Overview

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

2. Overview • Shifting gears from enforcement • What enables off-label reimbursement? • Reimbursement planning checklist • Additional information

3. 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

4. What Enables Off-Label Reimbursement? • Use not matched to label • Cost neutral • Pressure from 6 Ps • Published evidence • Legal mandates • Technology assessment

5. 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

6. 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

7. Pressure From 6 Ps • Prescribers are the most influential • Pharmacists influence formulary drugs • Patientadvocates affect decisions • Plaintiffs trigger reconsideration • Politicians occasionally impact payers • Press coverage can help

8. Published Evidence • Compendia • First source for most payers • AHFS DI • USP DI • Peer-reviewed journals • Influential with larger, national payers

9. Legal Mandates • Medicare – cancer • Medicaid – all rebate drugs • State laws – primarily cancer, AIDS

10. Medicare • “Anticancer chemotherapeutic regimen” • “Medically accepted indication” means that the off-label use • Is included orapproved for inclusion in compendia, or • Carrier determines based on “supportive clinical evidence” in peer- reviewed pubs

11. 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

12. State Laws • 39 states (1999) • Typically cancer or HIV/AIDS • N/A to ERISA regulated plans

13. 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

14. Medicare Coverage Process • National coverage (or non-coverage) decision – binding on contractors OR • Contractor (Carriers, FIs, DMERCS) decisions at local level

15. National Process is Slow But Transparent • Request for coverage policy • MCAC recommendation (sometimes) • CAG staff decision • Publication www.cms.hhs.gov/coverage • Reconsideration (?!) -- See Ocular Photodynamic Therapy With Verteporfin 4/12/00 – 3/28/02 on website

16. Contractor Process Can Be Mysterious • LMRPs are published www.lmrp.net • See e.g. Noridian Neupogen/Leukine Policyeffective 6/01/02 • But informal, equally conclusive decisions are not published • Notice via claim denials • Contradictory outcomes for no apparent reason are common

17. BCBSA TEC • Triggered by request from member plan • Advisory, not binding • Non-BCBS insurers subscribe • www.bcbs.com/healthprofessionals/tec.html

18. 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

19. 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

20. Additional Information • July 2002 Literature Search: TAGLAWDC@aol.com

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