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

Hope is high… expectation is high… the stakes are high Complex service planning to facilitate patient access ‘ 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 ’ UNNAMED PARTICIPANT, JP MORGAN HEALTHCARE CONFERENCE 2019 Long-term World class value needs to be communications demonstrated to connect & inform

We are entering an exciting period of high expectation Patients Unprecedented Sustainable challenges in the outcomes Bio- Pharma adoption of are needed for advanced all stakeholders therapies Health systems We are all learning together to change lives with advanced therapies

This booklet is designed to: OPEN Health brought together all of our capabilities to inform our thinking M E D I C A L P A T I E N T & B R A N D V A L U E , I N F O R M A T I C S C O M M U N I C A T I O N S C O M M U N I C A T I O N S & E V I D E N C E P H A R M E R I T L E A D I N G S H A R E D L E A R N I N G

Strategies in gene therapy need to be built on a foundation of: Patient first approach Communicating Demonstrating value the science

Patient First Divider Approach

Every patient is making a life altering decision How long will the effect last? How safe is it? What will happen at the COE? Where will I go? COE: Centre of Excellence

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

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

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

Principles underpinning PAG engagement and partnership 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 T R A N S P A R E N C Y individual disease areas. O F I N T E N T PAG – Patient Advocacy Groups

Communicating the Science

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

Communicating the Science Disease education Gene therapy education Pathway understanding 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 Education on the disease is an imperative communication pillar for No ‘one size fits all’ approach but core salient points are: any gene therapy because it sets up: • Appropriate patient identification Better disease management • Better treatment outcomes • Diagnosis Clinical consensus

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

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 of a gene therapy in rare disease: 1 2 3 4 Manufacturing Reimbursement The burden of Paucity of processes and pathways aren’t treatment must safety and supply chains well defined be considered efficacy data are complex

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

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

Case Study: Mobilising broad capabilities to deliver outcomes G E A R E D P O W E R E D with the by a technology patient platform at core Placing the patient at the centre of long term data collection and Outcomes follow up post gene therapy Monitoring D R I V E N SUPERCHARGED by exceptional with stakeholder project communications management

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