Cancer Drugs Have Worst Phase III Track Record

[Hopeful]

Elsevier Global Medical News. 2011 Feb 16, Sue Sutter, L Lamotta

Recent data on late-stage clinical success rates confirm that cancer drug development is a risky proposition for pharmaceutical and biotech companies, even before the Food and Drug Administration gets tougher on accelerated approval requirements.

A new study by BIO (Biotechnology Industry Organization) and the institutional research service firm BioMedTracker shows that oncology had the lowest phase III success rate among seven therapeutic areas, with only 34% of candidates succeeding at this stage over a 7-year period. The cardiovascular area had the next lowest phase III success rate (46%), and the autoimmune area had the highest success rate (63%).

Cancer also had the second lowest phase II success rate (29%), trailing only the cardiovascular sector by 1 percentage point.

The study examined the clinical phase status from year-end 2003 to year-end 2010 of more than 4,200 drugs and 7,300 indications in BioMedTracker's database. The study encompasses all companies - from big pharma to small biotech, both publicly and privately held - that are conducting development on therapeutics for approval in the United States.

A summary of the study findings was presented during a panel discussion on clinical trial success rates at the BIO CEO & Investor Conference in New York on Feb. 15. BIO said the full study would be published at a later date.

The overall success rate for all drugs and biologics from phase I to FDA approval was approximately 9%, with lead indications faring far better than secondary indications (14.5% vs. 3.2%, respectively).

Success rates in oncology tracked behind the overall numbers. Cancer therapeutics had an 11% overall success rate by lead indication, second worst only to the cardiovascular category. Only 2% of secondary indications for cancer therapeutics made it from phase I to approval, tying with the endocrine and respiratory therapeutic areas for the worst rate on this metric.
Infectious disease had the best overall success rate by lead indication (15%), whereas secondary indications had the best chance of success in the autoimmune sector (7%).

In a deeper look at the overall rates for oncology, treatments for head and neck cancer were most likely to succeed (19%), followed by renal cell carcinoma (15%). Treatments for non-small cell lung cancer had the worst clinical success rate, a dismal 2%.

"Strikingly, oncology drugs have the toughest time making their way through the clinic, despite [cancer's] being the most closely studied area in drug development," BioMedTracker senior biotechnology analyst Michael Hay said in a press release announcing the study results.

The study's findings come at a time when the FDA is contemplating tougher accelerated approval requirements for cancer treatments.

On Feb. 8, the agency's Oncologic Drugs Advisory Committee endorsed several FDA proposals for tightening the accelerated approval standards, recommending that sponsors generally be required to conduct randomized trials rather than single-arm studies, and that there be more extensive postmarketing studies to confirm clinical benefit.

The unattractive success rates in the therapeutic area, coupled with tougher regulatory hurdles, could give big pharma companies, biotech firms, and venture capitalists pause before they commit resources to those cancer indications with the highest failure rates.

Among the study limitations highlighted at the BIO meeting was its time frame; clinical programs that did not advance during the 7-year period and are still ongoing in a particular phase were not counted toward the success rate. The study also did not look at the reasons for failure, such as economic conditions vs. bad data.

Elsevier Global Medical News and "The Pink Sheet" are published by Elsevier.

Hopeful