Scientific Advisory Board. Report of the first meeting

Transcription

1 Scientific Advisory Board Report of the first meeting Utrecht, The Netherlands, May 15 and 16,

2 1) Introduction With this document, the Scientific Advisory Board reports on its discussions held in May 2017 on the scientific strategy of Lygature. This document consists of three parts. (1) On this page, the task and composition of the Board are introduced as well as the agenda of the first meeting. (2) On page 3, a summary of the conclusions of the two discussion sessions is provided. And finally, (3) on page 4 until 6, a more detailed write up is included of the discussions during the entire meeting. This report is made publicly available via the Lygature website to inform Lygature stakeholders. Task and composition On Monday, May 15 and Tuesday May 16, 2017, the first annual meeting was held of the Scientific Advisory Board (SAB). The SAB is appointed by the Lygature Board of Directors. The SAB task is threefold: - Advice on the coherence within Lygature s project portfolio on an annual basis. Provide feedback and identify chances for cross fertilization between projects in the various domains - Provide input on the development of new public-private initiatives from an international perspective, building upon the strengths of the Dutch life sciences and health sector - On an ad hoc basis: review of project proposals which will be submitted by Lygature towards various funders of consortia (e.g. Bill & Melinda Gates Foundation, GHIT (Global Health Initiative Technology) Fund, ) The members of the SAB together span the dimensions in which Lygature is active: scientific discipline (medtech, pharma), geographic scope (national, international), organizational nature (public, private), etc. The current members (May 2017) of the SAB are: - Daan Crommelin (chair), Emeritus-Professor Biopharmaceutics at Utrecht University - Rob Williams, PhD, Chief Drug Development Scientist at the Centre for Drug Development, Cancer Research UK - Matthias Gottwald, Head of R&D Policy and Networking at Bayer AG Pharmaceuticals Division - Laurent Degos, MD PhD, Professor Emerite of Hematology at University of Paris, Diderot Hopital Saint Louis, Paris, and past Vice President of the Pasteur Institute and Curie Institute - Sjaak Deckers, PhD, co-founder and CEO of G-Therapeutics BV Agenda The meeting was divided into three blocks: 1) Introduction to Lygature and its role in two major initiatives 2) Discussion on the evolution of public-private partnerships 3) Discussion on specific topics: a. biobanks, data and registries b. regulatory innovation Next to the Lygature Management Team consisting of Peter Luijten, Ton Rijnders and Jorg Janssen, five staff members joined the specific topics sessions: Remco de Vrueh, Erna Erdtsieck-Ernste, Eric Caldenhoven, Pieter Stolk and Jon de Vlieger. 2

3 2) Conclusions The two discussion sessions resulted in the following observations. Discussion on the evolution of public-private partnerships: - Public private partnerships have become mainstream, whereas the form, size and composition are very diverse. Bilateral versus multilateral, government-academia, industryacademia, etc. There is no one size fits all - It is of great added value, not only for the Netherlands, but also internationally, that Lygature has been able to secure the expertise built up in facilitating partnerships that ran within the CTMM and TI Pharma programs - Some further common language should be developed to distinguish between PPP s: precompetitive multilateral R&D partnerships, bilateral deals between pharma and academic institutions, product development partnerships, etc. - In Europe, PPPs are challenged by an overload of bureaucracy, whereas for example the ERC grants by the European Union are an example of an efficient decision -making process - PPPs can serve as a breeding ground for future scientists who are used to collaborating in multi-stakeholder platforms. This is also true for lawyers: train them through PPP involvement to broaden their view and horizon. - A difficult question is how to address scientists that are not aware of the complexity of PPPs and want to invent the wheel themselves. - PPPs in pre-competitive research & development form the majority of PPPs, PPPs in regulatory innovation are growing, and an untapped domain for PPPs are post-licensing studies. - PPPs should facilitate in helping the sector in the orphanization of diseases: the segmentation to distribute risks. This is not only true for drugs, but also for medtech. Discussion on specific topics: biobanks and regulatory innovation: - For biobanks, data and registry infrastructures, securing long-term sustainability should be a top priority. User-driven approaches, combined with business canvas exercises to develop business models, are essential elements to achieve this. - In regulatory innovation, the need for a neutral enabler such as Lygature is evident. Make sure to continue the focus on science-based discussions - Overall, the combination of infrastructure involvement and regulatory expertise, as well as the possibility to leverage this combination in operational roles, is providing Lygature with clear and unique added value in its progam management. - Additionally, a right balance has been reached in the Lygature project portfolio. This balance should be continued: between Dutch and European projects, combined with truly international initiatives in specific niches. - Make optimal use of the Netherlands as a hotbed for medical innovations. - Make sure to be able to initiate new initiatives in the future and hence strive for sufficient resources to be able to invest in future initiatives. - It would be useful to provide SAB with a comprehensive overview of current projects and the roles in these projects executed by Lygature. 3

