Global regulatory outlook and regional trends

Sarah E. Hardison, PhD

12.11.2019

© 2019 Clarivate Analytics 1

Agenda Industry trends that shape regulation

HTA and cost-effectiveness

Generics and biosimilars

Summary and conclusions

1.

2.

3.

4.

© 2019 Clarivate Analytics 2

Increased R&D innovation

Rare diseases

Facilitated regulatory pathways

• More novel drugs being approved faster

• Fewer patients addressed by each new drug

• Economic value assessment needed

Increased R&D productivity is outpacing regulation

Global outlook

© 2019 Clarivate Analytics 3

Number of new active substance (NAS) approvals has more than doubled since 2008

Growth in FDA approvals of novel medicines

Data from Cortellis Regulatory Intelligence

4

6

6

8

5

2

11

12

7

16

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21

21

15

22

32

27

31

33

14

38

42

0 10 20 30 40 50 60

2008

2009

2010

2011

2012

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2015

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2018 BLA

NDA

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Facilitated regulatory pathways accelerate time to approval

FDA approval times for novel drugs are decreasing

608

675628

569610

493533 521

485 472

717

514

668

507

456418 422 426

461

299337

302

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2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

Nu

mb

er

of

Day

s

Average Time to Approval, NDA & BLA

Known activesubstance

New activesubstance

© 2019 Clarivate Analytics 5Data from Cortellis Regulatory Intelligence

Innovative medicines are more often approved via priority review

Novel drugs approved via FDA facilitated pathways in 2018

12

7

8

22

17

7

4

0 5 10 15 20 25 30 35 40

Orphan Designation

Fast Track

Breakthrough Therapy

Accelerated Approval

NAS Applications Granted Designations in 2018

BLA NDA

13

4

31

11

0

5

10

15

20

25

30

35

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Priority Review Standard Review

Applications Granted FDA Priority Review in 2018

BLA NDAData from Cortellis Regulatory Intelligence

© 2019 Clarivate Analytics 6

And are correlated with commercially successful drugs

FDA designations accelerate approvals

© 2019 Clarivate Analytics 7

More novel drugs being approved every year

Global trends in NAS approvals

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HTA and cost-effectiveness

© 2019 Clarivate Analytics 9

• Attaining marketing approval is not enough to ensure a drug’s success

– Some developers are more concerned about access and reimbursement than marketing approval, launch, and sales

• Patient engagement is shaping approval and HTA decisions

– Patients and providers do not necessarily have the same metrics as regulators and developers

– Drug development happening end-to-end with the patient

– “Voice of the patient”—FDA patient-focused drug development

– Draft guidances available, final guidance expected end of 2021

• Working to bridge the gap between HTA and Regulatory

– Evaluation of risk and evaluation of value

Social, economic, organizational, ethical outcomes

Health Technology Assessment (HTA)

HIQA analysis

© 2019 Clarivate Analytics 10

© 2019 Clarivate Analytics 11

12

And advancing patient centricity

EMA calls for HTA preparedness

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The HTA process takes time And authorities rarely agree

© 2019 Clarivate Analytics 13

• 72 therapies assessed since ICER started its review program, and pricing was supported on just 21 drugs

Drug companies want to align with providers and payers

US cost-effectiveness watchdog ICER becoming more influential

0

10

20

30

40

50

60

Not supported Supported

ICER Recommendations 2006-2019

© 2019 Clarivate Analytics 14

• Criticisms include lack of harmonization with ISPOR– No patient engagement, QALY-focus– Analyses center around ICER results

Early bumps in the road

Japan established an HTA authority in 2019

© 2019 Clarivate Analytics 15

• 2019 DIA MERC (Middle East Regulatory Conference)

See it as a modernization of healthcare systems

First mention of HTA consideration in MEA

Head of Market Access, Novartis

Advisor to Minister of

Health, Saudi Arabia

Associate Professor of International Health Policy,

LSE

•Innovators collaborate with HTA agencies from the time of clinical investigation

•HTA not limited to new technologies but also biosimilars

•Need to transition from external reference pricing to deliberate value assessment system

© 2019 Clarivate Analytics 16

Generics and biosimilars

© 2019 Clarivate Analytics 17

Fewer drugs available and longer timelines

EU and US lead on biosimilars, but US is slower

Data from Cortellis Regulatory Intelligence © 2019 Clarivate Analytics 18

0

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2006 2007 2008 2009 2010 2013 2014 2015 2016 2017 2018 2019

Biosimilar approvals in EU and US

EMA approvals FDA approvals EMA approval time (days) FDA approval time (days)

To enter these markets, look for regulatory maturity

Biosimilars gaining large share of emerging markets

© 2019 Clarivate Analytics 19

Americas, Europe, Middle East, Asia, Australia

Global adoption of biosimilars regulation

© 2019 Clarivate Analytics 20

Countries that accept foreign-

sourced reference products

Countries that conditionally accept

foreign-sourced reference product

Countries that do not accept foreign-sourced reference products without a

bridging study (US/EU)

Sourcing enough reference product can be challenging to meet study requirements

IGBA calls for further convergence on biosimilars

© 2019 Clarivate Analytics 21

Released their final guidance on biosimilars in 2015

China has 5 NMPA-approved copy biologics on the market

© 2019 Clarivate Analytics 22

Chinese authorities, academia, and industry are growing into these regions

Belt and Road Initiative may signal future relationships

© 2019 Clarivate Analytics 23Nature Outlook: China’s science silk road, 2019

Summary and conclusions

© 2019 Clarivate Analytics 24

Increased R&D productivity and focus on rare diseases means more drugs are being approved faster

As new medicines come to the market, health economics outcomes need to be evaluated

Regulators , HTA authorities, and innovators need to work closely to involve patients in the development of new medicines

The generic and biosimilar market is increasing access to new therapies in the developing world

25© 2019 Clarivate Analytics

Regulatory landscape evolves to keep up with innovation

Access to new medicines is increasing across the globe

Thank you

Sarah E. Hardison, PhD

sarah.hardison@clarivate.com

+44 207 433 4445

clarivate.com/cortelllis

© 2019 Clarivate Analytics 26

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