EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma (onasemnogene abeparvovec) to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Spinal muscular atrophy is usually diagnosed in the first
We are pleased to inform you that the article “Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance” has been published on the European Journal of Health Law. The authors, Annagrazia Altavilla, Viviana Giannuzzi, Mariangela Lupo, Donato Bonifazi and Adriana Ceci, after having clarified the concept of translational
The European Medicines Agency defines “off-label” the use of a medicine for an unapproved indication or in an unapproved age group, dosage, or route of administration. The majority of marketed medicines world‐wide, including those commonly used or which could potentially be used in the paediatric population, have not been studied in the relevant paediatric age
Encouraging active participation and self-determination of patients and parents in the caring procedure, and promoting their involvement in scientific research and drug development process is the main objective of TEDDY Working Group on “Active engagement of children and adolescents in the themes of clinical research”. According to the TEDDY Statute, a Young Persons Advisory Group
Data collection in healthcare allows health systems to create holistic views of patients, personalize treatments, advance treatment methods, improve communication between doctors and patients, and enhance health outcomes. For this reason, the promotion of data collection, data sharing and data storing for secondary use is an important issue and is the goal of TEDDY Working
The European Commission is launching a new strategy to improve and accelerate patients’ access to safe and affordable medicines and to support innovation in the EU pharmaceutical industry, addressing mainly: the life cycle of medicines from R&D to authorisation and patient access how to put scientific and technological advances into practice how to fill market
The European Paediatric Formulation Initiative (EuPFI) works to improve the preparation of better and safer medicines for children. One area they are interested in is the “gadgets” or devices (e.g. measuring spoons, dosing cups, inhalers) that are used to help patients take their medicines. It is important that these devices are used properly to make sure
The Multi-regional Clinical Trials Center of Brigham (MRCT) in collaboration with Harvard Catalyst have developed a series of COVID-19 clinical research handouts that can be used as general informative material to prepare potential participants in advance of recruitment and consent. These introductory one-pagers are addressed to different potential target audience: patients, friends/family members, healthy proxies,
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
On May 14-16th over 1,500 people from 57 different countries, representing all stakeholder groups in the rare disease community, joined together at the 10th European Conference of Rare Diseases & Orphan Products — ECRD 2020. EPTRI, an EU-funded project coordinated by CVBF and in which TEDDY is involved, was glad to attend such an important event