Washington Report

New FDA Policies Shape Pharma Development and Production - continued

Risks and research Efforts to increase FDA oversight of pharmacy compounding are one of many issues that involve the development and proper use of safe medications. FDA is working to address these concerns on several fronts, as seen in a number of documents and programs recently unveiled. Final guidance on minimizing drug risk. FDA issued three final guidances in March designed to improve data collection on drug safety before approval, to enhance post- approval adverse event tracking, and to enhance the safe use of medications on the market. These documents finalize draft proposals that were debated and revised over three years and support broader agency efforts to ensure drug safety. Although the guidances focus on clinical trial design and postmarket risk management, they present many issues important to pharmaceutical manufacturers. The guidance on premarket risk assessment outlines additional testing and study approaches likely to enhance a sponsor’s understanding of product safety concerns. Its recommendations could affect demand for clinical supplies and encourage expanded clinical assessment of proposed packaging and product design to minimize the potential for medication errors. Another guidance describes the development and use of risk minimization action plans (RiskMAPs) to ensure that patients safely use certain high-risk products. RiskMAPs could include special educational programs, reminder systems, and limited access controls. Although FDA emphasized that only a limited number of products will warrant such efforts, the agency also notes that it has the authority to require product recalls, safety alerts, and enforcement procedures such as seizures or injunctions to prevent the possibility of serious risks to the public health if risk minimization efforts fail. A third guidance presents approaches for manufacturers to establish good pharmaco-vigilance programs for marketed drugs. In outlining strategies for identifying and analyzing safety signals, FDA recommends that manufacturers also investigate signs of confusion about a product’s name, labeling, packaging, or use. The guidances are available at www.fda.gov/cder/guidance. Submitting pharmacogenomic data. Another final guidance spells out how manufacturers should provide genomic data to the agency, either as part of a market application or through a new voluntary process. FDA aims to encourage pharmacogenomic research leading to more personalized medicine and notes that voluntary data submissions can help both regulatory and industry scientists keep abreast of new developments in this area. FDA encourages manufacturers to meet informally with agency staffers to discuss ways to use innovative genetic and genomic information, and assures industry that voluntary genomic data submissions will not affect the review and oversight of formal applications for new drugs. Instead, a new interdisciplinary pharmacogenomics review group will examine voluntary data submissions separately from the application review process. The guidance explains that it addresses only pharmacogenomics data related to ensuring appropriate clinical testing and use of certain drugs—and not genetic or genomic techniques for biological product characterization or quality control, a topic that FDA plans to address in future guidance documents. The pharmacogenomics guidance, along with additional information for submitting data to FDA and FDA’s assessment process, is available at www.fda.gov/cder/genomics/default.htm. Although the guidance emphasizes that broader assessment of pharmacogenomics data offers potential benefits in clinical trial design and drug development, the advent of more individualized medicines may bring significant changes to the drug industry. A report from PriceWaterhouseCoopers entitled, Personalized Medicine: The Emerging Pharmacogenomics Revolution predicts an erosion over time of the current blockbuster business model, a development that offers significant opportunities to smaller life sciences companies. Initial pharmacogenomic breakthroughs are most likely to occur in oncology, and developing and launching new drugs in this area will require further changes in regulatory and clinical practices to incorporate genetic testing as part of patient diagnosis and treatment. PT