clarifying the regulatory framework of off-label usage institute for international research...

Clarifying the Regulatory Framework of Off-Label UsageInstitute for International ResearchWashington, DCJuly 17, 2002

Reimbursement for Off-Label Uses

Overview

Shifting gears from enforcement

What enables off-label reimbursement?

Reimbursement planning checklist

Additional information

Shifting Gears from Enforcement to Reimbursement

Payer decisions are independent of FDA enforcement for same product

Unlike labeling and advertising, no legal limitations on off-label reimbursement But there are some helpful mandates

What Enables Off-Label Reimbursement?

Use not matched to label Cost neutral Pressure from 6 Ps Published evidence Legal mandates Technology assessment

Use Not Matched To Label

If there is no PA and the use is not “far off” enough to trigger software recognition E.g. SSRIs; oral antibiotics; some

cancer agents

Cost Neutral

Off-label reimbursement is almost always an economic issue Even when couched in clinical terms

If off-label use is drug cost neutral, reimbursement typically happens

Pressure From 6 Ps

Prescribers are the most influential Pharmacists influence formulary drugs Patient advocates affect decisions Plaintiffs trigger reconsideration Politicians occasionally impact payers Press coverage can help

Published Evidence

Compendia First source for most payers AHFS DI USP DI

Peer-reviewed journals Influential with larger, national payers

Legal Mandates

Medicare – cancer

Medicaid – all rebate drugs

State laws – primarily cancer, AIDS

Medicare “Anticancer chemotherapeutic

regimen”

“Medically accepted indication” means that the off-label use Is included or approved for inclusion in

compendia, or Carrier determines based on “supportive

clinical evidence” in peer- reviewed pubs

Medicaid

Rebate law requires coverage of off-label use if included or approved for inclusion in compendia

Not limited to cancer

No consideration of peer-reviewed pubs

State Laws

39 states (1999)

Typically cancer or HIV/AIDS

N/A to ERISA regulated plans

Off-Label Tech Assessments

All or nothing: Done once for product, rather than case-by-case

Tend to be clinically driven with cost undercurrent

E.g. - Medicare, BCBSA TEC

Medicare Coverage Process

National coverage (or non-coverage) decision – binding on contractors

OR

Contractor (Carriers, FIs, DMERCS) decisions at local level

National Process is Slow But Transparent

1. Request for coverage policy2. MCAC recommendation (sometimes)3. CAG staff decision4. Publication www.cms.hhs.gov/coverage

5. Reconsideration (?!) -- See Ocular Photodynamic Therapy With Verteporfin 4/12/00 – 3/28/02 on website

Contractor Process Can Be Mysterious LMRPs are published www.lmrp.net

See e.g. Noridian Neupogen/Leukine Policy effective 6/01/02

But informal, equally conclusive decisions are not published Notice via claim denials

Contradictory outcomes for no apparent reason are common

BCBSA TEC

Triggered by request from member plan

Advisory, not binding

Non-BCBS insurers subscribe

www.bcbs.com/healthprofessionals/tec.html

Devices

Closer scrutiny than drugs

Mfgr should expect that: Routine claims processing will identify

unlabeled uses All will be rejected unless supported

by solid published data

Reimbursement Planning Checklist Favored category (Ca; HIV/AIDS) Payer mix How far off (Dx; dose; route of

admin.) Treatment setting Prior authorized; case managed Formulary Cost – product, Rx budget, overall

Additional Information

July 2002 Literature Search:

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