On September 27, 2007, the Food and Drug Administration Act of 2007 (FDAAA) was signed into law. FDAAA 2007 included several key provisions, and gave FDA the authority to require a Risk Evaluation Mitigation Strategy (REMS) from drug sponsors. The primary objective of REMS is to enhance drug safety by ensuring that the benefits of a drug outweigh its risks. FDAAA 2007 became effective on March 25, 2008, when the FDA approved 16 drugs with existing RiskMAPs as meeting the requirements for REMS. REMS differ from previous risk management approaches (e.g., Risk MAPs) in several significant regards; they are well defined, assessed over time and legally enforceable.
REMS can range from a simple Medication Guide and Assessments of REMS implementation and effectiveness to programs that include Communication Plans with healthcare providers and complex distribution restrictions Elements to Assure Safe Use (ETASU). Of the various components of REMS, ETASU represents the greatest challenge. ETASU can include restrictions on various stakeholders (e.g., provider, physician, patient), and are ultimately distribution restrictions. Entereg was the first new product for which the FDA required this restrictive element (May 2008), although a number of drugs with RiskMAPs approved prior to 2008 were deemed to have met the requirements for REMS including ETASU (e.g., Accutane, Tysabri). Since then, the agency has required ETASU for >20 products: 4 in 2008, 5 in 2009 and 12 in 2010.
This report reviews 157 REMS for NDAs and BLAs implemented from March 2008 until the end of 2010. We analyze the yearly percentage of new molecular entity (NME), new drug application (NDA) and biologics license application (BLA) requiring REMS from 2008-2010. The data is also segmented by NDA chemical type (e.g., new formulation, new combination, new indication) and review classification (e.g., priority review, standard review, orphan drug).
We review historical trends, and forecast six future key trends in REMS requirements based on data in the last 3 years and interviews with industry experts. Trends discussed address issues such as REMS pervasiveness, change in REMS requirements, REMS streamlining, stakeholder input and more.
Given that REMS are likely to impact a significant number of drugs, we review the key implications for BioPharma, including drug approval rates and timelines, cost of commercialization and drug utilization/uptake. We discuss three distinct ways in which REMS can decrease a drug’s addressable patient population, as well as the impact of REMS on cash flow and drug valuation. Finally, we examine how REMS programs can be leveraged as opportunities for drug commercialization; we outline strategic, operational and organizational best practices adopted by sponsors to deal with REMS.
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