Partnering with Rare Disease Patient Groups Namrata Taak R&D External Communications, Rare Diseases
What we do Pharmaceuticals We develop and make medicines to treat a range of conditions including respiratory diseases and HIV/AIDS 4bn packs of medicines in 2014 Vaccines We research and make vaccines for children and adults that protect against infectious diseases 800m doses of vaccines in 2014 Consumer Healthcare We make a range of consumer healthcare products in four categories: Wellness, Skin health, Oral health and Nutrition 18bn packs of consumer healthcare products in 2013 2
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
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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
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
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, nonpromotional manner. GSK Chief Medical Officer. 7
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
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
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.
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.
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.
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
20 th September 2014 - The Day the Ph III drisapersen study results went public... Shock to the community... Complete shock We can't see him on a wheel chair May the good Lord guide you and your entire company in the 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
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
Umbrella organisations
17 THANK YOU Contact at GSK Rare Diseases Unit Fabrizia Bignami Head of Patient Engagement and Public Affairs, fabrizia.x.bignami@gsk.com