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Catapult is a Technology Strategy Board programme
Business-led Translation
Growing a UK cell therapy industry delivering
health and wealth
Keith Thompson CEO Terrapin Stem Cell, Cord Blood and Cell Culture Conference, Cambridge MA. October 2nd 2013 [email protected]
The Launch of Catapults
Hauser Report • Creating new manufacturing industries for the UK • Better exploiting the UK science base
• “Grow and stick”
Technology Strategy Board • £440m for innovation in 2013/14
• £250m+ assigned to TSB for 7 Catapults 2012 • High Value Manufacturing, Cell Therapy, Offshore Renewables, CDE, Satellite
Applications, Transport Systems, future Cities
• 2 new ones announced August 2013 • Stratified Diagnostics, Energy Storage
• Mandated to bridge the gap between research and commercialisation through industry collaboration
2
Cell Therapy -Mind the (translational funding) gap
3
Catapult
• Limited evidence yet that new cell therapies can be developed, licensed and adopted successfully
• Limited commercial investment
• Limited precedents for valuable exits via IPO or acquisition
• Large corporates mostly watching and waiting
• Operational SME’s lack finance and breadth of resources for rapid advance
Industry Barriers
4
• Health Economics • Business Models • Reimbursement • Partnering
Business
• Robustness & Reliability • COGS & Scale up • Characterisation & Analytical • GMP • CMC • Delivery
Manufacturing and
Supply Chain
• Complex Regulatory Landscape • Pre Clinical Packages • Clinical trial design • NHS partnering
Clinical And
Regulatory
Approach
Project led • Pathfind therapies into and through the Clinic and
out and into the market Ø Core projects with direct investment Ø Rounded phase II data package Ø Investible propositions
• Assist industry to progress to commercialisation Ø Collaboration Ø Contract research
• Technology platforms Ø Develop novel technologies and license to industry
• Train professionals though immersion
5
• Commercial Development Plan
Stage Gated
Proof of Principle • Scientific, clinical, regulatory, commercial
Non-clinical • Safety, toxicology, GMP proving, assays
Clinical • Safety and efficacy, investible data
Platform • Generic issues and large collaborations
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Suitability
Projects: Successful Path to Commercialisation -start with the end product in mind and deliver an integrated plan
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• 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
8 Working models and finance
Nature of project Paid for by: Carried out by: Ownership of outputs Finance
Core Project Catapult Catapult Catapult £70m/ 5 years
Contracted Development
Client Catapult Client £10m/ Per year
Industry Collaboration
Grant or Catapult and partner
Catapult and partner
Outputs shared
£10m/ Per year
Funding for Cell Therapy in the UK
Govt. Support
Medical Charities
Financial Investment
Corporate Venturing
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Commercial Development Plan
Assets -Facilities and Teams
Facilities
• Central London location • 1200 sq m on 12th floor Guys Tower • Clinical research cluster • Capacity for 100 people
Teams • Business
• Business development • Business models • Health economics
• Manufacturing and Supply • Process development • Analytical development • GMP process proving • Supply Chain
• Clinical Trial and Regulatory • Regulatory • Clinical trial sponsor • Clinical operations
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Catapult is a Technology Strategy Board programme
Manufacturing and Supply Chain Development
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Lab - Pilot - Scale
Commercial Scale
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Catapult GMP Proving Lab
Catapult Multi Functional development Pod
Tech t ransfer
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Non clinical development Phase I Phase II Phase III Market
Approval
• Cell therapy characterisation requirements change depending upon where a product is in the commercialisation process
• Goal is to define and quantify the critical quality attributes for a particular product (release criteria)
• Need to develop appropriate assays for product release which can be incorporated into the manufacturing process (rapid, simple)
Pilot GMP Process
Development Process
Commercial Process
Scale Up/Out
Product release criteria
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Non clinical development Phase I Phase II Phase III Market
Approval
Products in pre-clinical development often require more thorough/complex characterisation using more advanced analytical approaches to understand the product
Pilot GMP Process
Development Process
Commercial Process
Scale Up/Out
MOA Cell markers
Potential potency markers
Identification markers
Direct Surrogate
Process Comparability
Purity assay
process qualification
Cell therapy characterisation
15
Non clinical development Phase I Phase II Phase III Market
Approval
Cell markers
Identification markers
Purity assay
MOA
Potential potency markers
Direct Surrogate
Raw Materials
Sterility testing Mycoplasma Virus testing Endotoxins
Stability
Shelf life Genetic stability
Viability
Cell therapy characterisation
Pilot GMP Process
Development Process
Commercial Process
Scale Up/Out
the aim is to develop assays that are reliable, reproducible with low variance, inexpensive, simple, and rapid, with appropriate reference standards — that
also have relevance to the intended clinical activity.
