To strengthen regulatory and scientific support for innovative medicines and diagnostics development, in March 2020 EMA published its Regulatory Science Strategy to 2025.
As per the strategy report, the five main goals are:
The strategy document came to life following extensive external consultation. The recently published mid-point achievements report covers the period between March 2020 and December 2022 and serves as a reference summary which will be further complemented by the work to be performed through to 2025. The report can be found here, in which the following achievements concerning human medicinal products are highlighted:
These initiatives are of strategic value and address scientific questions of considerable relevance for the EU regulatory environment, with the mid-term report eliciting some reflections.
As was affirmed at the DIA in Basel in March 2023, EMA, sponsors, and EU member state authorities recognise that the introduction of CTIS has not been a smooth ride. However, by means of, amongst others, introducing an online training programme focusing on sponsors and member states and a monthly publication of key performance indicators to monitor the European clinical trials environment, improvements are noticeable. In addition, the user community is learning fast.
ProPharma has been using CTIS for clinical trial applications since the launch of this portal for a large and growing number of clients and has thus gained extensive experience in using CTIS in the most efficient way.
Recent reports in the media highlighting concerns that the EU is becoming less attractive as a location for innovative drug development, including conducting clinical trials, cannot have passed unnoticed. Part of the efforts to prevent these concerns becoming reality are clearly outlined in ACT-EU and the associated work plan. The focus in this plan lies on encouraging the development of innovative clinical trial designs that would increase the efficiency of performing clinical trials in terms of time and size, while maintaining acceptability for decision-making by the regulator and the health technology assessment institutes. Indeed, recent guidance from both EMA on complex clinical trials and FDA making reference to adaptive clinical trials shape the regulatory framework for discussion on cutting-edge drug development plans in the context of scientific advice and for robust benefit/risk assessment by the regulator. Further, with the recently announced impact-assessment of the eagerly awaited new pharma legislation, the ambition of the European Committee to maintain the position of the EU as a strong innovation-fostering scientific and regulatory environment is underscored.
ProPharma has extensive experience in partnering with clients to support scientific advice requests at both the national and the central level, from writing or reviewing briefing books to preparing and supporting discussion meetings. As can be expected, subject matter experts from the Biostatistics Unit at ProPharma are as such familiar with the discussions on innovative clinical trial designs and ready to advise clients to make best use of the new opportunities created by the ACT EU activities.
Lastly, if and how to use real-world evidence (RWE) for regulatory decision-making has been debated for years with focus on the requirements of, in particular, the quality of the data. While discussion is ongoing and limitations of the data in terms of the almost inevitable selection bias and an in general lack of standardized data collection, are recognised, the establishment of DARWIN and the pilot-studies and inventories planned for will allow to define exactly the space for use.
ProPharma subject matter experts are ready to explore with clients the possibilities to incorporate RWE into their innovative drug development strategy.
All in all, EMA seems to be on track and have outlined interesting next steps in their pursuit for the goals as set in their Regulatory Science Strategy. At ProPharma we continue to keep abreast of the latest global regulatory developments and look forward to work with clients to maximise the opportunities provided by EMA’s plans to maintain the EU as a stimulating environment for state-of-art drug development.
September 5, 2023
By now, most involved in the clinical trials disclosures field are aware of the upcoming reinstatement of the European Medicine Agency's (EMA) landmark transparency regulation: Policy 0070. Policy...
November 8, 2023
Randomized clinical trials (RCTs) are widely recognized as the gold standard for clinical and regulatory decision-making. Indeed, RCTs would be required in most cases, in particular when modest...
October 30, 2023
Are you truly up to speed on the most effective strategies to expedite the approval process for your innovative product in Europe's evolving regulatory landscape? In the United States (US), the Food...