1. Clarifying the Regulatory Framework of Off-Label Usage Institute for International Research Washington, DC July 17, 2002 Reimbursement 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 P s  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 P s Prescribers are the most influential Pharmacists influence formulary drugs Patient advocates 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 or approved 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 1. Request for coverage policy 2. MCAC recommendation (sometimes) 3. CAG staff decision 4. Publication www.cms.hhs.gov/coverage 5. 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 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

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

21. 1875 Eye St., N.W. - Suite 900 Washington, DC 20006 USA 202.785.3800 TAGLAWDC@aol.com www.tag-associates.com