Bridging the data gap
Once a new drug has been developed, it must be reviewed by both the regulators (for the decision on marketing authorisation) and health technology assessment (HTA) bodies (for the decision on access to patients). The regulators draw on data, mostly from clinical trials, to determine if a drug is safe and works well enough to be authorised for use in patients. For their part, HTA organisations assess more broadly the value of a new drug for a given healthcare system. For this, they need data to support the assessment of ‘relative effectiveness’: that is the extent to which a treatment does more good than harm when compared to one or more alternative treatments when provided under normal healthcare circumstances.
However, while there are clear, detailed guidelines on how to carry out clinical trials, there is little guidance on how to generate real world data and integrate this into drug development before launch. This is a serious issue in drug development – even if a drug is approved as safe and effective by the regulators, insufficient evidence supporting relative effectiveness at the time of launch may delay, or restrict (partially or entirely) patient access to new treatments. The challenge, therefore, is to incorporate relative effectiveness research into the earlier stages of drug research and development, and that is where the GETREAL project comes in.
Injecting real life data into drug development
The goal of GETREAL is to develop new ways of incorporating real-life clinical data into drug development. The benefits of this are manifold – not only will it help pharmaceutical companies take better decisions during drug development, it will aid healthcare decision makers when deciding how best to grant patients access to a new treatment.
Among other things, the project will analyse existing processes and methodologies for HTA. It will also generate a decision-making framework to help pharmaceutical companies design drug development strategies. The framework would include ideas for the design of trials and studies capable of providing information on the real world effectiveness of medicines, including relative effectiveness.
The project also aims to develop tools that will allow different stakeholders in drug development and approval to test different mathematical models in their decision making and assess their impact.
A significant part of the project is devoted to organising training activities for researchers, healthcare decision makers and other stakeholders on relative effectiveness concepts and how they can be applied.
Finally, the project will create a network of regulators, HTA organisations, companies, academics, healthcare professionals, patients and other societal stakeholders. This is important because the challenges faced by the pharmaceutical industry, regulators and other healthcare decision makers are linked.
By bringing together all key stakeholder groups (namely industry, academia, regulatory agencies, HTA bodies, reimbursement agencies, healthcare budget holders, and patient groups) to share their insights and know-how, GETREAL will help to generate a consensus on best practice in the timing, performance and use of real life clinical studies in regulatory and reimbursement decision-making. It will also help to create a strong platform for the communication of results and for future discussions in this important area.