Risk & Reward; FDA Expands Access to Investigational Drugs

By: Jordan Mummau, CIS Compliance Associate
jordanmummau@cis-partners.com

On August 12, 2009 the FDA issued two rules that are meant to bring clarity to the ways in which seriously ill patients can gain access to investigational drugs and biologics in cases where there may not be any other satisfactory treatments available. [1]

Unfortunately, some patients are faced with a serious illness that does not react positively to current treatment. Under these circumstances, a drug that shows promise and is under development can be very appealing to patients who have little other choice. The FDA had become aware of this in 1987 when it developed rules that would “allow patients to have access to investigational drugs under certain circumstances, even though the safety and effectiveness of the drug has not been fully established.” [2]

As previously stated, on August 12th the FDA instituted changes to the rules that were originally developed in 1987 so that the rules would apply to a broader population of people who have not had success with approved treatments, and who could have been previously denied access to investigational drugs and biologics. Furthermore, the FDA hopes these new rules will bring more clarity to physicians who are administering investigational drugs and the manufacturers who are developing them. This particular change was described in the Federal Register as a “change to the charging rule to explain when a drug manufacturer can charge a patient for an investigational drug, in a clinical trial or expanded access program, and what costs a manufacturer can recover when charging.” [3]

This could be a tremendous benefit to a sick patient who has run out of all other options. However, the FDA has referred to expanded access to investigational drugs as a “balancing act.” While the FDA understands a promising drug under development could potentially help someone in need, they also notice the risk associated with administering a drug that has not been fully tested. With that said, these new rulings ultimately give patients a choice that may not have been available to them before. The bottom line, per the FDA, is that the potential benefit must outweigh the potential risk. [4]

New Rules at a Glance
•Expanded Access Rule – to permit treatment of a patient with an investigational drug under an expanded access program
      oPotential benefit justifies potential risk
•Charging Rule – permits drug manufacturers to charge patients for an investigational drug in clinical trials or that’s being made available for expanded access. Charging in clinical trials will be allowed under very limited circumstances, but will be permitted for most expanded access uses.
      oMake process of obtaining authorization to charge more transparent
      oSpecify what costs can be recovered by drug manufacturers [5]



Sources:
1) "FDA Issues Final Rules to Help Patients Gain Access to Investigational Drugs." FDA US Food and Drug Administration. 12 Aug. 2009. Web. 1 Sept. 2009. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm176526.htm

2) "FDA Expands Access to Investigational Drugs." FDA US Food and Drug Administration. 12 Aug. 2009. Web. 1 Sept. 2009. http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm176845.htm

3) "FDA Expands Access to Investigational Drugs." FDA US Food and Drug Administration. 12 Aug. 2009. Web. 1 Sept. 2009. http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm176845.htm

4) "Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs." FDA US Food and Drug Administration. 12 Aug. 2009. Web. 1 Sept. 2009. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm172492.htm

5) "FDA Expands Access to Investigational Drugs." FDA US Food and Drug Administration. 12 Aug. 2009. Web. 1 Sept. 2009. http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm176845.htm