Health Science/Research PolicyGroup II

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

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

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

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

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)

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

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

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

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

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