Just Another Day at the Office?

10,000 drugs in the marketplace, 7,000 in development, and hundreds of applications to be reviewed each year: It's time to prioritize
By Kenneth I Kaitin and Christopher-Paul Milne

The priorities keep shifting, resources are static; staff is stretched thin, workplace tensions are thick; critics are many, friends are few. It's just another day at the office for the Food and Drug Administration.

Something has to change, but where to start? The FDA knows the answer: Prioritize. Development takes too long and costs too much because the processes, both regulatory and operational, haven't changed in 50 years. In response, the FDA launched the Critical Path initiative to establish this problem as a priority and has...

For some of these, the FDA must suggest potential funding sources for those interested in undertaking those opportunities, and for others, the FDA must identify institutions or organizations that could take stewardship of the opportunities, based on areas of expertise or previous government interactions. FDA funding for these activities could come from an increase in user fees, since the Critical Path will address premarket bottlenecks that could shorten both review and development times, ultimately benefiting drug sponsors (and patients).

To tackle postmarket concerns, the FDA must establish both a mechanism for ensuring appropriate justification and public health rationale for postapproval research requests, as well as a process for monitoring compliance by sponsors and evaluating research results. FDA should also establish an office of industry relations to ensure consistency of decision-making by review divisions.

A critical priority for the FDA should be to restore public confidence in the agency's accountability and integrity. This can be accomplished, in part, by increasing scrutiny and transparency for conflicts of interest. The FDA should have an external group evaluate the role played by outside experts in the agency's decision-making process. The agency should make the results publicly available, and when necessary, provide justification for instances where there is a need for industry expertise.

Something has to change but where to start? The FDA knows the answer: prioritize.

The FDA should also establish an office of patient advocacy to ensure balanced input from the multitude of patient perspectives, and to help depoliticize what should be a science-based public health mission. Some disease advocacy groups are well organized, and they effectively inform agency decision-making, while other patients may have little or no voice at the agency, thus exacerbating the problem of unmet medical needs. A case in point: Pain is the number-one reason that patients go to the doctor, yet there is little organized advocacy for pain research.

Kenneth Kaitin (left) and Christopher-Paul Milne (right) are director and assistant director, respectively, of the Tufts Center for the Study of Drug Development.

The FDA should also work to improve communication with doctors and patients, and to help educate them about the risk-benefit balance inherent in drug review and utilization. The office of patient advocacy, proposed above, could play an important role here. Patient advocacy organizations are adept at communicating with patients and health care practitioners, and an office of patient advocacy would provide the needed communication channel from FDA to the patient groups. Along those lines, the agency should figure out what patient- and prescriber-related factors may contribute to safety and efficacy problems that may have little to do with the basic pharmacologic properties of the drug.

Finally, the FDA must adapt to the rapidly changing environment for pharmaceutical and biotechnology innovation. First, the agency needs to establish a scientifically grounded and consistent process for the review and approval of biogenerics. Second, the FDA must not only continue its efforts to harmonize regulatory processes with EMEA, but it must also establish a harmonized approach to industry relations. This is especially important in light of the current trend toward multinational clinical trials and the near-simultaneous submission of regulatory dossiers in multiple markets. Third, as the research-based industry increases its focus on targeted and personalized medicines, and product type, process, and functional considerations become more important, the FDA will have to restructure its drug review division model, in ways that are likely to render current review practices obsolete.


Editor's note:The Scientist requested opinion pieces from two pharmaceutical companies - Millenium and Roche - describing their visions for the future of the FDA. Neither company would supply commentary that was specific enough to be published in the magazine, and both withdrew their pieces.

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