# Presentation: 197835

##  Evidence, HTA and Comparative Effectiveness in the U.S. Presentation at AMCP

**March 28, 2007**
**Peter J. Neumann**
**Tufts-New England Medical Center**

## Overview

- HTA around the world
- American exceptionalism?
- A new U.S. Center for Comparative Effectiveness?
- Looking ahead (implications for VA)

## HTA Around the World

## ISPOR MEMBERSHIP BY REGION 2006

## ISPOR Chapters

## American Exceptionalism?

## Explaining American exceptionalism in health care

- The values argument
- The “system” argument
- The political obstructionist argument

## The U.S. Landscape

- Medicare
- AHRQ
- Medicaid (the DERP)
- Regional efforts
- Other (VA!)

## Medicare spending trends

- Source: 2006 Medicare Trustees Report

**Total**

**Part A**

**Part B**

**Part D**

**Medicare Spending Per Beneficiary**

## A New Center for Comparative Effectiveness?

## Slide 12

## Activities of a Comparative Effectiveness Center

- Conducting and/or sponsoring research
- Setting research priorities
- Providing a forum for methodological and other issues

## Placement Options

- Within CMS
- Within AHRQ
- Within NIH
- Within FDA
- Creation of a new government agency
- Creation of a quasi-public organization

## Governance, Oversight, and Funding

- Institutional independence
- Separation of assessment from decision making
- Funding

## Substantive Issues

- Methodological rigor
- Analytic perspective
- Objectivity and independence
- Transparency
- Stakeholder input
- Appeals process
- Identifying research priorities
- Updating conclusions over time

## Implications

- For the collection of evidence
- For patient access
- For innovation
- For patient health

## Key issues

- Level of evidence required
- Tradeoff between rigor and timeliness
- The process for evaluation and decision making
- Role of CEA in HTA

## Will there be a role for cost-effectiveness?

## NICE in America?

## NICE Decisions by Cost-Effectiveness Threshold (1999-2003)

**< ****20**

**20****-30**

**>30**

**>30**

**>30**

**20****-30**

**20****-30**

**< ****20**

**< ****20**

**Notes:**
NICE has generally enabled to keep the cost per QALY below the 30,000 pounds

## Selected cost-effectiveness ratios for technologies covered by Medicare

- Implantable cardioverter defibrillators:  $30,000-$85,000/QALY
- Lung-volume reduction surgery:  $98,000-$330,000/QALY
- Left-ventricular assist devices: $500,000-$1.4 million/QALY
- PET for Alzheimer’s disease (Over $500,000)

- Source:  Neumann et al., 2005

## Why don’t Americans use  CEA?

- Conflicting/weak incentives
- Quality of evidence is problematic
- Regulatory/legal barriers
- Ethical concerns
- Lack of infrastructure
- Lack of cultural acceptance

## The role of the AMCP Format

## An audit of 115 dossier, 2002-2005

| Characteristics  | Total # of 
Observations | % Positive result (n) |
| --- | --- | --- |
| Statement on form of economic analysis (even if wrong) | 106 | 59% (62) |
| Statement of viewpoint of analysis | 106 | 38% (40) |
| Time horizon for costs and benefits stated | 106 | 78% (83) |
| Discounting if analysis 2 years or longer | 44 | 34% (15) |
| All assumptions are clearly stated
 | 106 | 20% (21) |
| Compared product to all relevant comparators
 | 106 | 37% (39) |

## Audit (page 2)

| Characteristics  | Total # of 
Obs. | % Positive results (n) |
| --- | --- | --- |
| Reports sensitivity analysis performed | 106 | 43% (46) |
| All conclusions follow from data reported  | 106 | 54% (57) |
| Conclusions accompanied by specific caveats | 106 | 18% (19) |
| Report mentions that comparators might be superior given changes in assumptions | 106 | 8% (8) |

## Six trends to watch...

- Rise of HTA organizations and evidence requirements
- Increasing parsing of clinical evidence
- Coverage with evidence development
- Cost-effectiveness requirements
- Flexible evidentiary standards
- Quantification of risks/benefits
- Adaptive clinical trials
- Linking evidence to payment