Regulatory science provides the Medicines Evaluation Board (MEB) with up-to-date data, knowledge and expertise based on current scientific insights, for optimal evaluation and marketing authorisation of medicinal products and novel foods. This ensures an even better assessment of risks, efficacy and quality.
Regulatory science opens the door to optimal evaluation
In the last quarter of 2018 the MEB has visited and/or organised:
- FIGON Dutch Medicines Day, 3 October 2018
- Meeting EPTRI project, 5 October 2018
- PRISM at the 6-9 October 2018 ECNP Congress
- Ephor project
- Topra Symposium 2018
- Lygature Meeting 1 november 2018
- The 2nd international symposium for young researchers in regulatory science, 13-14 February 2019
FIGON Dutch Medicines Day, 1-2 October 2018
The overarching theme of the FIGON Dutch Medicines Days was
Sustainable innovation in oncology; from molecule to man, societal sustainability & health economy
The two sessions that were organised by the Regulatory Science Network Netherlands (RSNN) aligned perfectly with this theme, and focused on regulatory challenges for Advanced Therapy Medicinal Products (ATMPs), and evidence generation for oncological products.
Session: Regulatory challenges for the next wave of cancer therapies
Increasingly more oncology treatments are progressing from bench to clinic, including ATMPs, such as the chimeric T cell receptors (CAR-T) cells. Questions that were discussed during this session were: Is the current regulatory landscape ready to facilitate registration of these innovative therapies to patients at the earliest appropriate time? Can we learn from the hurdles and barriers in the current ATMP regulation, to prepare ourselves for new cancer therapies such as CAR-T? During the lively discussion, it was visualised that especially for the ATMPs there are many potential products in early development, but only a few make it to registration. To prevent valuable products from failing, and to facilitate an optimal development process, several participants mentioned that during development, the focus should be on the end goal. As Dr. Hoefnagel stated: “The product is the process itself”. Furthermore, as most ATMPs arise from research in academia, academic groups could be better educated in the regulatory processes.
Session: Evidence generation for innovative products in oncology
Given the high unmet medical need, there is an imperative to provide access to promising products to patients in an expedient manner. With a number of potential breakthrough therapies in the pipeline, there is a high need to discuss the type and the volume of evidence that should be generated. What will future evidence generation strategies look like? What are the consequences of these developments for regulatory and HTA decision making? One of the important lessons that could be drawn from this session, and which nicely complements the main message of the previous session, was that the stakeholders involved should discuss together at an early stage what evidence is needed to register a product, reimburse and prescribe it. According to Dr. Hennik, a systems approach is needed to speed up development of new oncology drugs, which integrates regulatory and HTA aspects of licensing a novel drug. Two examples of registries were presented, in which the registry of the European Society for Blood and Marrow Transplantation (EBMT) provided a good example of a registry, for which several dialogues with the European Medicines Agency have taken place, whereas the Dutch Melanoma Treatment Registry (DMTR) was presented as an example where the health technology agency ZIN (National Health Care Institute) was involved at an early stage.
Dr. Christine Gispen-de Wied steps down as vice-chair of the RSNN
Last but not least, Dr. Christine Gispen-de Wied was thanked for her huge contribution in setting up the RSNN, and her continuous enthusiasm for this network. She received a very warm applause from all participants for her support. Her successor as vice-chair is Dr. Peter van der Meer, also from the MEB, who was already closely involved with the RSNN.
MEB meets EPTRI, 5 October 2018
On 5 October 2018 the Medicines Evaluation Board of the Netherlands (MEB) met with participants from the European Paediatric Translational Research Infrastructure (EPTRI) consortium for an informal discussion on questions related to paediatric drug development.
EPTRI aims to propose developmental models for a future research infrastructure focused on paediatric medicines, and intends to integrate the paediatric expertise of five different areas of pre-clinical research: early discovery and preclinical studies, biomarkers, pharmacology, formulation and underpinning paediatric studies. The main objective of EPTRI is to facilitate the paediatric pre-clinical research while reducing the time of its translation to the clinical phase.
