Advanced Therapies for Wales

Mark Briggs

02 July 2019

Drivers and Support for Transformation

National policies, expert reviews and strategies all recognise the need for innovation

“Doing ‘business as usual’ is unsustainable”Public Health Wales & WHO report Sept 2017

“We need to go beyond the delivery of traditional technical efficiencies to think innovatively”Wales NHS Confederation Briefing Nov 2017

“Harness innovation, and accelerate technology and infrastructure developments”Parliamentary Review of Health and Social Care in Wales Jan 2018

“New technologies will transform health and care in Wales when systematically deployed.”

MediWales Connects 20 June 2018 4

What are we talking about?

Therapeutic evolution has been driven by biological discovery

© GE Healthcare (BioWales 2017)

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Advanced Therapy Medicinal Products

“Advance Therapy Medicinal Products [ATMPs] are medicinal

products which are prepared industrially or manufactured by a

method involving an industrial process. ATMPs fall into three

categories…”

Somatic Cell Therapies

Tissue Engineered Products

Gene Therapies

Directive 2001/83/EC

And the interest in cell & gene therapies?

Gene therapy first to 'halt' common cause of blindness – AMD

Janet Osborne

Fergus Wales @ bbc.co.uk18 Feb 2019

Source: bbc.co.uk 21 June 2019

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“We have got one of the fattest populations in Europe...”*

• ~200,000 people living in Wales with diabetes

• 90% Type 2 due to lifestyle choice

• Estimate 500,000 by 2025

• Current cost to NHS ~£500M

• 80% managing preventable complications

• ~2000 people in Wales with foot ulcers; ~50% 5 year survival

• ~330 amputations per year

Also non-NHS cost (UK):

• Absenteeism: £8.4B pa

• Early retirement: £6.9B pa

• Social benefits: £0.15B pa

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By Jeremy Kemp at English Wikipedia, CC BY-SA 3.0, https://commons.wikimedia.org/w/index.php?curid=10547051

The ‘hype’ cycle…transformative therapies are not without pitfalls

Cellular Therapies:Mesenchymal Stromal Cells (MSCs)• Missing trial goals • BUT still have promise; knowledge lacking

demand (or greed?)Engineered T Cells (TCRs, CAR-T)• High % durable and ‘curative’ responses to

range of leukaemias• Adverse events (deaths) paused/halted

multiple trials – Kite, Juno, Cellectis etc

Gene Therapies:‘Antisense’ & Viral modification• Recovered from Jesse Gelsinger death

(1999) and momentum rapidly building• Gene editing – enter the CRISPR/Cas9

era?

Public demand and expectation is not diminishing….

Products being approved and licensed

09 Jan 2019NHS England CAR-T centre details announced

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New Pathways Require New Ways of Working

Presentation, Assessment,

Counselling & Referral

Treatment Plan:Suitability for Therapy->Selection

Clinical Trial/’Specials’/EstablishedApprovals & Patient Consent

Treatment:Patient Preparation, TherapyAdministration & Immediate

Aftercare

Product ManufactureOTS vs Bespoke

Allo vs Auto

‘Lab Tests’: Pathology, Genomics,Informatics, Radiology Etc

Tissue Collection Local vs Remote

Processing/ExpansionCharacterisation

Quality Control[Banking] Storage

Release

Transport & DeliveryEnd-user Formulation &

Processing

Patient

Product

Ancillary Services

Estates

Workforce

Finance

Organisation Structure & Commercial Partnerships Regulatory Clearance & Trials/Exemptions Approvals

Diagnosis

Intensive Care Readiness

Informatics Monitoring

Discharge & Release

Monitoring

Infrastructure & Resource Investment

Revenue vs Reimbursement

Aftercare vs Ongoing Treatment

Core Competency Development

Therapy Options Education

Therapy Release & Administration

Adverse Event Obs. & Control

Aftercare

Core InfrastructurePharmacy: GMP? Storage &

Handling FacilitiesIntensive Care Core Infrastructure

PathologyRadiology Etc

Governance

Commissioning

Risk Profiling & Screening

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My vision for precision medicine has two elements,

which are integrated diagnostics and advanced

targeted therapeutics. For both elements we have a

clear national vision that sets out how we will transform

our services to create sustainable, high quality, future-

facing care pathways that have space for increased

research, innovation and strategic partnerships.

In Wales, our journey towards precision medicine is well

underway and I am confident that we can be a front

runner in the global race to harness the potential of

precision medicine. It is vital that we have a connected

and collaborative approach towards precision medicine

and that our story is heard both in and outside Wales as

an example of good practice and of working together

towards a common set of goals…

Precision Medicine in Wales

Vaughan GethingMinister for Health And Social Services

https://gov.wales/written-statement-precision-medicine

Precision Medicine in Wales: The Four Pillars

A Vision for Wales…

Regulatory

Compliance,

Quality and

Safety

Clinical

Pathways Estates

Public and

Patient

Involvement

Informatics &

Information

Governance

Workforce

Research and

Innovation Strategic

Partnerships

Hosting and

commissioning

‘Our ambition is to translate the hope and potential of advanced

therapies into reality for Wales and to use this opportunity help people

to improve their health, education, employment and lives through

collaboration with industry and academic partners now and in the future.’

Innovate UK - ATTC Overview: 3 Centres across UK

Northern Alliance Advanced Therapy

Treatment Centre objectives :

Development of the clinical delivery

pathway & use of informatics to

achieve utility at scale in clinical

pathway

Midlands & Wales Advanced

Therapy Treatment Centre

objectives: End to end supply

chain logistics, network of

ATMP capable hospitals,

infrastructure for trial roll out,

validation with real world

manufacturing and trials,

economic model for ATMP

use

Manchester Advanced Therapy

Centre Hub objectives: co-

ordination of patient cell collection,

processing & storage, track & trace

solutions and education through

MRes platform iMATCH

Source: Cell and Gene Therapy Catapult

• Increase patient access to ATMPs

• Develop best practice for the safe and effective delivery

• Develop best practice for the manufacturing and final preparation of ATMPs using good manufacturing practice (GMP) within a clinical setting

• Assure consistency of quality, safety and efficacy of products if they are made at Different locations within the network

• Establish robust connected supply chains for manufacture and delivery of ATMPs

• Develop systems that enable long term patient follow up

• Develop systems and processes that can be applied to other hospitals

Midlands & Wales ATTC

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•

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Road to delivery

Implementation Plan:

• Based upon experience from Genomics, Pathology and Imaging

• Deliver SoI-AT Programme Objectives

• Secure funding

• Build upon ATTC structure and learning to inform recommendations

• Virtual – but co-ordinated with secretariat support

• Focus for expertise as it develops

• Evolve and iterate as sector matures; engage with external

parties

Pictorial representation of potential delivery approach

Implementation Plan submitted to

Welsh Government: 31st May 2019