AAN Issues Position Statement on Principles for Novel Neurologic Therapeutics

The American Academy of Neurology (AAN) has issued a position statement outlining its approach for advising policymakers on therapy types with potential neurologic benefits but limited evidence or with no approved indication from the Food and Drug Administration (FDA). While not offering clinical recommendations, the statement emphasizes the need for high-quality evidence and patient–physician discussions about risks and benefits. The objective of the principles are to guide neurologists in decision-making when faced with novel or unapproved therapies, off-label use, newly recognized safety concerns, or self-directed patient treatments, with the goal of maintaining objectivity and patient safety in clinical practice.

The position statement is organized around 4 common scenarios in which neurologists may encounter uncertainty, as listed below. For each, the document defines the situation, outlines ethical and professional considerations, and specifies how neurologists should approach discussions with patients and decision-making. The overall purpose is to provide a consistent framework for weighing risks and benefits, ensuring informed patient engagement, and supporting evidence-based practice across these diverse situations.

Key principles in the statement include:

• Novel, non–FDA-approved therapeutics: Patients and neurologists should review available evidence and discuss potential risks and benefits; unproven therapies should not be recommended without supportive data.
• Off-label use: Supported when high-quality evidence indicates benefits outweigh risks, with shared decision-making.
• Post-approval safety concerns: AAN generally defers final positions pending FDA review; supports postapproval monitoring when appropriate.
• Self-treatment by patients: The AAN cautions against unproven therapies and advises patients to consult their neurologist before starting any new treatment.