VAC2VAC annual meeting 2019
The 3rd annual meeting of the IMI project VAC2VAC was hosted by the Dutch National Institute for Public Health and the Environment (RIVM) in Bilthoven on 26-28 March. In VAC2VAC academic, industrial and regulatory partners from several EU countries collaborate to replace animal tests used in the release testing of seven vaccines (incl. both human and veterinary vaccines) with innovative animal-free methods.
About 80 participants represented the involved vaccine manufacturers, EU regulators, research groups and OMCL (official Medicine Control Laboratories). Also there were regulators from the US and Canada. This year’s meeting was opened by MEB director Hugo Hurts who gave an inspirational talk and articulated how well VAC2VAC fits in with the Netherlands’ aim to be a front runner in policy concerning the reduction, refinement and replacement of test animal use.
The innovative animal free methods under development within the project include physicochemical, immunochemical and cell-based methods. At this 3rd Annual Meeting significant progress was reported for the different Work packages for the different vaccines. While most methods are still in the exploratory phase, some promising results were presented. Some methods are further in their development and are moving to the next phase for suitability testing at the vaccine manufacturer’s site.
The focus of the regulatory partners involved in the project is to advise on and facilitate the implementation of innovative animal free methods. From the MEB Coen Stalpers and Marcel Hoefnagel are involved in VAC2VAC. Coen is exploring in vitro assay methods (using dendritic cells) to determine immunogenicity of Diphtheria, Tetanus and Pertussis antigens in adjuvanted vaccines. Furthermore, he is evaluating the use of animal testing to ensure consistency of vaccine immunogenicity. See also the overview of all PhD students and their research projects [link naar overzicht publicaties PhD].
VAC2VAC is financed for 5 years by the “Innovative Medicines Initiative” (IMI2), a joint initiative of the European Union and the European Federation of Pharmaceutical Industries and Associations.
ZonMW congress on Better Use of Medicines
The ZonMw programme Better Use of Medicines held its 7th annual congress on 4 April 2019: ‘Medicines in practice’. Presentations of recent research, panel discussions and brainstorming sessions inspired the participants to reflect on future developments. The Medicines Evaluation Board (MEB) contributed also to the programme.
The congress, held in the Beurs van Berlage in Amsterdam, focused on the appropriate use of medicines. How can we use medicines in such a way that patients benefit optimally? And what is the indispensable role of primary care professionals in this regard? The varied programme highlighted the results of several innovative projects.
The day started out with presentations about the value of preventive medicines, for example vaccines. Following these plenary presentations, there were multiple workshops. The MEB contributed to several workshops, that zoomed in on drug rediscovery and side effects of medicines. On the subject of drug rediscovery Carla Jonker indicated that the MEB assesses the balance between efficacy and safety for each medicine; drug rediscovery products do not differ from other reviews.
In the workshop about side effects of medicines, the Netherlands Pharmacovigilance centre Lareb and the MEB presented the identification process of side effects and the consequences for clinical practice. Furthermore, the MEB and Lareb elaborated on several pregnancy prevention programmes in a workshop about the use of medicines during pregnancy.
In addition, a plenary session was held about the use of opioids in primary care. Medicines such as oxycodone, fentanyl, morphine and buprenorphine - the so-called opioids - are easily prescribed. General practitioner Dr. Joost Zaat and pharmacist researcher and MEB board member Prof. Marcel Bouvy highlighted this theme, in which a panel consisting of several health care professionals, including vice-chair of the MEB Dr. Vera Deneer reflected on several statements.
RSNN Workshop 2019 – The future of clinical trials and evidence generation, and their use in regulatory decision making
On 17 April 2019, the fourth RSNN Workshop was organized. This multi-stakeholder event offered an opportunity to identify factors driving the need for innovation in clinical trials, an exploration of the options, and an overview of where we stand.
The golden standard for clinical trials, the randomised controlled trial (RCT), is no longer able to always deliver the answers needed to assess if a medicine is safe and effective. There is a clear necessity for innovation in clinical trials. During the workshop, several factors that drive the need to transform clinical trials were discussed. Several options that can accommodate these unmet needs also passed the stage. These include academia helping with designing trials that answer a specific question, using real-world data or evidence in pre- and post-marketing studies, and re-assessing the current rules and regulations to identify areas that allow for more regulatory flexibility.
The meeting was opened by Prof. Bert Leufkens, chair of the RSNN. Prof. Leufkens’ opening was followed by four speakers with different backgrounds (Prof. Joop van Gerven – Chairman Centrale Commissie Mensgebonden Onderzoek; Prof. Rolf Groenwold – Leiden University Medical Centre; Prof. Ton de Boer – MEB Chairman; Rebecca Lumsden, PhD – Director Europe and International Regulatory Policy at Pfizer) who shared their views on this subject in a plenary session. After the plenary session, the attendees split up into four smaller workshop groups to discuss the topic more in-depth. The full report of the meeting will be soon available on the RSNN website.
On 3-4 April 2019, a symposium was held by the BEAt-DKD consortium (www.beat-dkd.eu) on Precision Medicine in Diabetic Kidney Disease in Amsterdam, the Netherlands. The main aim of this symposium was to identify benefits, obstacles and challenges for registering and implementing biomarker tools into daily clinical practice, and to reach a consensus about a way forward to implement precision medicine in order to stop development of kidney impairments in diabetic patients. Seventy-two participants attended the two day symposium, including a good representation of regulators (National Competent Authorities among which the Medicines Evaluation Board, European Medicines Agency, United States of America Food and Drug Administration and Health Technology Agencies, see picture).
The attendees were generally very positive stating that precision medicine would benefit individual patients, and reduce the number of patients unnecessarily treated. Main challenges were implementation – requiring time and training of healthcare professionals - in clinical practice. Health Technology Agencies warned though that costs involved in diagnostic work-up will have to be considered in reimbursement discussions. Regulators indicated early interaction with trialists and industry is required to discuss value of biomarker-guided therapy, and how to deal with so-called ‘biomarker-negative’ patients that are supposedly less responsive to therapy. This requires early and step-wise development plans that make optimal use of explorative and possible adaptive study designs.
The BEAt-DKD consortium is a public private partnership of 20 academic institutions and eight biopharmaceutical companies supported by Innovative Medicines Initiative (IMI). BEAt-DKD's goal is to improve prevention and treatment of Diabetic Kidney Disease through precision medicine. Ways to reach this goal are to identify novel biomarkers, estimate individual renal and cardiovascular risk, and improve stratification of patients. An object of BEAt-DKD is to submit a proposal for biomarker qualification to regulatory agencies and development of clinical trials design to fit into precision medicine needs.
BEAt-DKD is organized in six Work Packages, out of them first four (WP1-4) concentrate on “discovery” and last two (WP5-6) concern “validation, integration and translation”. Specifically, Work Package 6 (lead by the University Medical Center Groningen) focuses on applying knowledge generated from other WPs into precision medicine approaches into clinical practice by developing an optimal clinical study design and integration in the regulatory process of drug registration.