Meeting report 2018

This year’s Partnerships MeetUp, which took place on November 1 at the Jaarbeurs Utrecht was opened by Lygature’s managing director Jorg Janssen with a short presentation emphasizing the important role that partnerships play in meeting unmet medical needs. Lygature, he explained, believes that there are three key ingredients to successful collaboration: a safe, open and honest environment for discussion; a mix of stakeholders; and a driving force to facilitate interaction. With its relaxed informal atmosphere, the aim of the Partnerships MeetUp is to provide that safe environment, in which participants can explore beyond the boundaries of their disciplines and connect with others to find out where opportunities for new collaborations lie.

Click here to download the program booklet with detailed information about the sessions and speakers.

Keynote speech
The highlight of the day was a keynote speech by Dr. Tomas Salmonson, who as former chair of the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) is one of the most visible and well-known regulators of this decade. Dr. Salmonson shared his vision on the future of regulatory innovation, starting with a simple statement: “Together is the only way forward”. He explained that a lot has already been achieved by working together but also offered a warning: “We can be happy with where we are now, but if we do not move forward, we will not be happy tomorrow.”

Among the actions that were needed to move forward, Dr. Salmonson highlighted the need to establish an external control agency, and the need to think about endpoints other than purely clinical ones. With the EMA having moved to the Netherlands, he stressed that the Netherlands’ track record of building competencies from different stakeholders, and its capacity to build strong networks between stakeholders, makes the Netherlands ideally placed to provide the future leadership that is needed in regulatory innovation. He also stressed the opportunities in regulatory science for the Netherlands and Lygature, and the importance of seizing such opportunities.

Morning Parallel Sessions – Bringing Better Medical Solutions to Patients
The morning parallel sessions explored some of those opportunities under the theme of ‘Bringing Better Medical Solutions to Patients’.

Extrapolation in Regulatory Science: Smarter use of available data
The session titled ‘Extrapolation in Regulatory Science: Smarter use of available data’ explored perspectives on extrapolation in clinical drug development and regulation, health technology assessment (HTA), and clinical practice in presentations by Kit Roes (Julius Center/UMC Utrecht), Wim Goettsch (Zorginstituut Nederland) and Saskia de Wildt (RadboudUMC) respectively.

Extrapolation, it transpired, is not a new topic but in the last few years more awareness has been gained about the uncertainties that are an integral part of extrapolation. The session highlighted the fact that if stakeholders are not involved to fill the knowledge gaps, those uncertainties remain in place and shift to clinical practice. The Dutch ‘Kinderformularium’, which translates clinical evidence and knowledge in the public domain into best-evidence drug dosages for children, was cited as an example of how these uncertainties can be addressed. In HTA, it was explained that uncertainty is addressed by moving towards a life-cycle approach, for example, leveraging post-licensing data collection to establish real-world data. The session concluded that it is always necessary to be explicit about the uncertainties and to specify the data requirements needed to address those uncertainties.

Registries: Real World Challenges in Real Life Data
The power of real-life data was highlighted in the parallel session titled ‘Registries: Real World Challenges in Real Life Data’, in which Professor Gerrit Meijer, CSO of Health-RI, introduced the hot topic of how to optimally use available data to provide evidence for better health and healthcare. He was followed by three further speakers, Dennis van Veghel from the Netherlands Heart Registry, Jacquelien Noordhoek from the Dutch Cystic Fibrosis Registry, and Iris Nagtegaal from the Public Pathology Database (PALGA), who shared their views and showed how their respective registries are successfully creating maximum value for patients and society. They clearly illustrated the common challenges that are faced when working with real life data and provided insights into the possible solutions to overcome these challenges.

In the second half of the session, Joep Rijnierse, chair of the Real Life Data working group from the Association Innovative Medicines, generated a positive discussion about the conditions registries should adhere to in order to sustainably provide valuable sources of information. Many stakeholders present supported these conditions and agreed that, in addition to technical innovations, a nationally integrated masterplan is needed to tackle the challenges at organizational and cultural level – a national collaboration clearly involving both public and private stakeholders.

