Work Package 1 has two core objectives:

1. Analyse and monitor activities that occur both within and outside of IMI programmes that provide and generate evidence about MAPPs;
2. Develop a gap analysis of the current and future needs of evidence generation to support MAPPs.

These objectives will be realised through a review of current and future research projects and assessment of tools and methods to support evidence generation and data interpretation throughout the product life cycle. The focus will include practical issues around data generation, validation of biomarkers and endpoints such as patient reported outcomes, interoperability of data standards and innovative methods for data generation such as use of registries and data mining of electronic Health Records (EHR). The challenges of pharmaceutical development will be also considered since modifications to the traditional CMC (Chemistry and Manufacturing Controls) development paradigm will need to be supported.

All stakeholders, including patients, regulators, HTA/payers, practitioners, and industry, must align on the evidence package and success metrics early in the development and access journey, and continue to assess the performance of the therapy through the life-cycle of the product.

The topics addressed in WP2 include the following:

• Develop a glossary of standardised terms to be used in MAPPs;
• Identify knowledge gaps that allow for a seamless pathway from pre-clinical research, to clinical development, and ultimately to a new therapy being used by patients;
• Define the selection criteria and the timing for using the MAPPs pathway in “beneficial treatment” and how stakeholders will be involved in that decision;
• Uncover any potential legal constraints to implementing a MAPPs pathway;
• Create the tools to ensure appropriate prescribing and appropriate use by patients in MAPPs;
• Integrate the findings of the EMA pilot project and other global initiatives that are relevant to MAPPs;

Share best practices, case studies, modelling, and simulations of pathway options in MAPPs.

It will identify all decision points in adaptive approaches throughout product lifecycle, and the steps required to align all the stakeholders around evidence needed and implications. It will also assess and communicate the impact of MAPPs on health ecosystem, including the dissemination of the learnings from early access pilots and on-going research initiatives.

This includes:

• R&D efficiencies and inefficiencies;
• Pricing arrangements, risk-sharing, effective patient access and business cases for sponsors;
• Ethical implications for patients and clinicians;
• Intellectual property and regulatory data protection;
• Legal implications for all decision makers;
• Personal data protection considerations.

The key objectives are to:

• Support and monitor the effective execution of the work plan including the control of deliverables, milestones and budgets;
• Enable collaboration between the various work packages, and making regular progress reports to the Navigator Group and IMI JU office as required;
• Organize and prepare meetings for project governance;
• Inform partners as well as European and global stakeholders on the project’s progress;
• Manage key interactions of consortium;
• Plan and coordinate a broad platform of external communication;
• Manage Intellectual Property rights and any foreground information generated.