The project is funded by the European Union’s Horizon 2020 programme and involves 26 partners from EU and non-EU countries. One of these partners is Prof.dr Saskia de Wildt, Radboud UMC (read her interview in this magazine).
Update MEB-Ephor project October 2018
The mission of Ephor (Expertisecentre PHarmacotherapy in Old peRsons) is to improve appropriate pharmacotherapy to older patients. To achieve this goal, information is collected on the number of elderly people studied in trials, the number of older men and women using the drug in the Netherlands, data on effectiveness and adverse effects in elderly, the interaction potential, whether pharmacokinetic studies have been performed in the elderly, if the dose for the elderly must be adjusted, the ease of use and information about stopping the drug.
The information is displayed in the Ephor app. This app is available since February 2018 and currently contains information (in Dutch only) about 65 medicines and 13 medicinegroups to compare drugs of one class with each other, like SSRI’s or benzodiazepines. The app can be downloaded for free from Google Play Store or the Appstore and is also available on the computer: ephorapp.nl. De information will also be implemented into the Farmacotherapeutic Kompas.
Precision Medicines comes to Neurosymptomatics
PRISM at the 6-9 October 2018 ECNP Congress
Martien Kas, PRISM project co-ordinator, spoke at the session on digital technology at the 31st ECNP Congress taking place on 6-9 October 2018 in Barcelona. His talk entitled “Passive remote behavioural monitoring in PRISM, an Innovative Medicine Initiative project” will introduce “BEHAPP”, a smartphone application aimed to provide longitudinal, objective, and quantitative measures for social withdrawal in patients suffering from depression, schizophrenia, or Alzheimer disease.
IMI-2 ROADMAP, 8-9 October 2018 in Madrid
IMI-2 ROADMAP initiative moves into end of Phase 1 as it holds General Assembly Meeting in Madrid
On 8 and 9 October (Madrid, Spain), the Real World Outcomes across the Alzheimer’s disease spectrum for better care: Multi-modal data Access Platform (ROADMAP) project held its 6th and last General Assembly Meeting. The meeting was chaired by Project Coordinator John Gallacher (University of Oxford) and Project Leader Frédéric Reydet de Vulpillieres (Novartis).
The aim of the meeting was to discuss future plans and to provide the Consortium members with updates on final activities of the different work streams. The project will conclude at end of October 2018.
Read more in the IMI newsletter.
TOPRA Annual Symposium, 1-3 October 2018
In advance of the 2018 TOPRA Annual Symposium a round table was organised around the topic of artificial intelligence (AI). Participants from industry, regulators, consultants and a representative of the Bill and Melinda Gates Foundation discussed the use of AI in the pharmaceutical field, from clinical trials to the simple variation process. During the meeting our MEB deputy director, Stan van Belkum as Chair of the Regulatory Optimisation Group (ROG), introduced the idea for supporting process optimisation with the use of AI.
- Artificial intelligence in clinical development and regulatory affairs – preparing for the future by Van Belkum et.al.
This article was first published in Regulatory Rapporteur – The International Journal for Professionals in Regulatory Affairs (2018;15(11):17–21)
Lygature Partnerships MeetUp, 1 November 2018
A keynote lecture by Tomas Salmonson (former CHMP chair).
Regulatory Science Session “Extrapolation in Regulatory Science: Smarter use of available data” with speakers Kit Roes, Wim Goettsch (ZIN), Saskia de Wildt (RadboudUMC) and Bert Leufkens as moderator.
The 2nd international symposium for young researchers in regulatory science
Following the success of last year’s edition, we are excited to invite you to the second international symposium for young researchers in regulatory science. We look forward to hosting you for two days of inspiring workshops and lectures on regulatory science. The symposium is combined with the Science Day of the Dutch Medicines Evaluation Board on 14 February in the afternoon.
Please mark your calendars:
13-14 February 2019,
@ Utrecht University/Medicines Evaluation Board
Stay tuned for an official invitation with the programme!
Please share this with colleagues who might be interested, and do not hesitate to contact us for further details.