Our lead features this month illustrate the vital connection between public policy and business practice. FDA Commissioner
Margaret Hamburg shares her commitment to faster, more efficient approval of drugs and vaccines, while our annual analysis
of the development pipeline highlights the raw material FDA must work with to deliver on that promise. With Big Pharma depending
less on in-house science and more on licensed compounds built around complex partnerships with a financially imperiled biotech
sector, the stakes for enlightened regulation are higher than ever.
It is encouraging that Dr. Hamburg sees the advancement of public health as dependent on FDA doing a better job of balancing
safety concerns against clinical benefit. The distinction is a fine one. Safety is the ultimate confidence builder, and a
necessity for a viable drug delivery system. But medical progress will not be made unless regulation is also focused on incentivizing
a commercial response to unmet medical needs through targeted drug therapy.
The list of unmet needs is long. (See tk, page 58.) A survey by the European Center for Disease Prevention and Control found
that one in five intensive care physicians in Europe is now seeing patients whose symptoms are "totally or almost totally"
resistant to antibiotics. A September conference hosted by the EU concluded that escalating safety and trial evidence standards,
lower pricing and reimbursement, eroding intellectual property protection, and the sheer difficulty of the science itself
account for the fact that only two new classes of antibiotics have reached the market since the 1960s.
The fight against cancer may be the litmus test for how regulation and private investment work together to address the disease
burden. Cancer, primarily a heritable disease, can be fought most effectively with new diagnostic tools that enhance the performance
of drug therapy by identifying the genetic mutations that drive tumor growth. With one in three cancer patients terminal within
five years, finding the best armamentarium of drugs—from the start of treatment—is critical to survival.
To unlock the potential of new drugs and personalized medicine, FDA needs to set a specific regulatory pathway for approval
of "companion diagnostics." The agency must treat these new technologies as full complements to drug therapy. Another step
is helping industry manage the complexities of clinical trial design for advanced cancer drugs. If regulators aren't clear
about the most desirable end points, and if survey requirements continue to rely on population-based data that fail to reflect
the individual nature of the human genome, then an inconclusive study result is guaranteed. Such studies are actually a disincentive
to investment in clinical trials, compounded by the fact that most of the drugs under review will end up being prescribed
for only a small number of patients.
And Hamburg shouldn't forget the vital "back end" of innovation. Payment standards for cancer drugs (especially diagnostic
tools that facilitate cost-effective use by targeting patients most likely to benefit) must be adjusted to reflect this hidden
cost. This starts with modernizing coding rules for CMS that have existed largely unchanged for more than 40 years so that
new technologies can be recognized and rewarded. Clarity is also needed on a payment pathway for off-label use, a key driver
of next-stage innovation in cancer.
Hamburg promises to forge closer links between the FDA and CMS. Let's hope the dialogue focuses on how that relationship enhances
individual patient access to the best drug, at the right time, for an appropriately incentivized price based on a system-wide
value perspective. These are the mutually reinforcing markers for therapeutic outcomes that deliver lasting improvement in