Regulatory Beat: Multiple Issues Challenge Biotech Industry in 2007

Published on: 
BioPharm International, BioPharm International-01-01-2007, Volume 20, Issue 1
Pages: 40–45

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

Democrats are back on top in the Congress and are mapping a broad agenda for change. Prescription drug pricing, medical product safety, and access to treatments are high on the priority list. Manufacturers will be on the hot seat answering questions about patent policies, high-risk products, and why new biotech therapies cost so much. However, the real challenge will be gaining approval of a bill to reauthorize the Prescription Drug User Fee Act (PDUFA) before the program expires on September 30, 2007. Such legislation would renew user fees for medical devices and continue the pediatric drug exclusivity program.

Jill Wechsler

Reaching agreement on these measures in a short time frame will require the industry to negotiate with some highly critical Congressional leaders. Reps. John Dingell (D-Mich) and Henry Waxman (D-Ca), both key players in shaping the original user fee program 15 years ago, will be orchestrating the debate. Dingell now chairs the House Energy and Commerce Committee, which oversees the Food and Drug Administration (FDA) and health care programs. Waxman is the senior Democrat on that panel. He will also be investigating industry practices and FDA programs as chairman of the House Government Reform Committee.

The more narrowly divided Senate will adopt a collaborative approach. Senate Health, Education, Labor and Pensions Committee chairman Sen. Ted Kennedy (D-Mass) has cosponsored drug safety legislation with now ranking minority member Mike Enzi (R-Wyo). The two plan to work together on PDUFA reauthorization and related measures.



FDA officials and industry representatives have been discussing user fee options for several months with the goal of presenting an agreed-on user fee plan to initiate the debate. The new five-year proposal includes requested fees, timelines for application processing, and the range of FDA activities that fees will support, including a significant expansion in FDA postmarketing safety oversight. The agreement is expected to boost user fees considerably, raising the cost of filing a market application near the $1 million mark.

With Democrats and Republicans unlikely to agree on many legislative proposals this year, both sides will regard any PDUFA bill as a ready vehicle for enacting a broad range of healthcare issues. Measures to enhance drug safety, to speed biogeneric products to market, and to expand drug importing are all likely to be hung on the PDUFA Christmas tree.

"All these issues will be in play," acknowledges Jim Greenwood, president of the Biotechnology Industry Organization (BIO) and formerly a leading Republican on the House Energy & Commerce Committee. Industry's task is to identify a working majority in the House and Senate that can "deal with these issues responsibly," he says; with PDUFA, "we can't play veto games."


A top priority for Congressional leaders is to enact drug safety legislation. The Kennedy-Enzi bill calls for stronger controls on the use of high-risk therapies, a more powerful drug safety oversight board to resolve disputes, curbs on new drug advertising, disclosure of clinical trial results, and penalties for failure to complete postapproval studies—but not an independent drug safety office, as others advocate. Enzi and Kennedy held a hearing on the proposal in November 2006 to jump-start deliberations on this issue.

Drug safety problems often involve inadequate product quality control, a topic that FDA continues to address through its "Pharmaceutical cGMPs for the 21st Century" initiative. Since its final report on this program in September 2004, FDA's Council on Pharmaceutical Quality has headed up agency efforts to adopt a more risk-based approach to GMP inspections, manufacturing supplements, data submissions, and system documentation. FDA is holding a workshop in late February 2007 to review progress and to update industry on the quality initiatives being implemented. Congressional panels and the HHS Inspector General are interested in knowing how well manufacturers comply with GMPs.


FDA officials and industry executives worry that mandates for more safety data and curbs on drug uses will slow down new product development and approval. FDA's "Critical Path" initiative and the more recent "Opportunities List" aim to offset such trends by modernizing the drug development process to keep pace with advances in biomedical research.

Over the past year, FDA has been busy forming consortia to implement many of the 76 opportunity projects that could yield new tools for evaluating test therapies, streamlining clinical trials, and modernizing manufacturing methods.

FDA also is encouraging a shift to personalized medicine, based on pharmacogenomics data available to identify those individuals most likely to respond to a medicine or to experience serious side effects. Several consortia seek to validate new biomarkers and develop diagnostics and screening tests that can identify certain enzymes and receptors linked to response. FDA is encouraging broader use of pharmacogenomic data by offering manufacturers early informal advice on how to analyze and submit genomic data that can facilitate regulatory review. Scientists from FDA and industry seek to develop standards for submitting and validating such information.


Both biomedical innovation and product safety stand to benefit from a number of FDA e-data initiatives. A new bioinformatics board headed by deputy commissioner Janet Woodcock is overseeing the development of data and technology standards, including a standard computerized medical vocabulary. Priorities are to overhaul the agency's adverse-event reporting system and to establish an electronic system for listing drug products and manufacturing sites.

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 about prescription drugs up front where it can be easily accessed by health professionals as well as patients. These data are establishing an electronic prescribing information database, operated by the National Library of Medicine and readily available to the health community.


FDA will continue to play a critical role in efforts to guard against terrorist events and emerging health threats. The agency is facilitating development and approval of new countermeasures for anthrax, plague, radiation emergencies, and infectious diseases, while also ensuring the quality of vaccines and treatments for seasonal and pandemic influenza. Such high-profile assignments not surprisingly draw outside scrutiny: the HHS Inspector General is examining FDA's progress in assessing and inspecting vaccine manufacturing processes and new vaccine production technologies.


Campaigning Democrats called for lower drug prices and improved access to needed therapies, largely by repealing Medicare's "noninterference" clause. Changes are easier to promise than to implement though, and they raise the specter of price controls.

Although pharma and biotech companies may be more enthusiastic about the Democrats' promise to expand federal support for more stem cell research, added support for biomedical research won't do much good if attacks on prices and practices dry up financial investment in the biotech industry, says Greenwood of BIO. "We need to persuade the new leadership that when they're making speeches on stem cell research, they have to make sure that biotech companies will be there to develop the promised new cures," Greenwood says. "We need to protect the biotech goose laying the golden eggs."

Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD 20815, 301.656.4634,