Morning parallel sessions
Morning parallel session 1
Complex drug products: new insights in development, production & characterization
Advanced Therapy Medicinal Products (ATMPs) and nanomedicines provide new opportunities to enlarge the medical toolbox. Yet the road from development to approval poses many challenges for scientists, developers and regulators. Only one or two cell therapies each year receive approval. Regulators struggle with the characterisation and therefore with the definition of complex drugs. Where the identity of a small molecule can be established by chemical analysis, that is seldom the case with more complex drugs. This proves a problem when you want to establish equivalence between the original drug and a generic replacement. Production and quality control can also pose many challenges. Collaboration is the only way forward.
Morning parallel session 2
Hands-on workshop: data management tools for translational research
In a successful hands-on workshop, participants were able to follow practical sessions lead by experts in various fields of data management in translational research. From clinical (OpenClinica - Jacob Rousseau) to imaging (XNAT - Adriaan Versteeg and Marcel Koek) to biobanking (OpenSpecimen - Erik van Iperen and Catalogue - Ricardo de Miranda Azevedo) research data, integration thereof (tranSMART - Mariska Bierkens and Wibo Pipping), as well as an application of the FAIR principles combining clinical and omics data (myFAIR analysis - Rick Jansen and Saskia Hiltemann). Participants were also able to obtain expert advice on their own data stewardship questions. Participants taking part in the practical sessions were involved and had good discussions.
Morning parallel session 3
Future topics for regulatory science in the Netherlands
During this session, moderated by Bert Leufkens (Utrecht University), the future regulatory science agenda in the Netherlands was discussed from four different viewpoints: the regulator, industry, academic and patient perspective.
Christine Gispen-de Wied (CBG/MEB) presented a topic list for future research based on multi-stakeholder discussions within the Regulatory Science Netwerk Nederland (RSNN) and internal priorities within the MEB. This list included amongst others the sharing of data across the health chain (from trial to reimbursement) and the use of real world data. Just Weemers (Dutch Association Innovative Medicines) accentuated the future challenge of collecting real world data that is of sufficient quality to create evidence. According to both Christine and Just, new sources of data and evidence should be explored in the future.
Dick de Zeeuw, professor at the UMCG, added to the future topic list the need for a personalized medicine approach in the regulatory environment, a viewpoint shared by Just Weemers. Patient’s responses may vary populations are becoming smaller, so and the unmet medical needs to become more personalized.
All speakers acknowledged the increased collaboration between stakeholders in the field of regulatory science, but more stakeholders should be connected in this dialogue. Patient advocate Cees Smit emphasized the importance of including patients in regulatory discussions, as outcomes of risk-benefit or cost-effectiveness studies are often divergent from patient experiences and expectations. In addition, societal dialogue is required for acceptance in further shaping the regulatory environment.
Overall, many topics for future research have been identified. Bert Leufkens concluded ended the session by underlining the importance of a safe research space to share and access data in multi-stakeholder collaboration.
Morning parallel session 4
Innovations for neglected tropical diseases: the next level
In this session, three different platforms were presented - from early discovery to a diagnostic field test.
From the various discussions about these presentations it became clear that in the field of NTDs, platforms are the way to go. They can be used, in case of discovery, to possibly tackle multiple diseases with similar targets at the same time, in case of model platforms to develop diagnostic markers for infection and drug and vaccine discovery, and in case of field diagnostics to develop multiple tests in rapid succession.
The keynote lecture was presented by Michel Rudolphie, former CEO of the Dutch Cancer Society (KWF Kankerbestrijding). KWF is the largest health fund in the Netherlands with an annual budget of €145 million from over one million donors. Every year over 45.000 people die from cancer in the Netherlands alone. KWF is determined to beat cancer, based on three goals: less cancer, more cure and a better quality of life for those affected by cancer, be it patients or family members.
