Katie Pascoe (Value and Access, ABPI)

22 September 2016

Following Cancer Drug Pathways from Inception to Launch: Partnership, prospects and Pitfalls

Partnering is a key factor in patient access to new cancer medicines

Pre-clinical

Phase 1

Phase 2

Phase 3

Post-launch

Search for drug targets

Solutions to improve care

pathways

Clinician and Patient input into

clinical trial endpoints

Clinician and Patient input into value proposition

Academic and cross-industry

partnerships to further develop

medicines

Studies of real world outcomes

Clinician and commissioning perspectives on current practice

Training: undergraduate and

post-graduate industry placements

European and UK joint scientific

advice from HTA and regulatory

bodies

New ABPI report highlights well established partnerships drive the development of new medicines

• Many examples of innovative partnerships that

focus on specific disease areas, cross-industry

research challenges

• Long-term open partnerships between industry,

academia, charities and other funders

Industry/Academic partnerships are strengthening; new initiatives are underway

Industry/Academic partnerships are strengthening; new initiatives are underway

The Experimental Cancer Medicine Centre combinations alliance – where CRUK, ECMCs, and industry work together to facilitate testing of novel combination therapy through investigator led studies Info here: http://www.ecmcnetwork.org.uk/combinations-alliance-industry

CRUK Stratified medicine programme – a large stratified medicines lung cancer trial led by CRUK, with treatment supported by AZ and PfizerInfo here: http://www.cancerresearchuk.org/funding-for-researchers/how-we-deliver-research/our-research-partnerships/stratified-medicine-programme

Genomics England – Pharmaceutical companies are working in a consortium to work with Genomics England to begin to scope how the data can be used for research into new diagnostics and treatments Info here: https://www.genomicsengland.co.uk/working-with-industry/

The new CDF will necessitate increased collaboration between stakeholders

Pre-clinical

Phase 1

Phase 2

Phase 3

Post-launch

Solutions to improve care

pathways

Clinician and Patient input into

clinical trial endpoints

Studies of real world outcomes

Training: undergraduate and

post-graduate industry placements

Search for drug targets

Academic and cross-industry

partnerships to further develop

medicines European and UK joint scientific

advice from HTA and regulatory

bodies

Clinician and commissioning perspectives on current practice

Clinician and Patient input into value proposition

CDF introduced to address poor patient outcomes and the low and slow access to cancer medicines

• 2005-2009: Evidence to show UK outcomes lagged behind other comparative

European nations

• 2010: CDF introduced

• 2016: Updated European analysis suggests UK outcomes have improved

• Despite increasing cancer incidence, since 1995 cancer mortality has decreased in

almost all countries, including the UK1

• UK male cancer mortality is the 8th lowest, but for females is the 4th highest1 (2012

figures)

• Wealthier countries tend to have better 5yr survival rates, but UK ranks 8th lowest of

28 member states2 (NB: figures for those diagnosed between 2000-2007)

• 10th lowest for breast cancer

• 9th lowest for prostate cancer

• 2nd lowest for lung cancer

• 9th lowest for colorectal cancer

The impact of the old CDF on patient survival and patient outcomes is yet undetermined; future comparative analyses will . The impact of the

new changes to the CDF will not be measureable for several years

Health spend on cancer per capita in the UK is low vs EU big 5 and Scandinavia (2014)1

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UK France Germany Italy Spain Sweden Norway Denmark

Collaboration between industry, NHS, NICE and PHE is key for new CDF

ABPI called for:

Appropriate CDF transition arrangements

Clearer alignment with the Early Access to

Medicines Scheme and the Accelerated

Access Review

Substantial evolution of NICE process and

methodology, especially for rare diseases

A fairer balance of risk between industry

and the NHS

Flexibility to accommodate global launch

processes and breakthrough medicines

• NHS E SOP live July 2016

• Industry engaged broadly with NICE, NHS

E, DH, Cancer charities to highlight areas

for concern

NHS E

NICE will now evaluate all new cancer medicines; appraisals should be concluded closer to licence

NICE ADVICE

Evidence summaries of new medicines (ESNM)

