Jill Wechsler

The personalized medicine bandwagon is on a roll, offering a new model for calculating reimbursement of high-cost biotech therapies. Strategies for identifying patients who will respond to a certain therapy, as well as those most likely to suffer adverse events, promise to improve healthcare quality while eliminating waste and inappropriate spending. Interventions based on individual genetic characteristics may have limited sales, but support higher prices and less costly clinical research methods.

Congress postpones debate on follow-on biologics while adopting new policies likely to reshape drug development

Federal regulation of biologics began more than 100 years ago with the enactment of the Biologics Control Act of 1902. That act required licensing of establishments to manufacture and sell vaccines, sera, antitoxins, and other similar products in order to prevent deaths from contaminated vaccines, as had recently occurred with the diphtheria antitoxin.

The new legislation authorizes the use of fee revenues for drug safety oversight and assessment throughout a product's lifecycle.

The main testing and regulatory provisions of the FOB legislation reflect multiple trade-offs between the demands of innovators and generics firms.

Now equipment makers are designing new systems that can be deployed rapidly, are less costly to build, and require less water and power to operate.

FDA's approach involves adopting efficient strategies for targeting inspections to more high-risk operations likely to have the greatest impact on public health.

Following the US Senate approval of the FDA Revitalization Act (FDARA, S.1082) in May, the debate over drug safety and the reauthorization of the Prescription Drug User Fee Act (PDUFA) now moves to the House.

The information provided by analytical testing is important in determining whether additional clinical trails are necessary to bring a follow-on to market.

The challenge will be to design a system that is flexible, yet appropriate, for the broad range of biological products and the varying quality control capabilities of different manufacturers.

An underlying theme of FDA's drug safety program is that new discoveries in biomedical science can detect risk issues earlier in clinical development.

The Bush administration's spending plan for 2008 treats the US Food and Drug Administration (Rockville, MD, www.fda.org) fairly well, considering the cuts directed at Medicare, Medicaid, and children's health programs and flat funding for the National Institutes of Health (NIH).

A series of ICH guidances are encouraging industry to adopt quality-based approaches for achieving the "desired state" of drug and biotech manufacturing: more efficient and flexible operations that can reliably produce high quality therapies with less regulatory oversight.

FDA is encouraging broader use of pharmacogenomic data by offering manufacturers early informal advice.

One of the last acts of former Senate majority leader Bill Frist (R-Tenn) was to push through confirmation of Andrew von Eschenbach as the official head of the Food and Drug Administration (FDA).

The prospect of any cost savings has been fueling efforts to establish a pathway for follow-on biopharmaceuticals.

FDA is working more closely with foreign regulators to keep abreast of drug and vaccine quality issues in their regions.

There is growing support for new partnership arrangements that seek to expand drug research by reducing the financial risk for manufacturers.

FDA did not gain any real teeth for regulating unsafe and ineffective products until a national health disaster in 1937 roused a public outcry.

Food and Drug Administration is encouraging public–private collaborations to more fully explore the physical and chemical characteristics of nanoparticles.

Glavin is preparing a plan to modernize ORA through organizational changes . . .

The Food and Drug Administration recently unveiled its long-awaited Critical Path Opportunities List, which maps out a number of "scientific projects" for improving the testing and production of biotech therapies. In its March report, FDA recognizes that problems in the characterization, testing, and quality management of medical products can delay clinical trials and even completely block drug development.

RFID is currently the most advanced tracking technology, but manufacturers need to pursue a multilayered approach . . .

As part of its campaign to facilitate research on drugs and medical products, the Food and Drug Administration (FDA) recently issued new policies to encourage sponsors to conduct more informative and less costly early clinical trials. A new guidance on exploratory investigational drug applications (INDs) explains how scientists in industry and academia may test very small doses of a candidate compound to detect any pharmacologic effect before investing in more extensive in vitro and animal studies required for conventional phase 1 trials. The goal is to quickly identify products that show some promise of efficacy, and to halt research on those that fail to hit preliminary targets.

The Food and Drug Administration's Prescription Drug User Fee program (PDUFA) has to be reauthorized by Oct. 1, 2007, and all the interested parties are fine-tuning their wish lists for "improvements." Although some consumer advocates and their Congres-sional allies blast user fees for extending industry control over the drug approval process, FDA officials, pharma companies, and patient disease groups applaud the program's success in ending "drug lag" and speeding new drugs and biotech therapies to market.

Andrew von Eschenbach, acting commissioner of the Food and Drug Administration (FDA), says that implementing the Critical Path Initiative is "one of my highest priorities," and is encouraging more FDA collaboration with other government agencies, academia, and industrial partners to find better ways to encourage innovation. As head of the National Cancer Institute (NCI), Von Eschenbach has supported joint projects with FDA to spur development of new cancer drugs, including efforts to qualify biomarkers for cancer detection in specific patient populations and to examine how imaging technologies can monitor the impact of therapies on cancer tumors.

This quality approach to biotech manufacturing may also establish a pathway for approving generic versions of biotech therapies.

Facts@FDA...ultimately will contain current product labeling information plus safety data gleaned from adverse event reports.

Health programs seek to establish secure systems to manage the fast-expanding quantities of drugs and medical products going to developing countries.

The Senate confirmed Lester Crawford in July as the official head of the Food and Drug Administration (FDA). Crawford's nomination had been put on hold as various legislators pressured FDA to approve an over-the-counter version of the "morning-after" pill, Plan B, and to support broader drug importing.