New wine in old bottles.

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249 The practice of medicine could be utterly transformed in as little as a decade. Research pioneers are opening up vast new territories in targeted, nontoxic cancer treatment—monoclonal antibodies and the dazzling Gleevec have arrived. Other signal transduction inhibitors, antiangiogenesis agents, gene therapy are almost at hand. But these new discoveries provoke a fundamental question: Are we not “putting new wine in old bottles” by attempting to evaluate radically new therapies with a decades-old drug-development system? Viewed from the financial angle, the drug development process may be headed into peril. Much of the innovation springs from small biotechs that may have only one product. It can be daunting indeed for a small firm to face spending half a billion dollars and ten years of testing to bring that product to market. Facing these obstacles, small biotechs may choose not to develop products that could help patients. This may happen to Bexxar, which has had a rough ride on the approval treadmill. The high cost of drug development will exact a great price from cancer patients if we do not make changes in the present system. For many years to come the most advanced and promising treatments will be available to only a select few in clinical trials. Those trials will be designed not to treat, but to satisfy FDA requirements for product approval. Consequently, researchers will continue to test new therapies in diseases which have virtually no standard treatment, because less approval problems lie along that path. Other diseases, such as lymphomas, will be ignored because “viable” treatments are thought to be available, although few patients would consider chemotherapy “viable.” Consequently, patients will not have access to those new therapies under expanded access rules because their disease is not the one being studied. Another cost to patients is that new drugs are often tested as monotherapies, to deliver clear results for the FDA—not in combinations—where they’d almost certainly be more effective. How many new products will get to patients is a simple mathematical calculation: If the pace of discovery quickens, the pace of product approval must do so also. If it does not, many useful modalities will fall by the wayside—or won’t be discovered at all.