The FDA Expands Access to Investigational New Drugs

James E. Hamilton — Fri, 30 Jul 2010 21:41:42 +0000

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:

Investigational New Drugs: The Patient’s Perspective

James E. Hamilton — Fri, 25 Jun 2010 18:21:57 +0000

In recent years, the FDA has expanded the use of investigational new drugs providing a lifeline to seriously ill patients who have exhausted all other options. Investigational new drugs are not yet approved for general use. However, they are under research by clinical investigators and practicing physicians using subjects who have given their informed consent to participate.

From the patient’s perspective, it is important to consider all of the facts. Patients should contact the manufacturer as well as the FDA for more information. ClinicalTrials.gov is a resource of information on expanded clinical studies. In addition, there are patient advocacy groups that provide assistance. The drug manufacturer, in addition to providing product information, can provide information about the cost of the new drug. It is important to note that most health insurance companies will not pay for investigational new drugs, no matter how seriously ill the patient may be.

There are regular news announcements of promising new drugs in the pipeline for various serious diseases and conditions. With the FDA’s expanded access and the increasing cooperation of the pharmaceutical manufacturers some patients might enjoy a more functional and perhaps extended life.

Part II – Investigational New Drugs: The FDA Expands Access

Helpful Links:

JE Hamilton & Associates

The FDA Expands Access to Investigational New Drugs

Investigational New Drugs: The Patient’s Perspective