Traditionally KWF was mainly concerned with funding research following a fund & forget strategy. The vision of Rudolphie when he started as head of KWF was to create more impact with integrated innovation. Scientific publications don’t cure patients. Rudolphie embarked on a quest to fundamentally change KWF from a charity fund to a cancer impact organisation. Still funding research, but through a ‘commit & integrate’ strategy. Many challenges still remain: how do you measure impact for example? How do you increase transparency? How to deal with strong emotions regarding change? How to grow in income as KWF?
Afternoon parellel sessions
Afternoon parallel session 1
Game changers in clinical research: wearable technology and e-monitoring
Samuel Smits and Lisa Vogelpoel from Gupta Strategists illustrated the benefits of moving patient care from hospital to home. There is a general belief that the concept of diagnosing and monitoring at distance strengthens healthcare and takes away a practical and financial burden for hospitals in the next decades. The most challenging tasks herein are to change current (more conservative) mindsets and train a new generation of physicians to fully implement the new roles for clinical personnel and patients in this system.
Hans-Peter Brunner-La Rocca and Josiane Boyne presented a practical example for healthcare assistance and monitoring at home: Molly, the virtual heart failure coach. It is expected that tools like this can majorly impact healthcare, not only through monitoring health and disease, but also as educative help for patients. Current challenges are to test the acceptance and usability of a nurse avatar in healthcare.
Faith Matcham pointed out that public-private partnerships in complex research projects such as RADAR-CNS provide multidisciplinary collaborations, ensure substantial financial investment and secure a sustainable study outcome. Regarding wearable devices for research, aspects of data storage, analysis and security and (inter)national regulatory requirements need to be carefully reviewed.
From this session we can conclude that the aim is to produce accessible and affordable innovative healthcare tools that can be standardized amongst multiple disease areas.
Afternoon parallel session 2
Sustainable business models in neglected tropical diseases
In this session, three spin-off companies in the field of NTDs presented their business models - finding a working business model is obviously difficult in a “not for profit” field.
“Break even” models can be accomplished by finding both government/industrial funding for each new project, the latter (industrial funding) often stemming from pharmaceutical CSR (Corporate Social Responsibility) programs. A model in which a (small) profit can be made, and a company can slowly grow, is hard to come up with. One company (TropIQ) however managed to develop a working hybrid model, combining further developing their core technology using government (national and EU) funding, with performing fee-for-service activities for e.g. PDPs and industry.
Afternoon parallel session 3
Dealing responsibly with ethical, legal and societal issues (ELSI) in Personalised Medicine research
Personalised Medicine promises to bring powerful new ways of improving treatment and health care, tailored to individual patients. At the same time, this leads to many different ethical, legal and societal issues. How, for instance, can privacy be protected when data sharing is the norm? Who decides how data is shared when research is funded by many different actors, including commercial companies? And what about patients’ rights and informed consent?
The national Health-RI Servicedesk could help practitioners to address these issues, by offering accessible advice, better opportunities for mutual learning and coordination, and an overview of areas for improvement. In this session, Susanne Rebers (Netherlands Cancer Institute, ELSI Servicedesk), explained the plans for the Servicedesk, a project which is currently in its start-up phase. By showing the different issues she faces in the AACR project GENIE, Mariska Bierkens (Netherlands Cancer Institute) illustrated the need for such a servicedesk. Participants engaged in an active discussion on the issues they face and the expectations they would have of the Servicedesk. The ELSI Servicedesk will be further improved using the valuable input gathered in the session. A beta version of the Servicedesk will be online from February 2018 onwards.
Afternoon parallel session 4
JIM session: ‘Transformation through collaboration’
One of the afternoon sessions was focused on the transformation of Healthcare through collaborative initiatives. Three motivated speakers presented both a vision but also actual achievements about the success of collaborations between different stakeholders. In a very open and active discussion it became clear that one of the main ingredients to make such a collaboration to a success is “trust”. Another ingredient that is sometimes needed is strong decisive leadership by the government which can help to speed up the process of collaboration. Another aspect that was discussed is how these collaborative achievements will or can affect current healthcare practices, or more specifically can reduce healthcare costs. The follow up on this is that we need transparency and a better communication to the public to show what is needed to develop new drugs or devices.