Washington Editor

WASHINGTON - Pressure from Congress and the public to gain access to more efficacious, less expensive drugs and biologics has caused the FDA to rethink its role in the drug development process.

Earlier this year the agency introduced the critical path initiative, a project to create a new generation of performance standards and predictive tools aimed at providing better and quicker results on safety and effectiveness of investigational products. (See BioWorld Today, March 19, 2004, and April 16, 2004.)

FDA officials over the last several months have taken the podium at conferences in Washington looking to sell the plan as a positive step for drug companies. And on Tuesday, the agency's annual science forum kicked off here under the theme "The Critical Path, From Concept to Consumer."

Janet Woodcock, former director of the Center for Drug Evaluation and Research (CDER) who now serves as the agency's acting deputy commissioner for operations, said the FDA believes companies will get to market with fewer hurdles if they use the critical path once they come up with an idea for a drug.

Research and development funding has doubled over the past five years, yet fewer innovative drugs have reached the market, said panel member Donald Stanski, a scientific adviser to CDER and a professor of anesthesia at Stanford University. "What is happening in drug development is not sustainable," he said. "We are spending more but producing less, which is why Janet [Woodcock] and Mark McClellan [former FDA commissioner] have been leading us into the critical path."

Beyond spending and failure, Stanski said new drugs often don't work as well. "The success rate of many classes of medicines is about 25 percent," he said.

Indeed, solving the problems will not be an easy task, panel members agreed.

Woodcock said the agency's goal under the critical path initiative is to "get more innovative products to patients and to achieve robust product development pathways that are efficient and predictable."

She said drug developers need better evaluative tools for early stage research so they can select candidates that are predicted to have a high probability of safety, effectiveness and industry potential. In the biotechnology and pharmaceutical world, researchers experience a high failure rate in the early stages of discovery and development.

Drug failures likely would decrease with better predictive tools that could evaluate a compound's impact on liver cell function or test for gene therapy vector potency. Furthermore, Woodcock said the industry and agency need to design better trials to identify the right dose or regimen for drugs.

While the FDA believes it can improve its processes and procedures, others think the industry has room for improvement, as well.

Stanski charged that the industry has competed more than it has cooperated in efforts to improve drug development.

"I think the industry hasn't been providing leadership and development in terms of moving the next generation of drug development forward," he said, adding that drug discovery has blossomed over the years, but clinical development has lagged behind.

As for the critical path initiative, each year the agency expects to have new guidance on different diseases or indications. For example, if an obesity drug were the subject of a critical path, the FDA would provide descriptive guidance about what is expected in the clinical and regulatory process from concept to bedside. Also, the FDA expects to provide scientists, investors and companies with information on the types of diseases needing new treatments and on research grants available through the National Institutes of Health in Bethesda, Md.

The initiative also calls for the FDA to work with the industry, academia and other government agencies to develop the "Critical Path Opportunities List," which is designed to identify areas that would most benefit from a modernized path of medical product testing and manufacture.