Adapting and Reshaping Medicine for an Ageing Population
The three other morning parallel sessions focused on current real-world challenges in bringing better medical solutions to patients. The session titled ‘Adapting and Reshaping Medicine for an Ageing Population’ addressed the fact that three million people in the Netherlands are already over the age of 65 years and face much greater challenges in maintaining good health than the younger population. The speakers, each of whom approached the challenges from a different angle, contributed to a valuable discussion about how the challenges could be met. Professor Sven Stegemann of Graz University of Technology provided a holistic overview of the patient experience in which key issues such as treatment adherence were addressed. Professor Eline Slagboom from Leiden University Medical Center highlighted the need for ageing biomarkers to monitor health across the life course and bring about lifestyle interventions, while Dr. Paul Jansen from the Expertise Centre for Pharmacotherapy in Old Persons (EPHOR) at UMC Utrecht provided his perspective of the difficulties general practitioners experience when prescribing medicines to patients suffering from multiple conditions and who therefore have to take multiple medicines simultaneously. Emphasis on the need to improve geriatric care was reflected in the Partnerships MeetUp keynote speech by Tomas Salmonson.

Crossing borders: moving towards One Health
The second session focused on the ‘One Health’ approach to designing and implementing programs, policies, legislation and research in which multiple sectors – for example, the human health and veterinary sectors – communicate and work together to achieve better public health outcomes. The first part of the discussion focused on understanding the scope of One Health – notably on the position different research fields and stakeholders take and the challenges faced. The conclusion was that one of the biggest challenges is setting up effective public-private partnerships to work together on One Health research.

The second half of this session focused on antimicrobial resistance (AMR), one of the areas in which the One Health approach is particularly relevant. The enormous impact of AMR was highlighted, and solutions were discussed to steer new research initiatives in the right direction by involving academia, industry and governments globally – crossing borders to find new international funding strategies, creating new public and private partnerships, and bridging different scientific disciplines.

Addressing the Needs of Poverty-Related Disease patients: Global Partnerships with a “Dutch twist”
The last session of the morning focused on the specific needs of poverty-related disease patients and the central role the Netherlands plays in R&D on neglected tropical diseases, which account for most of these patients. All four speakers, Remko van Leeuwen from Amsterdam Institute for Global Health and Development (AIGHD), Danielle Roordink from the Tuberculosis Vaccine Initiative (TBVI), Sake de Vlas from Erasmus University Medical Center, and Remco de Vrueh from the Pediatric Praziquantel Consortium, made it clear that developing therapeutic interventions, such as drugs or vaccines, is crucial but only one piece of a bigger puzzle. They emphasized that these efforts should be accompanied by programs and partnerships targeting health education, appropriate point-of-care diagnostics, proper hygiene, healthcare systems, etc. The conclusion drawn was that targeting the needs of patients with neglected tropical diseases, including tuberculosis and malaria, requires an integrated multidisciplinary approach to really make an impact.

Afternoon Parallel Sessions – Essentials for Successful Collaborative Innovation
With the morning sessions highlighting the importance of collaboration in bringing better solutions for patients, the afternoon sessions, which followed the keynote speech from Dr. Tomas Salmonson described earlier, focused on what is needed to create effective collaborations under the theme ‘Essentials for Successful Collaborative Innovation’

Post-innovation Innovation: Platforms for Drug Repurposing
The first session discussed the necessary conditions for breathing new life into old or dormant drug (development) compounds. Speakers from different types of organization – Adrian Freeman from pharma company AstraZeneca, Jean-Pierre Paccaud from the Global Antibiotic R&D Partnership (GARDP), Vincent van der Wel from the not-for-profit Fair Medicine Foundation, and Joris Langedijk from the Brabers Corporate Counsel consultancy – presented different models for drug repurposing. The many case studies they presented highlighted the fact that repurposing can provide a shortcut to meeting medical needs, and that this ‘post-innovation’ innovation can be realized in many different ways – from purely serendipitous to rational strategic data mining. However, it was also concluded that cost and effort of doing so are often underestimated, and that successful repurposing relies on legal, financial and regulatory frameworks and on sufficient funding for new (pre)clinical studies.

Why, How and What you Win when you Involve Patients
Another parallel session, moderated by senior advisor at PGOsupport Annemiek van Rensen, focused on the benefits of involving patients in setting research agendas by answering three simple questions: why do you win, how do you win, and what do you win?

According to speakers Jane Taylor and Harrie Weinans from the Innovative Medicines Initiative (IMI) APPROACH project, why you win is because patients can add valuable knowledge, experience and perspectives to research projects.

How you win, they explained, is by acknowledging the different roles patients can fulfill, such as advising, reviewing or co-producing research agendas and project proposals.

