Experimental Drugs for the Desperately Ill: A Progress Report

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Experimental Drugs for the Desperately Ill: A Progress Report

Less than a year ago, FDA established a regulation that offers a way to provide promising -- but still experimental -- drugs to desperately ill patients.

The regulation is known as the "treatment IND" (for investigational new drug) rule. It is based on the premise that there are times when an experimental drug shows such promise -- especially for a life-threatening condition for which there is no other hope -- that it seems unacceptable to withhold it from desperate patients. (See "Experimental Drugs for the Desperately Ill" in the June 1987 FDA Consumer.) The regulation, which went into effect last June 22, makes it possible to bring such promising and important drugs to desperately ill patients much earlier than was previously the case.

As we approach the one-year mark of the treatment IND rule, I am pleased to provide this progress report on its success to date. I think we can be gratified that the regulation has already proven its worth by providing new treatment choices -- and potentially life-saving ones at that -- for many critically ill patients.

As this column went to press, FDA had approved three important treatment IND requests.

The first approval was given last October to the Massachussets Department of Public Health for use of a biological product in patients undergoing kidney transplants. FDA felt there was reason to believe that the product, called cytomegalovirus intravenous immune globulin (CMV-IVIG), could prevent potentially life-threatening illnesses in transplant patients whose immune systems are artificially suppressed to prevent rejection of the new kidney.

CMV-IVIG is made from human blood plasma that contains high levels of antibodies to the cytomegalovirus. About half the U.S. adult population has been exposed to CMV. The virus can remain dormant in these infected people and be transferred via a donated kidney to cause severe, uncontrolled infections in the transplant recipient's lungs, liver, eyes, and other organs. With a suppressed immune system, the transplant patient may be helpless to fight off this normally benign infection.

There are few matched donor kidneys available (those are the only ones for which immune suppression would not be necessary). Since many donors would have encountered CMV infection at some time in their lives, this potential exposure of transplant patients to serious infection can't usually be avoided. Because there is no currently approved therapy. CMV-IVIG may provide the only protection until the immune system can be allowed to return to normal.

A small percentage of patients who receive CMV-IVIG can be expected to suffer reactions similar to those experienced with other intravenous immune globulins. The reactions tend to be related to the rate at which the immune globulin is administered. Symptoms might include flushing, chills, muscle cramps, back pain, fever, nausea and vomiting. Also, there have been rare reactions similar to anaphylactic shock, with symptoms that include wheezing and a drop in blood pressure.

CMV-IVIG is in very short supply and is available only in Massachusetts and Maine, where the plasma used to prepare the product is collected. The producer is working to increase the supply in the coming months, with the goal of being able to distribute it throughout New England and eventually nationwide. Department officials estimate that as many as 3,500 patients across the country could benefit from the expanded use of CMV-IVIG annually.

FDA has agreed to allow the Massachusetts Department of Public Health to recover its costs of producing CMV-IVIG as provided for in the treatment IND rule. It is not yet certain whether the costs will be passed along to the patients or whether health insurance companies will reimburse these costs.

The second treatment IND, requested by the National Cancer Institute (NCI), was approved last December. …