Similarly, FDA is taking action against pharma companies that market drugs for purposes other than approved indications. Lawyers
claim that such regulatory actions violate the right to free speech and interfere with the practice of medicine. But patients
are suing manufacturers for adverse events related to off-label drug use, payers are refusing to reimburse unapproved treatments,
and FDA is challenging off-label drug use as unsafe and illegal. An FDA public workshop this month aims to encourage sponsors
to add new uses to their labels to avoid such actions.
The danger is that demands for safer, risk-free medicines may raise development costs and kill research programs. FDA's Critical
Path Initiative and subsequent Opportunities List aim to offset such trends by modernizing the drug development process to
keep pace with advances in biomedical science. Over the past year, FDA has been busy forming consortia to develop new tools
for evaluating test therapies, streamlining clinical trials, and modernizing manufacturing methods.
All these initiatives support a shift to personalized medicine based on pharmacogenomics data able to identify those individuals
most likely to respond to a medicine or to experience serious side effects. Although this represents a major departure from
the blockbuster-drug development model, targeted therapy has been championed by FDA and federal health officials, and manufacturers
are realizing that new treatments for broad patient populations may be hard to find. Pharma companies thus are supporting
efforts to identify new biomarkers and diagnostics that can identify certain enzymes and receptors linked to response. FDA
is encouraging broader use of pharmacogenomics data by offering sponsors early informal advice on how to analyze and submit
genomic data that later can facilitate regulatory review.
Because personalized medicine often combines drugs with diagnostic tests, this development may have broader impact on how
FDA regulates medical products. Von Eschenbach recently noted that the emergence of more combination products may require
more integration of policies and procedures governing oversight of drugs, biologics, and medical devices.
Both biomedical innovation and product safety stand to benefit from trans-FDA initiatives to expand electronic submissions
and on-line communication by the agency. A new Bioinformatics Board led by deputy commissioner of operations Janet Woodcock
is overseeing the development of data and technology standards to automate communication of important information. One priority
is to overhaul FDA's adverse-event reporting system. The group also is establishing a standard medical vocabulary and an electronic
system for listing drug products and manufacturing sites.
Last month, FDA held a public hearing on the feasibility of requiring drug manufacturers to submit regulatory documents electronically.
Such a mandate could apply initially to applications for conducting clinical trials and marketing new drugs, biologics, and
generic drugs; eventually, it could extend to adverse event reports and post-approval amendments.
These efforts build on FDA's requirement that pharma companies file drug-labeling information electronically, beginning with
newly approved drugs and those filing efficacy supplements. The rule also revises the content and format of the package insert
to provide the most important information up front, where it can be better understood and more easily accessed by health professionals
and patients. This data will build an e-prescribing information database at the National Library of Medicine, which will make
information on appropriate drug use readily available.
Jill Wechsler is Pharm Exec's Washington correspondent. She can be reached at firstname.lastname@example.org