Pricing and Reimbursement Trends in the US: Impact of Comparative Effectiveness Research and Cost-Effectiveness Analysis
Public healthcare costs are rising. Governments need to get them under control. In Europe, that means the systematic use of health technology assessments (HTA) and reference pricing. The US, on the other hand, focuses on comparative effectiveness research (CER) to identify drugs that maximise clinical efficacy, minimise clinical harm and are more in tune with individual needs. Vastly different solutions - but are they working?
Report Overview
In Pricing and Reimbursement Trends in the US: Impact of Comparative Effectiveness Research and Cost-Effectiveness Analysis, FirstWord Dossier lays bare the US model by defining comparative effectiveness research and exploring its limitations with regards to drug trials. The report expertly examines direct versus indirect cost effectiveness analysis, the role of key government institutions and managed care organisations and the impact such research is having on blockbuster drugs. Based on au courant research, expert interviews and several compelling case studies, the report takes a critical look at US policy decisions and trends and offers a clear, uncluttered view of the US drug reimbursement system.
Highlights
Key Report Features
“Where is the risk/benefit quantitative standardised analysis that goes hand-in-hand with the CER analysis? It should never just be about avoiding a safety event in the absence of having a discussion of what is the married benefit. What's the right ratio we're looking for, not just the lack of a serious adverse event or the production of a clinically beneficial event? I think it's both.”
-John Doyle, Senior Vice-President and Practice Lead, Managed Markets at Quintiles
“There’s a paradox of lots of CER data collected, but the companies don't know what to do with it. But they are getting better at finding the right data sources and understanding how to analyse it. There is going to be a tipping point, I believe, when the very purpose of comparative effectiveness will become clear.”
-Samuel Wagner, head of Health Economics, Oncology, for Bristol-Myers Squibb
“There are more similarities than differences, in terms of the US and Europe, regarding most of the key issues. Accordingly, it is time for cooperation between the industry, HTAs and payers. The adversarial tones that we can read about currently, do not lead to a good place - for anyone.”
-Alicia Granados, Senior Director Global HTA Strategy at Genzyme
Expert Views
Report Overview
In Pricing and Reimbursement Trends in the US: Impact of Comparative Effectiveness Research and Cost-Effectiveness Analysis, FirstWord Dossier lays bare the US model by defining comparative effectiveness research and exploring its limitations with regards to drug trials. The report expertly examines direct versus indirect cost effectiveness analysis, the role of key government institutions and managed care organisations and the impact such research is having on blockbuster drugs. Based on au courant research, expert interviews and several compelling case studies, the report takes a critical look at US policy decisions and trends and offers a clear, uncluttered view of the US drug reimbursement system.
Highlights
Key Report Features
- Wide-ranging analysis of US drug and reimbursement policies and how they differ from European models
- Review of the use of CER in the US, with particular attention to Medicaid and Medicare
- Up-to-date insight into the role of branded biologics and biosimilars in the US market
- Insight into cost-containing managed care tools
- Examination of the benefits and drawbacks of head-to-head trials and combination therapy
- Analysis of future CER proposals and their implications
- Expert insight from ten industry voices on the effect of CER and CEA on the US system of drug reimbursement
- In-depth examination of pertinent policy decisions and trends in the US pharmaceutical industry, explaining the utilisation of CER and direct versus indirect CEA
- Multiple case studies and comprehensive references to key literature
- How does the US now control prescription drugs costs and what roles do reimbursement and pricing play?
- How are US and European models similar and how do they differ?
- How do branded pharmaceutical cost considerations influence reimbursement decisions and policy at the federal level in the US, as government costs outpace government revenue?
- What are the implications of these trends for pharma and how will it respond?
- Global health economists
- Global HTA directors
- Managed care/market directors
- Pricing and reimbursement managers
- Marketing, brand and sales managers
- Business development executives
- Regulatory and government affairs professionals
“Where is the risk/benefit quantitative standardised analysis that goes hand-in-hand with the CER analysis? It should never just be about avoiding a safety event in the absence of having a discussion of what is the married benefit. What's the right ratio we're looking for, not just the lack of a serious adverse event or the production of a clinically beneficial event? I think it's both.”
-John Doyle, Senior Vice-President and Practice Lead, Managed Markets at Quintiles
“There’s a paradox of lots of CER data collected, but the companies don't know what to do with it. But they are getting better at finding the right data sources and understanding how to analyse it. There is going to be a tipping point, I believe, when the very purpose of comparative effectiveness will become clear.”
