Below, please find a selection of the various patient advocacy activities and results achieved.
Many projects have a long lead time and are often not mentioned below. And, we are proud to share that there is also a lot in the pipeline for 2018.
The mentioning of an event means that a PSC Patient Europe representative was present. Special tasks, such as speaker or panel member, are listed per section. By participating in a wide range of activities, it is possible to increase awareness about PSC among a large group of stakeholders in the playing field.

 

International level

  •  AdaptSmart 
    Accelerated development or appropriate patient therapies: a sustainable, multi-stakeholder approach from research to treatment outcomes (ADAPT SMART project)
  • Asterix, Advanced in Small Trials dEsign for Regulatory Innovation and eXcellence
    * member Patient Panel
    * Presentation results at
    EMA, participant
    * final conference

Prior to 2017

  • Six lay summaries of scientificarticles,on Ground breaking PSC research at AMC and about Phase II Nor-urso and the Norwegain PSC Research center. other topics include Risk factors with PSC, scoring system for PSC and PSC in Japan.  Translated in 27 languages.
  • Growing number of National Liaisons
  • Excellent relationship with PSC drs. & researchers in Europe and beyond
  • Internal Newsletters for National Liaisons
  • Partner in two international PSC consortia
  • Partner in one prestige international innovative business consortium
  • Guest blog @PharmaViews on Importance of Lay Summaries
  • Attended various international, disease-transcending conferences, incl. HTAi Oslo 2015
  • Grants awarded for various visits / conferences / meetings
  • Consumer representative in the Cochrane Hepato-Biliary Group
  • ‘Dare to be Rare’ project
  • Speaker at Patient-Centric Clinical Trials Conference
  • Board member finished Eupati Patient Expert training and is now a Eupati Fellow
  • Presentation at Cochrane Hepato-Biliary Group session @ EASL International Liver Congress 2015 in Vienna

 

National level (NL) ,most ongoing

Please note: Often, these achievements are the seeds for projects to be rolled-out on an international level

Prior to 2017

  • PSCPE pitch at numberous academic medical centres: full collaboration
  • NFU: PSC Centres of Expertise, patient input (NL is only EU country integrating patient’s voice)
  • Ministry of Health:
    * Compassionate Use & Named Patient Program meetings
  • Ministry of Economic Affairs
    * InviteKnowledge & Innovation Agenda2016-2019 meeting: Patient Expert
  • Presentation ‘Dare to be Rare’ for 20 Medical Directors of pharma companies in the Netherlands
  • Meeting with politician on rare disease research (& funding): suggestions for improvement current regulations and funding system, future & need for disease-specific patient organisations
  • Publication: GP brochure on PSC, and roll-out internationally, currently in various countries, including Slovakia (under construction).
  • Initiating Project ’Quality Standard of Care PSC from a patients perspective’.
  • Project with potential roll-out on international level
  • ‘Third Senate Dinner’, topic; Compassionate Use & Early Access, co-initiator
  • HTA International meeting, ZonMw (The Netherlands Organisation for Health Research & Development)

Organisation

  • Establishing foundation, with a Board according to the Dutch  ‘Good governance of foundations’
  • By-laws
  • Chamber of Commerce registration & number
  • PBO (Dutch: ANBI): tax deductible perks for donations
  • Corporate ID, logo
  • Website & twitter account (@PSCPatientsEur)
  • Trello Cloud for all volunteers: easy & quick dissemination of information
  • Eurordis: full member
  • Health-RI supporter
  • First donations
  • Impressive international network
  • Quarterly Board meetings
  • Successful grant applications
  • Growing number of volunteers from around the globe including Australia, Israel, USA, Poland

 

We would like to thank all our dedicated volunteers and supporters who made it all possible!

And the PSCPE Board is happy to inform you there are quite a few very interesting projects in the pipeline, so please keep supporting us and stay tuned!