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

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

Building on growing attention to PID and previous campaigns; EU Expert Recommendations EU Advocacy Campaigns Joint Stakeholder advocacy 1. Political endorsement on a set of key priorities for PID community 2. Policy tool and basis for further campaigns including national ones… Recommendations on: 1. Awareness & Education 2. Screening & Diagnosis 3. Treatment & Management

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

Key messages & recommendations include: EU Advocacy Campaigns

Key messages & recommendations include: EU Advocacy Campaigns

Key messages & recommendations include: EU Advocacy Campaigns

Key messages & recommendations include: EU Advocacy Campaigns

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

Follow up National campaigns –SCID newborn screening meeting, UK, 2011 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 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) EU Advocacy Campaigns

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

Activities undertaken by Meeting with Health Commissioner John Dalli October 2011 & Vassiliou May 2008 Policy Meetings 2010: Plasma Protein Therapies in the Treatment of Rare Diseases, Chair: Miroslav Mikolasik Improving Care for Rare Plasma Related Disorders, Chairs: Jorgo Chatzimarkakis / Miroslav Mikolasik 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 IPOPI & PLUS

IPOPI actively participates in PLUS Stakeholders Consensus Meetings, Dublin Dublin Consensus statements published in Vox Sanguinis 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 IPOPI & PLUS

Participation in Eurordis Rare Diseases activities IPOPI & PLUS