Despite improvements in drug approval times overall, some therapeutic areas still lag behind others. Oncology, rare diseases, and infectious conditions have received the majority of Breakthrough Therapy designations and seen the fastest approval times, averaging 12 months. While review times have decreased overall since 2009, gastrointestinal, musculoskeletal, endocrine, and central nervous system drugs average longer times of 12-21 months. As the next user fee agreement approaches, there is an opportunity to identify ways to promote more consistent review times across all therapeutic areas.

1. Some Therapeutic Areas Still Need Improvement
Despite consistent decreases in average approval times, endocrine, gastrointestinal
and CNS therapies have lagged behind in recent years.
Breakthrough Designation
Pharmaceutical and biotechnology innovators
seek the breakthrough designation because it provides
a faster path to patients. Since its inception, breakthrough
therapies have been concentrated in oncology, rare diseases
and infectious conditions. That said, 2015 marks an increase
in the diversity of breakthrough designated approvals.
The FDA has an important and difficult job:
Ensuring timely and efficient drug review
processes while maintaining rigorous scientific
and safety standards. Overall, agency
performance has improved markedly, a
reflection of the work by FDA leadership,
industry, Congress and other stakeholders.
Refinements, such as the most recent user fee
legislation (the 2012 FDA Safety and Innovation
Act) and the breakthrough therapy mechanism,
have accelerated approvals. Still, there are
wide variations across therapeutic areas, with
some indications, such as oncology and
infectious disease (e.g., Hepatitis C), benefiting
more than others. As we approach the next user
fee reauthorization and recognize the
differences across therapeutic areas, there is a
real opportunity to identify managerial,
procedural and other practices that will
promote more consistency across the agency.
Tracking FDA Drug
Review Performance
Improved Review Times…
Since 2009, FDA review times have steadily declined.
…With a Caveat
Over the last ~15 years the fastest reviews have been in oncology, infectious and
rare diseases (although the gap has significantly narrowed over the last 5 years).
View online:
CALifeSciences.org/2016FDADrugReport
@CALifeSciences
SUBMISSION APPROVALS
Difference from average approval time,FY 2008-2015
Gastrointestinal
Musculoskeletal
Anti-infective
Dermotological
Endocrine
CNS
Oncology & Immunomod
SUBMISSIONS
-6 -3-12 -9 0 63
Months difference from average approval time of 12.7 months
3
8
21
4
10
17
52
33%
0%
76%
0%
0%
29%
58%
#
%
Expedited*
*Includes Priority,BTD,Priority + BTD and Orphan designated drugs together
Conclusion
The FDA has worked diligently to improve overall drug
review times and processes — a success to be applauded.
As Congress, the agency and the biopharmaceutical industry
look toward the next user fee round, we have a great
opportunity to replicate those improvements across all
therapeutic areas.
More than 2 years
1-2 years
Less than 1 year
Estimated review time (incl.in-process)
Review time (approved Rx only)
DRUG APPROVAL TIMES
Estimated review time to approval for NME/NBE submission cohorts,FY 2000-2015
Percentofapprovals
100%
75%
50%
25%
0%
’13’12*’11’10’09*’08*’07’06’05’04’03’02’01
U.S.Submission Cohorts (FY)
*Includes estimates of approval times for drugs still in process
’00 ’14 ’15*
Lighter shades indicate projections for drugs in-process Months
24
18
12
6
0
21 21
17
20
15
12 13 13
21 21
15
13
12 11
9 10
BREAKTHROUGH DESIGNATIONS BY THERAPEUTIC AREA
As of March 14,2016
Hematology
Ophthalmology
Rare disease
Respiratory
Infectous disease
Oncology
BREAKTHROUGH
DESIGNATED APPROVALS
2013-2015
100
80
60
40
20
0
PublicallyavailableBT
Dermatology
Gastroenterology
Cardiovascular
Sensory
Immunology
Blood
Respiratory
Neurology
Rare disease
Infectous disease
Oncology
TOTAL: 107
52
14
1
1
3
3
4
4
6
6
13
2013
2
1
3
2014
7
2
3
1
13
2015
9
3
3
3
2
1
21
1
SUBMISSIONS VS.APPROVAL TIMES
Breakdown of expedited and standard approvals,FY 2000-2015
0 6 123 9 15 2118 24
80
70
60
50
40
30
20
10
0
Average U.S.review time,in months
Numberofsubmissions
Blood
GastrointestinalMusculoskeletal
Genitourinary
Anti-infective
Other
Cardiovascular
Endocrine
CNS
Oncology & Immunomod
Expedited
(includes Priority,BTD,Priority + BTD and Orphan designated drugs together)
Standard
Oncology submissions are the most
numerous and achieve quickest review
SOURCES:BCG analysis based on FDA data,EvaluatePharma,literature reviews and
press releases
Data supplied by Boston Consulting Group,Feb.2016
Graphics by PAUL HORN / Special to CLSA
© 2016 California Life Sciences Association.All rights reserved.