Catapult is a Technology Strategy Board programme
Health Economics
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
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Successful commercialization depends on both regulatory approval and optimal market access
Quality Safety Efficacy Comparative Clinical & Cost-Effectiveness; Budget Impact
REGULATORY APPROVAL MARKET ACCESS
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Payers have a common aim: to achieve the greatest health care value for the money they spend
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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
20
Value-based pricing relies on the quantification of the added-value that a new technology delivers over SOC
• Reference Value of Standard of Care (SOC)
o Comparative data against the SOC is required: Ø H2H comparative data demonstrating superiority or
non-inferiority of Product X against the SOC is preferred Ø Indirect comparisons of high methodological standards
(NMA) sufficient for non-inferiority claims • Differentiating Value
o Clinical effectiveness o Economic impact: budget impact, cost-minimization,
cost-effectiveness, cost-utility • Value (V)
o For a given indication “V” varies depending on the intervention’s positioning in the treatment algorithm & the target patient profile
Reference Value (SOC)
Positive Differentiation
Value
Negative Differentiation
Value (NDV)
V
RV
PDV
V = RV + PDV - NDV
NDV
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• QALYs are the measure of clinical effectiveness Ø QALYs = Life expectancy (life years) x Quality of life (utility) Ø Utility ranges from 0 (death) to 1 (full health) Ø Utility determined by HRQoL instruments; incorporation in clinical trials is key for market access
• Costs Ø Direct (healthcare) and indirect (social care) costs
o Societal costs (e.g. time off work) not yet accounted by NICE
• Incremental Cost Effectiveness Ratio (ICER) Ø Cost B-Cost A / QALY B- QUALY A
QALYs gained (B vs A)
QoL
(ut
ility
sco
re)
Treatment B
Life Years 1 2
1.0
0.5
Treatment A
0
In markets like UK, cost-effectiveness is the measure of value and the determinant of reimbursed price
22
NICE ICER thresholds: • Set at £20K/QALY; up to £30K justifiable
if: Ø There is confidence in results Ø Technology is highly innovative
• Orphans: No defined threshold • Under VBP additional factors are likely to
impact ICER threshold e.g. Ø Disease burden Ø Level of unmet need Ø Size of target population
Main commercial preparations to optimize market access
Objectives
• Assess opportunity (e.g. epidemiology, disease burden, unmet need, clinical /pricing benchmarks, funding & supporting data requirements, competition)
• Generate insights on reaction to “Target Product Profile”, key value drivers, likely positioning, pricing, reimbursement, uptake, supporting data requirements
• Development of clinical value arguments and economic models
• Test value story with key market access stakeholders
• Identify areas to strengthen story and data
• Identify revenue maximising target price
• Inform asset valuation and market access strategy
• Enhance market access potential and strategic partnering
• Compile in a single document clinical and economic value proposition and corresponding evidence to support negotiations
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Enviromental Reviews
Qualitative Research with Key
Market Access Stakeholders
Value Story Development
Quantitative Pricing
Research Value Dossier Development
Development Suitability assessment Out-licensing or Launch
Catapult is a Technology Strategy Board programme
Clinical Trials 24
Databases of UK preclinical (<2 yrs from clinic) and clinical stage cell therapies
25
Category Number Comparision
Preclinical (<2 yrs from clinic) 37
More allogeneic therapies in preclinical stage
Larger variety of cell types in
preclinical stages
Larger range of indications for preclinical projects
Few commercially sponsored
projects in both pre clinical and clinical stages
Clinical (UK trial ongoing) 34
Total 71
http://ct.catapult.org.uk/
Regulation………
• Interact with regulators to improve clarity and speed
• Clinical access to NHS
• Clinical Trial Sponsor
• Use the existing flexibility
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27 Clinical trials for cell therapies
Patient population • Risk vs benefit • Disease stage • Paediatrics
Dose selection • Single vs multiple • Dose escalation
Safety • Risk mitigation • Monitoring • Duration follow-up
Efficacy • Recognised
endpoints • Head to head
comparison • HRQoL
Placebo and blinding
• In study vs • historical /parallel
Feasibility • Accompanying
device / surgical technique
• Manufacturing success rate
Logistics • Very close integration of
clinical and manufacturing teams
Catapult is a Technology Strategy Board programme
Project Examples 28
Identifying projects
• Pre-clinical and clinical databases • Technology transfer offices • Intermediaries
• Grant Funders • Industry Groups • Charities • Investors
• Direct contact • Inward investors • EU entrants
Collaborations
• Cooperation on manufacturing platforms
• Project sourcing and funding
• International collaboration
30
Project Examples 31
Large Cap Company
• New delivery device to reduce injection pain
• Share of expertise • Support for in house projects
• Phase 2 clinical trials • Scale up, Assays, Freezing and distribution
of cells
• Manufacturing partner, Regulatory, Clinical trial design and delivery
• Immunomodulation • Regulatory, Clinical trial design, business
models
Platform Project Example -Build a GMP iPS bank with Roslin Cells
GMP iPS cell bank
Known sub-population of triple HLA-A, B and DR homozygotes
Differentiated cells/tissues of chosen types and origins Existing registry
of volunteers for platelet and BM donation
Biopsy Reprogramming Cell therapy
Phase 1: Generate 6 cell lines: £2m
• Generate highly specified Master and Working Cell banks • Generate Research grade equivalents and distribute to bona fide researcher Phase 2: Expand to 20 cell lines: £3m • Milestone and success driven Phase 3: Expand to 100 cell lines: • Self financing dependent upon early clinical trial results
INVESTABILITY Market sen-ment for cell therapy investments improving
• Looking for rounded data package Clinical Data, Health Economics, Business Models, Market Access, Reimbursement, Defensibility
• Pricing target and COGS drive long term margin towards valuaHon
• Clinical Data incorporaHng H2H and HRQoL improves valuaHon
Transac-ons • Financial investment – Venture Capital
• Corporate Venturing
• Direct Corporate TransacHons
Health economics, pricing and value story built to support commercialisaHon
Strategic Goals
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
34
Catapult is a Technology Strategy Board programme
Industry
Cell Therapy Catapult
Investment
Researchers
NHS
[email protected] www.celltherapycatapult.org.uk
