www.efpia.eu

Committed to Health!

The contribution of the research-based companies to better health for European patients and sustainable healthcare systems

Bulgaria EU PresidencyMarch 6, 2018Sofia

Boris AzaïsDirector Public PolicyMSD

www.efpia.eu 2

Scientific advances and unmet medical needs are the key drivers of

pharmaceutical innovation.

Governments need to put in place strong “enablers”, such as IP rights, R&D

incentives, rapid access to market and a stable pricing environment.

IP

RIghts

MEDICAL NEEDS

SCIENTIFIC

PROGRESS

What drives pharmaceutical innovation?

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15.Augustyn C, Walker B, Goss TF; Boston Healthcare Associates. Recognizing the value of innovation in the treatment of rheumatoid arthritis. http://www.phrma.org/sites/default/files/pdf/BHARAWhitepaperMarch2013.pdf. Published March 2013. Accessed May 2017.

16.Centers for Disease Control and Prevention (CDC), National Center for Health Statistics. Health, United States, 2015: with special feature on racial and ethnic health disparities. https://www.cdc.gov/nchs/data/hus/hus15.pdf. Published May 2016. Accessed May 2017.

HIV/AIDS: Treatment Advances Over Time

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Medicines have transformed HIV/AIDS from a Death sentence to a manageable disease

0

2

4

6

8

10

12

14

16

1993 1998 2003 2008 2013

ASD

R p

er 1

00

,00

0

USA

Spain

Italy

France

Canada

Australia

Germany

United Kingdom

* Or latest year of available data: Italy (2012), France (2011), Canada (2011), Australia (2011), Germany (2011).

Note: HIV/AIDS ASDR extrapolated for Italy in 2004-2005 and Australia in 2005.

Source: Health Advances analysis; WHO Mortality Database (accessed February 2016).

Country

Decline in

ASDR

(1995-2013*)

-88%

-92%

-87%

-94%

-87%

-88%

-82%

-73%

HIV/AIDS Age-Standardized Death Rates (ASDR) By Country

HAART combinations introduced

HIV/AIDS

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Pawlotsky JM, et al. J Hepatol 2016; 62: S87–99; Manns M, et al. Nat Rev Dis Primers 2017;3:1–19.

The DAA Era

TVR

BOC

SMV

SOF

Identification

of HCV

IFN + RBV

The Interferon Era

IFN Peg-IFN + RBV

3rd Wave of DAA

SOF/VEL G/P

SOF/VEL/VOX

DCVLDV/SOF

OBV/PTV/r + DSV

EBR/GZR

2nd Wave of DAA

non-A, non-B

hepatitis

1984 1989 1998 2001 2013 2014 2015 2016 2017…2011 Elimination?

Hepatitis CFrom Discovery to Elimination in our Lifetime?

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Behind this success story, 77 investigational medicines failed in clinical trials and only 12 new medicines were approved between 1998 and 2014.

Source: Twenty-Five Years of Progress Against Hepatitis C: Setbacks and Stepping Stones; PhRMA 2014

HCV cure reduces risk of liver cancer

DAA-induced SVR is associated with a 71% reduction in HCC risk

Progress in HCV cure rates and improved treatments are supporting the global elimination strategy

2001-2010 2011-2013 2013-2014 2014-2016

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Pharmaceutical Research and Manufacturers of America (PhRMA). Researching Alzheimer’s medicines: setbacks and stepping stones.http://phrma.org/sites/default/files/pdf/alzheimers-setbacks-and-stepping-stones.pdf. Published 2015. Accessed May 2017.

What it means to be working at the frontier of science

Number of Alzheimer's Drugs in Development, 1998-2014

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With over 7000 medicines in development, medical innovation will play a key role in

addressing the challenges faced by patients and healthcare systems

Source: Health Advances analysis; Adis R&D Insight Database. March 2015, compiled by PhRMA

Where our R&D is focused

www.efpia.euSources: 1: Dirk Calcoen el al. 2014; 2: Biotech M&A Review, Windhover information 2008; 3: International Trade

Commission, 2016, Top Markets Report

Only 2 out of 10 marketed medicines generate returns that exceed average R&D costs.3

Attrition profiles across therapeutic areas

High Attrition Rates through the R&D Pipeline

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+ 2 + 1

EU=European Union; IP=intellectual property; RDP=regulatory data protection; SPC=supplementary protection certificateSource: 1. EFPIA, About IP incentives (2017)

IP incentives run in parallel and drive R&D to address unmet

medical needs

Patent (20 years)

RDP (8 years)

SPC (max.5 years)

Product available for patientsClinical trials (phases 1-3)

Orphan medicine (10 years market exclusivity)

Effective patent time lost

Paediatric research

Orphan designation

Timeline: -12 0 8 10 13 years

Paediatric extension(+ 6 months)

Paediatric extension(+ 2 years)

Research

For all medicines

For orphan medicines

The product is granted marketing

authorisation

The marketing authorisation is updated

for paediatrics

The patent is filed Generics enter the market after patent expiry

P&R delays

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Leading drug classes in 1995 were cardiovasculars, antibacterials, hypertension, pain, and a broad group of gastrointestinal products

All of these classes have been reduced to a much lower share of new and existing protected brand spend by 2015

However, existing protected brands is less than 50% of total expenditure

Latest innovation (<24months) has been less than 10% since 1996 [1]

Sources: [1] QuintilesIMS Institute (2017), Understanding the Dynamics of Drug Expenditure. Shares, Levels, Compositions and Drivers

Headroom for innovation:Last year’s innovation makes room for today’s

A complex policy framework requires partnership

TOGETHER

SUPPORT

Investment in Science:

- Academic centers

- Basic Science

- Higher education

- Promotion of partnerships

with industry

Investment in Healthcare:

- Financing

- Evidence-based medicine

- Focus on value

Favorable Environment:

- Intellectual Property Rights

- Smart regulatory framework

- Reward for innovation

- Rapid access to market

- Research tax credits

- Stability of the regulation

SCIENCE

POLICY

HEALTHCARE

POLICY

INDUSTRIAL

POLICY

Investments

in

Science

Investments

in

Public Health

Favorable

Business

Environment

Better Health

Human capital creation

Higher productivity

Economic growth

Policy Outcomes

New Drugs

Rapid Uptake

Sales

Cash Flow

R&D

Policy Decisions

Pharmaceutical innovation is the result of a strong partnership between science,

healthcare and industry.

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Thank You!