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DISCOVER • DEVELOP • DELIVER Medical innovation that helps millions of patients around the world EBE is a specialised group of European Federation of Pharmaceutical Industries and Associations, EFPIA ContentP4 • Introduction P5 • Value of Biopharma P5 • What role does biotechnology play in healthcare? P6 • How biopharmaceutical and small-molecule medicines differ P7 • Why are biopharmaceuticals complex to manufacture and to administer? P8 • Personalised Medicine – developing targeted treatments for patients P9 • Who are we? P9 • What we do? P10 • EBE members EBE • Biopharma • 2-3

The European Biopharmaceutical Enterprises (EBE) represents the voice of biopharmaceutical companies of all sizes in Europe and is a specialised group within the European Federation of Pharmaceutical Industries and Associations (EFPIA). Established in 2000, EBE is recognised as the leading biopharmaceutical association in Europe.
From developing new therapies that treat and prevent disease to helping people in
need, the Biopharmaceutial industry is committed to improving health and well-
being around the world. The industry develops, produces and markets medicines
that improve the lives of millions of patients across the world.
With a global ratio of 14.4% of investment in R&D compared to sales, the pharmaceutical industry is the most R&D intense sector at global level, and has the second largest R&D intensity in the EU (13.9%)1. On average, it takes 13 years and € 1,2 bn of investment to bring a medicine to the market2. Although € 30 bn is invested in research & development annually, many products do not make it to the market due to the high complexity of development. The biopharmaceutical sector employs
700,0003 people in Europe and 48,3304 in
biotechnology. The latter generates three
to four times the number of indirect jobs.
Taking the sector and the wider eco-system,
life sciences represent a key asset for the
European Union to achieve the EU 2020 goals
of smart, sustainable and inclusive growth5.
The majority of biotechnology companies
are SMEs. In fact, out of 2000 biotechnology
companies in Europe, 70% have less than 50 employees.
1 European Commission (2013), The 2013 EU Industrial R&D Investment Scoreboard; p. 452 EFPIA Facts 20133 EFPIA Facts 20134 Ernst&Young report "Beyond borders – global biotechnology report 2012"5 European Commission (2010), An Integrated Industrial Policy for the Globalisation Era. Putting Competitiveness and Sustainability at Centre Stage; COM(2010) 614, p. 26

Value of Biopharma: The biopharmaceutical innovation ecosystem is comprised of 325 million
multiple players and biotechnology has been recognised as from Biotechderived medicines one of the key drivers of innovation: Eight of the top-ten products in terms of market share are biological medicines6. 325 million patients benefit from Biotechnology derived medicines. These medicines represent 50 % of the pharmaceutical development pipeline and 20% of the medicines in the market. the pharmaceutical 50 persons
What role does biotechnology play in healthcare?
Biotechnology is a broad discipline in which biological processes, organisms, cells, or cellular
components are used to develop new medicines and diagnostics7. Modern biotechnology
provides breakthrough products and technologies to combat diseases. Currently, there
are more than 250 biotechnology healthcare products available to patients, many for
previously untreatable diseases such as cancer and rare diseases. Such products are
named "biological medicinal products" or simply "biopharmaceuticals" or "biologics".
Biotechnology is evolving rapidly in the areas of transgenesis,
nanotechnology and bio-informatics, thus fostering the development
of vaccines, which have been successfully used to prevent diseases,
as well as antibiotics, that are naturally produced by micro-organisms,
products such as insulin for diabetes, and other proteins which are
used in the treatment of cancers.
Through treatment with cellular material, including stem cells and gene manipulation, biotechnology explores new perspectives, and opens the door for the regeneration of damaged organs and the restoration of their original function. Innovation in this arena will continue and points to a promising future.
6 IMS Institute for Healthcare Informatics (2014), Assessing biosimilar uptake and competition in European markets. IMS Institute Inisght. October 2014; www.imshealth.com (accessed: 14/10/2014) 7 Biotechnology is part of our daily life, as derived from the definition used by the UN Convention on Biological Diversity which is "any technological application that uses biological systems, living organisms or derivatives thereof, to make or modify products or processes for specific use".
EBE • Biopharma • 4-5

