricing authorities that have mandated a formulary submission dossier as part of the technology assessment of new products, to establishing the standards required of a global dossier. Meeting the NICE and WPM requirements ensures that specific or targeted dossiers can be assembled to satisfy the requirements of other jurisdictions. A global dossier, therefore, if structured to meet the standards of NICE and WPM, will also meet the requirements of other jurisdictions - where individual formulary submissions are customized to meet the needs of individual health systems.

"a poorly constructed and self-serving case, where the modelled case has clearly been driven by the need to justify cost-effectiveness, seldom stands up to a critical review."

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Preparing Health Technology Submissions for Pharmaceutical Products

Table of Contents

Abstract