real world evidence (rwe) - · pdf fileagenda agenda presenter meeting objectives &...

Real World Evidence (RWE)

Deborah Wyatt

DIRECTOR

Office +44 (0) 1252 849074

Mobile +44 (0) 7712 333 844

[email protected]

talkhealthpartnership.com

Vaneet Nayar

Marketing Director

Mobile: +44 (0) 7429 592554,

Office: Tel: +44 (0) 1252 848848

[email protected],

www.svmpharma.com

Click here: Real World Evidence http://bit.ly/svmresources

mailto:[email protected]

http://www.talkhealthpartnership.com/


Agenda

Agenda Presenter

Meeting Objectives & Introductions Vaneet Nayar

Defining Real World Evidence?Why do we need RWE?

Group discussion

Patient engagement and experience Deborah Wyatt

“RWE” – the legal issues!

We will explore how the current RWE landscape must be carefully navigated to avoid the legal pitfalls – and why things aren't getting any easier

Simon Briskman

Driving Real World Evidence outcomes Vaneet Nayar

Summary & Close Vaneet Nayar

What you need to know

• Fire exit

• Refreshments

• Wash rooms

Meeting Objectives

What is the value to the NHS and Pharmaceutical industry of combining Real World Evidence and Patient Experience?

Introductions

Delegates

• Sophia Marsh

• Jane Paxton

• Stewart West

• Ingrid Omerovic-Smith

• Simona Aganovic

• David Marsden

• Mike Wakeman

Delegates

• Simon Briskman - Fieldfisher

• Deborah Wyatt – talkhealth partnership

• Catriona Williams - talkhealth partnership

• Vaneet Nayar - SVMPharma

• Gem Auddy- SVMPharma

• Fouad Lawal- SVMPharma

The growing importance of Real World Evidence

“It is increasingly recognised that conclusions drawn from classical clinical trials are not always a useful aid for decision making. Assessing the value of a drug or technology

requires an understanding of its impact on current managements in a practice, real life setting”

“Research conducted that professionals are becoming focussed on real world data, with over 70% of those surveyed expecting their time spent working with real world

data to increase over the next 2 years”

Real Life Data: A Growing Need - www.ispor.org/news/articles/oct07/rld.asp accessed 31 March 2014 Real World Data Report 2013

http://www.ispor.org/news/articles/oct07/rld.asp

Transitioning from clinical trials to “real world” clinical practice

Clinical Trials Real Outcomes (clinical and patient)

Prospective data collection Prospective and/or retrospective

Limited population are eligible for inclusion Broader patient population

Good patient adherence and compliance

Real world patient experience

Ideal for registration of medicines Demonstrates the benefits in usual clinical practice

Limited ability to demonstrate cost effectiveness Ideal for demonstrating value within local health economy

The problem with clinical trials: The need for ‘real world’ studies

• Can only describe the efficacy of an intervention under optimal conditions

• May not demonstrate the effectiveness of an intervention in the real world

• It is increasingly recognised that conclusions drawn from classical clinical trials are not always a useful aid for decision making

• Assessing the value of a drug requires an understanding of its impact on current managements in a practice, real life setting

• Engaging with patients everyday clinical experience

Committee on Comparative Effectiveness Research Prioritization, Institute of MedicineRealLife Data: A Growing Need - www.ispor.org/news/articles/oct07/rld.asp accessed 31st March 2014

http://www.ispor.org/news/articles/oct07/rld.asp

Traditional model for data collection

Clinical outcomes

Patient experience

Clinical trials Phase 1-3

Licence

Future model for data collection

Outcomes

Quality of life

PROMS

Clinical outcomes

Patient experience

Perception

Quality of life

Clinical trials Phase 1-3

Licence

What is RWE and its value to the NHS

• What is your definition of RWE?

• What is the value of Real World Evidence to the NHS and Pharmaceutical industry?

NB: please take post-it notes and write down your definitions and value and place them on the flip charts

Definitions

RWE definition

• Clinical data outside of traditional randomised clinical trials– Clinical and patient outcomes

RWE value

• Evaluating patients outcomes in ‘every day’ clinical practice?– Determining the impact on the NHS

– Patients experience within the NHS

Patient Engagement and Experience

Deborah Wyatt

What Patient Experience

Patient experience is about considering a disease, and the challenges of living with that disease, from a new perspective; the patients or carers point of view.

Listening to patients should be seen as a powerful motivator and driver for improvement and change.

It is not enough to make assumptions that we know what it is like for patients and their carers, or listening to second or third hand feedback. It is about taking the step of actively finding out and involving patients and carers which is critical when designing or changing a service.

http://www.institute.nhs.uk/qipp/joined_up_care/patient_centred_care.html

http://www.institute.nhs.uk/qipp/joined_up_care/patient_centred_care.html

Engaging Directly with Patients

• Why engage directly with patients?

