have fda expedited programs shortened drug development timelines? an analysis of newly approved...
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Have FDA Expedited Programs Shortened Drug Development Timelines?
An analysis of newly approved therapies and how FDA expedited programs impacted drug development timelines
September 8, 2014
Artisan Healthcare Consulting, Inc.+1 781-221-5656 [email protected]
We collected the following dates for each NME approved from 2009 to 2014:
IND(investigational new drug) submission date of the first approved indication
Date of the FDA end of phase 2 (EOP2) meeting where available or the start date for the pivotal trials
NDA (new drug application)/BLA (Biologics License Application) filing dates
NDA/BLA approval dates We stratified the NMEs by year of approval,
and type of FDA expedited program used. We categorized each NME by therapeutic
area: Allergy, Autoimmune, Cardiovascular, Central Nervous System (CNS), Diabetes/ Metabolism, Diagnostic, Infectious disease, Musculoskeletal, Oncology, Ophthalmology, Rare diseases, Respiratory system, Sexual Dysfunction, Skin, Supportive care, and Women’s Health.
1. We collected key dates for new molecular entities (NME) and biologics approved over the
past 6 years.
Methodology (1 of 2)
3. We compiled and analyzed the findings to determine the effect of FDA expedited programs on drug development timelines.
Fast Track is introduced to accelerate the approval for medications treating serious conditions with unmet medical needs or a drug that has been designated as a qualified infectious disease product.
Accelerated Approval allows for approval of medication treating serious conditions based on surrogate endpoints.
Priority Review is for medications that offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
Breakthrough therapy designation is for medications that treat serious or life threatening disease and the medications may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.
.
2. We looked at the 4 major FDA programs used to expedite clinical development to understand
impact on time to market
Methodology (2 of 2)
*Sources: “Approved drugs 2013”, “2011 Novel New Drugs”, “2012 Novel New Drugs Summary”
IND filing: As reported in FDA filings - where information not available dates extracted from the Federal Register
End of Phase 2 meeting/beginning of pivotal trial -As reported in FDA filings – where the information was not available start dates of the pivotal trial extracted from www.clinicaltrials.gov
NDA/BLA filing: As reported in FDA filings
NDA/BLA approval: As reported in FDA filings
Excluded NMEs from the analysis
Lumizyme and Corifact: Excluded entirely from the analysis as IND filings and EOP2 meeting data are not available
2014 approved NMEs Belsomra, Cerdelga, Jardiance, Jublia, Orbativ, Otezla, Pledridy, Sivextro, Striverdi Respimat, Tanzeum, Zydelig and Zykadia: Excluded as full FDA documentation is not published
Sources used
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NMEs That Used Any of the FDA Expedited Programs
Therapeutic area
Autoimmune
Cardiovascular
CNS
Diabetes/Metabolism
Diagnostics
Infectious diseases
Oncology
Rare diseases
Respiratory system
Supportive care
Ophthalmology
2009 2010 2011 2012 2013 2014*
*2014 FDA approvals by August 2014
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Which Programs Are Used the Most?
Accelerated programs
Fast track
Accelerated approval
Breakthrough therapy
Priority review
Any program
2009 2010 2011 2012 2013 2014*
1 of 25 (4%)
0 of 20 (0%)
15 of 30 (50%)
14 of 39 (36%)
10 of 27 (37%)
1 of 26 (45%)
2 of 25 (8%)
2 of 20 (10%)
3 of 30 (10%)
4 of 39 (10%)
2 of 27 (7%)
4 of 26 (15%)
-- -- -- -- 3 of 27 (11%)
2 of 26 (8%)
6 of 25 (24%)
6 of 20 (30%)
15 of 30 (50%)
16 of 39 (41%)
10 of 27 (37%)
8 of 26 (31%)
7 of 25 **(28%)
8 of 20** (40%)
17 of 30**(57%)
22 of 39 **(56%)
13 of 27 **(48%)
12 of 26** (46%)
*2014 FDA approvals by August 2014
Since 2011, ~50% Of All Approved Therapies Have Benefitted From An Expedited Pathway, With Priority Review and Fast Track Being the Most Heavily Utilized.
