EU Health Policy, Coherence, Stakeholder Diversity and Their Impact on the EMA

Abstract

Innovation is a major pillar in bringing new, targeted medicines to patients. In the health arena, this means the translation of knowledge into what we can call “value.” The latter covers the value to patients but must also take into account value to healthcare systems, society and, of course, manufacturers. The EU has recognised that innovations in healthcare can contribute to the health and well-being of citizens and patients through access to new products, services and treatments with added value. It is also aware that in order to stimulate development, there is a need to facilitate the translation of scientific advances into innovative medicinal products that meet regulatory standards, accelerate patients' access to new therapies and are affordable to Member States' health systems. Early dialogue between technology developers, regulators, health technology assessment and, where relevant, pricing bodies will promote innovation and quicker access to medicines at affordable prices, for the benefit of patients. But while uncertainties in healthcare policy still exist, a request by the European Ombudsman to the European Medicines Agency to provide more information about its early dialogue procedures questions the above “early dialogue” principal. It raises the issue of what the EU aims to do with its health regulation in bringing innovation to the patient. Is this added uncertainty about the hereto trusted role of the EMA a welcome development? Not necessarily.

Factors Affecting Citizen Trust and Public Engagement Relating to the Generation and Use of Real-World Evidence in Healthcare

“…Thus, ensuring evidence-based innovative medical research is challenging and it is important that it does not limit improvements to healthcare and citizen health. Strategic thinking regarding the current gaps for ensuring trust in the use of citizen data for RWE is required [19].…”

“…The Million European Genomes Alliance (MEGA) program-launched in 2018 and now the EU 1+ Million Genomes Initiative [19,20,24]-is a parallel initiative from health sector stakeholder groups across 22 EU countries, as well as the UK and Norway. It provides a potentially impactful model for building collaborations to share data, while at the same time inspiring trust among data owners as to how their data might be used.…”

Considering and communicating uncertainty in health technology assessment

“…Early dialogues with regulators and HTA bodies are well-established processes in which technology developers have the opportunity to discuss their research plans and gain advice and feedback on their planned approach. These activities can help identify uncertainties early in the process, along with the notion of how resolvable key uncertainties may be (19). This can influence the entire HTA process, from deciding if and when to conduct an HTA through to implementation of financial mitigation strategies such as MEA.…”