Just when it seemed as though Duke would launch its trial of a controversial blood substitute, PolyHeme, a little problem arose. Duke's Institutional Review Board approved the PolyHeme study last year despite questions about whether the research had to be conducted on subjects without obtaining their consent (see "Bloody Mess," July 28, 2004). Under the trial parameters, PolyHeme would be administered to severe trauma victims--those too severely injured to sign away their rights--instead of the standard saline and typed blood. But a concerned citizen pointed out to state regulators that the trial violated North Carolina's Patient's Bill of Rights, which states that "informed consent must be obtained prior to actual participation in such a program." With no way to bypass the hurdle, Duke put the trial on hold in January.
Now the university, joined by UNC and Wake Forest, is seeking an amendment to the Patient's Bill of Rights that would permit a waiver of the absolute ban under special circumstances. Federal guidelines already permit such waivers, and the U.S. Food and Drug Administration had given the go-ahead to the PolyHeme study, which is currently under way at medical institutions elsewhere in the country.
The state Medical Care Commission considered several possible amendments last week and will probably make a decision in early March. On the table is a narrowly written version crafted by Duke that would allow the PolyHeme trial, as well as a broader version supported by the universities that would bring state law more in line with federal rules (and would also permit Duke to proceed with the PolyHeme trial).
Neither of those satisfies the concerns of Nancy King, a professor of social medicine at UNC who has opposed the PolyHeme trial. For one, the need for a stricter standard than what the feds provide has gained weight in the wake of the Vioxx scandal and other indications that the FDA is perhaps more interested in greasing the wheels for drug companies than in protecting the public health. Indeed, the agency's approval of the PolyHeme study's design was arguably outside the intent of the federal waiver rules that allow for exceptions only if "available treatments are unproven or unsatisfactory." Though called an "ambulance study" that was intended to test PolyHeme in the field where alternatives have proved inadequate, the proposed PolyHeme trial would permit the continued use of the product up to 12 hours after the patient reaches the hospital--where blood transfusions have for years provided a reliable if not perfect standard of treatment.
Supporters of the study at Duke and elsewhere have said that having to obtain consent from trauma victims for such a trial in the hospital setting would be next to impossible given their condition. But that claim is confounded by the fact that Duke was running a consent-based PolyHeme study on heart surgery patients only a few years ago--a study that was pulled by PolyHeme's manufacturer for reasons that have yet to be explained.
King and others are proposing an amendment that would allow for consent waivers only in minimal-risk trials (such as one involving medical records, for example). At the very least, they want any amendment to include provisions for an independent review of a proposed consent-waiver study in order to ensure adequate public disclosure and community participation.
According to King, the Medical Care Commission is taking great pains to look at all the angles and is not likely to act precipitously. One possibility is to put the amendment question on hold and ultimately provide a more thorough airing of the issues involved before reaching any conclusions.
Though it makes sense to move slowly and deliberately on proposed amendments to something as sweeping and important as the Patient's Bill of Rights, that may not be fast enough for Duke--the university is still hoping to join the PolyHeme study before other institutions eat up the available subject enrollment (which has reportedly reached about 30 percent of the goal). Why the university hasn't backed off at this point may be a function of how much money Duke will receive for its participation.
Asked that very question during the commission meetings, Duke officials never answered directly. But PolyHeme's manufacturer, Northfield Laboratories of Evansville, Ill., has plenty of cash to get the study finished as quickly as possible. The company recently reaped a $77 million bonanza in a stock sale based in part on speculation over the study results for PolyHeme, Northfield's only product.
Anyone interested in expressing views on the proposed amendment to the Patient's Bill of Rights may send an e-mail to Mercidee Benton at email@example.com. The deadline is Friday, Feb. 18. Writers should indicate that comments are related to proposed temporary rule 10A NCAC 13B .3302.