**Number of drugs approved in particular year
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Which Therapeutic Areas Use the Expedited Programs the Most?Therapeutic area/ All programs
Oncology
Rare diseases
Supportive care
Diagnostics
Infectious diseases
Diabetes/Metabolism
Cardiovascular
Autoimmune
Ophthalmology
CNS
Respiratory system
Other
Total
3 of 5 2 of 2 7 of 7 10 of 11 8 of 8 4 of 4 34 of 37 (92%)
0 of 1 1 of 1 0 2 of 2 0 2 of 2 5 of 6 (84%)
0 0 2 of 3 1 Of 1 0 0 3 of 4 (75%)
0 0 1 of 1 2 of 2 1 of 3 0 of 1 4 of 7 (60%)
1 of 4 1 Of 1 2 of 4 3 of 3 3 of 4 3 of 6 13 of 22 (60%)
0 of 1 0 of 2 0 of 1 1 of 7 0 of 2 0 of 4 1 of 17 (59%)
2 of 5 2 of 3 1 of 4 1 of 3 1 of 3 0 of1 7 of 19 (37%)
0 of 3 2 of 4 3 of 3 0 of 3 0 1 of 4 6 of 17 (35%)
0 0 0 1 of 3 0 0 1 of 3 (33%)
1 of 4 0 1 of 4 0 of 1 0 of 3 2 of 3 4 of 16 (25%)
0 0 0 of 2 1 of 2 0 of 2 0 of 1 1 of 7 (14%)
0 0 0 0 0 0 0 of 8 (0%)
7 of 25 (28%)
8 of 20 (40%)
17 of 30 (57%)
22 of 39 (56%)
13 of 27 (48%)
12 of 26 (46%)
79 of 167 (47%)
2009 2010 2011 2012 2013 2014 Total
In Oncology, Rare Diseases and Supportive Care over 75% of Approved Therapies Utilized an Expedited Program.
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Which Expedited Programs Are Used the Most by Individual Therapeutic Areas? 2009-2014
Fast track Priority review
Accelerated approval
Breakthrough therapy
Any single Program 2 programs 3
programsAll 4
programs
Autoimmune 3 of 17 5 of 17 0 0 6 of 17 2 of 17 0 0
Cardiovascular 1 of 19 7 of 19 0 0 7 of 19 1 of 19 0 0
CNS 1 of 16 2 of 16 1 of 16 0 4 of 16 0 0 0
Diabetes/Metabolism 0 1 of 17 1 of 17 0 1 of 17 1 of 17 0 0
Diagnostics 0 4 of 7 0 0 4 of 7 0 0 0
Infectious diseases 9 of 22 11 of 22 2 of 22 1 of 22 13 of 22 8 of 22 1 of 22 0
Oncology 20 of 37 24 of 37 12 of 37 4 of 37 34 of 37 16 of 37 2 of 37 1 of 37
Rare diseases 2 of 6 3 of 6 1 of 6 0 5 of 6 1 of 6 0 0
Respiratory system 1 of 7 0 0 0 1 of 7 0 0 0
Supportive care 3 of 4 3 of 4 1 of 4 0 3 of 4 2 of 4 1 of 4 0
Ophthalmology 0 1 of 3 0 0 1 of 3 0 0 0
Other 0 0 0 0 0 0 0 0
Total40 of 167
(23%)61 of 167
(37%)20 of 167
(12%) 3 of 167 (18%) 79 of 167 (47%)
31 of 167 (19%)
3 of 167(18%)
1 of 167 (1%)
In Oncology, Infectious Diseases and Supportive Care, over 35% of NMEs Use Multiple Expedited Programs.
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Have Expedited Programs Shortened the Time from IND to NDA/BLA Approval?
2009 2010 2011 2012 2013 20140
2
4
6
8
10
12
8.810.1 10.2
8.7
6.6
8.79.0 8.6 8.9 9.2
7.5
5.6
NMEs Using 1+ Expedited Programs
NMEs Not Using Expedited Programs
# of
yea
rs fr
om IN
D to
NDA
/BLA
Ap
prov
al (a
vera
ge)
Year of NDA/BLA approval
When Considering the Time from IND to NDA Approval, Expedited Programs Do not Appear to Reduce Time to NDA/BLA Approval.
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Have Expedited Programs Shortened the Time from the EOP2 Meeting to NDA/BLA Approval?
2009 2010 2011 2012 2013 20140.0
1.0
2.0
3.0
4.0
5.0
6.0
7.0
3.64.0
4.9 5.0
3.6
4.7
5.3
6.3
5.25.8 5.7
5.0
Expedited programs
Non expedited programs
Year of NDA/BLA approval
# of
yea
rs fr
om E
OP2
mee
ting/
Star
t of
Pivo
tal t
rial t
o N
DA/
BLA
Appr
oval
(ave
rage
)On Average, NMEs Using Expedited Programs Reduced the Time from EOP2 to NDA/BLA Approval by 1.2 Years.
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Have Expedited Programs Shortened the Time from IND to NDA/ BLA Approval?
