1.6. Emerging opportunities

The Critical Path initiative document describes many opportunities to improve the efficiency of product development. We will mention just a few here. On better tools to assess a compound's safety, FDA states the need for new techniques to evaluate drug livertoxicity, new methods to identify gene therapy risk, better predictors of human immune responses to foreign antigens, new methods to further enhance the safety of transplanted human tissues, and efficient protocols for qualifying biomaterials. On better tools to demonstrate the medical utility of a compound, FDA shares the agency's successfully experience with biomarkers in HIV infection and duodenal ulcer healing. FDA states the need for more biomarkers and surrogate markers that can guide product development. In addition, FDA discusses the need for better animal models to combat bioterrorism, more clinically relevant endpoints, better imaging technologies, more innovative designs and analysis methods, and implementing the concept of model-based drug development. The latter involves building mathematical and statistical characterization of the time course of the disease and the drug effect using available clinical data.

The Critical Path initiative document also discusses the need for better methods to characterize, standardize, control, and manufacture medical products on the commercialscale. Since manufacturing expenses could exceed the research and development investment, there is a need for a better validation process that follows the risk-based inspectionparadigm advocated by the FDA in recent months. The latter includes more attention to setting specifications and shifting from detailed data analysis to overall process qualityassessment. The same philosophy suggests moving toward acceptance of a probabilistic definition rather than a pass or fail on the manufacturing process. Most important, FDA wants to encourage the manufacturers to integrate state-of-the-art science and technologyinto their manufacturing processes.

Following the issuance of the Critical Path initiative document, different centers within the FDA have further identified areas for innovations and have presented opportunities to FDA's Science Board on November 5, 2004. Since May 2004, many workshops have directed at least part of their agenda towards more efficient and effective ways to test and develop new treatments. Common to many of the discussions are the needs to apply quantitative thinking and techniques. Take the clinical phase of product development as an example, a major emphasis is to use mathematical and statistical models to help guide drug development and approval. The central idea is to pool data from multiple trials to augment our knowledge base and actively incorporate such knowledge in subsequent studies. Interestingly enough, the concept of pooling data has now been extended to pooling data of drugs that belong to the same class. The pooling of information across companies, while challenging, will undoubtedly facilitate the collective learning of the pharmaceutical industry.

The opportunities for statisticians to make substantial contributions at the strategic level are beyond what one could have imagined 20 years ago. Along with the opportunities expectations that statisticians will help solve the puzzle faced by modern-day scientists in the pharmaceutical industry.