• Ways of engaging with patients

• Innovative approach

Why Engage Directly with Patients

In the competitive UK healthcare market, engaging with patients directly may lead to…

– Understanding and improving patient experience

– Design, re-designing or refining service delivery

– An opportunity to engage directly with to patients to understand their disease from their perspective

Ways of Engaging with Patients

Traditional approach – qualitative and quantitative research

Innovative approach – patient engagement & insights

• Qualitative & quantitative together

• Using new digital technology

• Collaborative partnerships

• Insights that support changes to patient care and outcomes

MS Case Study – patient experience

The requirement

• Understanding MS patients’ experience and the day to day challenges of living with MS

The Methodology

• Cohort of 36 patients & 10 carers providing insights through online survey

The outcomes

• 38% of respondents considered the information and support was poor or fair at diagnosis

• 26% of respondents were “somewhat dissatisfied” or “very dissatisfied” that information about their condition had not been shared effectively between those involved in their care

Objectives

• Develop concept of patient experience survey

• Validate methodology

• Share results with MS charities

• Publish MS survey findings

Psoriasis Case Study – patient record study

The requirement• Discover how psoriasis patients manage their condition day to day • Gather insights about treatments used to control their condition• Gather insights about treatments patients wish they could have• Contact those who consented for general case studies

The result• 700+ patients with psoriasis completed detailed survey, providing insights • 31 patients agreed to be the subject of a case study

What did we do next?• Helped the client to evaluate their product range• Developed disease awareness programme based around education & adherence• Developed case studies – used in disease awareness campaign

Final thoughts …

“Do listen to your patients; always have them at the centre of your thinking, that’s what makes the big, big difference”1

1 www.institute.nhs.uk - Patient Centred Care

http://www.institute.nhs.uk/

Brussels / Düsseldorf / Hamburg / London / Manchester / Munich / Palo Alto / Paris / Shanghai / fieldfisher.com

Big Data

Simon Briskman, Partner – Technology, Outsourcing & Privacy Group

11 March 2015

Brussels / Düsseldorf / Hamburg / London / Manchester / Munich / Palo Alto / Paris / Shanghai / fieldfisher.com 22

Fieldfisher – converged thinking on data law

Regulation

Intellectual Property

Technology


Two worlds colliding


Big Data is about Data Monetisation

Data monetization … involves maximizing the revenue potential from

available data by institutionalizing the capture, storage, analysis, effective

dissemination, and application of that data. Said differently, it is the process

by which corporations, large and small, leverage data to increase profit and

efficiency, improve customer experience and build customer loyalty. The

practice, although common since 2000, is now getting increasing focus as

regulatory … and economic pressures increase on businesses...”


What risk?


What risk?

• Fines – 2% of worldwide turnover

• Consumer trust

• European employee privacy

• Data regulation: Financial firms, SOX etc


Big Data: Typical legal issues

• Context is everything

• Personal data and consent based approach

• Copyright and proprietary information

• Confidential information

• Social media

• Other restricted sources - HSCIC, clinical trial consent forms etc.


Recognise this lady?


Data protection reform 101

• Root and branch reform

• Key aims:

– improve harmonisation;

– reduce bureaucracy;

– Improve individuals rights;

– Enhance organisational accountability;

– Strengthen regulators’ powers.

• Expected to be in force around 2015/2016

• Fines (2% global turnover) and mandatory audits


Key reform issues for Big Data…

Issue Now Incoming

Data minimisation Data must not be “excessive” Data “limited to the minimum necessary”

International data

transfers

Prohibited unless “adequate” protection in place.

UK controllers can exercise discretion.

Generally prohibited unless “adequate” protection

in place. No discretion for UK controllers. Strong

emphasis on BCR.

Accountability No real concept of accountability, other than

limited transparency requirements.

Extensive accountability requirements inc.

transparency, data breach notification within 72

hours, DPOs, PIAs, PbD, audits.

Right to object Data subject must show “compelling legitimate

grounds”

Data controller must show “compelling legitimate

grounds”

Right to be forgotten Google v DPO Spain – very narrow rights to

erasure if data old and irrelevant

Data subject has mandatory right to require erasure

of its data

Right to be forgotten removed from latest draft

GDPR

Consent “Freely given, specific and informed” “Freely given, specific, informed and explicit”

Consent shall be purpose-limited


Managing big data risk

• Planning for flood

• Professional risk and impact assessment

• International risk

• Getting it right first time


Back to two worlds

Stakeholders in Big Data projects need to understand and assess the risks together:

• Reach out

• Find a common language

• Accommodate each other’s thinking

• Build long lasting solutions


Thank you!

