Rare Disease Dilemmas and Solutions
Filling the Knowledge Gap for Better Patient Outcomes

October 26 2023 

Climedo Connect_Rare Disease-1

Thursday, October 26 2023 | 4:00 – 5:00 PM CET 

What are the challenges posed by rare diseases and how can we address the essential needs of patients while navigating these conditions across all clinical phases? By leveraging the power of Quality of Life (QoL) data, personalized treatment algorithms and Real-World Evidence, we can build a comprehensive approach to managing these conditions effectively.

In our Climedo Connect on October 26, Domenico Merante and Henrik Schou of CSL Vifor presented their insights and expertise on how advancements in technology, data analysis and evidence-based strategies can reshape the landscape of rare disease management and close important knowledge gaps. They shared practical examples of how the integration of these elements can offer innovative solutions to the challenges faced by both rare disease patients and healthcare professionals. Together, we discussed:

  • Rare disease challenges, patient needs and opportunities
  • The importance of QoL data, treatment algorithms and RWE for patient outcomes
  • Best practices in rare disease data management during clinical development and in the post-approval phase
In addition to the presentations, attendees had the chance to join various live surveys, network with other professionals and, of course, ask our experts any questions they had. 

Meet Our Speakers

Laura Dosch
Laura Dosch (Host)

Account Manager

As an Account Manager at Climedo, Laura accompanies customers from the medical device and pharmaceutical sectors on their way to the successful digitalization of clinical trials. She advises companies on the use of Climedo's modular, decentralized and patient-centric solutions for EDC, eCOA/ePRO, eConsent and the integration of wearables and Telemedicine to bring the best treatments to patients.


Opinions are her own and don’t represent Climedo as a company.


Domenico Merante
Domenico Merante

Global Clinical Development TA Lead Nephrology and Orphan/Rare Disease Area
CSL Vifor

With 30 years’ medical experience as pharmaceutical physician, Domenico has proven his skills in global clinical development across various therapeutic areas (e.g. endocrine, diabetes, cardiovascular, haematology, immunology and rare kidney diseases). He combines this with 20+ years’ experience in Italy’s NHS as emergency doctor, the Italian Navy as medical officer and as a diabetic wounds specialist.
Opinions are his own and don’t represent CSL Vifor as a company.
Henrik Schou
Henrik Schou

Global Head Evidence Generation

CSL Vifor

As a Health Economist and Mathematician/Statistician, Henrik is a seasoned Global Pharma Executive. His career spans Novo Nordisk, Sanofi and CSL Vifor, excelling in RD, Regulatory, Medical Affairs, Pricing and Market Access. Currently Head of Evidence Generation at CSL Vifor, he chairs the Phase Four Research Board and is integral to the Senior Leadership Team.

Opinions are his own and don’t represent CSL Vifor as a company.


What was on the Agenda?

  • Intro (Laura Dosch)
  • Challenges and opportunities in clinical development and post-approval phases for rare diseases
    • Case study in ANCA-Associated Vasculitis and a discussion on gaps in QoL data and outcomes (Domenico Merante)
    • Real-World Evidence / Real-World Data (Phase IV) with historical examples and personalized treatments (Henrik Schou)
  • Where are we today and final remarks (Domenico Merante and Henrik Schou)
  • Discussion and networking (all)
  • Wrap-up (Laura Dosch)



A transformative perspective on leveraging technology and evidence-based strategies to revolutionize rare disease management.


Practical examples from the fields of rare and life-threatening diseases, based on historical examples. 


Talk to other industry professionals and find out how they are tackling challenges similar to yours.


The Digital Platform for Innovative Clinical Trial Execution.

Climedo offers an all-in-one eCOA and EDC solution with hybrid capabilities for non-interventional studies and real-world evidence. By using a patient-centric approach and leveraging real-time data insights and visualizations around a study’s current progress, Climedo empowers its clients to better engage with healthcare professionals and other key opinion leaders (KOLs). This boosts awareness, stimulates scientific dialogue and accelerates the launch success of new medical innovations, thus reaching more patients faster. Founded in Munich in 2017, Climedo is a leading trusted partner for pharma, medtech, CROs and academia with over 1.7 million patients enrolled to date. 

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