Rare Disease Day 2020 will be celebrated this year on Saturday the 29th of February - a fittingly rare date! Coordinated by EURORDIS in partnership with National Alliances, the aim is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives. Lead-up events throughout the month will be organised by members of the rare disease community including patient organisations, healthcare professionals, researchers, and policymakers across 101 countries.

Rare diseases will affect 1 in 20 people in their lifetime, but their rarity often leads to delays in diagnosis, and means that information and treatment options can be limited. Rare Disease Day is an opportunity to strengthen international cooperation, vital for identifying and classifying these diseases, coordinating research, and providing access to treatment and support for patients, their families, and carers.

For more information, go to the Rare Disease Day 2020 website.

 

The European Rare Disease Models & Mechanisms Network (RDMM-Europe) has been established by Solve-RD – an EU-funded research project. The overall aim is to boost research in rare diseases, discover new disease-causing genes and obtain evidence for pathogenicity through functional validation.

For more information, visit the following website.

Consultation on guidelines on Good Clinical Practice for Advanced Therapy Medicinal Products

Consultation on guidelines on Good Clinical Practice for Advanced Therapy Medicinal Products

The European Commission is launching an online targeted public consultation on the draft guidelines on good clinical practice for Advanced Therapy Medicinal Products (ATMPs) addressed particularly to small and medium-sized enterprises (SMEs), academia, hospitals and patient organisations.

The Commission has to draw up guidelines on good clinical practice specific to advanced therapy medicinal products. The document will focus on ATMP specificities only and will apply to the GCP Guidelines of The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).

The comments are invited to be sent by 31 October 2018. Responses should preferably be sent to sante-pharmaceuticals-B5(at)ec.europa.eu.

Find more information here.

E-Rare 2012 - Created by Toussaint Biger