Navigating the Uncharted: Advancements, Collaborations, and Hope in Rare Diseases

The conference was successfully organized on February 29th in Athens and online on March 1st, 2024, with the physical presence of 31 and the online presence of 32 distinguished Greek and foreign speakers, and more than 380 participants from 15 countries around the world in the context of the World Rare Diseases Day by the Rare Diseases Greece (RDG), member of EURORDIS – Rare Diseases Europe and BOUSSIAS Events. The Conference was held under the patronage of the European Parliament, and the auspices of the Greek Ministry of Health and the City of Athens, with the support of major international and Greek organizations, attracting the interest of the international community.

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Important speakers contributed to the success of the proceedings. Among others, the Greek Government was represented by Adonis Georgiadis, Minister of Health, Irene Agapidaki, Alternate Minister of Health, Aris Angelis, Secretary General of Strategic Planning, Hellenic Ministry of Health, Nikos Milapidis, Secretary General for Social Security, Ministry of Labor and Social Security, Pavlina Karasiotou, Secretary General for Fiscal Policy, Ministry of Economy and Finance, Lilian Venetia Vildiridis, Secretary General of Health Services, Hellenic Ministry of Health, while among important speakers were Stelios Kympouropoulos, Psychiatrist, MEP, Evangelos Manolopoulos, President of the National Organization for Medicines (EOF), Professor of Pharmacology, School of Medicine, Democritus University of Thrace, Dr. Nandia Gogozotou, NE President at EOPYY, President of the Drug Price Negotiation Committee and Dr. Pantelis Messaropoulos, President of the Board of Directors of the Hellenic Institute of DRG (KE.T.E.K.N.Y.). The participation of very important international speakers provided a very thorough international perspective of the ongoing projects and issues pertaining to the rare diseases landscape. Amongst them: Steffen Thirstrup, Chief Medical Officer of EMA, Affiliate Professor at the University of Copenhagen, former Head of Division for Medicines Assessment and Clinical Trials at the Danish Health and Medicines Authority, former CHMP EMA Member, Violeta Stoyanova-Beninska, Chair of Committee for Orphan Medicinal Products at European Medicines Agency (EMA), Division Europe and International Affairs at MEB, Vice Chair of Regulatory Science Committee of IRDiRC, Magda Chlebus, Executive Director Science Policy and Regulatory Affairs, EFPIA, Dr. Patrice Verpillat, Head of Real World Evidence (TDA-RWE), Data Analytics and Methods Task Force (TDA), European Medicines Agency (EMA), Stanislav Kniazkov, Technical Officer, Medical Product Regulation, WHO Europe, Anja Schiel, Special Adviser, Lead Methodologist in Regulatory and Pharmacoeconomic Statistics, Norwegian Medical Products Agency (NoMA), Valentina Bottarelli, Public Affairs Director & Head of European Advocacy, EURORDIS-Rare Diseases Europe and Maurizio Scarpa, Paediatrician, Professor of Paediatrics, Dept. of Women’s and Children’s Health, University of Padova, Italy Director, Coordinating Center for Rare Diseases, Azienda Sanitaria Universitaria Friuli Centrale, Udine, Italy, Founder and President of Brains for Brain Foundation, ERICA Board/ MetabERN Coordinator.

The President of the RDG Dimitrios Athanasiou stressed the need for the State and stakeholders to set Rare Diseases as a National Priority and to contribute to the efforts made in Europe for the creation of the European Action Plan. With Rare Diseases affecting up to 5% – 7% of the population, i.e., more than 500,000 families, it is important to sign and activate the National Registry of Rare Diseases immediately, but also to renew and eventually implement the corresponding National Action Plan within 2024. A holistic approach to RDs in our country is needed for prevention, equal access to treatments, actions for holistic support for patients and their families, as well as public awareness campaigns to eliminate stigma towards rare patients.


Under the Auspices of

About the Conference

Under the theme “Navigating the Uncharted: Advancements, Collaborations, and Hope in Rare Diseases”, the International Congress on Rare Diseases and Orphan Drugs aspires to stimulate dialogue and cooperation across all stakeholders – patient representatives, policymakers, clinicians, researchers, industry, payers and regulators – towards advancing a comprehensive rare disease strategy that promotes sustainability and equity within healthcare ecosystems.

The current policy momentum, the regulatory and digital health evolution in the European Health Ecosystem provide a big opportunity for co-creation and concrete solutions to rare disease patients’ unmet needs in the foreseen future.

Utilizing the experience and knowledge of international partners, the conference will open the dialogue with the current political developments in Europe and the drawing up of a National Strategy for Rare Diseases with the ultimate goal of creating and implementing the National Action Plan for Rare Diseases within 2024.

  • Foster Collaborations:
  • Explore Advancements:
  • Ignite Hope:
Foster Collaborations:

Network with professionals from academia, industry, patient advocacy, and government. Explore collaborative efforts in advancing the national access plan, rare disease data, registries, and patient care. Join thought-provoking discussions bridging gaps between unmet needs and practical solutions.

