The in-person event will take place in Sitges (Barcelona), Spain over four days from November 15th – 18th.

Patient groups can ask for free registration (subject to approval).

The Congress will address solutions to challenges around the political, commercial and scientific issues in manufacturing, clinical trials, strategy, regulation & policy, Market Access & Pricing, Science & Strategy, Pitch & Partner and Precision Medicine through a variety of keynote panels and interactive sessions (see agenda).

Dr. Daria Julkowska, Coordinator of the EJP RD, will moderate the panels on “Responding to the EU OMP review – how to strengthen the EU rare disease ecosystem” (November 15th) and “The European Expert Group on Orphan Drug Incentives – How do we develop a sustainable European ecosystem?” (November 16th).

More information and registration here: https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm.

The fully online workshop will take place over two days on October 20th – 21st from 14.00 – 18.00 CET.

The workshop aims at gaining insights into the future of the regulatory strategy for data acquisition and analysis. Therefore, a strong emphasis will be placed on presenting and discussing the work of three of the European Medicines Agency (EMA) Task Forces: Clinical Studies and Manufacturing, Data Analytics and Methods, and Regulatory Science & Innovation.

More information and registration here: https://events.eurordis.org/32ertcworkshop/identification?reg_type_id=183905&ref=aca_hcp

The fully online event will take place on September 17th from 15.00 – 17.30 CET.

The symposium aims to identify and explore the most pressing priorities in orphan drug innovation, and whether and how social science research, particularly legal research, can help meet these needs. The international showcase of speakers and panellists includes Terkel Andersen (EURORDIS), Jakob Wested (Danish Medicines Agency & CeBIL), Sarah Rickwood (IQVIA) and Sven Bostyn (University of Copenhagen).

More information and registration here: https://www.eventbrite.co.uk/e/orphan-drug-innovation-needs-and-priorities-registration-166028923981

The fully virtual summit will take place over two days from October 18th – 19th.

The summit agenda, which is still being finalised, features several plenary sessions as well as a choice of breakout sessions, on the following themes with both a U.S. as well as an international focus:

• Best Practices and Case Studies for Successful Partnerships
• Rare Diseases as a Public Health Challenge
• Impact of COVID-19 on Rare Disease Research, Clinical Trials and Drug Development 
• Designing Trials for Inclusivity, Equity and Engagement
• Drug Pricing: State and Federal Perspectives
• FDA Patient Engagement
• Gene Therapy and Gene Editing: Current Status and Potential Impact on the Rare Disease Community

More information and registration here: https://nordsummit.org/

The fully online event will take place on September 7th from 12.00 – 13.30 CET.

The event features a keynote address by Terkel Anderson, President of the EURORDIS Board, and a distinguished panel of speakers from Nordic countries who will discuss how to best ensure that the regulatory environment is both conducive to innovation and contributes to Nordic rare disease patients’ access to innovative therapies.

More information and registration here: https://zoom.us/webinar/register/WN_GzGuP0JlTXykqmlT8_JQ5Q

The webinar will take place on June 11th from 14.30 – 16.00 CET.

In this online event targeted towards the broad rare disease community, including research, academia, patient representatives, members of the investor community, rare disease companies and trade associations, the expert presenters and panellists will discuss a report with 14 policy proposals to improve the OMP innovation environment in Europe, present their recommendations, and discuss compelling ideas for the next decade of rare disease policies in Europe.

More information and registration here: https://events.euractiv.com/event/info/media-partnership-how-to-address-the-unmet-needs-of-rare-disease-patients-by-transforming-the-european-omp-landscape

Interested parties are invited to share their views and experiences on the main obstacles they are facing concerning treatments for rare diseases and children, on possible ways to overcome these obstacles and on how to make the current legislation future-proof.

The public consultation is currently accepting inputs as a questionnaire and will close on July 30, 2021.

More information here: https://ec.europa.eu/info/law/better-regulation/have-your-say/initiatives/12767-Medicines-for-children-&-rare-diseases-updated-rules/public-consultation_en

The webinar will take place on May 5, 2021 from 14.00 – 15.30 CET.

The event, in cooperation with BPI, is aimed at discussing how the revision of the OMP Regulation (N° 141/20000), 20 years after its adoption, could trigger new research for the benefit of rare disease patients.

The panellists, who represent a wide range of stakeholders involved in rare diseases research, include Ines Alves (ANDO Portugal President; ERN BOND ePAG), Rob Camp (Patient Engagement Senior Manager, EURORDIS CAB), Dr. Daria Julkowska (Scientific Coordinator, EJP RD), and Prof. Dr. Franz Schaefer (ERKNet Coordinator, Heidelberg University Hospital).

More information and registration here: https://alexion.zoom.us/webinar/register/WN_qjQPV2jiRB2IPLwdN83cBA