The Efficacy Challenge
Drug companies have always had three customers-physicians, patients and regulators. But they've focused on the doc, a customer who's allowed them to align customer requirements with research capabilities. But drug companies are now ever more conscious of the other two customers--consumers and regulators. They're courting the former; and the latter are forcing themselves upon their notice. In consequence, and for a variety of reasons--not the least of which is the regulators' recognition of the facts of consumer-driven prescribing--drug development will need more than a tune-up. Companies will have to figure out how to dramatically improve the efficacy data on which their drugs are approved and marketed. One essential tool: new diagnostics for identifying which patients will benefit from specific therapies.
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The most encouraging aspect of the series of speeches the FDA's new commissioner, Mark McClellan, MD, PhD, has been giving is not what he says--we've heard most of this before--but to whom he says it: pharmaceutical investors and companies.
In conjunction with the initial sales numbers for its sepsis drug, Xigris, manufacturer Eli Lilly cautioned that "the selection of patients with severe sepsis as indicated on the label has been complex for physicians initially." To address the knowledge gap, the National Institutes of Health has awarded a $6 million grant for a study of risk factors for sepsis, the first study of its kind and the biggest single R1 grant ever funded through NIH's National Institute of General Medical Sciences. The Xigris pivotal trial was the only one of 31 Phase III trials of sepsis drugs that didn't fail. "It's not just understanding how the drug works, it's believing in it," says the principal investigator for the NIH study, which should help move the process forward.
FDA drug review times are up, and the number of approvals are down, with a wary investment community taking note and adding a new element to their earnings projections. The lack of a permanent FDA commissioner, understaffing, and the recent recalls/withdrawals of approved drugs are among the factors that may be leading to a slowdown. But whatever the significance of any shift in approval patterns over the last year or two, there are longer-term regulatory causes of concern for the drug industry--some attributable to the agency, but others stemming from the industry itself. With novel drugs taking up an increasing proportion of drug company pipelines, the FDA's staffing problems are compounded by having to evaluate therapies for which there are no reference points. The agency, having learned the hard way that it's extremely difficult to manage risk once a drug hits the market, will be particularly careful before passing on groundbreaking medications. And that tendency towards caution is only likely to become stronger as consumers become a powerful force in affecting prescribing patterns.