FDA Issues Final Regulations on Expanded Access and Charging Patients for Investigational Drugs

By Kurt R. Karst –      

On August 12th, FDA announced the publication of final regulations concerning “Expanded Access to Investigational Drugs for Treatment Use” and “Charging for Investigational Drugs Under an Investigational New Drug Application.”  Both rules will be officially published in the August 13th Federal Register.  In addition, FDA launched a new website where patients and their health care professionals can learn about options for accessing investigational drugs. 

According to FDA, the “Expanded Access” final rule “clarifies existing regulations and adds new types of expanded access for treatment use.”  In particular:

Under the final rule, expanded access to investigational drugs for treatment use will be available to:

• individual patients, including in emergencies  
• intermediate-size patient populations  
• larger populations under a treatment protocol or treatment investigational new drug application (IND) 

It is intended to improve access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and who may benefit from such therapies. . . .

The “Charging Rule” revises FDA’s regulation, and will, according to FDA:

• clarify the circumstances under which charging for an investigational drug in a clinical trial is appropriate, 
• set forth criteria for charging for an investigational drug for the different types of expanded access for treatment use described in FDA's final rule on expanded access for treatment use of investigational drugs, and 
• clarify what costs can be recovered.

The rule permits charging for a broader range of investigational and expanded access uses than is explicitly permitted in current regulations.

Stay tuned for additional analysis of these new regulations . . . .