WASHINGTON, April 14 (UPI) -- There has been a slowdown in the medical product pipelines that deliver new, cutting edge drugs and devices to the U.S. market, and the Food and Drug Administration thinks researchers need updated tools to fix the problem.
The agency has prepared a paper that proposes a collaborative approach to modernize research procedures that underscores the agency's desire to have less of an adversarial relationship with the pharmaceutical and medical device industries it regulates.
"The pharmaceutical and device companies are able to hire the best basic scientists in the world," said Dr. Janet Woodcock, director of FDA's Center for Drug Evaluation and Research and an author of the paper. "The disconnect is in the science that is needed to bring those products along."
The FDA recently issued a report, "Innovation or Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products," which said there is "growing concern that many of the new basic science discoveries that have been made in recent years may not quickly yield more effective, affordable, and safe medical products for patients."
FDA officials think the current product development route is inefficient and too costly -- putting the country at risk of not being able to afford future research that translates into the "gee whiz" breakthroughs for which the industry is known.
"In FDA's view, the applied sciences needed for medical product development have not kept pace with the tremendous advances in the basic sciences," the report said. "The new science is not being used to guide the technology development process in the same way that it is accelerating the technology discovery process."
FDA wants to combine new performance standards with the latest in bioinformatics, genomics, imaging technologies and materials science to steer discoveries down a critical pathway. It wants the industry to be able to evaluate a discovery's potential success, safety and efficacy more effectively and quickly send the most promising to regulatory approval and production stages.
To do that, researchers must be able to use the newest in assessment technology, include pharmacogenomics in drug development, stem cells, cellular-based and bioengineered products to increase the likelihood of success during clinical trials, and new imaging technologies.
"We are looking now at imaging ... we saw for some products you can image where they go and what organ they are in and how long they stay in the body," Woodcock said. "We issued a guidance for using pharmacogenetics in drug development to predict who is at risk of side effects."
FDA wants industry to use pharmacostatistical, model-based drug development to gauge safety and efficacy based on preclinical and clinical data.
Woodcock said, as an example, a model of Alzheimer's disease allows researchers to see how it progresses. Combine that with a model of how well a drug might work and then ask the computer to factor in variability to predict how big a clinical trial is needed for a specific treatment.
"It's not like an art -- it's more like a science of what a disease looks like," she said.
Dr. Alan Goldhammer, associate vice president for regulatory affairs for the Pharmaceutical Research and Manufacturers of America, told UPI using genetic information to develop new drug targets, predict drug safety and personalize or tailor medicines to specific groups of patients represents "a very different approach ... to what's been followed for the past 20 to 30 years."
"We're getting there -- we're on the road," he said. "We know a lot more than we knew 10 years ago."
PhRMA members invested $33.2 billion in 2003 in research and development. It can take more than a decade for a molecule to move from discovery to drug trials and the approval stage, and the total cost of that process can be $1 billion or more. In 2003, 35 new medicines were approved, including 21 drugs and 14 biologics.
Goldhammer said it is "very difficult to predict what's going to be a blockbuster" drug and sometimes a discovery "leads to an awful lot of excitement but for a variety of reasons does not translate into a product."
"Companies continually are making predictions and decisions on drugs, and we clearly know some of the areas where we need to focus and develop new therapies," he said. There still are other areas where the disease is known but there just isn't a good target to pursue for treating it, he added.
Christopher-Paul Milne, assistant director at Tufts University Center for the Study of Drug Development, told UPI he agreed, overall, with the FDA's approach but added other areas to consider.
Scientists are concerned about drug delivery system problems, such as drugs that are not formulated so that they absorb properly when taken orally. "That is an area I would focus on in the development stage," he said.
Another concern is interdisciplinary training of scientists to "understand genomics and modeling so they can apply that to government and academic, as well as to industry studies."
"You have to have the tools but you have to have the scientists to use the tools," he said.
Woodcock said there are no regulatory approval-process changes planned to coincide with the initiative but rather internal changes would be made that "allows the science to evolve."
One FDA priority in recent years has been getting drugs and devices approved more quickly and efficiently.
To that end, Goldhammer said FDA "has really tried to refocus its approach by doing more by using regulatory guidances ... rather than going through the rulemaking processes."
He said it wasn't clear whether the agency would have to make any regulatory changes to accommodate the new technology.
Milne said the FDA report and initiative represent the agency's growing comfort level with the technological advances. He said FDA, which has a history of being "tepid about changing their processes," must be willing to act on new ideas evolving from its initiative.
He said the agency and industry will have to become more comfortable using conditional approvals, in which products are put on the market based on these new predictors of clinical benefits with testing continuing after approval is given.
Woodcock said the agency is asking for industry to comment on its report and work toward developing projects based on its recommendations.
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