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.
The MS Excel™ database used to generate data in this report is available upon request after purchase. Please contact
admin@decibio.com to request a copy of the file. |
