Partnering with Rare Disease Patient Groups Namrata Taak R&D - PowerPoint PPT Presentation

Partnering with Rare Disease Patient Groups Namrata Taak R&D External Communications, Rare Diseases

What we do Pharmaceuticals Vaccines Consumer Healthcare We develop and make We research and make We make a range of consumer medicines to treat a range vaccines for children and healthcare products in four of conditions including adults that protect against categories: Wellness, Skin respiratory diseases and infectious diseases health, Oral health and HIV/AIDS Nutrition 4bn 800m 18bn packs of medicines doses of vaccines packs of consumer healthcare in 2014 in 2014 products in 2013 2 Property of GSK – not for further use or distribution

Our business today Putting patients and consumers first Major research and development facilities in 3 continents 84 Manufacturing facilities in 36 countries in 2014 3 Property of GSK – not for further use or distribution

4 4 Property of GSK – not for further use or distribution

Why does GSK discover and develop medicines for Rare Diseases? • Treatment of the rare disease itself • Creates a window into human biology with potential application in more common diseases • For the goal of repositioning existing assets (generally for common diseases) for rare indications • To apply new technology approaches in a clear and tractable area e.g. ‒ Gene and cell therapy ‒ Oligonucleotides • Common diseases may become reclassified as an amalgam of rarer diseases 5 Property of GSK – not for further use or distribution

Key Success Factors – Innovation – Early validation process – Listening to patients, care givers and patient groups – Creative development program design – Transparency – Open and early engagement with regulators 6 Property of GSK – not for further use or distribution

CMO Message “We have an obligation to learn from physicians and patients and to make sure we provide accurate and complete information to them through appropriate channels; but we must do this in a careful, correct, non- promotional manner. ” GSK Chief Medical Officer. 7 Property of GSK – not for further use or distribution

Engagement with PAGs – Engage pro- actively “to gain patient insight and perspective of a disease, its impact on patients and carers. This will inform disease understanding, trial design, and advance development of products that best meet the needs of patients” Example : Ad-hoc Advisory Boards on specific topics – Upon unsolicited requests, provide key updates of clinical studies/plans Key Sources : GSK clinical study register, clinicaltrials.gov, congress plans, scientific publications, press releases 8 Property of GSK – not for further use or distribution

Case Study 1: MRI study in Hunter Disease (HD) • The objective of study was to determine whether a particular imaging technique could be applied to the case of HD patients in order to measure the effect of an investigational medicine. • Challenge was not only that HD is a rare disease, but also to identify the rare CNS affected HD patients still capable of taking part in the study Property of GSK – not for further use or distribution

Case Study 1: MRI study in Hunter Disease • An agreement with MPS Society UK signed to help: – identify suitable patients that meet the inclusion criteria – Arrange travel, accommodation, and reimbursements of out-of pocket expenses – provide individual support to patients and their parents/ carers throughout the study • Collaborative approach with support from experts • Study design and protocol received inputs both from patients and experts. This experience demonstrated collaborative work with patient groups to help drug development. More than in any other field rare diseases patient groups are truly centers of knowledge and expertise. Property of GSK – not for further use or distribution

Case Study 2: ADA SCID Gene Therapy • US and EU patient groups engaged in a dialogue to complete our understanding of patient and family journey from diagnosis to care and treatment • primary immune deficiencies (PID) • adenosine deaminase severe combined immunodeficiency syndrome (ADA SCID). • Also identified the needs and expectations of this community. Property of GSK – not for further use or distribution

Case Study 2: ADA SCID Gene Therapy Development of a post marketing evidence generation strategy. If gene therapy (GT) for ADA SCID is approved, an ADA SCID gene therapy registry will be required and will be:  The core of the ADA SCID GT Risk Management Plan,  The evidence generation tool that will track safety signals and will confirm and support the value of the product over the long term. Unique challenges compared to any existing drug registry. Property of GSK – not for further use or distribution

Case Study 3: Duchenne Muscular Dystrophy (DMD) Patient Group Meetings: Sharing GSK updates on the drisapersen clinical programme when public – keeping the patient community up to date with our progress. Patient Case Studies : Robust case studies use the experience of investigators and patients to highlight the key effects of a treatment in a format that is accessible. These emotive case reports prepared by drisapersen investigators showed the human impact of the disease and treatment, which were informative to GSK in drug development and may help payers to help secure reimbursement 13 Property of GSK – not for further use or distribution

20 th September 2014 - The Day the Ph III drisapersen study results went public.... Shock to the community............ “Complete “May the good shock” Lord guide you and your entire “We can't see him company in the on a wheel chair” right path” “ Please restart the program and save the life of the younger kids” “Falling “ Trying to come to grips with the apart” enormity of what has transpired over the last week ” 14 Property of GSK – not for further use or distribution

Sequence of Events – Rapid Patient Community Engagement – Day Zero: Press Release goes Public – Day Zero: E-Communication to Patient Groups Heads & Multiple 1:1 Phone calls to provide support – Day One: Start of multiple enquiries from patients and parents +++ – each individually and personally answered – Day One: Teleconference with Patient Groups to discuss results, how best to support them and planned next steps – Day 10 : External Q & A Provided to Patient Groups to support them with enquiries from their members – this is then posted onto multiple PAG websites – Day 11: WMS Congress Medical Booth to provide a face to face extension of our medical information service – Day 17: Global Webinar organised for the patient community to help answer any questions – recording and also slides provided for PAG websites – November : GSK invited to provide update at Patient Conference (Action Duchenne) 15 Property of GSK – not for further use or distribution

Umbrella organisations Property of GSK – not for further use or distribution

THANK YOU Contact at GSK Rare Diseases Unit Fabrizia Bignami – Head of Patient Engagement and Public Affairs, fabrizia.x.bignami@gsk.com 17