Catapult is a Technology Strategy Board programme Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical Manufacturing Hurdles Early Marcus Evans 11th Annual Commercial Translation of Regenerative Medicine Dr Stephen Ward, Chief Operating Officer 25 th November 2013
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Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical Manufacturing Hurdles Early A presentation by Chief Operating Officer, Dr Stephen Ward Nov 2013
Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical Manufacturing Hurdles Early A presentation by Chief Operating Officer, Dr Stephen Ward
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Catapult is a Technology Strategy Board programme
Autologous and Allogeneic Cell Therapy Industrialisation – Overcoming Clinical
Manufacturing Hurdles Early
Marcus Evans 11th Annual Commercial Translation of Regenerative Medicine Dr Stephen Ward, Chief Operating Officer 25th November 2013
The translational funding gap 2
Catapult
• Little evidence yet that new cell therapies can be developed, licensed and adopted successfully
• Limited investment from commercial sector
• Limited precedents for valuable exits via IPO or acquisition
• Most large corporates are observing and waiting
• Operational SME’s lack finance and breadth of resources for rapid advance
Mood is changing…….
Strategic Goals of Cell Therapy Catapult
Pipeline
• Increased cell therapies in UK clinical trial and clinical use
Value
• Investible propositions created leading to cell therapy companies that succeed and stay in the UK
Attractiveness
• Demonstrating that the UK is the place to do this work, with increased inward investment
Goals
• Build a £10bn industry
3
Cell Therapy Catapult
• Facilities • 1200 sq. m on 12th
floor • Capacity for
80-100 people
• Open end Q1 2014
• Currently in temporary facilities in KCL
4
Number of Staff at Cell Therapy Catapult
0
10
20
30
40
50
60
70
80
No of Staff Projected Staff
Addressing Key Sector Barriers
6
• Health Economics • Business plans • Manufacture & Supply vs Service • Reimbursement • Investment Risk
• Efficacy and safety hypothesis and evidence • Definition and characterisation of cellular product
• Patient population, unmet medical need, differentiation • Safety • Robust evidence of efficacy • Dose and dosing regimen
• GMP manufacturing process; release; comparability assays • Supply logistics • Scale-up / scale –out; Control of cost
• Pricing and reimbursement plan • Defensibility (IP, know-how…) • Commercialisation partner
Science
Clinical
Manufacturing
Business
Reg
ulat
ory
agen
cy d
ialo
gue
on
plan
s at
eac
h st
age
Transition from Discovery Science to Manufacturing Science
8
Pre-Clinical FIM Phase II-III
Manufacturing Science
Discovery Science
Cell Therapy Catapult Strategy for industrialisation of cell therapy processes
9
CONTRACT MANUFACTURING
GROW UK SECTOR
INNOVATION
SME
NHS
ACADEMIA
CT CATAPULT
NON-CLINICAL
PROCESS DEVELOPMENT
ASSAY
DEVELOPMENT
MANUFACTURING
GMP PROVING LAB
Lab - Pilot - Scale
Commercial Scale
10
Catapult GMP Proving Lab
Catapult Multi Functional Pod
Core Themes to ensure Sector success 10
Theme 1: For sector to mature we need to be driven by ‘market need’, including COGs
Theme 2: Collaboration across industry sectors
Theme 3: Build robustness into supply chains
Catapult is a Technology Strategy Board programme
12
Theme 1 • Commercial driver for product • Not driven by scientific interest • Manufacture to a sensible price point • Reimbursement strategy • Ask question; will product ever be used?
For healthcare products, price, reimbursement and demand are interlinked determinants of profit
All these have to be effectively addressed to reach commercial goals…
PROFIT
PRICE
DEMAND REIMBURSEMENT
13
Payers have a common aim: to achieve the greatest health care value for the money they spend
14
Does it add value
over SOC?
Comparative clinical
effectiveness
Is it worth it?
Cost-effectiveness Price comparison
Should we control its use?
Restrictions
Can we afford it?
Budget impact
Is the product needed?
Unmet need
Do we need to fund it?
Political imperative
The emphasis on these elements differs across markets, most notably the use of
cost-effectiveness in decision-making
Payers are key market access decision-makers (with input from clinical and economic advisors)
Cost-based Competitor-based Value-based
What is it?
• Price is set by assumptions on costs, expected sales volumes and margins
• Price is driven by the pricing of competitor products
• Price is based upon therapeutic / economic value to the customer
Examples • Cost-plus pricing • ROI based pricing
(e.g. PPRS in UK)
• Penetration pricing • Reference group
pricing • Value-based pricing
Comments • Becoming obsolete;
no longer resonates with payers
• Enforced by many reimbursement systems for “undifferentiated” products
• Typical approach for differentiated products
Pricing approaches in healthcare are shifting towards value-based models
15
Value-based pricing relies on the quantification of the added-value that a new technology delivers over SOC
Catapult is a Technology Strategy Board programme
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Theme 2 • Theme 2: Collaboration across industry
sectors • Common challenges already solved: • Biopharmaceutical • Common challenges already solved: • Vaccine
Significant Opportunity to Leverage knowledge from related sectors
17
Synergies
Spill-over Benefits
18
Established Biologics Knowledge
New Cell Therapy Knowledge
Creating Innovative Manufacturing Critical Mass
Leverage Existing Capabilities and Capacity
Re-innovate Prevent Wheel- re-invention
UK PLC
Cell Therapy Catapult cross-sector strategy
• Hired development/production staff from Biopharma and Vaccine industry
• Interact with High Value Manufacturing Catapult
• National Biologics Manufacturing Centre • Fill finish
• Formulation
• Volume reduction
• Centre for Process Innovation • Micro-electronic printing: stability and integrity