10 October 2018
The European Medicines Agency (EMA) and the European Commission’s Directorate-General for Health and Food Safety (DG SANTE) have published a joint action plan to support the development of medicines for children in Europe.
The action plan addresses challenges identified by the European Commission’s ten-year report on the implementation of the Paediatric Legislation. It also takes into account the ideas on how to better apply the Paediatric Regulation to boost the development of medicines for children collected during the multi-stakeholder workshop organised by EMA and the European Commission in March 2018.
The actions in the plan are clustered around five key areas:
It is expected that the implementation of these actions will increase the efficiency of paediatric regulatory processes in the current legal framework and boost the availability of medicines for children.
The plan was originally intended to be completed within a two-year timeframe. However, due to the impact of Brexit and the Agency’s relocation to the Netherlands, some actions are not expected to be completed by the end of 2020, as specified in the plan. The plan will be updated over time as necessary, depending on prioritisation and availability of resources.
The Paediatric Regulation came into force in the EU in 2007 to encourage manufacturers to research and develop medicines for children’s specific therapeutic needs by using a system of rewards and obliging developers to specifically plan the development of their medicine for children (e.g. by integrating it into the development for adults) and submit a corresponding PIP. PIPs are scientifically reviewed and agreed by the EMA’s Paediatric Committee (PDCO), which was also introduced by the same legislation.
31 October 2018
Scientists unveiled a more effective approach for assessing drug response
Scientists from Eli Lilly and Company, the Icahn School of Medicine at Mount Sinai (New York, USA) and Sema4 (Stamford, USA) released results from a proof-of-concept study demonstrating that patient-derived cells offer a more effective approach for assessing drug response than conventional methods.
30 October 2018
Researchers developed an AI approach to identify antibiotic resistance genes
Researchers at the University of California San Diego (USA) have developed an approach that uses machine learning to identify and predict which genes make infectious bacteria resistant to antibiotics. The approach was tested on strains of Mycobacterium tuberculosis – the bacteria that cause tuberculosis (TB) in humans. It identified 33 known and 24 new antibiotic resistance genes in these bacteria.
29 October 2018
Expanding the reach of gene editing with a new CRISPR enzyme
The CRISPR-Cas9 gene editing system has been widely studied because of its potential therapeutic applications, but limitations in the number of locations on the genome it can target remain a major drawback. Now scientists at the Massachusetts Institute of Technology have identified a new Cas9 enzyme that they say can help CRISPR reach more gene mutations.
26 October 2018
Biotech Backed by Bain, Pfizer loads prime CNS assets into new biotech
Pfizer has followed through on its pledge to divest a hunk of its neuroscience R&D, spinning several programs into a new company called Cerevel Therapeutics backed by $350 million in venture funding. Pfizer is contributing a trio of clinical-stage drug candidates—including a Parkinson’s therapy due to start phase 3 testing next year—plus a clutch of earlier-stage programs, while Bain Capital and affiliates stumped up the initial funding.