Università Cattolica del Sacro Cuore

The role of Regulatory Affairs

in drug development

Rome, May 12 2017

Francesco Mazza,

Director of Legal, Fiscal & Compliance Affairs in Farmindustria

Who we are

Who we represent …

200 pharmaceutical companies holding MAs

40% italian owned

60% foreign owned

Our mission: Represent the voice of companies to

promote competitiveness and scientific

development, communicate their value to

Institutions, to public opinion and to all

stakeholders at national and European level.

What we do

represents the legitimate

needs, and provides support

service

Farmindustria,

being an Association of pharma companies, based upon

the principles of

direct participation of companies

and open and shared dialogue

Source: elaboration of Istat, Eurostat data

Pharma industry in Italy

by nationality of ownership

(% on total)

Note: average of turnover, employees, investments

in manufacturing and R&D, foreign sales, taxation

Italian-owned

companies

foreign-owned

companies

40%

60%

Key figures of pharma sector in Italy

€ 30 billion of manufacturing value,

73% of which due to export (€22 billions)

€ 2.6 billion in investments,

€1.4 of which in R&D

and €1.2 in hi-tech plants and machinery

6,100 R&D employees, 53% women

200 companies, member of Farmindustria,

representing 90% of industrial value

of pharmaceutical sector in Italy

63,500 employees (90% graduates),

43% women, and 66,000 in upstream sectors

Pharmaceutical and upstream sectors

by Regions

Source: elaboration of Farmindustria, Istat data

28,000 direct employees, Lombardia ranks 1st for pharmaceutical and biotech

firms; 18,000 employees in upstream industries (chemicals, machinery, paper)

Lombardy

16,000 direct employees47% of regional export (87% of hi-tech export)

6,000 employees in upstream industries(chemicals, packaging)

Lazio

Tuscany

3,000 employees, 7,000 in upstream industries (machinery, chemicals, packaging)

Veneto

3,600 direct employees and 7,000 in upstream suppliers (machinery, chemicals, glass). Parma: pharma ranks third for export

Emilia Romagna

Centres of excellence of major international companies (3,000 employees in pharma companies and related industries). Bari and Brindisipharma export accounts for 25% of total

Puglia

Campania

SicilyAbout 1,000 direct employees and 2,500 in related industries. Catania: pharma accounts for 14% of total export

1,400 direct employees (100 in R&D) and 1,300 in allied industry

Abruzzo

900 direct employees and 3,000 in upstream industries

2,900 employees in pharma companies and upstream suppliers. Ancona: export to more than 40 countries Ascoli Piceno: pharma accounts for 70% of total export

Marche

6,000 direct employees

Piedmont and Liguria2,000 direct employees

and 7,000 in upstream industries (machinery, chemicals, packaging)

4,000 employees in upstream industries (glass, chemicals)

Fact&figures of pharmaceutical industryand upstream sectors in Italy

3,4 € bln of R&D, plants and machinery investments

130,000 employees

6.6 € bln of salaries (including social contributions)

44 € bln of production value

Source: elaboration of Istat, Efpia data

35

30

15

25

20

10

5

0

In 2015 Italy ranks second in Europe

for pharmaceutical production,

with the expectation to become the first

manufacturer in the medium-term

Among the Big Eu Italy accounts for

26% of total pharma production

and 19% of total sales

Italy is the second pharma manufacturer in EU

(aiming at becoming the first)

Pharmaceutical manufacturing

overall value (€ billion, 2015 estimates)

The role of pharmaceutical industry in Italy

for the economy of the country

Source: elaboration of Istat data

GDP and pharmaceutical production

(var. %)

12%

10%

4%

8%

6%

2%

0%

-2%

-4%

-6%

2010-2014 2015 2010-2015

The comparison with GDP shows

the importance of the pharmaceutical industry

for Italy’s economy

Furthermore, from 2010 to 2015

the pharma ranks first among manufacturing

sectors for growth of:

industrial production (+11%)

(manufacturing average: -7%)

export (+57%)

(manufacturing average: +23%)

productivity* (+21%)

(manufacturing average: +5%)

Pharmaceutical production

GDP

(*) labour productivity: added value per employee

The largest growth of export in Europe:

export as the engine of pharma companies in Italy

Source: elaboration of Istat, Eurostat data

10% 20% 30%0% 50% 60%40%

Pharma export: trend 2010-2015

for the big Eu countries

(% change)

From 2010 to 2015 pharma export in Italy

has grown more than all big Eu countries

and more than Eu 28 average

The performance of export is the result

of an increased quality of medicines

and vaccines exported all over the

world: in 2010-2015 the average value

at export registered a growth of +34%

(compared to +22% of Eu average):

+57%

+33%

Exporting a paradigm shift

(this slide was shown to Global CEOs at a meeting with the Italian

Government, May 2016)

Farmindustria long term strategy

Over the last 5 years Farmindustria has decided to play attack

to shift a paradigm in the pharmaceutical sector

that definitively needed to be changed

A cost to society A winning card for the economy

Pharmaceuticals as

a “cash machine” for NHS

No cuts and pharma savings

reinvested for innovation

A silos-based approach

to healthcare

A holistic view of health,

outcome-based

FROM TO

Priorities

• Transparency

• Compliance

• Value of Research and

Development and manufacturing

• Intellectual Property (Patent and Trademark)

