New European Consortium - AGORA - launches to ensure access to life-saving gene therapies for children with rare diseases


By AGORA Founders - Claire Booth, UCL Great Ormond Street Institute of Child Health London and Arjan Lankester, Leiden University Medical Centre, Netherlands

A new Europe-wide academic initiative, named AGORA (Access to Gene therapies fOr RAre disease), has been launched to create sustainable solutions for the (growing) economic and regulatory hurdles that are preventing access to proven effective life-saving gene therapies in children with rare- and ultra-rare diseases for non-medical reasons. The small numbers of affected patients present huge challenges for making these therapies available through traditional market authorisation and commercial routes since the projected financial costs of the late stages of development and authorisation will not be economically viable or commercially feasible. As most of the early-stage research into these therapies is funded through national and European research programmes, these ‘dropped’ treatments also represent a significant waste of public money.

AGORA will focus initially on ultra-rare diseases in the field of inborn errors of immunity and inborn errors of metabolism for which no equally effective alternative curative treatments are available. In the short-term, AGORA will explore the barriers that currently exist and identify areas of potential evolution and actions required to enable sustainable access to affordable treatments. Its main role lies in harmonisation of pan-European national activities, acting as a central body providing support to academic centres/not for profit organisations to seek regulatory approval for gene therapies at a multinational level. Ultimately, the aim is to explore the creation of an independent, sustainable, not-for-profit entity that can support marketing authorisation, delivery and affordable access to therapies which are not commercially sustainable and would otherwise be unavailable to patients.

The first AGORA meeting, held on September 22nd at London’s Great Ormond Street Hospital for Children (GOSH), was attended by founding members from six countries representing academic groups, regulators, funders, patient advocacy groups and drug developers. The group is currently exploring the next steps to achieve its goals.