The US Food and Drug Administration (FDA) has proposed changes to the rules so that patients who are seriously ill and have no other treatment options can obtain “promising” experimental treatments and drugs more easily. The FDA wants access to experimental drugs to be broader, fairer, and less cumbersome than the current rules allow.

Since the 1970s the FDA has granted access to some experimental treatments in particular circumstances for compassionate use. For instance it has allowed emergency use of experimental drugs that have not yet gone to market in individual cases and for large groups of people who are very sick with HIV and AIDs, cancer and cardiovascular diseases.

The proposed changes should make it easier for emergency and non-emergency patients and healthcare professionals to get hold of a much wider range of experimental treatments. There is a delicate balance to be struck between risks and benefits, while at the same time ensuring that charges are reasonable for both the consumer and the manufacturer who needs to recover costs.

“Another equally important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market,” said Dr. Andrew C. von Eschenbach, Acting FDA Commissioner.

The FDA are not proposing to increase access to experimental drugs if other alternatives are available, or if the patient’s disease is not serious or immediately life-threatening. And neither will it allow experimental treatments that impede the drug’s development to go ahead.

Requests for use of experimental drugs will have to include clear evidence about the severity of the disease and the numbers of people involved.

The proposed changes will be available for public consultation for 90 days.

American Food and Drug Administration (FDA).

Written by: Catharine Paddock
Writer: Medical News Today