• Sustainability

• Big Data/Health Data

• Data Privacy

• The actors

• The fundamental role of Regulatory

Affairs

• From the very beginning

Why the pharmaceutical sector is an asset for the

Country

Pharmaceutical

sector

Vaccines and

prevention

Less surgical

procedures

Less

hospitalization

Lower risks of

disability

Higher educational

levels

Upstream

Direct Contribution

Higher

productivity for

healthier

workers

Innovation, appropriateness,

compliance to therapies,

access to treatments,

freedom to prescribe

More Health

Eeconomic

growth Saving for

Welfare

Welfare in the Pharma

sector

€

HCS sustainability

Considerable increase in medical innovation

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

with over 7000 medicines under development

CAR-T therapies

Combination therapies

Gene therapy

Cell therapy Antibacterial treatments

Alzheimer’s treatments

Clinicians

Society

Payers

Patients

&

Families

Pharma

Policy

Makers

Healthcare

providers

INNOVATION

BENEFITS

Many meanings for “access to innovation”

challenges and benefits for

all stakeholders

increased quality of life

better health outcome

better disease management

more effective treatment

options for individual needs

proactive healthcare agendas

reduced welfare costs

effective use of resources

incentive to invest in innovation

Challenges for the future

What can be done to get prepared

Understand what is coming through the

industry pipeline and Horizon Scanning at

country level

Discuss with all stakeholders involved

the various implications

Inform policymaking, healthcare service

management and budget planning

Healthcare systems are required to provide rapid,

effective, fair, equitable and sustainable patient access

to new medicines

Develop new knowledge in selected areas

early and ongoing consultation on clinical trial

design and manufacturing processes;

supporting temporary access while waiting

confirmation of clinical benefit

adapting regulatory pathways

valuing + rewarding innovation flexible HTA and P&R processes would allow

for provisional access to be provided in case of

uncertainties at launch

adapting financing models overcome limitations of annual and siloed

budgets with new finance schemes

new approaches to treatment

and delivery of services

facilitating the optimal delivery of innovation,

with integrated care and manufacturing

facilities and early-diagnosis centres

evidence through Real World

Data

robust data capture systems to generate RWE

on patents outcomes for temporary access

Source: Farmindustria, EY, PhRMA

Pharma research in Italy: a network of excellence

for health and quality of life

the first medicine in Europe based on

stem-cells is Italian

More than 7,000 medicines in development

globally, with the contribution

of research in Italy: in biotech

(324 products in development), vaccines,

plasma protein therapeutics, advanced

therapies, rare diseases and sex-difference

medicine

Innovation is the result of the network

between companies and public-private

partnership, non-profit, universities. As

demonstrated by three examples of excellence:

the first gene therapy medicine

“ex-vivo” is Italian

N°of medicines in development globally

(for many diseases)

Cancers 1,813

1,329

1,256

1,120

599

511

159

475

Neurological

disorders

Cardiovascular

diseases

Mental health

disorders

Diabetes

HIV/AIDS

Immunological

disorders

Infectious

diseases

the first immunogene therapy (HSCT) for

adult patients with high-risk heamatological

malignancies

Source: elaboration of Farmindustria, Istat, Bain&Company data

R&D pharma investment in Italy are growing

also thanks to a more attractive ecosystem

R&D pharma expenditure in Italy

(€ million)

1,450

1,350

1,300

1,250

1,200

1,150

1,100

1,400

+15%

2010 2012 2013 2014 20152011

In 2015 pharma companies invested

€ 1,4 billion, the 7% of national total

In the last two years investments have grown

by 15%, and 75% of companies claim an

additional increase in the near future

Pharma companies in Italy invest 15% of

their added value, 10 times more than the

national average. The sector drives the country

to Europe 2020 goal (3% of national

added value)

Source: elaboration of Farmindustria, Aifa data

Clinical trials in Italy: share of Eu total

(% on total)

Italy could become a hub for clinical

trials, thanks to the scientific expertise and

the excellences in industry, universities and

NHS structures

Clinical trials: the door of innovation,

opportunity and resource for the country

In 2015 pharma companies invested

€ 700 million in clinical trials,

in NHS structures

Investing in clinical trials not only means

new medicines and innovative

therapies for patients: it also

guarantees to NHS important

resources and savings, since

companies bear all the costs connected to

clinical trials (for 1€ invested in oncology

trials, NHS saves 2,2€)

18.5

17.5

17.0

16.5

16.0

15.5

15.0

18.0

2010 2012 2013 20142011

Conclusion

• Certainty of the rules

• Centrality of the decisions taken.

• Respect of intellectual property, patents and brands,

where clarity, in compliance with all rules, constitutes

an advantage for all the players of the sector, for the

Administration, and last but not least, for the

safeguard of citizens’ health.

• Safeguard of Research, that means offering innovation

and better access to medicines.

Without patents there would be no research and without

Research there would be no new medicines.