Collaborating with patients: learning from psoriasis and other diseases

Transcription

1 Collaborating with patients: learning from psoriasis and other diseases Marilyn Metcalf, PhD Sr. Dir., Benefit Risk Evaluation Chief Medical Office EveryLife Foundation for Rare Diseases September 15, 2015 Washington, DC On behalf of Kay Warner, Manager, Focus on the Patient Fabrizia Bignami Head, Patient Engagement and Public Affairs, Rare Diseases Unit Namrata Taak, Global Pipeline Comms Dir. Sue Vallow, Head, Patient Focused Outcomes

2 From our Chief Medical Office 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. - Scientific Engagement Global Standards 2

3 Listening to and learning from patients and carers 11 years ago GSK established the program Focus on the Patient Brings patients to GSK sites Main component is awareness seminars Hear directly from patients, caregivers and advocates Raise general awareness of the multidimensional aspects of a disease clinical course, effects on daily life, personal experiences Provide summary of GSK efforts to meet patient needs holds a mirror up to us on where we re hitting the mark and where we re falling short 3

4 Patient and employee feedback The timing was perfect since we are working on one of our projects for Scleroderma. Having a chance to meet with the patients ahead of this was an excellent experience for me. John, Discovery Medicine, BioPharm 4

5 Recent and future Focus on the Patient topics 2013: Neglected tropical diseases, COPD, type 2 diabetes, gene therapy, HIV, chronic hand ezcema, Duchenne muscular dystrophy 2014: Cancer care in Africa and our alliance with NGOs in the region, severe aplastic anemia, motor neurone disease (MND) including amyotrophic lateral sclerosis (ALS), eosinophilic granulomatosis with polyangiitis (EGPA), pediatric ITP 2015: Breast cancer, zoster/vaccines, Parkinson s Disease, severe eosinophilic asthma, respiratory, metachromatic leukodystrophy (MLD) 5

6 23 July 2014, BERLIN Case Study: Psoriasis December 2012 to January 2013 Focus on the Patient partnership with Biopharm Discovery Medicine & Commercial Strategy Held individual interviews at GSK, Learned patient insights and perspective of their disease, its impact on them and carers Influenced study endpoints for clinical trials, target patient population, device design changes Sent letter to patients thanking them for participation and outlining key collective findings More teams seeking patient insight for a variety of reasons 6

7 Learning to meet challenges Identifying patients: good networks are key Written language in legal contracts: more reader friendly Understanding internal polices and the external Code of Practice we can and should talk to patients Early phase drugs are less likely to succeed, so earlier involvement will include work on drugs that have less chance of success BUT learning isn t wasted we carry it forward 7

8 Votrient in advanced soft tissue sarcoma trial design Patient voices improved the Phase II first line maintenance study of Votrient in advanced soft tissue sarcoma The drug development team was better able to select disease symptoms measured as a trial outcome, along with the time-frame in which data was collected. The early discussion with patients improved the collection process of patient reported symptoms Streamlined design Trial less burdensome but more relevant to patients 8

9 Case Study: 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: Experience of investigators and patients highlight the key effects of a treatment in an accessible format. Case reports prepared by drisapersen investigators showed the human impact of the disease and treatment, which were informative to GSK in drug development and could inform payers to help secure reimbursement. 9

10 From Case Reports, EUPATI WORKSHOP MEANINGFUL PATIENT INVOLVEMENT IN INDUSTRY-LED MEDICINES R&D 23 July 2014, BERLIN 10

11 20 th September 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 11

12 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) 12

13 The Community Advisory Board (CAB) of patients and caregivers was loud and clear about what was important to them, to us prioritize slowing or halting disease progression, recommend no or minimal exposure to placebo, encourage trials that include all individuals (young, adult, walking and non-ambulatory), use regulatory tools such as Accelerated Approval a loud urgent voice for unmet needs. GSK was privileged to be one of many contributors to the June 2014 community led guidance that preceded FDA s post 13

14 Drisapersen under review for treatment of some Duchenne patients 14

15 Case Study: ADA SCID Gene Therapy US and EU patient groups engaged in a dialogue to enhance our understanding of the patient s journey and that of their family from diagnosis to care and treatment primary immune deficiencies (PID) adenosine deaminase severe combined immunodeficiency syndrome (ADA SCID). Also identified needs and expectations of this community. 15

16 An application is under consideration 16

17 Our gratitude to our patients and their families, and to our other collaborators Patrick Vallance, President of Pharmaceuticals R&D at GSK, said: I am very pleased that we are now at a stage to file for approval of this gene therapy for ADA-SCID a devastating rare disease which drastically limits and shortens patients lives. Development of this therapy would not have been possible without the immense support of the ADA-SCID patients families and the work of so many pioneering scientists who have worked in this cutting edge area of medicine for decades. We are privileged to take forward this submission. Press Release 05 May 2015: GSK, Fondazione Telethon and Ospedale San Raffaele announce EU regulatory submission for gene therapy to treat rare disease ADA-SCID 17

18 Case Study: 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. 18

19 Growing collaborations, more patient leadership EUPATI Patient-led five year project under the Innovative Medicines Initiative (IMI) launched in February 2012 GSK provides in-kind contribution Helps patients prepare for participation in scientific, ethical and regulatory committees that can accelerate and improve clinical trials, drug development and access strategies. FasterCures Center of the Milken Institute, an action tank with the goal to save lives by speeding up and improving the medical research system September 2014 Benefit-Risk Boot Camp, led by and designed for patient organization staff members, patient advocates, policy makers, and other stakeholders to hear directly from experts about enhancing patient-centered decision-making about medical products GSK participated in Boot Camp and continues on FC s Benefit-Risk Advisory Council 19

20 Implementing through internal efforts, e.g., Patient Engagement Plans Contents Patient Engagement Plan for [Disease] Programme Introduction Understanding the [Disease] patient journey Defining the [Disease] patient care-path General [Disease] Patient Engagement Communication Plan Summary of Activities Immediate Implementation Appendix A: [Disease] Patient Advocacy Groups & Foundations Appendix B: Communication Structure to Support Programme 20

21 What next? Continue participating in patient-led and patient co-led efforts Continue to point drug development teams to patient engagement resources Look for new opportunities moving forward 21

22 THANK YOU 22