Breakthrough therapy
Fast track
Priority review
Accelerated approval
Any program
0 2 4 6 8 10 12
2009 2010 2011 2012 2013 2014
Average time: 7.4 years
Average time: 4.5 years
Average time: 8.6 years
# of years from IND to NDA/BLA Approval (average)
Average time: 9.0 years
Average time: 8.8 years
Breakthrough Therapy Has the Greatest Impact on Time to Approval of All Accelerated Programs; Accelerated Approval Also Provides Benefit.
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Have Expedited Programs Shortened the Time from EOP2 meeting to NDA/ BLA Approval?
Breakthrough therapy
Fast track
Priority review
Accelerated approval
Any program
0 1 2 3 4 5 6
2009 2010 2011 2012 2013 2014
Average time: 4.7 years
Average time: 2.5 years
Average time: 4.5 years
# of years from IND to NDA/BLA Approval (average)
Average time: 4.4 years
Average time: 4.7 years
When Considering Time from EOP2 Meeting to NDA/BLA Approval, Only Breakthrough Therapy Significantly Improves Time to Approval Compared to Other Expedited Programs.
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In Which Therapeutic Areas Have the Expedited Programs Shortened the Time from IND to NDA/BLA Approval?
Autoimmune Cardiovascular CNS Diagnostics Infectious diseases
Oncology Rare diseases Supportive care0
2
4
6
8
10
12
14
16
11.5
8.2
9.9
8.6
7.0
8.6
6.1
15.0
8.0
10.0
11.6
7.9
6.5
7.8 7.8
12.9
Used 1+ Expedited Program No Expedited Programs Utilized
# of
yea
rs f
rom
IND
to N
DA/
BLA
Appr
oval
(ave
rage
)
Reduction in Time From IND Submission to NDA/BLA Approval Is not Consistent Across Therapeutic Areas.
13
In Which Therapeutic Areas Have the Expedited Programs Shortened the Time from Time from EOP2 meting to NDA/BLA approval?
Autoimmune Cardiovascular CNS Diagnostics Infectious diseases
Oncology Rare diseases Supportive care0
1
2
3
4
5
6
7
8
4.65
5.24
6
2.73
3.724.25 4.19
7.19
5.06
6.566.88
4.66 4.52 4.5
3.293.59
Used 1+ Expedited Program No Expedited Programs Utilized
# of
yea
rs y
ears
from
End
of P
hase
2 m
eetin
g/St
art o
f Piv
otal
tria
l to
ND
A/BL
A Ap
prov
al(a
vera
ge)
For Most of the Therapeutic Areas Using Expedited Program Has Shorten the Time from EOP2 to NDA /BLA Approval.
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Conclusions
Since 2011, ~50% Of All Approved Therapies Have Benefitted From An Expedited Pathway, With Priority Review and Fast Track Being the Most Heavily Utilized.
✔
When Considering Time from EOP2 meeting to NDA Approval FDA Expedited Programs Reduced the Time from EOP2 to Approval by 1.2 Years. The Expedited Programs Do not Shorten the Time from IND Submission to NDA/BLA Approval.
✔
✔ Breakthrough Therapy Was introduced the Latest but Seems to Have the Biggest Impact on Approval Time Compared to Other Expedited Programs.
In Oncology, Rare Diseases and Supportive Care over 75% of Approved Therapies Utilized an Expedited Program. In Oncology, Infectious Diseases and Supportive Care, over 35% of NMEs Use Multiple Expedited Programs.
✔
Brad Payne - Bio
Brad Payne is a Team Leader for Artisan Healthcare Consulting. He has over 5 years experience in the healthcare consulting space, with expertise in domestic and international reimbursement and market access. • Lead of the global market access practice• Extensive knowledge of national and regional payers in Europe, Canada, Latin America, and
Asia/Pacific• Advanced understanding of U.S. market access, with a proven track record of developing strategy
that influenced national and regional payer coverage and payment decisions• Proven ability to develop market access strategies utilizing experience across a number of
therapeutic areas including oncology, immunology, infectious diseases, rare diseases, and devices• Understanding of health economics, with extensive expertise in Health Technology Assessment
(HTA) analyses
Prior to joining Artisan, Brad was a consultant at Trinity Partners, a pharmaceutical and biotech consulting firm, where he provided insight on forecasting projects, licensing and acquisition opportunities, and provided recommendations for managed care contracting strategies.
Brad graduated with a degree in economics Cum Laude from Harvard University and received an MBA from the W.P. Carey School of Business at Arizona State University.
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Brad PayneTeam Leader
+1 781.996.7338 office+1 702.250.3136 cell