4 3) Detailed write up Introduction to Lygature and its role in major initiatives Lygature is the result of the merger of two large public-private partnerships (PPP): the Center for Translational Molecular Medicine (CTMM) and Top Institute Pharma (TI Pharma). Lygature has secured the public-private program management expertise built up within these two PPPs and has made these alliance management capabilities available to other public-private partnerships. In the discussions, the term alliance management was brought up as another way of describing program - or research management. The Health RI (Health Research Infrastructure) initiative was presented. In Health-RI, the common goal is to interconnect biomedical resources such as data, biobanks, to empower researchers to develop better personalized medicine and health solutions. Health-RI is the interconnected infrastructure for personalized medicine & health research in the Netherlands. As such, it has also the ambition to form the single point of contact towards various biomedical European infrastructure initiatives. Through Health RI, researchers should feel comfortable that for example all legal procedures are arranged professionally. In addition, combined with clear communication to the public it can help to increase the number of people (patients) willing to provide access to their private data. Experience in France could be taken as an example in this case in how they address the question of getting people committed to allow the use of their data. The European Lead Factory (ELF) was presented. This initiative has seen rapid progress since its start in After ramping up with compounds from the seven participating pharma companies, the chemistry part of the consortium has delivered new compounds to the Joint European Compound Library. To date, 85 public screening campaigns have been run or are underway. They have resulted in evenly distributed hits over the library, indicating the quality brought in by the various companies. In size, with 17 screening campaigns executed in 2016, the ELF effort is comparable with a small large pharma company. The public screening campaigns show a large percentage of screens in the area of infectious (including AMR) and neglected tropical diseases. The results of the ELF are broad: drug candidates, start-ups, patents and publications, knowledge transfer and more. Its impact on the European life sciences sector is evident and its impact on public health is promising. Sustainability is now the prime focus. Various models are worked on and discussed with the SAB. Sustainability should come from a mix of funding sources including EU funding, charities, as well as investors to fund follow up work. Discussion on the evolution of public-private partnerships PPPs are found in various forms: from bilateral partnerships between a company and university in the competitive sphere, to multilateral pre-competitive partnerships with a range of stakeholders, for example to validate tools and processes. Any combination of two organizations from either government, industry, academia or societal organizations, is currently viewed as a PPP. Trends are more cross functional PPPs, for example in combining medtech and pharma, such as the complementary drug-device interaction. Other examples of partnerships are repurposing initiatives 4

