Editor

Data presented at last week's annual meeting of the American Society of Clinical Oncology (ASCO) confirmed that more clinical trials focus on cancer than any other disease, sparking debate about how to control the costs of those trials and – ultimately – of the many new cancer drugs coming onto the market.

Duke University scientists reported that 22 percent of interventional studies registered on the clinicaltrials.gov database after September 2007 were focused on oncology – more than any other indication. A separate report from Citeline Inc. found that sponsors initiated 428 new mid-to-late-stage oncology trials in the 12 months between October 2009 and 2010 – again, more than any other disease.

The most popular indication for new late-stage oncology trials, according to Citeline, was non-small-cell lung cancer. Breast cancer came in second, but single studies looking at multiple hematological cancers came in third, and single studies looking at multiple solid tumor cancers came in sixth.

Phase I oncology trials often wrap multiple tumor types into a single study. But extending that approach into Phase II trials was twice as common in 2009-2010 as it was in 2008-2009, which may indicate drug companies are seeking more cost-effective ways to figure out where a drug really works and increase the chance of success in Phase III. Exelixis Inc. used the tactic with cancer drug cabozantinib (XL184), reporting data at ASCO from a single Phase II trial in 13 tumor types. (See BioWorld Today, June 7, 2011.)

Meanwhile, researchers from Paris-based Institut Gustave Roussy and the Mayo Clinic reported data at ASCO showing that traditional trial design strategies using large sample sizes to definitively establish superiority yielded smaller expected survival gains over a 15-year time horizon compared to trial design strategies using a smaller sample size and larger alpha. The findings prompted the researchers to conclude that the current risk-averse trial design strategy may slow drug development and deserves re-examination.

While it may be a stretch to expect the FDA to totally overhaul traditional clinical trial requirements, biotechs are increasingly looking to adaptive trial designs as a way to decrease sample size and save money. Oncolytics Biotech Inc. is using an adaptive design for its Phase III trial of oncolytic virus Reolysin, as is EpiCept Corp. for its Phase III trial of leukemia drug Ceplene (histamine dihydrochloride) and Anthera Pharmaceuticals Inc. for its Phase III trial of cardiovascular drug varespladib. (See BioWorld Insight, Nov. 2, 2009.)

But one of the most provocative ways to potentially decrease clinical trial costs was unveiled last week, not at ASCO, but by Pfizer Inc. According to the Wall Street Journal, the big pharma firm is conducting the first drug trial with no brick-and-mortar clinical trial sites. Patients will enroll online, receive study drugs through the mail, and report outcomes via a mobile phone app (although they will have to get lab work done either at a clinic or by a technician during a home visit).

The FDA has signed off on the trial, which will seek to validate the online-only approach by testing Pfizer's already-approved overactive bladder drug Detrol (tolterodine ER) against placebo and comparing the results to data gathered in a similar study in 2007.

While the all-electronic approach may be difficult to implement in cancer trials – particularly those in which very sick patients require frequent physician monitoring and radiological assessments – some of the approaches used in Pfizer's trial might turn out to be broadly applicable.

And that's a good thing, considering the dozens of posters and presentations at ASCO focused on the costs of cancer medicines. Some offered up value-based pricing models that incorporate survival benefit and a county's ability to pay, while others focused on specific drugs: researchers from the University of Miami and Johns Hopkins Singapore International Medical Center argued that adding Avastin (bevacizumab, Roche AG) to paclitaxel is not cost-effective in the treatment of metastatic breast cancer, while a study conducted by RTI Health Solutions and Novartis AG questioned the cost-benefit of Xgeva (denosumab, Amgen Inc.).

The take home message seemed to be that – regardless of how it happens – clinical trial costs have to come down.