Network

Building the UK ecosystem for the

delivery of ATMPs

The Potential

Global ATMP landscape

Investment, partnerships & commercialisation: UK Strength

70+ companies in 2019 and rapidly

growing

85 clinical trials

- More advanced, more commercial

sponsors

Opportunity

>70 UK companies

2019 and 2024 forecasted difference for UK C&GTemployees

2,284 - 4,803 =+2,519 (110% increase)

50% increase

The number of clinical trials

with ATMPs are increasing

by ~50% each year, in recent

years there has been an

increase in industry led trials

£10 Billion

UK economy estimated at £10 billion in revenue by 2035 if 15% of the global market is captured

30-60d trial approval

Regulators have introduced

a range of fast-

track programmes to enable

patients to benefit from new

treatments more quickly

73% VC increase

Substantial increase in deals

done (VC investments, IPOs,

M&As and partnerships) –

73% increase in global finance

invested in 2018 cf 2017

3rd global cluster

UK is 3rd global cluster for ATMP development –London 80% of new products

The adoption challenge: Increasing patient demand

2018

Mainly clinical trial ~200 patients per year

2019

Licensed products for larger patient numbers

2021

Increased patient delivery ~2500 patients per year

2023

Routine patient delivery ~5000 patients per year

2028

Embedded patient delivery ~10000 patients per year

The adoption challenge: Increasing patient demand

Cell based Immune-oncology Landscape Gene Therapy Landscape

Figures courtesy of John Bishai, Wells Fargo

The adoption challenge: Scale up needed

Challenges

• Tracking

• Patient condition

Cells taken

from patient

Manufacture Challenges

• Cost of goods

• Product release

• Transport

Supply chain Challenges

• Temperature

• Time restrictions

• Patient specific

Arrival at

hospital

Challenges

• Thawing

• Registry

• IT

• Internal logistics

Delivery to

patient

Challenges

• Pharmacy

• Tracking

• Training

Payment

The adoption challenge: Scale up needed

Why develop the Ecosystem?

The ATMPs are still

developed by SMEs (90% of

the market) and the big

pharma PNL is still not

affected by ATMPs (less than

1% of revenue) though the

industry is growing rapidly and

3 unicorn companies*

coming from the sector in the

last few years

* Orchard, Autolus and Adaptimmune have each reached over $500M in value

The healthcare system is

not yet ready for a major

scale up required with

only a handful of products

currently routinely

delivered to patients in the

UK

* Yescarta, Kymriah, Strimvellis, Holoclar in routine use in the

UK with more anticipated soon

Innovative solutions are

required to address key

challenges facing ATMPs

in the UK

The industry The NHS The innovation

Northern Alliance

Advanced Therapies

Treatment Centre

iMATCH – Manchester

Advanced Therapy

Centre Hub

MW-ATTC - Midlands &

Wales Advanced Therapy

Treatment Centre

Cell and Gene Therapy

Catapult

Northern Alliance

Advanced Therapies

Treatment Centre

iMATCH – Manchester

Advanced Therapy

Centre Hub

MW-ATTC - Midlands &

Wales Advanced Therapy

Treatment Centre

Cell and Gene Therapy

Catapult

Advanced Therapy Treatment Centre network

• Disseminating the

learnings and systems from

initial centres to enable more

widespread adoption of these

innovative therapies across

the NHS

• 30 delivery partners –

collaboration between NHS,

Industry and Academia

• 200-250 people working on

the programme

• The network of Advanced

Therapy Treatment Centres will

develop and deliver systems

for the routine supply delivery

of these disruptive therapies.

London Advanced Therapy

Network

ATTC partnerships – crucial for success

Objectives:

• Development of the clinical delivery pathway

• Achieve utility at scale in clinical pathway through use of informatics

NHS providers

ATMP suppliers

Academia

Supporting industries

Industrial engagement in network

Industry Advisory Group

ATTCs: Aims

Build the UK

ecosystem for the

delivery of these

disruptive therapies

Build knowledge

and shared

learnings to provide

a favourable

environment for

developers

Increase patient

access to these

potentially life-

changing

medicines

Develop new

technologies and

approaches to

make adoption

efficient, and

facilitate novel

payment practices

Make UK attractive

to investment by

accelerating UK

adoption

Using exemplar products to:

• Create easily run and ready to use systems and solutions that can be rolled out more widely to

the NHS

• Increasing institutional readiness and patient access to ATMPs

Impact of ATTCs to date

Knowledge SharingOrganisational readiness toolkit for ATMPs

Platform with guidance, checklists & SOPs

>8000 views since January launch

Industry EngagementIndustry advisory group covers

major players in UK ecosystem

StandardisationNational & international partners collaborating

with best practice

PharmacyNetwork expanded to cover all UK

Guidance issued for chief pharmacists

TrainingSingle platform to host NHS ATMP education

ATMPs MSc launched at Manchester University

Supply ChainPlug and play supply chain solutions for ATMPs.

Asymptote investment in a pilot scale-up Hub

Number of trialsFivefold increase in ATTC

GovernanceSharing of Governance procedures to

accelerate hospital adoption of ATMP trials

Health EconomicsWorking with key stakeholders to

explore novel payment mechanisms

FOCUS - Plan for future

Innovative digital solutions Utilise integrated NHS data

Registry support/devlopment

Innovative tracking and tracing models

Universal data integration and exchange

Rapid alert systems & follow-up

Pull through the pipelineSupporting companies & NHS to adopt therapies

earlier

New treatment modalities

Disruptive technologies with system challenges

Driving best practiceStandards framework for Industry/NHS Innovative

Models of Tissue Procurement/Distribution

Smart supply chainComplex logistics & tracking and tracing -

leverage existing systems

Prepare for massive & rapid scale-upNew treatment modalities

Disruptive technologies with system challenges

Identify technological innovations

Production of a skilled workforce Confidence to promote physician adoption

Coordinated, multidisciplinary training

Innovative payment mechanismsWork with NHS

Unknown business models

Risked based, outcomes driven

.

Companion Diagnostics Identification of patients

Implementation of Companion Diagnostic tools

and pathways for patient identification and follow-

up

Public and Patient EngagementUnique patient, carer & public challenges

Confidence to promote public adoption

Ensuring equitable access

The Ecosystem - route to beneficial adoption

Vibrant research base

Systems to pull developers

throughDrive innovation

Scale of impact, and overall cost

Bring down the price cost

Adoption at scale, competition,

Domestic Industry

Budget impact, and verification of efficacy and

benefits

Pay as benefits realised

Outcomes based value sharing

Readiness, and rapid

technological change

Prepare for one, 100X; OR,

prepare for 100, ONCE

Innovation, knowledge

sharing, and interoperability

Challenges in the adoption of ATMPs

• SME led industry doesn’t yet have capacity to implement solutions at a scale required

• Enormous potential for patient benefit that needs to be realised

• The scale and dynamics require a new approach

• Extremely disruptive to current healthcare setting, but could be transformative (with supply

chain and logistics bringing additional barriers)

• Ongoing and long-term data requirements requiring data integration and novel collection

methods

• The budget and adoption barriers are manageable but need to be proactively managed

• Continue to build the internationally recognised UK leading ecosystem for the

development, manufacture, trial and delivery of ATMPs to make the UK the

most innovative healthcare provider for their rapid adoption and treatment of

patients in the UK

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