Dr. Scott Gottlieb prepared this white paper for the Food and Drug Law Institute's Colloquium on Access to Unapproved Drugs, held on February 27, 2007.

Click here to read the complete white paper as an Adobe Acrobat PDF.

In the coming months, Johnson & Johnson is scheduled to unveil the results of a large, final stage trial of its new cancer drug Zarnestra. Some argue that this targeted medicine, part of a new class of cancer drugs called farnesyl transferase inhibitor, could have been available years ago for a group of cancer patients that remain poorly served by existing drugs. The results of J&J’s new study will come almost two years after the experimental medicine first came before the Food and Drug Administration for consideration for early or “accelerated” approval based on the results of a smaller clinical study that examined the drug for treatment of elderly patients with aggressive acute myeloid leukemia. AML is an often-deadly disease where there are few medical options for the frail. If the new results show the drug is very effective, it will be vindication to those who believe that this earlier trial showed the drug worked, but small solace to thousands of patients who have forgone treatment altogether over the last few years, or opted for less tolerable regimens, because Zarnestra was not available to them. . . .

Click here to read the complete white paper as an Adobe Acrobat PDF.

Scott Gottlieb, M.D., is a resident fellow at AEI.