Investigational Drug Therapies – FDA Changes

Access to investigational therapies is a highly debated topic in the medical world.  Investigational therapy involves drugs that are being scientifically tested but not yet approved by the Food and Drug Administration (FDA). Often these drugs are offered through programs such as “compassionate use programs,” and “expanded access programs” to allow seriously ill patients access to new drugs before they have obtained FDA approval.

The processes of acquiring FDA approval can, in some instances, take up to ten years.  In the interim, because of where they are offered (typically in large cities), the associated costs (many insurance companies do not cover experimental treatments), and the strict eligibility requirements, most patients have limited access to these potentially life-saving therapies.

The FDA has realized the need for an overhaul.  In the past year, representatives from the FDA, the pharmaceutical industry and the medical profession have convened to re-interpret the rules and regulations of investigational therapies to form a new framework that allows greater access to more patients.

One such change centers on whether drug manufacturers can charge for the investigational trial drug.  Before, the regulations were unclear on what the manufacturer could charge and for what.  Under the new guidelines, the FDA has made it very clear that manufacturers can recover the cost of the product but can not make a profit.   The FDA hopes that with costs down, more people will seek out investigational therapies as a treatment option.

Additionally, the FDA is now requiring full-safety reporting for all patients in compassionate use programs, a requirement that exceeds the usual reporting protocol for serious adverse events.  While in theory this is good medical practice, companies could be resistant to this regulation since it would require more staff to follow up on patients, thereby raising a company’s bottom line, which could turn a company away from implementing compassionate use programs. 

While it is a positive development that the FDA has recognized the need for change, it is still a complicated process to gain access to investigational, but potentially life-saving therapies.  

To learn more about the new FDA guidelines and expanded access programs, please visit. http://www.medscape.com/viewarticle/711719.  You should also read the National Cancer Institute’s Fact Sheet on investigational drugs at:  http://tinyurl.com/ybmh4mk.