Explore Advancements:

Explore rare disease breakthroughs, access pathways, and therapeutic innovations. Esteemed keynote speakers, including world-renowned experts, will discuss cutting-edge technologies, innovative funding models, and pharma legislations reshaping the rare disease landscape.

Ignite Hope:

Immerse yourself in inspiring stories of resilience and progress from individuals living with rare diseases and their families. Learn about patient advocacy initiatives, support networks, and how collective action is bringing hope to those navigating the unexplored waters of rare diseases.

Don’t miss this unique opportunity to be part of a transformative event that is paving the way for advancements, collaborations, and hope in the realm of rare diseases.

To discover new worlds and bring about change, all pioneers set out on uncharted waters. Join us to navigate them together!

Main Streams will focus on

  1. The Policy Landscape on Rare Diseases
  2. ATMPs in the New PharmaceuticalStrategy for Europe
  3. New Regulatory Models for Orphan Drugs
  4. Defining and Measuring Unmet Needs in Rare Diseases, a European Discussion
  5. RWE for informed and impactful Decision Making
  6. Access and Reimbursement Strategies for Orphan Drugs in Europe
  7. Innovations in Rare Disease Therapeutics
  8. European Reference Centers (ERNs) in the National Health Systems and their Research Potential
  9. Building a Holistic and Supportive Ecosystem for Rare Diseases
  10. Navigating Access Pathways for Orphan Drugs in Greece
  11. RD Patients Registry and the value of data in Decision Making
  12. Working together for a Sustainable Future – SFEE Rare Diseases Task Force
  13. Strategic Planning for Rare Diseases in Greece and the Greek National Action Plan
  14. Foresing 2030 – Stay on top of the biggest the initiatives that will change the RD landscape!


Confirmed Speakers

Who should Attend?

Healthcare Stakeholders • Government representatives and officials • Pharmaceutical industry executives (Market Access, Government Affairs, Health Economics) • Public and Private Health services providers • Health economists • Health Policy Experts • Healthcare Professionals • Nurses • Academics • Researchers • Journalists • Patients and Patient Representatives


Organised by

Member of

Grand Sponsor

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit


Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. Chiesi operates in 30 countries and counts more than 6,000 employees.

GENESIS Pharma was one of the first pharmaceutical companies in Europe to specialize in the marketing, sales and distribution of biopharmaceutical products and is currently the largest by turnover among Greek companies focusing on innovative medicines. Through long-standing strategic partnerships with some of the leading global pharmaceutical companies committed to cutting-edge R&D, GENESIS Pharma has created a strong portfolio of innovative and high therapeutic value pharmaceutical products for more than 30 severe and rare diseases.

IQVIA (NYSE:IQV) is a leading global provider of information, innovative technology solutions and contract research services dedicated to using analytics and science to help healthcare stakeholders find better solutions for their patients. Solutions are powered by the IQVIA CORE™, which combines big data, advanced technology, analytics and extensive industry knowledge. Formed through the merger of IMS Health and Quintiles, IQVIA has approximately 70,000 employees worldwide.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. Our mission is to provide access to best-in-class treatments for patients with little to no treatment options. For 25 years, we have been successful delivering on our mission by pursuing groundbreaking science and technology to bring multiple innovative therapies that are transforming patient’s lives, globally.

Throughout our 125-year history, Roche has grown into one of the world’s largest biotech companies, as well as a leading provider of in-vitro diagnostics and a global supplier of transformative innovative solutions across major disease areas. Our commitment to our people, partners, stakeholders and, most importantly, our patients remains as strong as it was on the first day of our journey.

At Sanofi we are driven by one purpose, we chase the miracles of science to improve people’s lives and we are dedicated to transforming the practice of medicine by working to turn the impossible into the possible.

Takeda is the largest pharmaceutical company in Japan and has a strong presence in over 80 geographies across the world. With a history for over two and a half centuries, we focus on innovations that contribute to making a difference in people’s lives in our core therapeutic and business areas, including gastroenterology and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines.

UCB innovates and delivers solutions that make real improvements for people living with severe diseases. We partner with and listen to patients, caregivers and stakeholders across the healthcare system to identify promising innovations that create valuable health solutions.



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CONEQ provides technical equipment and services to any kind of event. With our Simultaneous Interpretation Systems, Conference Systems, AudioVisual Equipment we support events and provide the best service to our customers. Our professionalism, our high quality of service and state of the art equipment offered to over 600 large and of great significance conferences during the last 5 years have certainly contributed to our constant uptrend. Call us at (+30) 211 2163453. or email us at [email protected]

Official Publication

Contact us

Registrations & Sponsorships
Chrysoula Kordouli, Τ: +30 210 6617777 (ext. 204), +30 6946236795, E: [email protected]
Conference Info
Natalia Toubanaki, T: +30 210 6617 777 (ext. 289), +30 6947936708, E: [email protected]
Conference Content and Program
Dimitrios Athanasiou, T: +30 6944604292, E: [email protected]

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