- Keywords > clinical trials
- Keywords > marketing authorization
- Keywords > pharmaceutical gaps
- Keywords > pharmaceutical innovation
- Keywords > policy - priority issues
- Keywords > priorities for pharmaceutical research
- Keywords > priority diseases
- Keywords > priority medicines
- Keywords > research - cost of drug development
- Keywords > research and development
(2013; 25 pages)
The costs of pharmaceutical R&D are high, with clinical trials being a major component of these development costs. There is an urgent need to address therapeutic gaps in order to be able to respond to unmet medical needs. To help resolve this problem, there is a need to increase efficiency and to bridge bench and clinical research with real-world practice. Making better use of real-world data can improve the efficiency of the whole medicine development chain: HTA bodies, regulators, clinicians and patients will be able to make better-informed decisions, and companies will be able to design better and more efficient development strategies that provide the appropriate evidence to decision makers.
Additionally, data obtained from health information systems can be used to
support priority setting, detect safety problems and assess the real-world
effectiveness of medicines. Moreover, policy initiatives such as adaptive
licensing, value-based pricing and comparative effectiveness studies are
critically dependent on the efficient use of Electronic Health Record (EHR)
data. However, the resources available in Europe are fragmented, and good
quality data are often only available for limited disease areas or geographic
In the 2004 Priority Medicines Report, the use of electronic health records was highlighted as an area of high importance. It was suggested as "a way of creating post-marketing "randomized epidemiology" studies to better understand comparative effectiveness and cost-effectiveness." Although progress has been made since then, the potential is still largely unfulfilled.
Real-world data are now more widely available than ever before and offer new opportunities for research and health systems development. Information in the health care system is increasingly processed electronically in many countries across the world. Databases that collate health care information from larger numbers of patients are becoming available for research. The information that is available for research in these databases has been changing over time and the possible contribution of health care databases to pharmaceutical innovation has been evolving.
We will provide examples in this background paper on how these databases can inform priority setting and support pharmaceutical innovation by assisting in, amongst others, the prioritisation of research needs, better disease understanding, safety monitoring, comparative effectiveness research and the evaluation of health care policies. Examples will be given on how these health care databases can help to identify the greatest challenges with pharmaceutical treatments and test their effects in real life.