PhD theses published
The Escher platform
Escher’s mission is to promote research and international debate in the field of policy and regulations, relating to development, market authorization, reimbursement, and use of medicines and medical technology. To this end, Escher initiates and conducts its own research into regulatory and health technology assessment topics. The platform also plays an active role in assignments undertaken by Lygature for external parties, for example within the ADAPT SMART project. In all cases, outcomes are translated into publications and/or practical proposals for regulatory and policy innovation, in order to facilitate patient access to medicines that address public health needs.
Providing neutral ground for multi-stakeholder dialogue and research
Scientific research and collaboration between public and private partners have been essential to Escher from the start. The platform reaches out to many different stakeholders, with partners from industry academia, government, NGOs and elsewhere. The broad range of activities that TI Pharma provided to support multi-stakeholder dialogue and research will be continued under the Lygature brand from January 2016, and include:
- Governance and coordination. Lygature is the legal entity responsible for the Escher platform and provides the chair of the Steering Committee, which decides whether and how a research project should be executed. Lygature also involves Escher when coordinating third-party assignments on regulatory topics, such as ADAPT SMART.
- Research activities. The Escher platform can offer an independent perspective on policy and regulatory topics. It initiates and conducts research projects, such as ‘Improving the EU system for marketing authorization’ and ‘Traceability of biologics’ (both now completed).
- Platform for dialogue. Lygature allows Escher to provide a multi-stakeholder home for regulatory innovation. The platform promotes dialogue on relevant policy topics, fueled by the evidence generated during research projects.
Escher is a unique platform that brings together a wide variety of stakeholders in strategic policy projects and research initiatives. It generates benefits by applying an understanding of both scientific and regulatory issues. Escher is a significant component in the wider Lygature mission to coordinate multi-stakeholder dialogue, and to help create more efficient and effective regulatory system.
Escher is currently working on one large project, executed in collaboration with research institutions in the Escher network and supported by expertise and data from public and private stakeholders. Still more projects are progressing through the planning phase.
In order to maintain a strategic focus, Escher concentrates on projects that align with four main research themes. Within each main theme, a number of projects have been completed:
- The UK BIO-TRAC study: An evaluation of product and batch traceability in UK clinical practice and reporting of adverse drug reactions
- Evidence generation methods and evidence requirements
- Scientific dialogue and stakeholder interaction
- The decision-making process and benefit-risk assessment
- Evaluating the regulatory system
Outputs from Escher
Escher is focused not only on generating new evidence, but also on disseminating it as effectively as possible. The goal is always to do everything possible to help translate research findings into real-world regulatory improvements, creating benefits for patients.
Results are therefore presented in many different ways. These include formal scientific papers in leading journals and PhD theses, but they extend much further. Events are hosted to encourage face-to-face discussion, policy reports are written to stimulate debate and press releases raise awareness of the progress made in the projects.
Final call ATMP survey: Help improve product development and regulation
Advanced Therapy Medicinal Products (ATMPs) are medicinal products including gene therapies, somatic cell therapies and tissue engineered products. They are characterised by their innovative character. Despite considerable development activity in this dynamic field, a limited number of ATMPs have received Marketing Authorisation in the European Union. There is a growing urgency to understand which factors are associated with the advancement of ATMPs through the medicines life-cycle.
End of March 2017, Lygature, a Dutch public-private collaboration catalysing new medical solutions, the European Biopharmaceutical Enterprises (EBE), together with EFPIA (European Federation of Pharmacetical Industries and Associations (EFPIA) and Utrecht University (UU) launched an open inivitation to European ATMP-developers to help product development, regulation and commercialization.
ATMP developers – past and present – were invited to participate in a survey investigating factors, impacting the development ATMPs within the EU. Amongst others, scientific challenges, manufacturing issues, clinical considerations and regulatory requirements are explored. This is the first of its kind research investigating the developer challenges in a systematic way.
The response to the survey continues to increase, with new participation requests still coming in. This shows the commitment of developers, and their willingness to improve ATMP product development and its regulation. Recognizing the need for comprehensive information and acknowledging the willingness to contribute, the survey remains open until the end of June.
Interested ATMP developers can still contact Lygature to participate at firstname.lastname@example.org.
- The survey is conducted on-line and takes approximately 30 minutes to complete.
- Results are expected late 2017 and will be shared with survey respondents.
- Optimising confidentiality of participating developers Lygature acts as intermediate (as honest broker) between the ATMP developers and the Utrecht University research team, ensuring data security and anonymizing responses. Results will only be published using aggregate data, making it impossible to trace back information to individual developers. The intent is to publish the results in a scientific journal.
The research is carried out with an unrestricted research grant from EFPIA and EBE.
UK BIO-TRAC study off to a good start
The UK BIO-TRAC study was launched in December 2016, to assess the traceability of biologics in clinical practice and adverse drug reaction (ADR) reporting. After a successful start of the project, the study is now moving forward, requesting official approval to survey hospital pharmacists and to study the United Kingdom’s ADR database.
Within the framework of the UK BIO-TRAC study, hospital pharmacists from the National Health Service trusts throughout England will be surveyed. In addition, a comprehensive assessment of the status of the traceability of biologics in the Medicines & Healthcare products Regulatory Agency’s ADR database will be performed.
The UK BIO-TRAC study is the result of a collaboration between Escher, the Lygature platform for regulatory innovation, and the Drug Safety Research Unit (DSRU) in the United Kingdom. It is a follow-up to an earlier Escher study in The Netherlands, concluding that brand names and batch numbers are not routinely recorded in clinical practice.
The results of this study are expected towards the end of 2017, and will be presented in a multi-stakeholder workshop.
Open Invitation for ATMP-developers to help improve product development and its regulation
Despite considerable development activity, a limited number of Advanced Therapy Medicinal Products (ATMP) have received Market Authorisation in the European Union. There is a growing urgency to understand which factors are associated with the advancement of ATMPs through the medicines life-cycle.
ATMP developers – past and present – are invited to participate in a survey investigating these factors, impacting the development ATMPs within the EU. Amongst others, the following factors will be explored: scientific challenges, manufacturing issues, clinical considerations and regulatory requirements. The survey will be executed by the Escher platform of Lygature together with Utrecht University.
Interested ATMP developers can contact Lygature to participate: email@example.com