And finally, they explained that what you win is greater participation in clinical trials through more patient-friendly clinical protocols and better patient information – for example, newsletters written by and for patients or discussions based on individual patient experiences that create focus and thereby increase the impact of the research.

Consortia Branding: How to Connect to Stakeholders?
In relation to creating exposure and creating impact, a parallel workshop session presented by media communications expert Bas Douma and science journalist and presenter Marloes ten Kate, provided useful tips on consortia branding and connecting to stakeholders. They highlighted the fact that most research consortia are temporary in nature, yet to have the greatest impact they need to be properly defined and widely recognized. The workshop focused on what research consortia can learn from marketing: how can you give something as complex as a consortium real-world meaning. Bas Douma’s biggest tip was to focus on creating awareness, not by relying solely on communications people, but by getting researchers and scientists actively involved in making consortia visible. Marloes ten Kate contributed useful tips on how to do this, explaining that to get your message across, you need emotion as the vehicle for your facts, and that a good story needs to have a main character, a problem and a solution. Most scientists are not used to thinking about their research this way, but getting them actively involved in communicating a research consortium’s goals will certainly help to get journalists and other stakeholders interested.

The other two sessions in the afternoon focused on two essentials for successful collaborative innovation, the first on data collection and privacy and the second on partnership strategies for affordable and accessible healthcare.

Conflicting? Big Data and Privacy
In the session on data collection and privacy, moderated by Nico van Meeteren from Health~Holland, presenter Martijn van Oijen from Amsterdam UMC highlighted the potential value and shortcomings of using ‘Big Data’, collected via patient reported symptom monitoring, wearable devices and social media. One conclusion was that social media is a valuable source for identifying signals at population level, which could trigger ideas for setting up studies and give caregivers greater insight into how patients experience a particular disease.

Birgit Wouters from Maastricht University went on to explain the foundations of the General Data Protection Regulation (GDPR) and the issues and consequences surrounding Big Data based research and protection of the data subject’s privacy. Acknowledging that guaranteeing privacy is essentially impossible with all available data, Birgit proposed that personal data should be collected based on a contract (confidentiality) basis rather than on consent (privacy) basis.

The audience was polled on their opinions about Big Data and data privacy before and after the discussion, with several changes of opinion triggering a lively follow-on discussion. Everyone agreed that big data should be used in health research, but regarding the use of personal data, the consensus was that the benefit-risk ratio should be evaluated.

Partnership Strategies towards Affordable and Accessible Healthcare
In the session on partnership strategies for affordable and accessible healthcare, Lygature board member Michel Rudolphie outlined the challenge of finding new (business) models that strike a balance between innovation and costs when developing new medicines. Representatives from four initiatives – Jon de Vlieger representing the European Lead Factory, Hans Büller representing the FAIR Medicine initiative, Irene Kanter-Schlifke representing the Oncode Institute, and Netty Dorrestijn representing the Utrecht Center for Affordable Biotherapeutics – shared their own ways of involving all relevant stakeholders in a public-private initiative at different stages of the drug discovery and development phase. Current best practices were shared during the session, with a ‘helicopter view’ discussion suggesting areas for alignment and collaboration.

The general conclusion was that a greater number of innovative partnerships were needed, but each one is now an experiment in itself with the solution, if there is one, yet to be found. It was agreed that transparent, evidence-based constructive dialogue is an essential element for success, allowing the community to further explore ways of leveraging investments already made in fundamental and translational science by public and private partners for the benefit of population health and economic growth.

Closing Remarks
With all the parallel sessions very well attended, many to maximum capacity, everyone came together to listen to a closing speech by Lygature’s Scientific Leadership Team member Bert Leufkens. Summing up the day, he proposed that healthcare and medicine development require a collaborative multidisciplinary approach, exemplified by the activities of Lygature and the Partnerships MeetUp itself. He highlighted that every session and speech had talked about the need to cross the borders between disciplines and stimulate opportunities for collaboration. The Partnerships MeetUp had, he said, successfully provided the safe environment of open and informal discussion that it aimed to create, because what was apparent throughout the day was the open-mindedness of the participants and their willingness to respect and understand each other’s views.

Citing Lygature’s project portfolio, he explained that bringing disciplines together definitely works, and that organizations like Lygature are a driving force for building trust and facilitating collaboration between stakeholders. Multidisciplinary collaboration is at the heart of Lygature, he said, and the Partnerships MeetUp clearly demonstrates that Lygature is successful in making partnerships work.