In an Associated Press story Monday about the creation of the Reagan-Udall Foundation, an FDA research institute funded by the drug and medical device industries, the Union of Concerned Scientists director Francesca Grifo voiced her concern. “Given FDA's track record in the past, I'm not confident in their ability to create something that is free of influence from industry," she said.

 

This statement was aimed at what is the bi-partisan crown jewel of current FDA reform legislation. Also known as “the Crtitical Path Foundation,” the institute was approved by Congress and signed into law late last month. It is charged with streamlining and improving the development of drugs and medical devices and will allow the FDA to work with both industry and academe to accelerate the nascent sciences of genomics and proteomics to help realize the very real potential of personalized medicine – a new kind of medicine capable of tailoring highly effective treatments against the underlying causes of specific diseases in individual patients – and perhaps even preventing those diseases from occurring or progressing in the first place.

According to Senator Ted Kennedy, D-Massachusettes, the Reagan-Udall Foundation "will make new research tools and techniques available to the entire research community, shortening the time it takes to develop new drugs and reducing costs for patients."

Just what track record is Ms. Gifo referring to anyway?

 

The hard truth is that many of the most dramatic scientific advances in basic research are difficult to translate into safe and effective medical treatments for patients. Isn’t this something that “concerned” scientists should be concerned about?

 

Despite the increase in R&D spending, the number of new innovative products being submitted to the FDA for approval is decreasing and the rate of failure is increasing. Almost half of potential drugs are failing in late-stage trials. This costs companies millions of extra dollars and is driving up the cost of successfully bringing a new drug to market. In 2003, researchers at Tufts Center for the Study of Drug Development estimated these costs to be $802 million, and some sources suggest that the total cost is closer to $1.7 billion.  

 

Twenty-five years ago, the success rate for a new drug used was about 14 percent. Today, a new medicinal compound entering early-stage testing – often after more than a decade of pre-clinical screening and evaluation – is estimated to have only an 8 percent chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product's success is even lower. 

 

Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process. 

 

New development tools in these areas will enable better through-put to commercial product development and will act as a productivity multiplier, increasing the returns on public and private investment in basic research. With improved scientific methods and a new, shared effort by all of us, we can develop and improve standards for product characterization and product safety testing, for both traditional and innovative products.

 

Today only about 1 percent of the proteins in blood have been identified. Of that 1 percent only a fifth have FDA approved diagnositic utility. These proteins, after we understand them, could help predict disease remission. Currently academics and private companies collect data and establish correlations, but no one is responsible for organizing this information into the broader knowledge that could lead to generalized principles industry and FDA could use for broader, faster, and more accurate product evaluation.

 

Think about the millions of dollars that would be saved by all types and sizes of companies and governments if publicly discussed and vetted biomarkers could be used and used predictably in the drug approval process. Using the lower end of the Tufts drug development number, a 10 percent improvement in predicting failure before clinical trials could save $100 million in development costs. Similarly, shifting 5 percent of clinical failures from late-stage to early-stage trials reduces out of pocket costs by $15-$20 million.

 

The Critical Path must be blazed in partnership. Regulators and industry, patient groups and legislators, FDA and European Agency for the Evaluation of Medicinal Products must work together to help bridge the widening canyon between bench and bedside. We are in the right place at the right time to make a real difference to global public health in the 21st century. One of the most pressing issues that confronts the FDA is learning how to better address and assist in medical product development. FDA needs to prepare today so the agency can efficiently evaluate the technologies of tomorrow.

 

The Union of Concerned Scientists thinks  the FDA has “an agenda” – and they’re right – it’s to protect and advance the public health.

 

 

The Journal of Life Sciences welcomes commentaries. To submit a piece, contact Daniel S. Levine at daniel.levine@tjols.com or 415-591-5449.