retaining independence; dealing with competition & staying in - PowerPoint PPT Presentation

Working with Pharma – retaining independence; dealing with competition & staying in control Tanya Collin-Histed Chief Executive, UK Gauchers Association & CEO European Gaucher Alliance (EGA) Find a Cure Friday 12 June 2015 Find a Cure, Friday 12 June 2015

Setting the scene • Established in 1991 • One company, one drug • Now three ERTs and two oral treatments for Type 1 GD and visceral symptoms of Type 3 – 301 patients • High cost drugs, cost savings by NHS and challenges getting drugs through HTA – Even more important that Patient Organisations retain their independence and ensure the needs of the patients are at the centre Find a Cure, Friday 12 June 2015

Independence Financial support 1. One company 2. Multiple companies 3. Specific project funding Find a Cure, Friday 12 June 2015

Interpretation of the ABPI Code Different Interpretations • Supporting events • Patient Organisations representatives attendance at scientific meetings Find a Cure, Friday 12 June 2015

Dealing with Competition • Pressure on patient organisations • Resources • Skills • Trust – use of CDA • Code of conduct Example ; Eastern European Country patient group struggling to become independent Find a Cure, Friday 12 June 2015

Staying in Control Patient need V Pharma sales – Different agendas – Pay the Parent role – Always try to work with other stakeholders – What is Pharma’s role? Example ; • Raising awareness in the Jewish community • Pharma Patient Information literature/websites Find a Cure, Friday 12 June 2015

Humanitarian Aid • EGA has a history of supporting patients who are not able to access treatment through their health system – Genzyme’s European Access Programme (ECAP) – Shire’s Charitable Access Programme (CAP) Find a Cure, Friday 12 June 2015

Patient Need v Pharma Challenges Drug Shortage • In 2009 there was a virus at Genzyme’s plant in the US that effected the production of Cerezyme to the Global patient community • EWGGD and EGA convened a meeting of all companies • Genzyme clinical trial with oral drug, comparison arm • Patient community and clinicians challenged the use of the drug for it’s clinical trial • ERT released back to patient community Find a Cure, Friday 12 June 2015

Developing a New Drug • In early 2015 the Gauchers Association gained Orphan Designation for Gene Therapy for GD • Affordable accessible treatment for unmet need • Started to talk to a pharma company that could help us to progress this, using our disease as a proof of concept for a larger disease area. • Intellectual Property (IP) • Patient organisation submitting business case not academia • Scientific advice, protocol assistance, clinical trials • Project group – lead and funded by PO Find a Cure, Friday 12 June 2015

Working with Pharma • • Trial recruitment Proving work placements • Linking Pharma to Academic • Patient Information Leaflets • Lobbying Government • Diagnosis pathway surveys • Educating Doctors and Nurses • Research design • Developing new Patient Groups • • Burden of Disease questionnaire Representing the patient community on steering groups, • Patient Related outcome panels • Educating staff • Acting as a communications hub • • Patient Awareness Literature Humanitarian Aid – Developing company programmes – Getting drug to patients Find a Cure, Friday 12 June 2015

Summary • They need patient involvement, make it very clear you are not there to simply tick a box but to make a valuable contribution • Don’t be intimidated, they don’t know what patients and parents go through everyday, you do • Always ask questions and ensure they provide the answers even if its after the event • Accept the fact that you will not always agree, be honest and always be the patient’s advocate they will respect you or that • Despite everything they will sometimes do what they want anyway, don’t say I told you so, reiterate what you said and move on • Always know what you want out of a meeting/project and be very clear at the beginning what this Find a Cure, Friday 12 June 2015