IPOPI Advocacy & Media Training Workshop EU Expert Recommendations & The work of PLUS

1. IPOPI Advocacy & Media Training Workshop EU Expert Recommendations & The work of PLUS Johan Prevot Executive Director 14-15 September 2012 Belgrade Art Hotel (BAH) Belgrade, Serbia

2. EU Advocacy Campaigns • It all started with…… • European Parliament STOA Panel, 17 March 2004 2010

3. EU Advocacy Campaigns • Building on growing attention to PID and previous campaigns; EU Expert Recommendations Joint Stakeholder advocacy 1. Political endorsement on a set of key priorities for PID community2. Policy tool and basis for further campaigns including national ones… Recommendations on: 1. Awareness & Education2. Screening & Diagnosis 3. Treatment & Management

4. EU Advocacy Campaigns • Building on growing attention to PID and previous campaigns; EU Expert Recommendations • Key messages & recommendations include:

5. EU Advocacy Campaigns • Key messages & recommendations include:

6. EU Advocacy Campaigns • Key messages & recommendations include:

7. EU Advocacy Campaigns • Key messages & recommendations include:

8. EU Advocacy Campaigns • Key messages & recommendations include:

9. EU Advocacy Campaigns • PID Forums – SCID Newborn Screening & HTAs in 2011: • National RD Plans PID Forum 2012: 26 September Recommendations, Parliamentary Questions and EP resolutions Building political momentum around PID issues

10. EU Advocacy Campaigns • Follow up National campaigns • SCID newborn screening meeting, UK, 2011

11. EU Advocacy Campaigns • IPOPI has been involved in a number of other initiatives at EU level • To promote the need to take into account the specificities of PIDs in rare diseases policies with regards to: • Diagnosis rates/ Awareness • Access to therapy - challenges linked to unique treatment plasma protein therapy (immunoglobulins); cost/benefit • Varying treatment levels in Europe • Awareness and education

12. EU Advocacy Campaigns • IPOPI active on following key EU dossiers : • Council Recommendations on Rare Diseases – July 2009 & National Rare Diseases Plans – an opportunity for the PID community • Pharma package: Patient Information, Counterfeit and Pharmacovigilance • Cross-border healthcare directive • European Commission Blood Directive & Reports on Voluntary Donations • European Commission Committee of Experts on Rare Diseases to advise and assist in formulating all legislation that affects the treatment and care of rare diseases • EMA Patients and Consumers Working Party (PCWP)

13. IPOPI & PLUS • IPOPI works with other patient organisations representing rare plasma related disorders under a coalition group called consisting of:

14. IPOPI & PLUS • Activities undertaken by • Meeting with Health Commissioner John DalliOctober 2011 & Vassiliou May 2008 • Policy Meetings 2010: • Plasma Protein Therapies in the Treatment of Rare Diseases, Chair: MiroslavMikolasik • Improving Care for Rare Plasma Related Disorders, Chairs: JorgoChatzimarkakis / MiroslavMikolasik

15. IPOPI & PLUS • PLUS Call to Action: • Better diagnosis rates for plasma related disorders • Equal treatment levels • Recognition of unique nature of plasma protein therapies • in national policies • Value of strong patient groups • Patient registries • Reference Centres • Value of national Rare Diseases plans • Supply of human plasma • MEP Interest Group

16. IPOPI & PLUS • IPOPI actively participates in PLUS Stakeholders Consensus Meetings, Dublin • Dublin Consensus statements published in VoxSanguinis • EU Parliament Fly-In meetings and meeting with Commission services (2011), Blood Directive Report • Active participation in EU Commission survey of the blood, blood components and plasma derivatives market

17. IPOPI & PLUS • Participation in Eurordis Rare Diseases activities