-Samuel Wagner, head of Health Economics, Oncology, for Bristol-Myers Squibb
“There are more similarities than differences, in terms of the US and Europe, regarding most of the key issues. Accordingly, it is time for cooperation between the industry, HTAs and payers. The adversarial tones that we can read about currently, do not lead to a good place - for anyone.”
-Alicia Granados, Senior Director Global HTA Strategy at Genzyme
Expert Views
- Leela Barham, MSc, Independent Health Economist
- Marc Botteman, Managing Partner, PharMerit
- John Doyle, DPH, Senior Vice President and Practice Lead Managed Markets, Quintiles
- William Gerth, MBA, Principal, WC Gerth & Associates
- Alicia Granados, MD, Senior Director Global HTA Strategy, GMA Genzyme
- Larry Gorkin, PhD, President, Gorkin & Cheddar Consulting
- Rob Nauman, Principal, BioPharma Advisors
- Samuel Wager, PhD, Executive Director, Bristol-Myers Squibb
- Jeff Waite, Pharmapricing.org
- Leslie Williams, MBA, President and CEO, ImmusanT
EXECUTIVE SUMMARY
INTRODUCTION
US SHARE OF GLOBAL BRANDED PHARMACEUTICALS SALES
Diminishing impact of US: Revenue for branded biologics
Tumour Necrosis Factor-Alpha (TNF-a) Inhibitors
USE OF CER IN THE US
Medicare
Medicaid
The Food and Drug Administration (FDA)
The FDA's response to GSK's Avandia in type 2 diabetes
New criteria for the approval of biosimilars
Managed care tools to restrain utilization and costs
The Managed Care Formulary
Prior Authorization
Open-label naturalistic studies: Focus on Medco's study of Plavix and Effient
Impact of the managed care tools
Disease indications that mitigate the impact of managed care
Oncology drugs
Amgen's Xgeva
Roche's Avastin
Orphan drugs and the lack of alternative treatments
Atypical antipsychotic agents via the Medicaid connection
Governmental agencies practicing CEA indirectly: Case studies
Age-related macular degeneration: Avastin versus Lucentis
Erbitux in advanced/metastatic colorectal cancer
FDA delays me-too with a Novel Mechanism of Action
Pros and cons of conducting head-to-head clinical trials
The combination therapy compromise
Implications of combination therapy on branded drug costs
Fixed-combination drugs
Future considerations
Contentious CER proposals
Cooperative CER Proposals
Potential renaissance in drug development
Implications of the renaissance in drug development
REFERENCES
ACKNOWLEDGEMENTS
APPENDIX 1
Use of CER by international governmental agencies
Health Technology Assessment: The UK's NICE
Reference pricing
Risk-sharing strategies
Risk sharing in the US
INTRODUCTION
US SHARE OF GLOBAL BRANDED PHARMACEUTICALS SALES
Diminishing impact of US: Revenue for branded biologics
Tumour Necrosis Factor-Alpha (TNF-a) Inhibitors
USE OF CER IN THE US
Medicare
Medicaid
The Food and Drug Administration (FDA)
The FDA's response to GSK's Avandia in type 2 diabetes
New criteria for the approval of biosimilars
Managed care tools to restrain utilization and costs
The Managed Care Formulary
Prior Authorization
Open-label naturalistic studies: Focus on Medco's study of Plavix and Effient
Impact of the managed care tools
Disease indications that mitigate the impact of managed care
Oncology drugs
Amgen's Xgeva
Roche's Avastin
Orphan drugs and the lack of alternative treatments
Atypical antipsychotic agents via the Medicaid connection
Governmental agencies practicing CEA indirectly: Case studies
Age-related macular degeneration: Avastin versus Lucentis
Erbitux in advanced/metastatic colorectal cancer
FDA delays me-too with a Novel Mechanism of Action
Pros and cons of conducting head-to-head clinical trials
The combination therapy compromise
Implications of combination therapy on branded drug costs
Fixed-combination drugs
Future considerations
Contentious CER proposals
Cooperative CER Proposals
Potential renaissance in drug development
Implications of the renaissance in drug development
REFERENCES
ACKNOWLEDGEMENTS
APPENDIX 1
Use of CER by international governmental agencies
Health Technology Assessment: The UK's NICE
Reference pricing
Risk-sharing strategies
Risk sharing in the US
