The Regulation Atmosphere

The pharmaceutical industry is cautiously optimistic that the new Bush administration and Congress will continue efforts to streamline the drug discovery and approval process. But the federal regulatory and licensing landscape will also be shaped this year by patent reform and gene therapy issues. In addition, two major pieces of legislation--one affecting drug approval times and the other market exclusivity--will either be phased out or reauthorized in the coming 18 months. "We really can't say much about where the [Food and Drug Administration] may be headed under the Bush administration" until a new commissioner is named, says Jeff Trewhitt, spokesperson for the Pharmaceutical Research and Manufacturers of America (PhRMA). But he believes the American public is "pretty comfortable" with current drug standards and procedures, and notes that the drug industry is not seeking major changes to the process. The industry is heartened by the Republican National Committee's party platform, which posits the "need to build on the strengths of the free market system ... and make sure there are incentives for the private sector to develop new and inexpensive drugs." Trewhitt comments that overall, the drug industry "has been pretty pleased with many of the provisions of FDAMA [Food and Drug Administration Modernization Act]."Passed in 1997, FDAMA contains 70 sections of law and mandated a sweeping overhaul of FDA's drug review and licensing procedures. Many provisions have been enacted, but others are still being implemented. Among other things, FDAMA included a five-year re-authorization of user fee payments to FDA by sponsoring companies seeking to reduce new drug approval times. 1 Prior to user fees, FDA approval of new molecular entities (NMEs), those drugs that are truly novel in composition, took about 30 months. But the average time had dropped to about 12 months in 1998 and 1999 to approve 30 and 35 NMEs, respectively. In calendar year 2000, however, approval time had jumped back up to a mean of 17.6 months for only 27 NMEs. "We're a little concerned" about this, says Trewhitt. "We're tying to determine if it was regulatory creep or just a one-year anomaly." (Priority or so-called "fast-track drugs"--intended to treat serious or life-threatening conditions--were not affected and continued to be approved in an average of six months.) The drug approval process, to which sponsoring companies have paid $137 million in user fees in fiscal year 2000, has saved the drug industry $2 billion annually by shaving drug development time, notes FDA commissioner Jane E. Henney. "It's clear that user fees have brought many benefits," she adds. But with the Prescription Drug User Fee Act [PDUFA] expiring in September 2002, congressional action will be needed to reauthorize it. Thus far, government and industry appear supportive. "The resources PDUFA provides are essential to the vitality of FDA's program assuring the safety and quality of the nation's prescription drugs," states former HHS secretary Donna E. Shalala. A bit closer in the regulatory pipeline is the pediatric exclusivity provision of FDAMA that grants an additional six months of marketing exclusivity to manufacturers who conduct clinical studies of drugs in children. The purpose of the legislation is to provide needed dosing and safety information to physicians in product labeling. But so far the law has failed to have the desired effect for at least three reasons: scientific and ethical problems inherent in pediatric testing, stringent FDA requirements, and a lack of economic incentives for older antibiotics and other drugs that lack patent or market exclusivity. The pediatric exclusivity provision expires on Jan. 1, 2002. The FDA and the pharmaceutical industry both would like to see it extended, but the FDA wants modifications that would provide additional incentives for clinical testing in the youngest age groups and for drugs that no longer have patent protection or exclusivity. Meanwhile, the U.S. Patent and Trademark Office (PTO) released new guidelines in January for patent applications on genes, gene fragments, and other "compositions of matter." These new standards make it more difficult to patent a gene or gene sequence and require the discoverer to describe in some detail the gene's "tangible" and "demonstrable" use. "We've raised the bar" for patenting genomic-related products, acknowledges John Doll, director of biotechnology for the PTO. "That's probably where the new guidelines will have the biggest impact." Previously, patents have been issued for incompletely sequenced genes or genes of unknown function. Many researchers in academia and industry have long criticized this practice, especially when patents were issued for expressed sequence tags (ESTs)--research tools principally used to assist in the discovery and identification of full-sequenced genes and gene function. The controversial new guidelines will face court challenges this year that should help define how they are to be applied in real cases. 2 According to Doll, the PTO is working closely with several biotech companies to develop test court cases. Through the appeals process, he says, "we can answer the difficult questions where applicants think they have a utility and we don't think they do." Finally, the Clinton administration in January proposed a new rule that would make publicly available information on all new or ongoing clinical trials involving either gene therapy or xenotransplantation. The rule grew out of concerns following the September 1999 death of 18-year-old Jesse Gelsinger, a volunteer in a gene therapy trial at the University of Pennsylvania. 3 Many industry and university researchers argue that existing government regulations are sufficient. "Gene therapy is subject to greater oversight than virtually any other medical technology," asserts Carl B. Feldbaum, president of the Biotechnology Industry Association. In announcing the proposed new rule, Commissioner Henney claims the new procedures pose "a remote, but unique risk" to those involved in the trials. "Our proposal will ensure that the public is fully informed as we investigate these new public health opportunities and challenges." Ted Agres (tagres@usa.net) is a freelance writer in Washington, D.C. References 1. S. Bunk, "FDA and industry improve cooperation," The Scientist, 14[5]:17, March 6, 2000. 2. See also, D. Steinberg, "Will genomics spoil gene ownership," The Scientist, 14[17]:1, Sept. 4, 2000. 3. P. Smaglik, "Gene therapy death may delay new trials," The Scientist, 13[22]:9, Nov. 8, 1999.

The Regulation Atmosphere

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Ted Agres

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March 2001

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