Addressing PNH unmet medical needs: a policy roadmap to aspire for change 

About Partnering4PNH

Partnering4PNH is a multistakeholder policy initiative that aims to highlight the gaps and challenges faced by people living with an ultra-rare disease called paroxysmal nocturnal hemoglobinuria (PNH) when accessing   care and adequate treatments. It aims to examine national and international policies accompanying the patient journey, from presentation of symptoms to diagnosis, to access to treatment and care, as well the impact on mental health and quality of life.

Our work:   As research in PNH is evolving and the therapeutical benefits are improving, healthcare policies around the world must adapt and evolve with science. Partnering4PNH members worked in developing a global policy consensus paper to highlight the gaps and challenges faced by people living with this disease when accessing care and adequate treatments and issued a set of policy recommendations to support this scientific journey.

The mission of this initiative is to ensure that the challenges faced by patients living with PNH are not overlooked. The group aims to develop policy recommendations collaboratively and drive change both at global and national level.

Partnering4PNH is a global multistakeholder policy initiative which brings together PNH experts with a range of perspectives. Its members are representatives of patient associations, clinicians, nurses and industry from different European countries such as UK, Spain, Ukraine but also from Canada and Australia. Partnering4PNH has two political Co-Chairs, who are the Members of the European Parliament (MEPs) István Ujhelyi (Hungary, Socialist and Democrats) and Stelios Kympouropoulos (Greece, Christian Democrats). The Partnering4PNH initiative is organised and funded by the Swedish Orphan Biovitrum AB (Sobi), whilst the Secretariat is led by Evoke Incisive Health.

The members of Partnering4PNH


If you have any question or information please E-mail here: Link

Call to action

On 1 March 2023, Partnering4PNH and Sobi, in collaboration with the Parliament Magazine and EUCOPE, organised an event hosted by and in partnership with MEP István Ujhelyi (S&D, Hungary), MEP Stelios Kympouropoulos (EPP, Greece) and MEP Maria Garça Carvalho at the European Parliament.

During the event participants were invited to sign a call to action to pledge their commitment to continue the search for much needed solutions to address the many unmet medical needs of the rare disease patient community.


Job code: NP-25272 | Date of preparation: December 2022

Intended for healthcare professional only