Translating the science and value of gene therapy to key stakeholders - - PowerPoint PPT Presentation

Succeeding with Star Wars medicine: Translating the science and value of gene therapy to key stakeholders

UNNAMED PARTICIPANT, JP MORGAN HEALTHCARE CONFERENCE 2019

‘ S T A R W A R S M E D I C I N E I N T H E A G E O F F L I N T S T O N E R E I M B U R S E M E N T ’

Complex service planning to facilitate patient access Long-term value needs to be demonstrated World class communications to connect & inform

Hope is high… expectation is high… the stakes are high

We are all learning together to

change lives with advanced therapies

We are entering an exciting period of high expectation

Unprecedented challenges in the adoption of advanced therapies Sustainable

• utcomes

are needed for all stakeholders Patients Bio- Pharma Health systems

L E A D I N G S H A R E D L E A R N I N G

This booklet is designed to: OPEN Health brought together all of our capabilities to inform our thinking

M E D I C A L C O M M U N I C A T I O N S P A T I E N T & B R A N D C O M M U N I C A T I O N S V A L U E , I N F O R M A T I C S & E V I D E N C E P H A R M E R I T

Strategies in gene therapy need to be built on a foundation of:

Patient first approach Demonstrating value Communicating the science

Divider Patient First Approach

How safe is it? Where will I go? How long will the effect last? What will happen at the COE?

Every patient is making a life altering decision

COE: Centre of Excellence

A patient first approach

Key early strategies to underpin a patient first approach Patient Centred Outcomes Patient, care-giver and HCP journeys Burden of Illness

A patient first approach to decision led communications:

Enabling informed consultations through insight driven communications grounded in health psychology Connecting HCPs across pathway to ensure positive patient experience at every stage Creating informative and accessible materials to inform on process, stakeholders and experience

Educating Connecting Informing

A patient first approach to value demonstration:

Engaging patients early in clinical development to ensure relevant PROMs are selected Gathering data on patient and carer burden of illness and patient preference to support value dossier Building an aspirational value proposition and testing this with the patient community to ensure messages resonate

Consulting Collecting Assessing

In engaging with the patient community, thought needs to be given to compliant activities particularly when there is only limited numbers of treatment and companies working in individual disease areas.

T R A N S P A R E N C Y O F I N T E N T

Principles underpinning PAG engagement and partnership

PAG – Patient Advocacy Groups

Communicating the Science

Best practice scientific communications

Map the therapeutic ecosystem Collaborate to build deeper insight Optimise digital channels Maximise your KOLs Led with peer-to-peer communication

Communicating the Science

The core principles of medical communications are the same in gene therapy as they are in all other medicines: demonstrating efficacy and safety is the basic requirement Disease education Pathway understanding Gene therapy education

No ‘one size fits all’ approach but core salient points are:

Clinical consensus Diagnosis

Disease Education

Education on the disease is an imperative communication pillar for any gene therapy because it sets up:

• Appropriate patient identification
• Better disease management
• Better treatment outcomes

Gene Therapy Education

Encompass the basic building blocks of: Crucial to this communication is that it is delivered by trusted, well-respected KOLs This will go a long way in reassuring HCPs

Mode of action Efficacy Safety

Pathway understanding

• Centres of Excellence (COE) and their physicians are fundamental to the safe and effective

uptake of a gene therapy

• They will need to:
• Share valuable experience from the clinical programme
• Build capacity to accommodate a safe and effective flow of patients
• Educate referring physicians on optimal patient identification
• Support patients and care-givers make final decisions on whether gene therapy is right for them
• Initiate long term outcomes follow up programmes

Pathway understanding

• Whilst COE physicians are vital for safe and effective initiation of treatment referring physicians

are critical stakeholders to support sustained uptake

• They will need to:
• Identify eligible and optimal patients
• Confidently answer patient and care giver questions
• Understand the referral pathway to the COEs
• Monitor and potentially treat re-emerging disease symptoms
• Be connected to long term follow up outcomes monitoring

Demonstrating Value

Demonstrating Value

There are four core challenges to demonstrating the value

• f a gene therapy in rare disease:

Manufacturing processes and supply chains are complex Reimbursement pathways aren’t well defined The burden of treatment must be considered Paucity of safety and efficacy data

1 2 3 4

Best practice recommendations to help demonstrate value beyond the clinic

• Collaborate with PAGs to define patient value
• Co-create innovative financial models
• A patient-led approach to understanding the burden of disease and treatment
• Co-create patient and health system centred approaches to outcomes monitoring

– Managed access – Innovative financial models – Long term follow up

Delivering cohesive strategies to deliver better outcomes

Meeting hope and expectations through:

Innovative tools to facilitate positive communications Interconnected communications strategies Clear and sustained value argumentation Put the patient at the centre of all thinking

OPEN Health

• A global healthcare communications, evidence and access company with extensive

experience and expertise in rare disease and oncology

• Our best-in-class practices work together seamlessly to provide bespoke solutions that

meet the unique challenges and deliver world-class communications and tools to enable them

• OPEN Health has lots of experience in commercialising gene and other advanced therapies
• Our practice experts are mobilised to accelerate their learning and capabilities and be

fully prepared to deliver for our clients

Placing the patient at the centre

• f long term data collection and

follow up post gene therapy Outcomes Monitoring

P O W E R E D by a technology platform SUPERCHARGED with stakeholder communications G E A R E D with the patient at core D R I V E N by exceptional project management

Case Study: Mobilising broad capabilities to deliver outcomes

For further information, please contact:

Gavin Jones +44 (0) 7795 038952 gavinjones@openhealthgroup.com

Delivering world-class tools and communications to enable outcomes with advanced therapies