NEWSLETTER DECEMBER 2020
Designing the framework for a new European paediatric research infrastructure
Manifesto for Paediatric Research
Orphan and paediatric medicines share very similar challenges; however, some overlaps currently exist between the Regulation for medicines for rare diseases and the Regulation for medicines for children.
EPTRI is participating in the EC Consultation on the revision of the Paediatric and Orphan Regulations (Ref. Inception Impact Assessment Ares (2020)7081640 – 25/11/2020) to promote more paediatric and orphan medicines and the inclusion of the themes of paediatric research in the EU Agenda. EPTRI supports the implementation of joint solutions to harmonise and optimise procedures, incentives and rewards addressed to paediatric and orphan medicinal products.
Here below are some of the main points.
- A public paediatric research plan should be developed as integral part of the ‘First Strategic Plan for Horizon Europe’, building on the most relevant EU funded paediatric and paediatric relevant research initiatives and leading to the set up of a European Paediatric Medicines Partnership.
- Paediatric research activities should be included in the EU Strategic Plan for Horizon Europe, by identifying paediatric topics to be funded and allocating dedicated funds for paediatric research within topics of general interest. Priority should include unmet paediatric needs and orphan paediatric indications.
- Research on ethical and legal issues and social impact of both paediatric and rare diseases also in a children/fundamental rights perspective should be promoted and funded to comply with the EU recognised principles and rights.
- Unmet therapeutic needs specific to children of different ages should be identified, by revising the “Therapeutic Needs” Lists prepared by the EMA-PDCO and engaging experts from paediatric clinical and nonclinical research initiatives (e.g. c4c, ERNs, EJP-RD, EnprEMA Members EPTRI, EUPFI, etc.) with the active participation of patients/children organisations.
- PRIME scheme adoption should be expanded once adapted to paediatric specificities.
- The current system of rewards and incentives should be implemented and harmonised in case of paediatric & orphan medicines.
- Technological and scientific advances should be embraced by adopting criteria based on mechanism of action instead of disease classification (also leading to revise waivers criteria).
- Effective and efficient procedures should be provided by accelerating and simplifying the PIP applications and by revising waivers and deferrals criteria.
- The implementation of regulatory-compliant innovative studies’ methodologies (Bayesan, adaptive design, RWD, etc) should be supported to develop paediatric and orphan medicines.
Best wishes from EPTRI
this very challenging year is coming to an end and I would take this occasion to thank all of you for your continued collaboration and engagement that has allowed EPTRI to reach great achievements. As you know, EPTRI ended the design phase and just entered in the preparatory phase.
The European Commission launched a consultation to revise the Orphan and Paediatric Regulations
EPTRI Webinar on the “EPTRI and EUPFI surveys: tools to investigate paediatric needs”
The EPTRI virtual meeting was held virtually on December 1st 2020. The meeting was addressed to all the 112 Research Institutions which signed the EPTRI Memorandum of Understanding aimed at organising the large existing community of paediatric researchers and facilities to impact the R&I in the drug discovery, design and development processes.
First meeting of the Italian Node (EPTRI-IT)
The first meeting of the EPTRI Italian Node (EPTRI-IT) was held online on October 28th, 2020. The 3 hours meeting was attended by more than 60 representatives coming from the 24 Italian Institutions – members of the Node.
The European Commission launched a Manifesto for EU COVID-19 research
The European Commission has launched a manifesto to maximise the accessibility of research results in the fight against COVID-19. The manifesto provides guiding principles for beneficiaries of EU research grants for coronavirus prevention, testing, treatment and vaccination to ensure that their research results will be accessible for all and guarantee a return on public investment.
EPTRI supports the Share4Brain project
In March 2020, the Erasmus+ program “Exchange of Good Practices for Brain Education in Europe” (Share4Brain) was kicked off with the aim to draw the attention of academics, relevant public institutions and the general public to the importance of basic and clinical research in neuroscience for better prevention, diagnosis and treatment of neurological and psychiatric disorders.
