Working Groups were thematic. The groups were focussed on specific topics of common interest and were formed by expert members from the different projects (plus invited experts from related initiatives). Working Groups discussed and sanctioned guidelines and white papers, recommended the creation of Task Forces pertaining to their area, evaluated new developments and alternatives.
From the outset, a group of foundational Working Groups was set up (Data Sharing and Re-use, Health Technology Assessment /regulatory, Ethics and Patient Privacy, Sustainability), Working Groups met as needed over the course of the project.
- To capture the lessons learned concerning data sharing in IMI projects.
- To capture the best practices and examples of collaboration frameworks and data access approaches from IMI projects.
- To develop guidelines and recommendations aimed at facilitating the sharing of and access to data, biological tools and other materials amongst IMI projects, as well as with other interested researchers at a European and global level.
- Develop tools to support effective engagement with regulators, HTA agencies and payers in the EU.
- Identify projects’ needs and knowledge gaps in relation to specific procedures and processes where external expertise may be sought.
- Provide a forum to share lessons learned from previous HTA and regulatory engagement.
- Support the projects in the development of their regulatory, HTA and payer strategy, as applicable.
- Encourage and facilitate cross-project collaborations on ethics and patient privacy issues.
- Map existing procedures and capture best practice for informed consent and data privacy.
- Develop guidance and templates which projects can use to support their work on ethics & data protection.
- Develop guidance to support sustainability and exploitation of projects’ results and assets.
- Identify projects’ needs and knowledge gaps in relation to sustainability.
- Provide a forum to discuss experiences and share lessons learned about sustainability planning.
- Support the projects in the development of their sustainability strategy as much as possible.
There is a wealth of scientific data buried in the archives of hospitals, academic institutions, private sector, and other stakeholders that has not yet been leveraged to its maximum. The sharing of data useful for research and clinical practice is increasingly viewed as a moral duty, especially in the neurodegeneration field where major breakthroughs and interventions being brought to market are still pending. However, despite the willingness and general ethos of data sharing to advance the field, in practice, it still proves to be quite challenging due to various technical, ethical, legal, financial, cultural and even psychological issues that typically hamper data sharing. Another challenge is the variability (lack of standardization) in how data are being collected and the format of these datasets (lack of harmonization).
Within and between various Innovative Medicines Initiative Joint Undertaking (IMI JU) projects (beyond neurodegeneration), similar challenges have been identified and hamper project progress. Various IMI consortia have been created to address parts of the global challenge of data identification, standardization, harmonization and eventually large-scale community use.
To maximise the impact of the IMI neurodegenerative disease portfolio, Neuronet created a Working Group (WG) for data sharing and reuse. The main aim of WG was to develop specific guidance to aid projects on data sharing policies and tools, incentives, value propositions, infrastructural solutions, etc. This WG developed guidelines aimed at facilitating the sharing of and access to data amongst IMI projects, as well as with other interested researchers at a European and global level. Our results aim to not only be of high value to the IMI Neurodegeneration portfolio, but also to any other initiatives that have the ambition of or rely on establishing Big Data-driven research platforms.
Health Technology Assessment/Regulatory
Regulatory agencies, HTA bodies and payers all play key roles in enabling people to access new drugs. This includes approving and assessing the quality, safety and efficacy of medicines (regulators); reviewing clinical and cost effectiveness of medicines compared with standard treatment (HTA bodies); and ensuring access to healthcare and support for people with neurodegenerative disease (payers). The outputs from most IMI projects might have relevance for regulatory, HTA and payer settings. Timely and effective engagement with these stakeholders is therefore crucial to ensure their requirements are included in the evidence development strategy to support new treatments. This is particularly relevant in the area of neurodegenerative disease, for which despite high levels of research investment, there remains a lack of effective treatments.
To maximise the impact of the IMI neurodegenerative disease portfolio, Neuronet created a Working Group for HTA and Regulatory liaison. The Working Group provided a cross-space forum for projects to share lessons learned and to discuss challenges and needs in relation to HTA and regulatory engagement. Through the Working Group, projects also had the opportunity to access external expertise on specific HTA and regulatory procedures and processes, where common needs and knowledge gaps are identified.
Alongside the Working Group, Neuronet has developed a decision tool to support the projects to identify the key opportunities to engage with EU regulatory and HTA agencies at key timepoints during the development of an ‘asset’. The Working Group interactions are expected to provide further insight into the individual needs of projects.
Data that is gathered in clinical research studies and during the delivery of “real-world” care includes simple variables such as age, gender and BMI (body mass index) as well as more complex data such as blood test results, cognitive assessments, medical imaging and genetic data. This is sensitive information which requires a high level of protection to ensure it is not unnecessarily disclosed, as loss or misuse of health data can expose patients to substantial social and personal harm. Consequently, protecting patient data is a core value in healthcare, linked to the fundamental human right to privacy enshrined in the 1948 United Nations Universal Declaration of Human Rights. Moreover, respecting patient privacy also shows respect for patient autonomy – a prerequisite for trust between patients and healthcare providers, and between participants and clinical researchers.
To ensure that patient privacy is respected, data protection and ethics form an integral part of EU-funded health research. Indeed, compliance with ethical requirements and data protection regulations is viewed by the EU as pivotal to achieve real excellence in health research. On the other hand, patient privacy concerns can be perceived as barriers to health research and, in particular, research that involves the secondary use of patient data.
The vast majority of the IMI neurodegeneration projects supported by Neuronet involved the use of patient data, ranging from projects that re-use existing clinical datasets to those which are carrying out clinical research studies that generate new datasets. Neuronet therefore created a Working Group on “Patient Privacy and Ethics”, that provided a cross-project space for experts to discuss common issues and compile best practice in data protection and ethics across the breadth of the IMI Neurodegeneration portfolio.
Sustainability is a challenging undertaking for most research projects. In fact, during the preparation of NEURONET’s proposal, nine IMI neurodegeneration projects were asked to list the project areas in which they would appreciate support, and sustainability was ranked as a high interest top priority. A key issue for project survival and sustainability often centers around access to diverse, long-term sources of continued economic funding, especially if the project is not able to generate steady revenues as a consequence of its activities, and considering that current funding mechanisms do not foresee that project budgets can be topped-up during the project lifetime; funding ends after the defined time period of the grant, and only occasionally extensions involving extra resourcing can be applied for.
Sustainability is a complex topic involving a huge variety of considerations. Of special note, different asset types (such as preclinical tools, clinical datasets, cohorts, etc.) may require completely different viability roadmaps. Each asset type involves different considerations that would need the input of different kinds of experts along the way. One of the aims of the Working Group was to help delineate those variables for creating a tailored and actualized sustainability plan. With this objective in mind, the NEURONET Working Group on Sustainability compiled recommendations and best practices for sustainability planning as a resource for current and future IMI projects to improve the chances of successful sustainability decisions.