FDAnews Drug Daily Bulletin


Dec. 1, 2005

Safety concerns are setting up new barriers for drug indication expansions, according to a recent report by industry analyst Datamonitor.

"Recent high-profile drug safety concerns are also threatening to reduce the potential for companies to profit from off-label use of their products in an expanded patient population," Datamonitor analyst Adele Schulz said in the report.

This poses a problem because "some of the most successful forays of products into new indications in recent years have been driven by off-label usage," Schulz said. For example, the success of blockbuster cancer drug Rituxan (rituximab), which is jointly marketed by Genentech, Biogen Idec and Roche, largely stems from off-label prescribing to leukemia patients, Schulz said.

In the wake of recent drug safety scares such as the late-2004 withdrawal of painkiller Vioxx, the FDA plans to ramp up drug safety regulation with a number of reforms, including the Drug Watch website, which Schulz said "may have particular implications" for off-label prescribing because it will draw attention to "adverse events experienced during off-label use of products." The FDA may also "become more active in enforcing penalties for breaching of its guidelines to industry," Schulz added.

Meanwhile, the UK Parliament's Select Health Committee this year issued a report recommending that clinical trial results — one of the main ways drug companies encourage off-label drug use — be published in an "independent register" so as to "avoid publication bias," the Datamonitor report said.

Moreover, new U.S. and EU regulations have curbed brand drugmakers' ability to extend drug lifecycles through on-label indication expansion. The Medicare Modernization Act of 2003, for example, now limits brand drug companies to one 30-month stay of generic approval per product rather than multiple stays. In Europe, new regulations prevent drug companies from filing for new indications in different European markets to fend off generic competition, Schulz said.

In this new environment, drug firms should plan and implement indication expansions earlier than they do now, and look at indication expansion as a "mid-lifecycle ROI [return on investment] boosting strategy," rather than a "late-lifecycle generic defense strategy," the report said. Drugmakers will also switch from indication expansion to indication "targeting," Schulz said.