ICMRA 'Rare'


16 September 2024
Lugano, Switzerland


A very warm ‘welcome’ to our dedicated event website for the ICMRA ‘Rare’ Symposium 2024.

You can register as an on-site or virtual participant for the Symposium, find the agenda concept, venue and travel information as well as a hotel booking platform below.


Join us for the ICMRA ‘Rare’ Symposium and Workshop 2024. A multi-stakeholder event focusing on rare diseases, organised by the International Coalition of Medicines Regulatory Authorities (ICMRA) and hosted by Swissmedic, open to selected stakeholder organisations and to regulators.

The symposium will take place on September 16, 2024 and will be followed by a regulators workshop on September 17, open to participating ICMRA authorities only. The symposium and workshop together aim to appreciate evolving technological and regulatory developments encountered with (ultra-)rare diseases.

Key objectives:

  • To better appreciate the main challenges and opportunities encountered with (ultra-)rare diseases in translation and clinical development

  • To critically compare existing regulatory requirements in this space and identify opportunities for innovators/investors, in particular the utilization of DHTs (digital health technologies) for data generation and recruitment.

  • To hear from patients and caregivers about their journeys and how we can better involve them

Photo credits: ©LAC 2015 - Foto Studio Pagi

Event Location

Lugano Arte e Cultura

Piazza Bernardino Luini 6, Lugano
6900 Lugano


September 16
Check-in & Welcome Beverages

Agency & ICMRA Heads (tbc)
Claus Bolte (Swissmedic) & Kerry Jo Lee (FDA)

Keynotes 1: Clinical insights – High-level overviews
  • More opportunities than challenges today!

    Alessandro Aiuti, Immunologist (I), HSR Research (Link)

  • How to utilize registries

    Bernhard Landwehrmeyer, Neurologist (D) (Link)

  • Basic & translational research

    Tiina K. Urv, NIH (US) (Link)

  • Pharma R&D (clinical development)

    Thomas F. Miller, Industry (US), InsideScientific (Link)

Panel 1: Hands-on experience – Clinical evidence generation
  • Academic clinicians and investigators

    Keynote 1 speakers

  • Lessons learned, expectations, suggestions

    Lars Hemkens (CH), Pragmatic Evidence Lab (Link)

  • (Ultra-)Orphan Drug Designation?

    Daniel Michaeli (D), Nat. Center for Tumor Diseases (Link)

Brain and Bio Break
Keynotes 2: Moving the needle? – Digital Health opportunities
  • Showcasing exciting tech developments

    Jennifer Goldsack (US), Digital Medicine Society (Link)

  • How rare diseases became common

    Dan O’Connor (UK), ABPI (Link)

  • (What) can we learn from Oncology?

    Blythe Adamson (US), Flatiron (Link)

  • Linking regulators and tech

    Klaus Romero (US), Critical Path (Link)

Panel 2: Managing innovation – Innovation at interfaces
  • How can DHT help (recruitment, evidence)?

    Keynote 2 speakers

  • Regulatory Science evolving

    Elisabeth Konkoski, FDA (US) (Link)

  • Low- and middle-income countries

    EU and RoW Regulator

Lakeside chat with caregivers
  • How to better appreciate and integrate their journeys and experience?

    Bob Stevens (UK), MPS Society (Link)
    Christoph Poincilit (CH), NPD Suisse (Link)
    Klaus Rose (CH), Pediatrics, Sturge–Weber syndrome (Link)
    Julia Vitarello (US), Mila’s Miracle Foundation (Link)


Claus Bolte (Swissmedic) & Kerry Jo Lee (FDA)

Adjourn @18:30 (CET)

ICMRA Steering Committee & Faculty Dinner @20:00 (CET)

Symposium draft agenda & concept (V.6)


Please note that the agenda provided is pre-final and subject to change. Any updates or modifications to the agenda will be promptly reflected on our website. We encourage you to check the website regularly for the most current information.


Hotel booking platform

Through our official partner agency, Amiconi Consulting SA, Swissmedic has pre-booked rooms in various hotels in the city of Lugano. All hotels are located within a walking distance of max. 15 minutes from the venue. The hotels have different star categories, locations and prices in order to meet the different needs of the participants.

All participants are responsible for arranging and paying for their own travel to and accommodation during the meeting.

Rooms will be offered on a first-come, first-served basis. The room contingents will be available until 15th August 2024 depending on the hotel you choose. After this date, all hotel rooms will expire and Swissmedic has no longer the possibility to organize accommodation and availability can no longer be guaranteed.

The number of rooms is limited. It is therefore worth booking your room early.

Book your hotel here: https://ticino.e-vent.online/web/50E817DA9B9F4FFFB44C4C18D7A62D04/EN

Travel information

Arriving by air

We recommend flying to one of the following airports:

• Zurich Kloten Airport (CH)

• Milan Malpensa Airport (IT)

The airports of Zurich and Milano are connected to the Swiss railway network and have their own stations.

Trains to Lugano run every 30-60 minutes from all cities. We recommend traveling to Lugano by public transport. Cabs/Taxis are very expensive in Switzerland.

Link to the Swiss railways (SBB/CFF) online schedule: www.sbb.ch

Arriving by train

Tickets for the train can be purchased either online via www.sbb.ch or at one of the ticket machines at the station.

Travel times to Lugano by train

• From Zurich Kloten Airport: approx. 3 hrs

• From Milan Malpensa Airport: approx. 1 hr 30

Arriving by car

Switzerland – and Lugano – is also easily reachable by car from its neighbouring countries as it is linked to the entire European highway network. The Swiss motorway ticket is required when driving through Switzerland in your own vehicle. Click here to buy your Swiss motorway ticket. Rental cars already come with a ticket.

Travel times to Lugano by car:

• From Milano: 80 km, approx. 1 hr

• From Zurich: 220 km, approx. 3 hrs

Useful links

www.myswitzerland.com: Information for travelling to Switzerland

(such as passports & visa, customs entry regulations and electricity)

www.myswitzerland.com: General information about Switzerland and Lugano

www.sbb.ch/en: Swiss Railway System SBB

www.swissmedic.ch: About Swissmedic


Riccardo Luigetti
Meeting coordination
Mari Vihertola
Organisation and Administration
Iliana Perera
Organisation and Administration