Pharmacoeconomic Analysis in R&D Decision Making

Introduction

This report gives in depth analysis of the ways in which the pharmaceutical industry can ensure that it collects sufficient data to support health technology assessments for new products and how it can apply economic analyses to support decision making during the R&D process.

Features and benefits

• Understand the importance of health technology assessment in reimbursement decisions and how to optimize data to get a positive outcome.
• Compare different methods that use health economics early in product development and can enable decisions to be made that will reduce R&D costs.
• Understand how trials can be designed to provide good evidence to support positive reimbursement decisions.
• Understand the impact of increasing interaction between the various stakeholders in the reimbursement decision making process.
• Discover the views of experts in the field of pharmacoeconomics including the Associate Director of the Scientific Advice Programme at NICE.

Highlights

• Pharma companies should think earlier about potential HTA requirements for any new drug. Understanding patient perspectives, existing practice, including treatment heterogeneity, unmet needs, and the costs associated with a condition will help to frame discussions of cost-effectiveness lbefore a new product comes to market.
• Phase III trials can be designed in such a way as to provide data that supports positive reimbursement decisions. Studies that provide evidence to support reimbursement will typically include a comparator, be longer, include a broader patient population, and realistic outcome measures.
• In the light of healthcare reform in the US, discussion of the costs – and cost-effectiveness – of treatment will be raised more and more frequently. The FDA and Centers for Medicaid and Medicare Services have announced their intention to work together to explore a parallel review process.

Table Of Contents

About the authors
Disclaimer

EXECUTIVE SUMMARY
Defining pharmacoeconomics
Pharmacoeconomic analysis to support decision making in drug development
Studies to support pricing and reimbursement
Future directions for value-based drug development
Defining pharmacoeconomics
Summary
Defining pharmacoeconomics
Health Technology Assessment
National authorities
Global collaboration for HTA
Assessing value
Pharmacoeconomics in drug development
Pharmacoeconomic analysis to support decision making in drug development
Summary
Introduction
The MATCH project
The Headroom Approach
The Health Economic (HE) Evaluation Tool
Bayesian decision theory and the value of information
Portfolio optimization
Conclusions
Studies to support pricing and reimbursement
Summary
Understanding pharmacoeconomics in early drug development
Cost-of-illness studies
Epidemiological studies
Patient perspectives
Measuring utility
Including health economics in Phase III study designs
Comparators
Study design
Endpoints
Post-approval studies and data analyses
Observational studies
Pragmatic clinical trials
Data synthesis to support HTA
Costs of comparative effectiveness research
Conclusion
Future directions for value-based drug development
Summary
Harmonization of HTA requirements across jurisdictions
Multi-stakeholder dialogue in Europe
The NICE Scientific Advice process
Multi-stakeholder dialogue
Cost-effectiveness and the US healthcare system
Private sector
Minimizing the gap between efficacy and effectiveness
Progressive approvals
Pharmacogenomics
Prescribers and patients
Global development teams
Future directions
Appendix
Research methodology
Acknowledgements
Abbreviations
Glossary
References

LIST OF TABLES

Table: Influential HTA and reimbursement authorities in different regions
Table: Potential benefits from innovative medicines
Table: Trials described in approval packages in the US and EU
Table: Possible Phase III study designs to support HTA
Table: Sample sizes for a 3-arm randomized controlled trial or an adaptive Phase III/IV trial
Table: Post-approval study types that support HTA

LIST OF FIGURES

Figure: Evidence required for regulatory approval and reimbursement decisions
Figure: Areas of potential benefit from innovative medicines
Figure: Definitions of the Quality-Adjusted Life-Year and the Incremental Cost-Effectiveness Ratio
Figure: Decision-making supported by early health economic analysis
Figure: Applying the Headroom method for a regenerative medicine for surgical treatment of bladder cancer
Figure: An iterative, Bayesian approach to integrating health economic data into the drug development lifecycle
Figure: Relationship between the size of Phase IIb and the probability of success in Phase III
Figure: Relationship between the size of Phase IIb and the product’s expected Net Present Value
Figure: Using health economic evaluation in drug development and post-approval
Figure: Designs of studies for efficacy and effectiveness
Figure: Adaptive Phase III/IV trial design
Figure: Variation acceptance of surrogate outcomes by disease and HTA body
Figure: Costs of different study types
Figure: Evaluation factors influencing reimbursement decisions in Australia and England
Figure: Functions required in Global Development Teams from the early stages of drug development