The challenges of global reach for a mid-size Biopharma company

May 2014

Nathalie Forrer

ן Helping thousands of patients across

87 countries

ן FY 2013 revenue: €3.4 billion

ן More than 8 500 employees globally

ן R&D Spend: 25% of revenue

ן Listed on Euronext

UCB: A patient-centric biopharma leader

2

Focusing on severe diseases in

Immunology and the Central Nervous System

Stephanie, living with rheumatoid arthritis

Agenda

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Biotech Product Development Considerations

Case Study - China

Case Study - Japan

Biotech Product Development

Vial

PFS

Device

4

MARKET ENTRY -

COMPETE

FOR PATIENTS WHO

CANNOT SELF-INJECT

AND/OR REQUIRE

CLOSE MONITORING

DIFFERENTIATE

PATIENTS WHO

WANT CONTROL

BECOME ICONIC

PATIENTS WHO

WANT TO GET ON

WITH IT

Product

Portfolio Life Cycle

Management

D

e

v

e

l

o

p

m

e

n

t

Biotech Product Development

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Evolution over Time

Drug Substance Drug Product

Increased

number of

Markets

Increased

Demand

Increased

complexity

Final form

I I

I

I I

I I I

LCM

6

Biotech Product Development

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Increasing complexity during development of product

Regulatory restrictions

■ Some countries don’t accept more than one Drug Substance site (or only recently changed)

■ Some countries have different Drug Product sites associated with different applications

■ Long approval timelines and big differences in assessments

■ Limited guidance from local Health Authorities available, compliance requirements and point of

focus perceived differently in different area’s

Mid sized company restrictions

■ Only affiliates in main countries, need to use partners for most ROW countries

■ Partners work for multiple companies

■ Difficult to retrieve feedback, at least one extra layer in the communication (sometimes more)

Case study

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China - Regulatory Framework

Regulatory process in China is lengthy process

■ Only accepts one Drug Substance and one Drug Product manufacturer

• Change in regulation has been discussed for a long time

■ Need to perform clinical trial first, clinical trial approval can take up to 3 years

■ Between submission and approval, many changes applied:

• Drug Substance process changes

• Analytical method changes

• Specification changes

■ No formal process for submitting changes during clinical trial approval or when clinical trial is

ongoing

■ Need to keep supply in line with the initial submission (over 3 years ago)

Case Study

9

China - Supply Constraints

“Dinosaur” Process to be maintained @ the manufacturing site

Need to plan production campaign per process

Reduced Batch Output

Specific testing methods may still be required

“Dinosaur” Process to be maintained in inventory

Batch scale may not be adequate anymore leading to potential discard

Although Demand is low, no possibility to phase out

Expected to manufacture locally

Case study

10

Japan - Regulatory Framework

Implementation restrictions

■ Change needs to be submitted as a prior approval change (PCA)

• Requirements for documentation different from other countries

■ Once approved the change needs to be implemented in the product released to Japan immediately

• Approval can take anywhere between 6-12 months

• Need to have product available with and without “change” to be able to supply Japanese market

■ Implementation can be defined in many ways

■ AQL doesn’t exist in Japanese regulations 100%inspection - no defect

Case Study

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Japan - Development Strategy

Global vs Local Product

A subset of a

ROW process

2 different

Products

1 Product taking

JP requirements

into account from

the start

Case Study

12

Japan - Supply Constraints

Visual Inspection Process

Case Study

13

Japan - Local Testing

Local Testing to be performed on BioPharma Final Presentation

BEST CASE: Incoming ID test must be performed in JP, provided MOU country will support release

testing

FULL TESTING required should any activity such as packaging and/or distribution

are made out of a non-MOU country

Even if the company can demonstrate that the operations performed in the non-MOU country do

not impact the product quality manufactured and tested in the MOU country, the testing will be re-

performed in Japan.

How to deal with local testing results?

Take away

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Global harmonization would bring relief!

Change

Change

Thanks!