Clinical Guidelines (CCP)

Commissioning support documents (CSD)

NICE GUIDANCE

Technology appraisal (TA)

guidance

Highly specialised technologies

(HST) guidance

S

TA

M

TA

A

TA

Regional medicines

optimisation committees

Specialised commissioning

annual prioritisation

NEW MEDICINE

- All cancers

- Those referred

from DH

- Ultra-orphan

conditions

- Non cancer

- Rare diseases

- Non cancer

- Non TA/HST

- Non TA/HST

- Primary care

NHSE FUNDING

NHSE FUNDING

Spec. CommCommittee

UKMi/

Other

1 of 4 RMOCs

CCG FUNDING

FUNDING NOT MANDATED

CDF

NICE timelines necessitate earlier evidence submission for cancer medicines

Regulatory submission

Yr -3 Yr -2 Yr-1 Yr +1 Yr +2 Yr +3

CDF study protocol

CHMP

MA

NICE

committee

meeting

CDF study proposal

Study approval

Data collection

Evidence

submission

- NICE scoping workshop would be early, potentially soon after Regulatory submission

- NICE evidence submission would occur ~1yr prior to launch

- CDF study proposal would need to be discussed very early, ideally prior to Evidence submission

- Timelines for CDF review should be mindful to allow sufficient time to recruit patient numbers and

collect outcomes relevant to cancer

- Very short timeframe to analyse and incorporate results in updated evidence package

NICE

guidance

Ongoing study to deliver data

ScopeCDF

reviewNICE

guidance

Analysis

Horizon Scanning

Early and consistent dialogue between industry, NICE, NHS E, PHE

Opportunities for partnership to accelerate patient access to new cancer medicines

Regulatory submission

Yr -3 Yr -2 Yr-1 Yr +1 Yr +2 Yr +3

CDF study protocol

CHMP

MA

NICE

committee

meeting

CDF study proposal

Study approval

Data collection

Evidence

submissionNICE

guidance

Ongoing study to deliver data

ScopeCDF

reviewNICE

guidance

Analysis

Horizon Scanning

Clear process flow between EAMS, AAR and CDF

“The purpose of the Early Access to Medicines Scheme (EAMS) is to support access in the UK to unlicensed or off-label medicines in areas of unmet medical need, thus enhancing the landscape for developing, licensing and procuring innovative medicines.”Government response to the UK Early Access to Medicines Scheme Consultation –March 2014

- Strong feedback from companies on the value of EAMS

- some recommendations for process improvements

- Data collection governance, process and infrastructure needs clear process

Since launch, EAMS has awarded 9 positive scientific opinions and provided early access for over 250 patients with clinical unmet needs

31 promising innovative medicine (PIM) applications received

20 PIM designations granted

12 Scientific Opinion (SO) Applications

9 SO awarded

Clear process flow between EAMS, AAR and CDF

“The purpose of the Early Access to Medicines Scheme (EAMS) is to support access in the UK to unlicensed or off-label medicines in areas of unmet medical need, thus enhancing the landscape for developing, licensing and procuring innovative medicines.”Government response to the UK Early Access to Medicines Scheme Consultation –March 2014

- Strong feedback from companies on the value of EAMS

- some recommendations for process improvements

- Data collection governance, process and infrastructure needs clear process

Since launch, EAMS has awarded 9 positive scientific opinions and provided early access for over 250 patients with clinical unmet needs

31 promising innovative medicine (PIM) applications received

20 PIM designations granted

12 Scientific Opinion (SO) Applications

9 SO awarded

Accelerated access review implications

???

