Washington Editor

Blood substitute companies and others developing emergency-use products might be facing new clinical study regulations.

The FDA is looking at the rule that allows for clinical emergency research when informed consent cannot be obtained. That regulation, 21 CFR 50.24, lets studies test experimental emergency treatments on patients with specific life-threatening medical conditions such as head trauma, cardiac arrest and stroke, even though neither patients nor their families are able to give informed consent. In those cases, patients in life-threatening situations have been enrolled in research studies before arriving at a hospital, sometimes receiving treatment while in an ambulance or in the emergency room.

Since the rule went into effect in 1996, the FDA has received about 60 requests to conduct clinical investigations, but "many fewer" have been conducted, said Sara Goldkind, a bioethicist at the FDA, during a conference call. The agency has issued a new draft guidance for therapeutic companies working in the field of emergency medicine, institutional review boards and clinical investigators.

"We've had 10 years since the publication of this regulation," said Janet Woodcock, the FDA's deputy commissioner for operations, "and we would like to have a public evaluation and discussion of the outcomes of this."

In its review of the emergency-research rule, the FDA is seeking input at a public hearing scheduled for Oct. 11 and through written comments on the draft guidance at http://www.fda.govdockets/ecomments. In its Federal Register announcement, the agency included a number of questions on the matter and identified challenges related to this type of research, such as consulting with representatives of the communities in which the studies will take place and from which the subjects will be drawn, public disclosure prior to initiation, as well as results after studies end.

The FDA's draft guidance better defines the oversight roles and responsibilities of sponsor companies, institutional review boards and clinical investigators than previous guidance, includes a section on in vitro diagnostics and "gives additional points to consider" on responsibilities around community consultation and public disclosure, Goldkind said.

Since adopting the regulation a decade ago, the FDA has reviewed concerns expressed by investigators, ethicists and other interested parties, in addition to issues raised in the literature and its own experiences. Regulators plan to use the information obtained at the public hearing to help develop strategies to address challenges that have been identified and will consider comments received on the draft guidance, as well as comments and suggestions received at the public hearing to determine whether the regulation's current framework is adequate or if modifications are needed.

It's always been planned this way, Goldkind said, adding that the agency "recognized that it would be critically important" to review the rule at some point after it went into effect. "We're at the point now where we feel that we have the body of information that we need to do so."

Any changes in the regulation could have an impact on biotech companies developing blood substitute products, also known as oxygen therapeutics, in addition to device firms external defibrillators and clot removal products.

The agency is carrying out its review as part of its Human Subject Protection and Bioresearch Monitoring Program, an ongoing effort to ensure scientific and ethical rigor in clinical research. More broadly, the initiative includes a series of new policy and regulatory developments aimed at strengthening its oversight and protection of study patients and the integrity of resulting data to facilitate the modernization of the regulation of clinical trials and bioresearch monitoring.