In celebration of the worldwide Rare Disease Day 2023 (RDD23), the global multistakeholder policy initiative Partnering4PNH is delighted to invite you to the event “Addressing the unmet medical needs of people living with a rare disease: What will it take?”.
During the event, a multidisciplinary panel of experts from the rare disease community will discuss how the European Union and other countries can continue to play a central role in the rare disease space by stimulating research and innovation, development of new therapies, and ensure access for patients.
We stand at a crossroads for setting new standards for people living with a rare disease. We need to rise to the challenge and develop a suitable framework to address their unmet needs.
Whilst reflecting on the ongoing reviews of the General Pharmaceutical Legislation, the Orphan Medicinal and Paediatric Regulation, expert speakers will present their views on what is necessary to expedite research and innovation.
Furthermore, to increase awareness on what is at stake, patients and healthcare professionals from Partnering4PNH will present their policy recommendations on how to address the unmet needs of people living with paroxysmal nocturnal haemoglobinuria (PNH).
We warmly invite you to join the event and take an active part in this collective journey to improve the quality of life of people living with a rare disease.
Our speakers include:
MEP István Ujhelyi, S&D, Hungary
MEP Stelios Kympouropoulos, EPP, Greece
MEP Maria da Graça Carvalho, EPP, Portugal
Louise Arnold, PNH Specialist Nurse, Leeds Teaching Hospital, United Kingdom
Dr Morag Griffin, Haematologist PNH Specialist, Leeds Teaching Hospital, United Kingdom
Dr Jelena Malinina, Data Director, EURORDIS
Alexander Natz, Secretary General, EUCOPE
Dr Jameel Nazir, Vice President, Patient Access and Community Engagement (PACE), Swedish Orphan Biovitrum AB (Sobi)
Olena Wagner, Chair, PNH Ukraine.