Evidence-based Formularies

1. Evidence-based Formularies Pete Penna, PharmD Formulary Resources, LLC

2. The Formulary Process: Who Decides Coverage? The P and T Committee or The �Pharmacy Business Committee� - P and T determines clinical merit - If equivalent, then business issues are the determining factor (A key issue is the capabilities of the �staff support pharmacist�)

4. Three Basic Questions: Is the drug under review more effective, equal to, or less effective than comparators?

5. Three Basic Questions: Is the drug under review safer, equal to or less safe than its comparators

6. Three Basic Questions: If equivalent to the comparators, which drug is the best overall �deal?�

7. Current Method of Evaluation Anecdote and bias Incomplete information Drug impact only (cost, rebate) Evidence based decision-making Total medical costs: cost offsets Total health impact Total value

8. Factors That Can Impact a Decision

9. The Environment: Current Limitations Often inadequate safety and efficacy data Evidence-based assessments difficult Lack of unpublished studies Lack of off-label use data Lack of QOL, functional status, and cost-effectiveness data Committee capabilities Excessive staff time required Typically, safety and efficacy data has been well researched and documented, but this information may still be inadequate, particularly in the case of drugs that are fast-tracked by the FDA. The vast majority of products lack a description of their effectiveness in actual practice. Accordingly, P&T committee members must either ignore the economics and outcomes implications of products under review, attempt to express efficacy data in terms of effectiveness, contract for their own PE studies to gain the necessary perspective. Typically, safety and efficacy data has been well researched and documented, but this information may still be inadequate, particularly in the case of drugs that are fast-tracked by the FDA. The vast majority of products lack a description of their effectiveness in actual practice. Accordingly, P&T committee members must either ignore the economics and outcomes implications of products under review, attempt to express efficacy data in terms of effectiveness, contract for their own PE studies to gain the necessary perspective.

10. Other Issues Status of decision making - increasing challenges to formularies Consumer demand and appeals Manufacturer pressure Foreign formulary processes FDA regulations and unsolicited requests AMCP Format for Formulary Submissions

11. What�s Needed to Make the Best Decisions? EBM expertise Appropriate committee membership Capable staff support Evidence A rigorous process Support of the organization

12. What�s Meant by �Evidence?� Randomized controlled trials Peer reviewed publications Data on file Anecdotal information Comparator information Economic information

13. Is the Evidence Relevant? Are studies (of any type) well designed and conducted? relevant study populations? Are head to head comparisons relevant to clinical practice? Are multiple measures of efficacy and effectiveness presented? Is focus on intermediate endpoints or final outcomes?

14. Is the Evidence Relevant? Are costs associated with introduction of the drug addressed? Is long term impact addressed (beyond clinical trial periods)? Is impact on sub-groups addressed? differing cost-effectiveness characteristics (age, sex, physiological response)

15. The Need for Improvement Given the current environment there appears to be a real need for change Given the diversity in evaluation processes, there is a substantial need for standardization

16. AMCP�s Format for Formulary Submissions

17. Content Product Information Supporting Clinical and Economic Information Modeling Report Product Value and Overall Cost Supporting Information

18. Product Information Product description - Approved Indications - Off label Uses - Pharmacology - Adverse Reactions and Interactions - Pharmacokinetics

19. Product Information (Cont.) Place in Therapy - Epidemiology - Pathophysiology - Approaches to Treatment - Alternative/Comparator Products - Place in Treatment - Expected Outcomes

20. Supporting Clinical and Economic Information Key Clinical and Economic Studies (Published or not) in evidence tables Disease Management Intervention Strategies Outcomes Studies and Economic Evaluation Supporting Data

21. Modeling Report To predict system-wide consequences of the drug Helps define the drug�s role in the MCO Should be relevant to the MCO, based on their - Costs - Demographics Overall, aids the decision-making process

22. Model Output Examples The cost to treat a case of AOM using drug A = $10.50, vs $59.00 for drug B We need to invest $3 million in bisphosphonates to prevent 1 fracture Using �Wonderstatin� for all lipid lowering will increase drug spend $0.75 pmpm, but will decrease TME by $2.50 pmpm.

23. Product Value and Overall Cost Develop a Value Argument Based on: Summary of all the data presented Expected unit cost Estimate of the expenditures for the drug Anticipated effects on clinical and HRQL outcomes Economic consequences for the MCO, members, and clients

24. Supporting Information References (Copies of publications or other documents) Spreadsheet models Submission check-sheet. Anything else that may be pertinent but does not fit elsewhere.

25. TYPICAL DOSSIER REVIEW

26. AMCP Format Perspective The Format is not the Answer to rational formulary or coverage decisions, but it is a major step in the right direction