Trying to resolve a controversial, complicated, long-standing issue, the U.S. Food and Drug Administration (FDA) has proposed significant rule changes that would make experimental drugs more accessible to critically ill patients. According to the agency, the regulatory changes would offer “expanded access for experimental drugs … to individual patients, small patient groups, and larger populations under a treatment plan when there is no satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.”

“This proposed reform is carefully designed to balance several objectives,” said Dr. Andrew C. von Eschenbach, Acting FDA Commissioner. “One goal is to enable many more patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need for safeguarding the individual patient. Another equally important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market.”

At the core of the issue is a category of medications known as investigational new drugs, or INDs. These include drugs that are in development by pharmaceutical companies and that have begun to be clinically tested, but that haven’t yet won approval from the FDA. In most cases, INDs have not gone through adequate safety testing and therefore may pose unforeseen risks to patients who are using them. The new regulations would even allow access to experimental therapies that are still in “phase one” of testing; the large majority of drugs that reach phase one do not ultimately win FDA approval.

However, for seriously ill patients without any other treatment options, the risks involved often do not outweigh the potential benefits of drug treatment. In the past few decades, INDs have been considered successful in the treatment of ailments ranging from HIV/AIDS and cancer to heart disease.

It is a rather tricky balancing act for the FDA: resolving the need for rapid development of new therapies to treat critically ill patients with the desire for disciplined, scientifically rigorous safety studies. Two watchdog groups, the Washington Legal Foundation and the Abigail Alliance for Better Access to Developmental Drugs, brought a suit against the FDA in 2003 that seeks to broaden access to drugs not yet approved by the agency. The case is still in federal court and will be heard again sometime in 2007.

There are two main components to the proposed regulatory changes. The first deals with expanded access to investigational drugs. According to the FDA, this change would “clarify existing regulations and add new types of expanded access for treatment use. Under the proposal, expanded access to investigational drugs for treatment use would be available to individual patients, including in emergencies; intermediate-size patient populations; and larger populations under a treatment protocol or treatment investigational new drug application.”

The second rule change is related to how the drug companies can charge for the use of these drugs. The new rule would “clarify the circumstances in which charging for an investigational drug in a clinical trial is appropriate” and “clarify what costs can be recovered for an investigational drug.” The agency said that this change “is intended to permit charging for a broader range of investigational and expanded access uses than is explicitly permitted in current regulations.”

“FDA hopes this proposal will increase awareness in the healthcare community of the range of options available for obtaining experimental drugs for seriously ill patients,” said Dr. Janet Woodcock, FDA’s Deputy Commissioner for Operations. “By clarifying and streamlining the processes, FDA also hopes to encourage companies to make such drugs available and reduce barriers for healthcare practitioners in obtaining them.”

This entry was posted on Tuesday, December 12th, 2006 at 10:19 am and is filed under Health Concerns, Legal News.