The Food and Drug Administration has announced the launch of a docket to require inputs and comments from patients, patient advocates, the scientific community, health professionals, regulatory and health authorities and general public regarding practical steps to establish a rare diseases clinical trial network.
The issues related to the support of the innovation in clinical research, the facilitation to medicines access and the promotion of a patient-centred approach become even more relevant in the field of paediatric and rare diseases. For this reason, TEDDY, as methodologic Network at EnprEMA, intends to candidate to provide its contribution in US new initiative. Donato Bonifazi, TEDDY Representative in the Enpr-EMA Coordinating Group, explained the reasons of this willing: “TEDDY Network is always proactive in the promotion of paediatric pharmacological research with a particular interest in the populations and research fields requiring more attention like the rare diseases. Moreover, TEDDY is among the beneficiaries of the European Joint Programme on Rare Disease (EJPRD), aimed to create a comprehensive, sustainable system allowing virtuous circle between research, care and medical innovation in the rare diseases field. For this reason, TEDDY welcomed enthusiastically this initiative, offering the opportunity to link together the most relevant initiatives worldwide including EnprEMA.”
The overall aim of the FDA initiative will be to provide a more cooperative approach and common strategy to support the conduct of clinical trials in rare diseases populations. In particular, with this request for information and comments, FDA is interested in investigating what work is currently being done and what work needs to be carried out to improve the design, conduct and completion of rare diseases clinical trials.
As underlined also in FDA docket, the goal is to join efforts and create a global network connected with the most relevant initiative in the world related to rare disease research and clinical research activities in order to support innovation and quality in the drug development pipeline for rare diseases.
For this reason, TEDDY has proposed the setting up of a new EnprEMA working group aimed at addressing the FDA requests of comments and more in general to prepare the framework for an EU paediatric and rare disease research initiative parallel to FDA action. This could have a more relevant value in view of the expected revision of orphan and paediatric medicines regulations, announced by the European Commission.
Regarding the current COVID-19 crisis, Donato Bonifazi added: “This emergency has underlined the importance of medical innovation as an essential component in tackling global health challenges, above all in fields, as paediatrics and rare diseases, where the lack of adequate drugs and formulations, the difficulty in medicines access and clinical trials conduct is very relevant. This kind of initiative can provide the possibility to better face these challenges and overcome possible future emergencies”.