Christopher-Paul Milne, DVM, MPH, JD and Kenneth I. Kaitin, PhD
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In March 2004, FDA issued a provocative white paper: Innovation and Stagnation—Challenge and Opportunity on the Critical Path
to New Medical Products. The Critical Path was identified by FDA as those parts of the R&D continuum that constitute bottlenecks
in the drug development process. These bottlenecks are the cause of rising clinical costs, stagnating development times, decreasing
success rates, and inconsistent productivity. FDA acknowledges that there are inefficiencies in the current system for developing
new medicines. Because most sources of variability in human response are not understood and thus cannot be controlled for,
demonstrating medical utility is highly empirical. The degree of uncertainty inherent in current techniques can result in
the setting of overly conservative safety standards. FDA has provided the following three rationales to justify its initiative:
1) the FDA's mission is to protect the public health by promoting medical innovation; 2) the agency has a critical role in
setting standards, which in turn guide development programs in the industrial sector; and 3) the FDA is in the best position
to evaluate objectively what works well and what doesn't work well in the drug development process.
The FDA has called for an aggressive collaborative effort to create a new generation of performance standards and predictive
tools to improve the efficiency of the three development and review dimensions of the critical path: ensuring product safety;
demonstrating medical utility; and improving the industrialization process. FDA's Acting Commissioner refined the FDA role
as one in which FDA would take information developed by industry, NIH, and academia and formulate it into generalized principles
to guide development and review. FDA took the lead by announcing the development of a Critical Path Opportunities List, in
consultation with stakeholders, and internal changes to support high-priority critical path research efforts. FDA acknowledged
that it has little in the way of resources to address what will inevitably be a massive and coordinated undertaking to avoid
wasting time and money. 
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It has now been well over a year since the FDA released the Critical Path Initiative. In the intervening months, Vioxx was
withdrawn amid considerable media and congressional scrutiny. FDA resources were diverted to addressing whether its new drug
review standards were adequate, whether it had the capability to monitor spontaneous drug safety reports, and whether its
relationship with industry had become too "cozy" in the decade since passage of the Prescription Drug User Fee Act (PDUFA).
FDA's Critical Path initiative was back-burnered in order to address these and other issues. The Critical Path Opportunities
List, which was to be the showpiece on which FDA would demonstrate its commitment to the initiative, has now been delayed
at least six months. Understandably, industry buy-in has been lukewarm. They are on the outside looking-in and getting a very
fuzzy picture of the shape of things to come. FDA is sending a mixed message, and leading many to question, "Is the agency
willing to lead, or only cheerlead?"
It is worth noting that FDA has taken some small steps to date, by establishing a docket to collect comments from stakeholders,
forming several inter/intra-agency working groups, and cooperating on small projects with academia. FDA will have to manage
how best to turn confidential data into lessons learned, but it already does that at workshops and public meetings, as well
as when it develops guidances or responds to stakeholders' commentaries and queries. A bigger challenge for FDA, however,
will be to implement its grand plan for the way forward, which calls for the agency to specify the work needed, prioritize
opportunities, coordinate the Initiative, work on new tools and regulatory guidance, and update stakeholders to identify unmet
needs and opportunities. These tasks highlight the critical need for a point person at the agency to ensure that progress
is being made, and a formal partnership with industry and academia.
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