5 and cohort pooling. Cohort pooling represents a rather new type of partnerships of which the IMI- EPAD project and the new pediatric clinical trial network established with IMI funds are mentioned as examples. The establishment of PPPs can be viewed historically as a result of safety issues with blockbuster drugs. The public debates regarding these issues can be interpreted as prelude to the end of drugs for large populations. The dynamics move from treating large populations with a single drug to treating orphan diseases with more targeted drugs, and steps further to personalized -, and ultimately precision medicine. This is the overarching trend of the past decade in which large pharma decreased its R&D efforts and moved to sourcing of ideas at academia. In addition, the past decade saw the onset of discussions with a broad range of stakeholders to improve regulatory processes to enhance speed and quality of the approval process. An example of the latter is the design of the adaptive pathway concept. Looking at the adaptive pathway concept, more evidence will have to be gathered once drugs are on the market to provide proof for safety. The post licensing studies needed to obtain the data to provide further proof for safety is yet an unexplored terrain where PPPs may prove to be the answer to address these issues. The involvement of industry is essential and its specific role in these PPPs has to be discussed. In France, the forfait innovation between the Haute Autorité de Santé (HAS) and medtech industry was recently initiated, giving the opportunity of an early reimbursement under conditions. Next to scientific topics for PPPs, the way in which PPPs are organized is changing. More and more, they move to finding solutions for, and now also together with, patients. For the involvement of patients however, there is no clear protocol on how to reimburse them for their roles. This should be addressed, for example by or within IMI. On the other hand, there is also a trend to avoid working with pharma companies, e.g. with some patient organisations using statements like pharma free. This will work contra-productive. Openness and transparency is needed among all partners in a PPP, to make best use of the respective expertise for the benefit of patients. With respect to Europe, the bureaucracy in the Netherlands is, relatively speaking, less of a burden than in the EU. The Netherlands should work on bringing this strength of the Dutch sector to Europe. Additionally, especially for funding schemes beyond pre-competitive research partnerships it should become normal in Europe to pay back the public funding in case of commercial success. As a last point, the social element is discussed as one of the key success factors for PPPs. The rise in PPP initiatives over the past decade has contributed to an increased mobility of people moving between jobs in academia and industry. In turn, this increases the understanding between the various cultures and contributes again to the success of PPPs. 5

6 Two sessions on specific topics In the specific topics session, the experience in various projects was shared with the Scientific Advisory Board. Biobanks, data and registries: Through its program management roles in connecting biobank catalogues (BBMRI), clinical biobanks and data collections (Parelsnoer) and a toolbox for translational research (TraIT), as well as in the development of the Health RI initiative, Lygature has an added value in connecting these initiatives. In addition, the expertise and possible connections are valuable for other research projects facilitated by Lygature as they can benefit from using these research infrastructures. Trends in biobanking include the need for inclusion of phenotypic data, being able to create larger cohorts and multi-source datasets and long term follow up. The introduction of dynamic informed consent, and continuous communication with patients in cohorts, will add to the flexibility and usage of these cohorts and biobanks. Ultimately, this will benefit these patient groups. The TraIT initiative was discussed in more detail: starting as a bottom-up initiative to deliver a toolbox for translational research for CTMM projects, it is now being used by a much wider community of more than 3000 researchers. This impressive uptake is a clear indication of the quality and user driven setup of TraIT. The toolbox is constantly being updated (including removal of tools that are not broadly used). Currently the development of a registry in a box solution is worked on. Ideally, this should also connect with registries at insurers and allow to couple data with biobanks. With its involvement in hands on projects, Lygature is able to combine policy requirements with a user driven setup. The user driven setup is an essential element for future success. Sustainability of these infrastructures is, also in European projects, a challenge. User driven approaches and business canvas exercises may be helpful in securing infrastructure availability for the field and ensure efficient use of resources. The SAB advices to brand the program management role by Lygature better: be good but also tell it. Regulatory innovation: As a second specific topic, various projects that either focus on regulatory innovation (Adapt-Smart, NBCD, the Escher platform), or projects where regulatory expertise is brought in (such as RADAR- CNS) were discussed. In the first type of project, the neutral enabler or honest broker role by Lygature is an essential element for the success of these projects. It is good to see that the EMA is taking a leading role in for example the adaptive pathway development and that payers are involved in these discussions. As concluded in the general discussion, post approval evidence studies will become of vital importance for concepts like the adaptive pathways. Overall, the combination of running research infrastructure and regulatory innovation initiatives, as well as the possibility to leverage this combination in operational roles for other projects, is providing Lygature with clear and unique added value in its alliance management capabilities. 6