Driving Innovation

Stratified Medicines

Innovation Platform

Graham Bell

Lead Specialist Stratified Medicine

Driving Innovation

New strategy launched 2011

Driving Innovation

Challenge Lead Innovation

Driving Innovation

Challenge Statement

• How to place the UK at the centre of a

new era of stratified medicines:

• Approach: to help accelerate the

development and uptake of treatments

based on the combination of diagnostic

test and drugs

... to benefit the healthcare industry

... to improve patient outcomes

... and for wider UK economic benefit

Driving Innovation

Programme Partners

• Technology Strategy Board

• Department of Health

• Scottish Government Health Directorate

• National Institutes for Clinical Excellence

• Medical Research Council

• Cancer Research UK

• Arthritis Research UK

Driving Innovation

Together the partners will invest over £200

million over a 5 year period will be invested

to accelerate Stratified Medicine in the UK.

The aim is to bring the government,

researchers and business together in a

major initiative that will place the UK at the

centre of a revolution in the diagnosis and

treatment of disease.

Stratified Medicine Innovation Platform

Driving Innovation

First calls

• Tumour Profiling & Data Capture (£5.8m)

– Working along side CRUK programme

– Funded 6 projects

• Inflammatory Biomarkers (£4.3m)

– Working along side MRC:ABPI programme

– Funded 4 projects

• Business Models and Value Systems (£0.9m)

– Funded three projects

Driving Innovation

Technology Roadmapping

• Build a UK vision for stratified medicine

– ..... that the UK can deliver

– ...... supported by the SMIP and PMG

• Build a community of people who will help

• Take a strategic view of investment options

– Identify the barriers

– Programme activity to overcome them

– Investments in support of the programme activities

Driving Innovation

Vision for 2025

• The UK should be the best place to develop, and have adopted, stratified medicine. This will

benefit patients, provide cost-effective solutions for the NHS and other healthcare providers and

create opportunities for business

• There should be an increased collaborative culture throughout the sector based around shared

resources, and systems should be in place for effective data collection, sharing, governance and

use across sectors (including NHS, business, academia, regulators and NICE)

• It should be quicker and less expensive to develop new drug-diagnostic combinations and have

them licensed, and success should be reflected in increased UK economic growth

• It should be possible for all NHS patients to be involved in medical research if they wish, including

through use of patient information and records, in order to inform the next generation of successful

therapies

• There should be a smooth reimbursement process for stratified therapies and diagnostics, and an

intellectual property (IP) framework that encourages innovation

• The UK health system should have established stratified care pathways, and evidence should be

available to show that patient outcomes are improved where stratified medicine is used

Driving Innovation

Key Themes

• Incentivising adoption

• Increasing awareness

• Patient recruitment – consents and ethics

• Clinical trials

• Data – collection, management and use

• Regulation and standards

• Intellectual property

• Bio-banks and biomarkers

• Increasing the impact of R&D investment

Driving Innovation

Incentivising adoption

Key message:

Adoption of products into the healthcare

system was seen key during the

workshops.

There are many activities ongoing in this

area reflecting it’s importance to both

Industry and health service

Sound demonstration of value for money

for products is seen as the key

deliverable

Driving Innovation

Increasing awareness

Key message:

Need to develop a sound information

base and ensure that professionals

and public are provided with evidence

and a rational for Stratification.

For professionals proof of effectiveness

and value

For public avoid the message that

stratification is a form of rationing

Driving Innovation

Patient recruitment – consents and ethics

Key messages:

Often patient consents are restrictive and

do not allow use in research projects

beyond the immediate study. Broader

consents are preferred.

Ethical consideration needs to be given to

later incidental findings particularly in the

area of whole genome screening.

Driving Innovation

Clinical trials Key messages:

Delays around identifying the appropriate

patients and recruiting into trials is one of

the key bottle necks in development.

Any systems to pre-identify patient

cohorts could save significant resource.

New types of trial design will need to be

discussed and approved by regulatory

agencies

Driving Innovation

Data – collection, management and use

Key message:

There is a significant quantity of highly

relevant data already in existence within

the NHS and in Pharma companies.

Finding some way to share this data

appropriately could benefit all.

Will require significant effort in data

standardisation to enable open sharing.

Driving Innovation

Regulation and standards

Key message:

Regulation is a key gatekeeper in drug

development, all products must be safe

and effective (but how should trials be

designed to show efficacy in preselected

groups)

There was a desire for more regulatory

guidance around both trial design for

stratified products and in the validation of

tests and test protocols.

Driving Innovation

Intellectual property

Key message:

This was raised as a concern by diagnostic

companies who were concerned that

“homebrew” tests did not face a level

playing field.

Quality systems being implemented

nationally may help to improve this but

companies would like to see strong IP

protection for tests.

Driving Innovation

Bio-banks and biomarkers

Key messages:

There is a recognition that access to

appropriate samples with clinical

annotation is key and that having linked

Bio-banks with standardised systems

would help

Clarity or standardisation around what

information is required to “validate” a

biomarker

Driving Innovation

Increasing the impact of R&D investment

Key message:

The desire is to increase the impact of

everyones R&D spend, public, private

and third sector

There was a widespread acceptance that

no single organisation could make a

difference alone. The key is coordinated

activity in multiple areas simultaneously.

Only by working together

Driving Innovation

Summary

There was a widespread acceptance that

no single organisation can make a

significant difference working alone. The

key is coordinated activity in multiple

areas simultaneously.

Driving Innovation

Adverse Events and Non-Responders

( up to £7.5m)

Where Next?

• Developing tests to predict risk of suffering

adverse events, or non response to

current therapies.

– Phase 1: Health economic valuation of product

and impact on clinical care pathway

– Phase 2: Funding of development of most

successful projects from phase 1

– SBRI in partnership with Dept of Health

Driving Innovation

Contact details

• Dr Graham Bell (graham.bell@tsb.gov.uk)

• Dr Alasdair Gaw (alasdair.gaw@tsb.gov.uk)

• https://ktn.innovateuk.org/web/stratified-medicines-

innovation-platform