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Incorporating real-life clinical data into drug development

Ziel

When a new medicine reaches the market, it is accompanied by an extensive data package that provides information about the safety and efficacy of the medicine in a clinical trial setting. However, assessing the expected future value of the medicines when used in “real world” clinical practice requires additional information next to traditional (pre-authorisation) clinical trials. Regulatory, HTA agencies and other healthcare decision makers have to make decisions on authorisation and access under conditions of uncertainty. Currently, data packages which aim to minimise uncertainty on safety and efficacy may leave significant uncertainty in assessments of real world effectiveness of new medicines. This results in further research commitments required post-authorisation (PASS, PAES, Reimbursement with Evidence Generation), and wide variability in access to medicines between countries.
The subsequent uncertainty relating to the reimbursement, and thus market implementation of new medication once approved by regulatory authorities, is negatively affecting the value of the drug development pipeline. The costly clinical developments to get to market approval, no longer seem to be a guarantee for market entry. This new risk presents a serious additional hurdle for drug developers that already face pipeline attrition. It also affects the speed and level of patient access, and therefore the extent to which patients and society might benefit from new medicines.
In recent years there has been considerable attention paid to the post-authorisation evaluation of treatments in real world clinical practice: study design and analytical methodology for assessing relative effectiveness; and use of registries and electronic healthcare data. It may be possible to improve the value of information available at initial market authorisation by incorporating these techniques into pre-authorisation drug development. HTA bodies, regulators, will become able to make better-informed decisions, and developers of new medicines will be able to direct development efforts to areas where value is most likely to be delivered to patients and health care systems, improving the efficiency of the whole medicine development chain.
However, the adoption of real world / relative effectiveness objectives in a pre-authorisation development strategy has many operational, methodological, regulatory, and ethical issues and Pharmaceutical R&D organisations need more certainty as to: the impact of development choices on the regulatory review process; the value of different programmes to HTA bodies and other decision makers; the best balance of pre-launch and post-launch effectiveness research and the coordination of various post-authorisation commitments. There is little guidance on how to incorporate alternative study designs into a development programme to optimally meet the needs of all stakeholders over time.
The GetReal consortium aims to improve the efficiency of the medicine development process by better incorporating real-life clinical data into drug development and to enrich decision-making by regulatory authorities and HTA bodies through:
• Bringing together regulators, HTA bodies, companies, patients and other societal stakeholders
• Assessing existing processes, methodologies, and key research issues
• Proposing innovative (and more pragmatic) trial designs and assessing the value of information
• Proposing and testing innovative analytical and predictive modelling approaches
• Assessing operational, ethical, regulatory issues and proposing and testing solutions;
• Creating new decision making frameworks, and building open tools to allow for the evaluation of development programmes and use in the assessment of the value of new medicines;
• Sharing and discussing deliverables with, among others, Pharmaceutical companies, regulatory authorities, HTA / reimbursement agencies, clinicians and patient organizations;
• Developing trai

Wissenschaftliches Gebiet (EuroSciVoc)

CORDIS klassifiziert Projekte mit EuroSciVoc, einer mehrsprachigen Taxonomie der Wissenschaftsbereiche, durch einen halbautomatischen Prozess, der auf Verfahren der Verarbeitung natürlicher Sprache beruht. Siehe: https://op.europa.eu/de/web/eu-vocabularies/euroscivoc.

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JTI-CP-IMI - Joint Technology Initiatives - Collaborative Project (IMI)

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GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LIMITED
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Private for-profit entities (excluding Higher or Secondary Education Establishments)
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