Advanced Therapy Delivery from NHS -clinical infrastructure requirements

Patrick Ginty PhD, RAC(US)Head of Regulatory Affairs Cell and Gene Therapy Catapult

The scale up challenge

2018Mainly clinical trial ~200 patients per year

2019Licensed products for larger patient numbers

2021Increased patient delivery ~2500 patients per year

2023Routine patient delivery ~5000 patients per year

2028Embedded patient delivery ~10000 patients per year

Conf

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Many products– different product types and indications

Immune-oncology Landscape Gene Therapy Landscape

Source: Wells Fargo Securities/CryportSource: Wells Fargo Securities/Cryoport

Source: Wells Fargo Securities/Cryoport

The scale up challenge

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

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Northern Alliance Advanced Therapies Treatment Centre

iMATCH – ManchesterAdvanced Therapy Centre Hub

MW-ATTC - Midlands & Wales Advanced Therapy Treatment Centre

CGT Catapult manufacturing centre

London Higher Education Funding Council for England Connecting Capabilities

• The network will increase the ability of the NHS to deliver disruptive medicines

• The centres will develop systems and processes within the trusts and hospitals capable of delivering advanced therapies at scale to patients across the NHS

• The learnings and systems from the initial centres will be rolled out to other centres in the UK

Advanced therapy adoption network

The network of Advanced Therapy Treatment Centres will develop and deliver systems for the delivery of cutting edge cell and gene therapies.

Northern Alliance Advanced Therapies Treatment Centre

iMATCH – ManchesterAdvanced Therapy Centre Hub

MW-ATTC - Midlands & Wales Advanced Therapy Treatment Centre

CGT Catapult manufacturing centre

London Advanced Therapy Network

ATTCs – different but complementary objectives

Midlands and Wales ATTC• Develop end to end supply chain

logistics• Create network of ATMP capable

hospitals

• Validate with real world manufacturing and trials

• Generate economic model for ATMP use

Northern Alliance• Development of the clinical

delivery pathway • Achieve utility at scale in

clinical pathway through use of informatics

iMATCH

• Co-ordination of patient cell collection

• Processing and storage, track and trace solutions

• Education through MSc/MResplatform

The ATTC network will work together with its partners to address the identified priorities to ensure they provide easily-run and ready to use systems and solutions that can then be

rolled out to other NHS centres, leading to enhanced patient access to ATMPs

ATTC – Industry/NHS/Academia partnerships

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

IUK funded ATTC – CGT Catapult role

Coordination of IUK funded ATTC activities and outputs

• Unifying centre projects

• Network projects• Ensure meets overall

programme outcome

• Supporting collaboration between centres

• Working with London ATN• Coordinating network

streams

• Network projects

• Communication regarding ATTC activities• Stakeholder engagement;

Regulators, Industry, Payers, International

• Dissemination of outputs

• Front door for companies

What is required to accelerate adoption

IUK ATTCs, London ATC and

other centres

Establish clear pathways to gather industry feedback:

Establishing streamlined information flow (e.g. through

industry partner group) to ensure requirements of industry are taken

into account.

Define common framework: Help companies navigate with harmonised procedures and

practices: e.g.: apheresis, treatment regimens,

patient selection.Work with companies and their

specific protocols to produce common frameworks and systems.

Clinical scale up requirements:

Understanding what is required at organisational level e.g. number of

apheresis units, ICU beds.

Contract standardisation:To ensure timely implementation

and adoption, contracts need to be standardised.

Speed of access: Streamline both procurement of starting material and delivery to product into health care system.

Define courier best practice.

Patient data collection and sharing

Bring data together, capture number of interventions post

treatment, capture data in real time, agree where data should be

held, connect with Digital Innovation hubs, development of Registries to meet both regulatory and reimbursement requirements. Patient monitoring:

Development of companion diagnostics, validated analytical methods and patient monitoring, staff training for safe patient

management will need to take place.

Access to expert knowledge by industry: Need for experts, education and physicians to

help trial design, formulating a database of knowledge network.

Patient engagement: Engaging the patients, patient

groups and charitable associations further.

The scale up challenge

2018Mainly clinical trial ~200 patients per year

2019Licensed products for larger patient numbers

2021Increased patient delivery ~2500 patients per year

2023Routine patient delivery ~5000 patients per year

2028Embedded patient delivery ~10000 patients per yearATTC & other centres

Accelerated Access Pathway/EAMS

Widespread adoption

Standardised methodology & training programmes established

Systems embedded in NHS for delivery & reimbursement

Reimbursement mechanisms established for licensed products. Rollout to other centres

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

Advanced Therapy NetworkCreating easily run and ready to use systems and solutions that can be rolled out to other NHS centres, increasing institutional readiness and patient access to ATMPs.