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

Find a Cure, Friday 12 June 2015

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

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

Staying in Control

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

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

Patient Need v Pharma Challenges

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
• Patient Information Leaflets
• Diagnosis pathway surveys
• Research design
• Burden of Disease questionnaire
• Patient Related outcome
• Educating staff
• Humanitarian Aid

– Developing company programmes – Getting drug to patients

• Proving work placements
• Linking Pharma to Academic
• Lobbying Government
• Educating Doctors and Nurses
• Developing new Patient Groups
• Representing the patient

community on steering groups, panels

• Acting as a communications hub
• Patient Awareness Literature

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