Wednesday, July 23, 2008

Stemming Medical Tourism (part 1)

The July 17 issue of Nature reports that a patient participating in a Vienna-based cell transfer study for urinary incontinence won a lawsuit against the University Hospital in Innsbruck for not being "told... the procedure was experimental."

The case was described in an earlier post in my blog (May 27, 2008: Bladder Trouble at the Frontier).  There are a number of cell transfer strategies that are being applied in clinics, despite absence of reliable data showing efficacy. This should worry conscientious investigators who are trying to shepherd this promising technology platform to clinical application.

Here's one example: Bankok based company TheraVitae offers cell transfer to advanced heart patients. In future posts, I will explore concerns about an emerging overseas industry in non-validated therapeutics that 1- involve cutting edge therapies, and 2-involve investors, and scientists, whose countries of origin have not yet licensed such therapies for clinical application. (photo credit: Olivander 2006)

Tuesday, July 22, 2008

Ethics, Phase 1 Trials, and the Developing World

Under what conditions is it ethical to perform phase 1, translational clinical trials in low and middle-income countries? Together with lead author Alex John London (Carnegie Mellon, Philosophy), I attempt to answer that question in the July 5 issue of Lancet ("Justice in Translation: From Bench to Bedside in the Developing World").

The article makes three main points. First, we note that much discussion about international research has centered on post-trial access to interventions (that is, should patients enrolled in trials be provided with free drug even after a trial is completed?) However, we argue that this question is irrelevant for phase 1 studies, which test safety rather than efficacy.

Second, we note that most major national and international ethics codes indicate that trials conducted in disadvantaged communities should be responsive to local health needs. We suggest that this is the appropriate standard for determining whether phase 1 trials are consistent with international standards of fairness for clinical research.

Third, we attempt to specify what international policies actually mean by responsiveness. We suggest that a trial is responsive if it addresses an urgent, umet health need of a host community, and if it is part of a process of developing knowledge that can reasonably be projected to be applied in a host community. (photo credit: VCU Tompkins-McCaw Library Collections, 2008)

Thursday, July 3, 2008

Are Phase 1 Volunteers Vulnerable?

In the January 2008 issue of Archives of Internal Medicine, an NIH team led by Christine Grady reports the results of a study (Participants in Phase 1 Oncology Research Trials: Are They Vulnerable?) surveying the demographics of patients who participate in phase 1 cancer studies. They report that these volunteers are overwhelmingly white, have pretty good physical functioning, earn above average incomes, have above average educational attainment, and do not lack for health insurance coverage. The authors then suggest that phase 1 volunteers "do not fit into any of the categories of vulnerable populations addressed by specific regulations."

This is an informative paper, though the results should not surprise anyone who has followed the field of phase 1 cancer research. Their use of National Cancer Institute (NCI) data is clever and comprehensive (though their anemic sample of 11 non-NCI studies is puzzling). And I agree with the tacit message: that regulators and ethicists should avoid infantalizing persons with terminal illness.

On the other hand, the article contains some misleading or debatable statements. The suggestion that phase 1 cancer volunteers "do not fit into any categories of vulnerable populations" is not exactly true: the Belmont Report implicitly categorizes the "very sick" as vulnerable. The World Health Organizations CIOMS policy states quite clearly: "Persons who have serious, potentially disabling or life-threatening diseases are highly vulnerable."

The view of vulnerability and autonomy espoused in this paper will strike many readers as thin and reductionistic– as if it were a box on a census form rather than an experience. So too is the suggestion that phase 1 study participants are not "in need of special protection." Nevertheless, this paper does help direct our concerns away from income and demography towardvariables that might really matter, like dependence, fear, and flux. (photo credit: Lutz-R.Frank 2007).