In August 2009, the FDA clarified 20 year old guidelines governing access to investigational new drugs expanding availability to patients. Prior to the clarification, the FDA had been operating under loosely defined rules that limited patient access and public awareness.  The FDA’s aim was to increase public knowledge and availability of these drugs while ensuring the safety and well-being of patients. In addition, the new guidelines clarify when a manufacturer can charge for the use of the drug and what costs can be charged to the patient.

Under the Expanded Access rule, the FDA must be assured that:

• The patient’s condition has no suitable approved treatment
• Approved treatments tried have not been successful
• The possible benefit to the patient overrides the possible risks
• The expanded use of the unapproved drug will not hinder its development

The Expanded Access rule ensures that the patient safety is paramount and explains the difference between the types of access available.

Under the Charging Rule, the FDA allows manufacturers to charge the patient for certain expenses accrued with expanded access. The rule also allows fees for drugs used in clinical trials.

Additional information is available on the FDA’s website to patients and their providers about when and how to access investigational new drugs.

Part I – Investigational New Drugs: The Patient’s Perspective

Helpful Links:

• http://www.fda.gov
• http://www.phrma.org
• http://www.patientadvocate.org
• http://www.hospicepatients.org
• http://library.uchc.edu/departm/hnet/advocacy.html
• http://rarediseases.info.nih.gov/
• http://www.cms.gov