1Cost-Effectiveness and HTA in The USApplications to Regenerative Medicine

3rd Health Technology Assessment International Symposium, University of Tokyo

Peter J. NeumannTufts Medical CenterSeptember 16, 2014Boston, MA

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Overview

• The changing health care landscape in the US• The role of cost-effectiveness analysis• Applications to regenerative medicine• Where are we going?

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The changing US health care landscape…

Insurance expansions/reforms

Payment and delivery reforms

Better information Health information technology Data systems Comparative effectiveness

research (PCORI)

The PCORI

The role of cost-effectiveness analysis

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(Intervention isless effective and

more costly)IncreasesCosts

Decrease in QALYs

DecreasesCosts

Increase in QALYs

(Intervention ismore effective and

less costly)

$

The Cost-Effectiveness Paradigm

Laupacis A. et al., Can Med Assoc J 1992;146:475

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www.cearegistry.org

The Tufts CEA Registry

The Cost/QALY Ratio

Costs

Quality-adjusted life years

CEA Registry ‐ a resource for:

Research

Decision making

Contents (through 2012)

3,772Cost‐utility analyses 

(CUAs)

>10,300Cost‐utility ratios

>14,200 Utility weights

Over 10,300 standardized

cost-utility ratios

CUAs by intervention type

0

100

200

300

400

500

600

# C

UA

s

Year

Other DiagnosticsMedical device Pharmaceutical

CUAs by country

US40%

UK17%

Canada7%

Netherlands6%

Sweden4%

Australia2%

Other24%

CUAs by disease

Cardiovascular17.3%

Cancer14.2%

Infectious10.6%

Endocrine6.6%

Musculoskeletal/ Rheumatologic

9.5%

Program/ Organizational

9.1%

Neuro-Psychiatric and Neurological

9.7%

Other23.5%

Contents

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Cost-effectiveness ratios Methods Utilities

• Intervention• Target population• Comparator• Cost data• Effectiveness data

• Perspective• Discounting• Time horizon• Sensitivity analysis• Study quality

• Health state• Elicitation method• Measurement scale• Sample population• Sample size

What is more cost-effective?

A. Diet and treatment for CHD reduction in 35–44-year-old non-smoking men with normal cholesterol

B. Diet and treatment for CHD reduction in 65–74-year-old non-smoking men with elevated cholesterol

What is more cost-effective?

A. Osteoperosis screening followed by alendronate in women aged 80-90 years

B. Osteoperosis screening followed by alendronate in women aged 60-70 years

Examples: cost saving interventionsCost-saving $0 $20,000/QALY

• Disease-modifying drugs for arthritis on symptom onset

• Gene testing to refine colorectal cancer treatment.

• Pharmacy-led education and self-management program for COPD

• Chemotherapy for low-risk bladder cancer

• Chest x-ray & sputum microscopy in TB suspects

• Abdominal aortic aneurysm screening

• Diabetes education program

•Peginterferon alfa + ribavirinin chronic hepatitis C

Examples

• Implantable cardioverterdefibrillator

• Clopidogrel plus aspirin in atrialfibrillation

• Valsartan for moderate hypertension

• Posterior laminectomy for spinal stenosis

• Monthly degarelix in metastatic prostate cancer

• Intensive nurse support for heart failure

$20,000/QALY $50,000 $100,000

• Methotrexate + Leflunomide for early RA

• Dasatinib in CML

• Universal microalbuminuriascreening every 2 years

$500,000

Examples of CEA use in the US

Advisory Committee Immunizations (ACIP) Public health insurance (e.g., Medicare) Private health insurance

Varied landscape Some use of CEA (e.g., Premera, AMCP dossiers) Formulary restrictions without CEA (e.g., Express

Scripts)

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August 28, 2014

Why isn’t cost-effectiveness analysis used more in the US?

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Key questions

• Mistrust of methods and motives

• Antagonism to explicit rationing

• Arguments that payers (and the government) should not get between doctors and patients

• ?

Joe Selby, MD, MPHExecutive Director of PCORI(December 2011)

“PCORI has no interest or intention to ever fund a cost-effectiveness study.”

Oct 14, 2010

Key ethical questions

• Discrimination against disabled persons?

• Does not give priority to the sickest or most vulnerable?

• The “fair chance” vs. “best outcome” dilemma

• Should small benefits to a large number of persons receive priority over large benefits to a small number?

Applications to Regenerative Medicine

CEA in Regenerative MedicineComparator

Regenerative Medicine

What costs to include?• Intervention• Staff• Facility• Other medical costs• Non-medical costs

What time frame?• Short term• Long term

What perspective?• Health Payer• Health System• Limited Societal• Full Societal

Is CEA of regenerative medicine different?

Methods What is patentable? One-time treatment Cure Value-based pricing Ethics

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Where are we going?31

Looking ahead• Intense interest in value

• But explicit use of CEA uncommon

• Instead, a focus:• Clinical evidence• Outcomes• Subgroups• Guidelines• Payment reform

• And much debate!

pneumann@tuftsmedicalcenter.org