Generating comparative evidence on new drugs and devices before approval

Area of Science:

• Health economics
• Pharmaceutical policy
• Medical device regulation

Background:

• Limited comparative data exists for new drugs and medical devices at the time of regulatory approval in Europe and the USA.
• Existing active-comparator trials may not yield clinically or policy-relevant data.
• Expedited approval pathways in Europe and the USA can further reduce evidence generation.

Purpose of the Study:

• To propose five key principles for regulatory agencies and payers to incentivize pharmaceutical and device companies to generate robust comparative data.
• To ensure the timely availability of evidence that supports informed decision-making for patients, clinicians, and payers.
• To address the paucity of comparative evidence for both drugs and medical devices.

Main Methods:

• Proposing five key principles for regulatory bodies (e.g., European Medicines Agency, US Food and Drug Administration) and payers.
• Recommending enhanced labeling to inform about comparative data availability.
• Suggesting more selective use of expedited approval programs with incomplete data.
• Advocating for increased use of randomized trials with active comparators and network meta-analyses.
• Proposing payer-led incentives through pricing and payment decisions based on proven added benefit.

Main Results:

• The proposed principles aim to create incentives for generating comparative data on drugs and devices.
• Implementation could lead to more meaningful evidence for clinical practice and health policy.
• Ensures timely availability of useful comparative evidence for decision-making.

Conclusions:

• A multi-faceted approach involving regulators, payers, and clear labeling is crucial for improving comparative evidence generation.
• Regulatory agencies should be more judicious with expedited approvals and promote robust trial designs.
• Payers have a significant role in incentivizing evidence generation through economic policies.