Beyond Borders: Global biotechnology report 2010

oral anti-diabetes medications Januvia
and Janumet if patients take the drugs as
prescribed. Additionally, CIGNA will receive
further discounts/rebates if patients lower
blood sugar levels, even by methods other
than taking Merck’s diabetes medications.
These pricing models — where firms get
paid based on outcomes rather than sales
volume — are uncharted territory for most
companies, and present new sources of
risk. As these arrangements become more
prevalent, companies will need to focus
on ways to address challenges such as
FDA orphan drug approvals and designations, 1990–2009
Number of approvals
30
25
20
15
10
5
effectively managing data and appropriately
accounting for these transactions. (For
more information, see A closer look on
page 92.)
Orphan drugs
The FDA approved 17 orphan drugs in
2009, including 11 NMEs or BLAs and six
products with expanded indications — the
largest total since 2006, when 22 drug
indications received approval. The agency
also gave more than 160 indications orphan
Orphan drug approvals Orphan drug designations
0
1990 1991 1992 1993 1994 1995 1996 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009
40
Source: Ernst & Young, FDA
94 Beyond borders Global biotechnology report 2010
drug designation; slightly lower than the
165 in 2008 but markedly higher than the
levels seen earlier this decade.
Orphan drug approvals have been on an
upward trajectory over the last decade.
According to a Tufts University study,
approvals of orphan drug designations in
the US more than doubled from 208 in
2000–02 to 425 in 2006–08.
Biotech firms have traditionally been
significant players in this segment. To some
extent, this was because the economics of
160
140
120
100
80
60
Number of designations