FDA announces reform plans

The Food and Drug Administration (FDA), citing a gap between investment in pharmaceutical research and new drug approvals, announced plans Tuesday (March 16) to reform the regulatory path to therapy development.The plans represent the agency's attempt to reduce the cost of developing drugs by using new diagnostic and imaging technologies to better design clinical trials. “We're not seeing the increases in new products that we expected based on all the advances in science,” said Janet Woodcock, the FDA's director of cross-center initiatives. “The professional staff at the agency is also concerned about how the new scientific knowledge is going to be worked up to apply to product development.”The FDA estimates that a new drug costs from $800,000 to $1.7 billion to bring to market, but most drugs do not work on most people, Woodcock told The Scientist. According to the FDA report, despite a growth in government and private investment, the number of new drugs with novel chemical structures has fallen from roughly 70 in 1993 to less than 30 in 2003. New biologics applications have fallen from just under 30 to just under 20 during the same time period. “The big story, I think, here, is that if you were to write a description of how drugs are developed in 2004, that really hasn't changed very much in the last 30 years,” said Alastair J.J. Wood, assistant vice chancellor for research at Vanderbilt University. “This is an early attempt on the FDA's part to make the case that maybe we should at least start a conversation on how we should change that,” said Wood, who was at one time considered a candidate for FDA commissioner.Reducing the cost of drug development, as proposed in the FDA's plan, could help open opportunities for smaller biotechs daunted by the huge costs, Wood told The Scientist. It would also give the pharmaceutical industry, under tremendous pressure to develop new drugs, a needed boost. Officials from both sectors welcomed the proposed changes. “The proposed FDA initiative is timely and significant,” Merck spokeswoman Anita Larsen told The Scientist. “The FDA is in a unique position to identify new processes, technologies, and best practices and to spearhead this improvement initiative.”Carl B. Feldman, president of the Biotechnology Industry Organization, in a written statement, applauded what he called a “courageous statement… recognizing the serious problems that are preventing new, innovative drugs and biologics from getting to the patients who need them.”The FDA report outlines three phases in drug development—ensuring safety, demonstrating efficacy, and manufacturing effective treatments—that may require reform. Tools for testing safety have changed little in the last few years, the report says. Trials to determine efficacy are a source of “innumerable failures,” and problems in designing, characterizing, and scaling up products “routinely derail” or delay development programs and keep needed treatments from patients.The report outlines no concrete plans for change, but lists “opportunities” to use new techniques and technologies. For example, the report cites “proteomic and toxico-genomic approaches” that could better test safety, and suggests they be used early in the drug-development process. The agency also advocates targeted toxicology research. “Take liver toxicity… it's one reason that drugs fail,” Woodcock said. “It's very rare… why does one in 10,000 fail?”The agency hopes that by improving safety testing tools, companies can identify patients who will most likely experience liver toxicity, exclude them from trials, and inform them of the dangers of taking the drug once they are on the market. “We can't make it safer by testing it,” Woodcock said. “We can only make it safer by getting information about who can take it.”In streamlining drug development, FDA hopes to work in tandem with the National Institutes of Health (NIH), and the report says the agency's work will complement the NIH Roadmap, a plan to invest an estimated $2.1 billion over the next 5 years to linking basic research with clinical benefits. The FDA also invites industry and advocacy officials to help develop a list of “critical path opportunities,” a kind of a task list for change.Woodcock said that she expects the first phase—gathering information for its task list—to be complete in 6 months and that the agency has already started internal “refocusing” to align its research with the new approach.Jamie Love, director of the Consumer Project on Technology, said that the most critical opportunity would be improving the financing of research and development so that drugs could get to people who need them but who cannot afford their high costs. Those costs have also prompted a flurry of realignments and mergers in the pharmaceutical industry. Merck's Larsen said that the company has already developed alliances with diagnostic technology or biotechnology companies that can streamline the development process. The pharmaceutical giant signed 10 such deals and alliances in 1996 and closed 46 of them in 2003, she said.Wood called the FDA report “an opening salvo” in a long “conversation” that could eventually be beneficial to both industry and patients. He stressed that it is important for the discussion to take place outside the FDA, because industry officials tend to treat the agency as many Soviet officials once regarded the Kremlin, trying to decode every word as if it was an important pronouncement. Still, the agency's willingness to change is a good sign, he told The Scientist: “The bottom line is that it's in everybody's interest to reduce the cost of drug development and to increase the likelihood of success.”

FDA announces reform plans

Agency hopes to reduce development costs by using diagnostic and imaging technologies

Interested in reading more?

Become a Member of