How biopharmaceutical and small-molecule medicines differ: Vaccines, allergen immunotherapy, blood components, and gene therapies are examples of biological products. Most biologics are produced in living organisms, such as plant or animal cells, whereas small molecule medicines (most pharmaceuticals) are typically manufactured through chemical synthesis. The inherently complex nature of biologics makes them also more complex to develop and administer as well as challenging to replicate. However, similar versions of biologics (called "biosimilars") that offer comparable quality, safety and efficacy to a reference product can be brought to the market after the patent of the originator biologic has expired, and thus offer cost savings to the healthcare system8. Large molecules (Biological)
Chemical: Aspirin - a traditional, Biopharmaceutical: Human Biopharmaceutical: Antibody chemical molecule (21 atoms) Growth Hormone ( 3.000 atoms) (IgG molecule, 20.000 atoms) A biopharmaceutical is a large molecule with a complex three-dimensional shape derived from a biological source. Some of these are already present in the human body. Examples include proteins such as insulin, growth hormones and erythroproteins. The ‘active substances'9 of biological medicines are larger and more complex than those of non-biological medicines. 8 European Commission, what you need to know about biosimilar medicinal products, April 20139 Active substance (or active ingredient) causes the desired effect and make the medicine work

Why are biopharmaceuticals complex to manufacture and to administer? Cells need a very specific environment to thrive.
At every step of the biopharmaceutical production process, a specific and complex environment is created and maintained. Even subtle changes in pH or temperature can affect the production process and alter the proteins being produced. Even minor changes in the structure of the product may result in inefficiencies and undesirable side effects of the medicine. For this reason, strict controls are necessary to ensure the quality and consistency of the final product. Due to the specificity of the molecules, the vast
majority of biopharmaceuticals are administered
by direct injection, usually through the intravenous,
subcutaneous or intramuscular routes.
EBE • Biopharma • 6-7 Personalised Medicine – developing targeted treatments for patients Personalised medicine (PM) is a promising new approach to healthcare that has enormous potential to help develop more effective treatments, with improved safety profiles, by offering the right medicine to the right patient at the right time. PM is based on significant scientific advances in understanding the underlying causes of diseases, driven by breakthroughs in molecular biology and the ‘omics' revolution. Because of these new technologies, we are now able to more accurately identify the causes of some illnesses, and thus develop therapies targeted to treat the specific cause. Molecular diagnostic tools play a crucial role in PM, as they help identify the patients who will respond best to a given medicine, for instance in breast cancer. Companies involved in developing these new technologies are instrumental in making Personalised Medicine a reality for patients. Today, the European Biopharmaceutical Enterprises, EBE, is recognised as the leading
biopharmaceutical organisation in Europe. 52 pharmaceutical companies are
members of EBE and the majority of them are small and medium sized companies.
As a specialised group within the European Federation of Pharmaceutical Industries
and Associations (EFPIA) EBE is the "Europe's expert voice for emerging bioscience
& technology and the leading platform for the health innovation ecosystem."
EBE focuses on enhancing emerging healthcare technologies, promoting science innovation and advocating for policies that foster innovative health solutions in Europe via expertise in: • Innovation eco-system and funding models
• Emerging biotech & science - regulatory advancement and advocating
on following priorities
PUBLIC AFFAIRS • REGULATORY & TECHNICAL AFFAIRS EBE • Biopharma • 8-9 EBE • Biopharma • 10 DISCOVER • DEVELOP • DELIVER Medical innovation that helps millions of patients around the world Rue du Trône 108 • Leopold Plaza Building • B-1050 Brussels, BelgiumT: +32 2 626 25 55 • [email protected] • www.ebe-biopharma.eu EBE is a specialised group of European Federation of Pharmaceutical Industries and Associations, EFPIA

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