1. Health Science/Research Policy Group II

2. Problem Statement Safety and effectiveness of prescription drugs in a real-world environment are uncertain because post-marketing surveillance is inadequate –Fails to capture data on bad outcomes –Limited drug-interaction data during a pre-market trial –Lack data on off-label usage –Subpopulation effectiveness is unknown –Don’t have comparative efficacy to already existing drugs

3. Rationale – Policy Protect the public safety; reduce harm associated with drug therapy Ensure optimal drug effectiveness Maintain public trust and confidence in the FDA –82% of adults think the FDA’s decisions were to a great extent or some extent influenced by politics rather than science

4. Rationale – Why Now? Recent scandals and federal investigations created a strong public awareness Public demand for greater drug safety –80% of adults are concerned about the FDA’s ability to make independent decisions –58% think the FDA does only a fair or poor job of ensuring the safety and efficacy of prescription drugs Media coverage PDUFA coming up for reauthorization

5. Stakeholders - Pro Consumer groups (e.g., Public Citizen, Families USA, AARP) Health plans and PBMs Disease-specific patient advocacy groups CMS AHRQ IOM Physicians State Medicaid programs

6. Stakeholders - Con PhRMA and individual drug manufacturers Other technology manufacturers (e.g., AdvaMed, MDMA, NEMA) Privacy Advocates? AMA? (reporting burden on physicians) AHA? (reporting burden on hospitals)

7. Plan of Action Improve post-market surveillance of drug safety and effectiveness, drug interactions, and off-label use –Computer tracking/ Health IT Demonstration project at VA and/or Kaiser State-level Medicaid demonstration project –Post-market clinical trials –Post-market limited clinical trials –Post-market drug registry –Mandatory adverse events reporting

8. Plan of Action Enforce the provisions of the FDAMA of 1997 requiring supplemental applications for approval of new (off-label) use Improve FDA enforcement of Phase IV clinical trials conduct and reporting

9. Plan of Action Build a coalition among stakeholders and other potential allies –Pro stakeholders –Quality improvement groups (e.g., IHI, Leapfrog, NQF) Create incentives for con stakeholders –Expedited coverage decisions –Potential protection against liability (tied to meeting the safety requirements) –Payment incentives to encourage enrollment in the registry

10. Plan of Action Link to PDUFA reauthorization in 2007 by entering into negotiations with the pharmaceutical industry in 2006 regarding: –User fee negotiation between FDA and Industry –Creation of incentives as described in previous slide