NEW YORK, July 14 - In testimony before the Joint Economic Committee of the US Congress last week, FDA Commissioner Mark McClellan warned that innovative biomedical technologies such as genomics, proteomics, and bioinformatics may lead to an increase in the overall costs of healthcare, but noted that "the potential benefits of new treatments could grow even more dramatically."
McClellan noted that "genomically based drugs, and gene and tissue therapies based in genomic sciences, are making up a growing number of the new drugs entering clinical trials," in a statement delivered to the committee on July 9, and touted the promise of proteomics, gene expression studies, and bioinformatics to "increase the future potential for more effective, more targeted, even individualized medical treatments that can cure or at least slow or halt disease progression." However, he cautioned, "With these unprecedented technological achievements have also come unprecedented concerns about the total spending on healthcare and, in particular, about the rising spending on these new medical technologies."
Despite the mounting costs of the drug development process over the last decade, drug approvals have slowed down, McClellan said. The trend is "directly related to a decline in the number of new applications for drugs and biologics coming into the agency," he added. While genomics and other emerging technologies promise to fill the depleted drug development pipeline, McClellan estimated that an additional decade or more of increased R&D spending would be required before these technologies have a measurable impact on productivity. "At this point in genomics, for example, scientists are still primarily gathering information, sorting out patterns, and only starting to understand what the turning on or off of hundreds of genes by a new drug means for whether it is safe and effective in patients," he said. "So far, genomics has mainly added steps at the front end of the development process, through microarray testing of gene responses, and has not reduced the costs of clinical research significantly."
McClellan cautioned that the intense pressure on policymakers to make health care more affordable might cause them to focus only on reducing medical costs in the short run, which could reduce the necessary long-term investments in R&D.
"This combination of rising costs of product development and pressures to control costs rather than increase value is not a good one for keeping the United States at the forefront of biomedical innovation," McClellan said, "and more importantly it's not a good combination for affordable and high-quality, innovative health care for our population."
Calling for "policy solutions that both support innovation and make healthcare more affordable," McClellan provided an overview of the steps the FDA is taking to speed the translation of biomedical innovation into healthcare products. As an example, he noted, the agency is developing new guidance for emerging technology areas such as pharmacogenomics, where it has set up a "research exemption" program for product developers and academic experts to share data that could help predict clinical benefits and risks, thereby reducing the costs of demonstrating safety and effectiveness, he said.
McClellan also highlighted other FDA initiatives that have the goal of reducing the time and cost of product development, such as new performance measures for its review procedures, reforms in the generic drug approval process, and revised good manufacturing practices.
"Translating the new biomedical sciences into new kinds of treatments for patients requires major new investments, and it seems plausible that such investments may take many years to reach fruition," McClellan said. "But the fact remains that developers of biomedical products are not producing drugs particularly faster than they were before all these innovations came along. From a public health standpoint, with millions of Americans suffering from diseases that may be curable or at least manageable in the not too distant future, we cannot afford to wait many more years for all these investments to become valuable products."
The complete transcript of McClellan's testimony is available here.