The European Commission adopted the “Pharmaceutical strategy for Europe”
On the 25th November 2020, the Commission adopted a “Pharmaceutical strategy for Europe” to ensure patients have access to innovative and affordable medicines and to support the competitiveness, innovative capacity and sustainability of the EU’s pharmaceutical industry. EPTRI participated in the consultation phase underlining the necessity to stress new adequate health policies and actions promoting the development and the access to safe and efficacious medicines specific for children.
EPTRI at the Europe Biobank Week 2020
From 17th to 20th November the European Biobank Week 2020 took place in a virtual format. The conference was a joint effort of ESBB and BBMRI-ERIC and for the first time it was fully digitized. Within the event, the abstract “Paediatric patients’ involvement fostered by EPTRI” was chosen for an oral speech held by Dr. Donato Bonifazi within the session “The roles of patients in personalised medicine development – from participation to involvement”.
EPTRI contribution at the ASCAT Conference
The 15th Annual Sickle Cell & Thalassaemia (ASCAT) Conference was held online on the 26th-31st of October 2020 together with the 1st European Sickle Cell Conference (EHA). Donato Bonifazi, EPTRI’s Coordinator, participated in the writing of an abstract proposed by Carsten Lederer and named “Catching them early: progress, economy and ethics of prenatal and childhood application of advanced therapies”.
EPTRI at the EAPS Meeting 2020
The 8th Congress of the European Academy of Paediatric Societies (EAPS 2020) was held virtually on the 16th – 19th of October, 2020 and was organised by three societies: the European Academy of Paediatrics (EAP), the European Society for Paediatric Research (ESPR) and the European Society of Paediatric and Neonatal Intensive Care (ESPNIC) with the eleven other European paediatric societies joining as collaborating societies.
EPTRI in the European Biotech Week
From the 28th of September until the 04th of October, the 2020 Edition of the European Biotech Week took place. In occasion of this event, EPTRI organised three webinars aimed to increase knowledge on how biotech has contributed to improve our life offering solutions to the major societal challenges.
Don’t miss the EJP-RD open calls
The European Joint Programme on Rare Diseases (EJP RD) has launched the EJP RD Joint Transnational Call (JTC) 2021 and will fund multilateral research projects on rare diseases under the EJP-COFUND action.
RE(ACT) Congress and IRDiRC Conference
January 13th to 15th 2021, Online
The 6th edition of the RE(ACT) Congress and the 4th IRDiRC Conference is organized in collaboration with the European Joint Program Rare Diseases and in partnership with Eurordis (European alliance of patient organizations) and RDI – Rare Diseases International (global alliance of people living with a rare disease). The meeting will be performed online from the 13th to 15th of January 2021. It will approach the theme of rare diseases and the application of diagnostic, artificial intelligence, new technologies and advanced therapies. The registration is open with an early discount fee applied until the 31st December 2020.
EPTRI submitted an abstract on the application of the sodium dichloroacetate in acute and chronic treatment of acquired and congenital lactic acidosis in the paediatric population.
Child Health Technology Conference 2021 – CHT2021
March 2nd to 5th 2021, Online
The NIHR Children and Young People MedTech Co-operative (NIHR CYP MedTech) and Marketing Sheffield organise the UK’s first Child Health Technology Conference – CHT2021.
It will be held online from the 2nd to the 5th of March 2021 with inspirational keynote talks, seminars, live Q&A sessions, interviews, cutting edge research updates, and live technology demonstrations on child health technologies.
The registration will remain open until the 26th of February 2021 with an early bird fee until the 18th of December.
EPTRI submitted an abstract on the actions performed to identify the paediatric framework of medical device research and the activities foreseen to foster the research in this field, that has been accepted as poster presentation.
International Meet & Expo on Surgery and Surgical Techniques – SURGERYMEET2021
September 13th to 15th 2021, Porto (Portugal)
SURGERYMEET2021 will comprise interesting scientific sessions, workshops and symposium seminars specifically dedicated to relevant fields in surgery, and sessions of proffered papers in Plenary and Keynote presentation. The meeting foresees an early bird registration within the 28th of December 2020. The EPTRI Coordinator Donato Bonifazi has been invited as Speaker with a talk in the Plenary Session on the EPTRI approach to tackle the challenges in paediatric medical devices research including the surgical ones.