Every early phase trial begins with a series of predictions: that a new drug will show clinical utility down to road, that risks to study volunteers will be manageable, and perhaps, that patients in trials will benefit. Make a bad prediction here, and people potentially get hurt and resources wasted. So how good a job… Continue reading Tea Leaves: Predicting Risk and Benefit in Translation
Tag: risk
Age of Risk: Biologicals
Approving new drugs is a risky business. Despite best efforts (and frankly, some less than best efforts), newly approved drugs frequently turn out to have unexpected toxicities. One example is unexpected heart toxicity associated with the use of the common pain-killers like rofecoxib (i.e. Vioxx). Another is the surprising heart toxicity associated with the wonder… Continue reading Age of Risk: Biologicals
The Future of Pharmaceutical Regulation
The October 2008 issue of Nature Reviews–Drug Discovery contains a very informative perspective piece on how drug regulators negotiate uncertainty, risk, and benefit when making approval decisions (“Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma”). I have long argued that novel biologics like gene transfer will require… Continue reading The Future of Pharmaceutical Regulation
The Octopus of Reference Standards
When gene transfer researchers perform an experiment, how do they measure the dose of their vectors? For that matter, when investigators perform a study using any novel biologic, how have they characterized their agents? Few would think the questions are ethically significant. But consider the fact that research teams often use different techniques to determine… Continue reading The Octopus of Reference Standards
In the Dark?
Most cancer patients who enter phase 1 clinical trials are motivated by the prospect of controlling their cancer. Increasingly, however, such studies, in the words of one ethicist, “take without giving in return” by involving biopsy procedures in which tissue is collected before and during the study in order to gauge whether a new drug… Continue reading In the Dark?