Safety & Efficacy

The clinical development process by which sponsors of new therapies gain FDA licensure is estimated to take from five to eight years, and require literally thousands of pages of documentation. Following submission, unless sponsors received so-called "Fast Track" review, FDA's process for review and approval can take from six to ten months.

There is considerable momentum for accelerating the process, some of it from the new FDA Commissioner Mark McClellan. In a report, "Improving Innovation in Medical Technology: Beyond 2002," he called for improved communication between his agency and drug applicants, fellowships that would bring in new expertise from the outside, and tapping the expertise of practicing cancer doctors to establish "appropriate endpoints for clinical trial design."

In addition, the FDA is seeking public commentary on its new draft Guidelines for Pharmacogenomic-based Products, which may offer advantages in predicting which patients will experience the best efficacy and lowest incidence of side effects based on their genetic profiles. FDA officials have stated publicly their desire to have as much information submitted to them by sponsors on such patient profiling as early in the development process as possible.

While it has been suggested that FDA should regulate only safety, and let physician experience guide the question of efficacy, others believe that medical progress would in fact be stymied by an absence of the regulatory obligation to demonstrate efficacy. If, for example, the rate of efficacy of a particular drug is not documented, then against what standard will future drugs be evaluated?

Key Questions:

• How can the regulatory process be streamlined while protecting patients?
• Is safety an adequate criterion for drug approval or must efficacy always be demonstrated?
• What are the implications of pharmacogenomics and pharmacogenetics on the regulation of new therapies?
• Is the FDA moving rapidly enough to define guidelines for drug sponsors?
• Is FDA going to an extreme with drug safety requirements? Has it removed the patient's right to choose?
• How do FDA and the industry work together to streamline regulatory requirements on new therapies that are based on vast quantities of genetic, genomic and proteomic data?
• How much of an effect does the perception of increased study requirements by FDA have on the slowdown in new drug applications?
• Can Phase IV trials substitute for a rigorous process prior to approval?

Read on to Patients & Clinical Trials.