EAMS and AAR will also impact timelines and stakeholder dialogues

Regulatory submission

Yr -3 Yr -2 Yr-1 Yr +1 Yr +2 Yr +3

CDF study protocol

CHMP

MA

NICE

committee

meeting

CDF study proposal

Study approval

Data collection

Evidence

submissionNICE

guidance

Ongoing study to deliver data

ScopeCDF

reviewNICE

guidance

Analysis

Horizon Scanning

Early Access to Medicines

Scheme (EAMS)

Accelerated Access Review

(AAR)

Stakeholder alignment is needed on principles across new access processes (CDF, AAR, EAMS)

16

Enhanced Horizon

Scanning

Early and broader

NICE value

assessment

Managed Access

Agreements for

some products

Incentives in the

system for

appropriate use

Measure and

reward outcomes

Spot significant new

medicines and

classes earlier

Improve long term

national and local

budget planning

Early identification

of opportunities for

system change

(redesigned care

pathways)

Flexible type of

appraisal depending

on medicine (Full

NICE technology

appraisal (TA) /

Abbreviated TA /

Other non-TA

evaluation)

Developing a fuller

value assessment

and optimising NICE

efficiency/capacity

New route to market

needed for products

where specific

uncertainties need

to be resolved

Clear rationale for

value of MAA

Enhanced data

collection pathway

Executed well, UK patients will get access to modern medicines in a way that ensures value for money. But more

alignment is needed across parallel processes (EAMS, AAR and CDF) and their implementation should be closely

monitored

Recognition that

cost effective

technologies should

be rapidly taken up

by the system

Remove additional

layers of

assessment after

national appraisal

Local incentives to

collect long term

outcomes to

demonstrate value

Robust data

infrastructure to

measure outcomes

A single arbiter of

quality in the

system.

Independent

monitoring of health

outcomes

Emerging NHS

reforms in vanguard

sites focus on

outcomes &

integrated budgets

Industry partnership for enhanced, earlier horizon scanning

MHRAEuropean Medicines

Agency

Industry

National Horizon

Scanning Centres

Department of Health

NHS England

NICE

Pharmascan provides robust

infrastructure

Earlier signals to the system for game

changing medicines (5-7 years prior

to launch)

Better, earlier stakeholder dialogue to

plan for high impact

medicines/classes

Clear assessment pathway and

alignment between different access

assessment routes

NICE ADVICE

Evidence summaries of new medicines (ESNM)

Clinical Guidelines (CCP)

Commissioning support documents (CSD)

NICE GUIDANCE

Technology appraisal (TA)

guidance

Highly specialised technologies

(HST) guidance

STA

MTA

ATA

Regional medicines

optimisation committees

Specialised commissioning

annual prioritisation

NEW MEDICINE

- All cancers

- Those referred

from DH

- Ultra-orphan

conditions

- Non cancer

- Rare diseases

- Non cancer

- Non TA/HST

- All other new

medicines?

NHSE FUNDING

NHSE FUNDING

Spec. CommCommittee

UKMi/

Other

1 of 4 RMOCs

CCG FUNDING

FUNDING NOT MANDATED

CDF

Evolving value assessment to be fit for the future

Industry and the ABPI very keen to partner on future research: HTA decision

making, the NICE threshold and assessment methods and process

The NICE appraisal process needs to evolve to robustly evaluate the

benefits of current and future medical innovation

ensuring patient outcomes are optimised by recommending the right

medicines for the right patient

Industry would like to partner with NICE, NHS England and other

stakeholders to shape the future NICE TA process and methodology so that

it remains the cornerstone of medicines evaluation for decades to come

Partnership for co-creation of implementable managed access agreements

Flexibility

• Wide range of commercial schemes

• Outcomes-based

• Long term solutions

Clear Timelines

• Early dialogues

• Well defined process

• Regular monitoring

Measurable

• Data infrastructure

• Outcomes

• System impact

Industry partnership in improving medicines uptake and measuring outcomes

Data infrastructure

Monitoring medicines

uptake

Analysing and

Reporting outcomes

Changing the care pathway

Partnership is key to success of CDF and other new access processes

Existing

• Well established partnerships drive success in early medicines development

• Strong collaborations are developing to find solutions for priority areas of health research

CDF

• Stronger partnerships underpin new CDF process (Horizon scanning, HTA Assessment, MAAs, Data collection, Implementation)

Alignment

• Alignment of process and timelines with other key routes to access (EAMS, AAR, NHS E driven evaluations)

• Broad stakeholder alignment to embed new process