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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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History of risk management
Description of REMS
Criteria for REMS
Historical REMS trends
Future REMS trends
Implications for BioPharma
Impact of REMS
Likelihood of receiving REMS
Strategic consequences for BioPharma sponsors
Opioid class REMS
REMS by NDA review classification
REMS elements by application
Complexity of REMS over time
List of REMS analyzed in this report
List of exhibits
Exhibit 1: Risk management timeline (1990-2010)
Exhibit 2: Six components of REMS requirements
Exhibit 3: ETASU distribution restrictions by stakeholder
Exhibit 4: ETASU key challenge by stakeholder
Exhibit 5: Characteristics of drugs requiring a patient registry
Exhibit 6: Sponsor responsibility for Implementation System by items
Exhibit 7: Atypical timetable for submission of REMS Assessment
Exhibit 8: FDA hierarchy for REMS decision-making process
Exhibit 9: List of RiskMAPs converted to REMS in March 2008
Exhibit 10: Cumulative number of REMS by application type
Exhibit 11: Cumulative number of REMS by number of REMS elements
Exhibit 12: Approved BLA requiring REMS (2006-10)
Exhibit 13: Approved NME requiring REMS (2006-10)
Exhibit 14: Approved new formulation requiring REMS (2006-10)
Exhibit 15: Approved new combination requiring REMS (2006-10)
Exhibit 16: Approved NDA and BLA requiring REMS (2006-10)
Exhibit 17: Percent of drugs with at least one additional REMS elements
Exhibit 18: Percent of drugs with a ETASU
Exhibit 19: Average number of elements for REMS for BLAs, NDAs and all approvals . 22
Exhibit 20: List of key REMS trends
Exhibit 21: Timeframe and impact of REMS trends
Exhibit 22: Setup and monthly operational costs associated with REMS elements
Exhibit 23: Effect of REMS on patient population
Exhibit 24: Impact of REMS on cash flow and NPV
Exhibit 25: Number of programs with REMS requirements
Exhibit 26: BLA approved with REMS requirements (2008-10)
Exhibit 27: REMS elements for approved BLAs
Exhibit 28: Details of BLAs with ETASU
Exhibit 29: Philosophy for REMS investments
Exhibit 30: Opportunities of REMS elements for drug commercialization
Exhibit 31: NDA with priority review requiring REMS (2006-10)
Exhibit 32: NDA with standard review requiring REMS (2006-10)
Exhibit 33: NDA with orphan drug status requiring REMS (2006-10)
Exhibit 34: Comparison of REMS elements required for approved BLAs and NDAs
Exhibit 35: Complexity of REMS elements by quarter (2008-2010)