Food for thought.... Not sure what it means for a company that hangs out in phase II for 3-4 years and still only has 80 patients and 1.0 MM PII studies, but an interesting perspective none the less. I guess when you don't have robust funding support you could take the cost efficiencies of PII approach, but.....
NATURE MEDICINE | NEWS
Analysis of drug failures underscores value of robust phase 2 testing Roxanne PalmerNature Medicine 18, 477 (2012) doi:10.1038/nm0412-477bPublished online 05 April 2012
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In the realm of drug development, many pharmaceutical companies—and their shareholders—are especially eager to reach phase 3 clinical trials, where candidate drugs are tested for their ultimate effectiveness. But an analysis presented last month at a meeting of the New Jersey chapter of the Licensing Executive Society emphasizes that companies are wiser to spend more resources working on fine-tuning their therapy in phase 2, lest they falter just in sight of the finish line.
“It's not sexy to hang out in phase 2 for three to four years, but that may be best way to develop a drug,” says Lisa Natanson, an analyst at Deloitte Recap, the San Francisco area–based, biopharma-focused intelligence arm of the international consulting firm Deloitte Touche Tohmatsu.
Last year, Natanson and her colleagues combed through information in the Deloitte Recap database from 190 mostly US-based biomedical companies on 97 compounds originated in house that reached a final clinical outcome in the US within the past decade. The analysis revealed that the 64 approved drugs enrolled a median of 171 participants in each phase 2 program and conducted two phase 2 trials, on average. By comparison, the late-stage failures—which were terminated at the phase 3 stage or rejected by the US Food and Drug Administration for problems with efficacy or safety reasons—enrolled a median of 69 people per phase 2 program and conducted an average of only 1.2 such trials, according to the Deloitte report. Moreover, smaller phase 2 trials did not reduce the time it took to help companies reach a final result.
Steven Paul, who retired in 2009 as president of the Lilly Research Laboratories in Indianapolis, says that the Deloitte data are consistent with his experience. He explains that business attitudes focused on near-term results are partly to blame. A primary reason for premature advancement to phase 3, he says, is “wishful thinking.”
But rolling out larger phase 2 studies is easier said than done for cash-strapped startups. To that effect, John Arrowsmith, an advisor at Thomson Reuters Life Sciences Consulting in New York, suggests that small companies that might lack funding for large phase 2 trials can conduct a preliminary version to get an early indication of efficacy as a way to attract investor interest. “It isn't a case of all or nothing in phase 2. You can get a sniff of success by running an investigative trial rather than a full-blown phase 2 study,” Arrowsmith says. |