Simon BriskmanPartnerTechnology, Outsourcing & Privacy Group

E: [email protected]: +44 (0)20 7861 4145

Cayston – Postive NHS outcomes

Real World Evidence

Background

Data gaps in Phase 3 cystic fibrosis studies

• Lack of combination antibiotic regimens

• Patient cohort very restrictive in clinical trials and evidence is lacking for patients generally seen in clinical practice

• Duration of therapy shorter

Clinical studies and access

• Specialist commissioning England

• No funding in Wales and Scotland

• No new clinical studies in pipeline

Project overview & timeline

• Project was conducted over 24 months from January 2012 to January 2014

• Establish a ‘Steering Committee Group’ (9 centres: Ireland and UK)

• Develop online Cayston Treatment Evaluator

• Data was extracted for 1 year pre- and up to 1 year post-Cayston

Develop online treatment evaluator Identify steering group

Define data collection parameter with each centre

Data collectionData entry by centres, analysis & review by steering group

Data analysisFinal data entry by centres, analysis & review by steering

group

Abstract submission

Feb 2012

Dec 2012

Jan 2013

Jan 2014

Positive resubmission to SMCJan 2015

Patient confidentiality

• All patient data was pseudo-anonymised and a unique patient ID created by the database

• Each centre was only able to see their own (anonymised) patients details and aggregated anonymised for the other centres

• Each centre was responsible for gaining local approval to share their data with the group

• The data was owned by the steering group, and managed by a third party (SVMPharma Limited)

Positive resubmission to SMC

• The clinical data used in the analysis came from the Cayston Treatment Evaluator (CTE), a retrospective database which collected data from 53 patients across nine cystic fibrosis centres in Ireland and the UK

• It should be noted that a subgroup analysis was also conducted in 31 patients who were not obtaining satisfactory therapeutic benefit with an inhaled tobramycin based regimen.

• SMC restriction: When inhaled colistimethate sodium and inhaled tobramycin are not tolerated or not providing satisfactory therapeutic benefit (measured as ≥2% decline in forced expiratory volume in 1 second [FEV1]).

Xiapex: Postive NHS outcomes

Real World Evidence

Xiapex (Collagenase) Timeline

2011 Xiapex Licenced in EU

Minimal Use in UK

Most decision makers awaiting

further advice

2011 ELHT performs Cost Analysis and Commissions Drug

18 Month RWE Data Collection of

Use Begins

AWMSG Approval

SMC Approval

2014 Findings of RWE Data Collection

Presented

Data contributed to NICE Review

Background

• Xiapex (Collagenase) is the only non-surgical treatment for Dupuytren’s Contracture

• It is administered directly via injection

• It was licensed for use in the EU in 2011.

• Dupuytren’s contracture is a condition that affects the hands and fingers. It causes one or more fingers or the thumb to permanently bend into the palm of the hand.



Minimal Use in UK


further advice

2011 ELHT PCT Cost Analysis and Commissions Drug


Use Begins

AWMSG Approval

SMC Approval


Presented


Local commissioning body takes the lead

• East Lancashire PCT undertook a cost analysis in 2011 which found that Xiapex pathway was more cost-effective than surgery (£1700 versus £3000)

• They implemented a change in their policy for the purposes of: – Promotion of innovation and efficiency

– Encouraging emerging outcomes

– Supporting positive patient experience

• They initiated a 18 month Real World Evidence (RWE) data collection process for all patients using Xiapex

• Following this both AWMSG and SMC both approved use in 2012



Minimal Use in UK


further advice

2011 ELHT PCT Cost Analysis and Commissions Drug


Use Begins

AWMSG Approval

SMC Approval


Presented


Results of RWE Data collection

• Results of RWE data collection programme was presented in 2014

• Collagenase (Xiapex) injection has been the treatment option of choice for 68 patients since 2011 approval

• Results demonstrate – Significant improvements in the extension of the digits

– Quicker return to normal function

– A shorter period of hand therapy

• The results are “very promising” with high patient satisfaction.

• Results part of the NICE HTA



Minimal Use in UK


further advice

2011 East Lancashire PCT Cost Analysis and Commissions Drug


Use Begins

AWMSG Approval

SMC Approval


Presented


Visualisation In Big Data & Healthcare Analytics

A Cystic Fibrosis Case Study

Why do we need visualisation tools?

• Traditional written, tabular and graphical formats can be inadequate in conveying the findings

• Key details can be lost in summarising and aggregating the data.

• There a number of visualisation tools and solutions which have been proven to be valuable in identifying relationships and conveying information.

Cytoscape Gephi Nodebox

Tableau Circos

Cystic Fibrosis visualisation using Gephi

• SVMPharma carried out a data visualisation exercise using Gephi to show the multi-disciplinary nature of Cystic Fibrosis (CF) by highlighting its co-morbidities and associated health problems.

• 15,000 Cystic Fibrosis patients who visited hospital from 2010-2014 were selected by their ICD-10 code (diagnosis code) from the Hospital Episodes Statistics (HES) dataset.

• This provided 3,800 different ICD-10 codes and 25,000 different connections between them. The data was entered into Gephi, with each ICD-10 code represented by a node, and each different connection between them via a line.

From tables to visualisation

A Closer Look

Diseases grouped via position and

colour

Node size and line width represents

number of connections

Contact us

Deborah Wyatt

DIRECTOR

Office +44 (0) 1252 849074

Mobile +44 (0) 7712 333 844

[email protected]

talkhealthpartnership.com

Vaneet Nayar

Marketing Director

Mobile: +44 (0) 7429 592554,

Office: Tel: +44 (0) 1252 848848

[email protected],

www.svmpharma.com

Click here: Real World Evidence http://bit.ly/svmresources


http://www.talkhealthpartnership.com/


http://www.svmpharma.com

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