Some Therapeutic Areas Still Need ImprovementDespite consistent decreases in average approval times, endocrine, gastrointestinaland CNS therapies have lagged behind in recent years.

Breakthrough DesignationPharmaceutical and biotechnology innovatorsseek the breakthrough designation because it providesa faster path to patients. Since its inception, breakthroughtherapies have been concentrated in oncology, rare diseasesand infectious conditions. That said, 2015 marks an increasein the diversity of breakthrough designated approvals.

The FDA has an important and difficult job:Ensuring timely and efficient drug reviewprocesses while maintaining rigorous scientificand safety standards. Overall, agencyperformance has improved markedly, areflection of the work by FDA leadership,industry, Congress and other stakeholders.Refinements, such as the most recent user feelegislation (the 2012 FDA Safety and InnovationAct) and the breakthrough therapy mechanism,

have accelerated approvals. Still, there arewide variations across therapeutic areas, withsome indications, such as oncology andinfectious disease (e.g., Hepatitis C), benefitingmore than others. As we approach the next userfee reauthorization and recognize thedifferences across therapeutic areas, there is areal opportunity to identify managerial,procedural and other practices that willpromote more consistency across the agency.

Tracking FDA DrugReview Performance

Improved Review Times…Since 2009, FDA review times have steadily declined.

…With a CaveatOver the last ~15 years the fastest reviews have been in oncology, infectious andrare diseases (although the gap has significantly narrowed over the last 5 years).

View online:CALifeSciences.org/2016FDADrugReport

@CALifeSciences

SUBMISSION APPROVALSDifference from average approval time, FY 2008-2015

Gastrointestinal

Musculoskeletal

Anti-infective

Dermotological

Endocrine

CNS

Oncology & Immunomod

SUBMISSIONS

-6 -3-12 -9 0 63Months 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

ConclusionThe FDA has worked diligently to improve overall drugreview times and processes — a success to be applauded.As Congress, the agency and the biopharmaceutical industrylook toward the next user fee round, we have a greatopportunity to replicate those improvements across alltherapeutic areas.

More than 2 years1-2 yearsLess than 1 year

Estimated review time (incl. in-process)Review time (approved Rx only)

DRUG APPROVAL TIMESEstimated review time to approval for NME/NBE submission cohorts, FY 2000-2015

Perc

ent o

f app

rova

ls

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-processM

onth

s24

18

12

6

0

21 2117

20

1512 13 13

21 21

1513

12 119 10

BREAKTHROUGH DESIGNATIONS BY THERAPEUTIC AREAAs of March 14, 2016

HematologyOphthalmologyRare diseaseRespiratoryInfectous diseaseOncology

BREAKTHROUGHDESIGNATED APPROVALS2013-2015

100

80

60

40

20

0

Publically available BT

DermatologyGastroenterologyCardiovascularSensoryImmunologyBloodRespiratoryNeurologyRare diseaseInfectous diseaseOncology

TOTAL: 107

52

14

113

34

4

6

6

13

20132

1

3

2014

7

23

1

13

2015

9

333

21

21

1

SUBMISSIONS VS. APPROVAL TIMESBreakdown 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

Num

ber o

f sub

mis

sion

s

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 mostnumerous and achieve quickest review

SOURCES: BCG analysis based on FDA data, EvaluatePharma, literature reviews andpress releases

Data supplied by Boston Consulting Group, Feb. 2016

Graphics by PAUL HORN / Special to CLSA

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