Medicine development decisions are complex, involving many different stakeholders and based on many different factors (e.g. legal, ethical, clinical and societal). The following initiatives / tools have been developed to consider the impact of alternative evidence generation activities, such as an earlier use of real-world evidence (RWE).
The Sure-Real simulation and interactive visualisation tool was developed as part of GetReal, in a collaboration between NICE and the Massachusetts Institute of Technology (MIT) NEW Drug Development ParadIGmS (NEWDIGS) programme (see Key Important Initiatives). The purpose of Sure-Real is to assess the impact of alternative evidence generation strategies in clinical drug development using RWE.
Who is it for?
This tool is designed and envisaged to be used by different stakeholders, including manufacturers, health technology assessment (HTA) agencies and academics, and to provide visual output for any others involved in developing or assessing health technologies.
How does the tool work?
Sure-Real enables users to visually design and explore multiple trial scenarios when creating evidence development plans. It provides insight regarding the use of RWE in clinical drug development and assesses the impact of implementing alternative evidence generation strategies.
The tool consists of a data repository, models of different study designs, and patient costing and clinical data.
The user interface of Sure-Real is built on Microsoft Visio, and can be accessed via customised menus both in Microsoft Excel and Visio.
Three types of interface are incorporated into the main Sure-Real interface: the Timeline Designer, the Patient Experience Manger and the Population Manager. The tool allows interaction through these visual interfaces, by selecting and changing an element on the Visio page. The tool manages the underlying data by making corresponding modifications including updating the various models, in turn showing results and updated metrics in the visual interface.
The Janus Initiative
The Janus Initiative (Trusheim et al., 2014) is a multi-stakeholder process and simulation tool that was set up by the Massachusetts Institute of Technology (MIT) NEW Drug Development ParadIGmS (NEWDIGS) multi-stakeholder collaboration (see Key Important Initiatives) to help users to better understand the consequences of different combinations of actions by different stakeholders. The overall aim is to help users find the best action to increase patient access to safe and clinically effective medicines.
The Janus Initiative has created an open-access tool that allows stakeholders to map out decision pathways. It uses quantitative information to simulate how a decision might impact on each stakeholder, and it identifies what evidence is needed and how to generate this for each option. It maps out any uncertainty and considers the importance of different factors, illustrating the balance that needs to be made between these factors. Although it does not prescribe a specific solution, the tool helps users to consider and communicate possible decisions and their consequences in a transparent way.
The main principles of the Janus initiative include:
- Integrate the best available models and develop new models.
- Maintain transparency in their development and provide open access for their use.
- Use alternative scenario management and comparison.
- Foster collaboration
Joost de Folter and Heather Stegenga, NICE