Towards Better Pharmaceutical Provision in Europe-Who Decides the Future?

Significant progress has been achieved in human health in the European Union in recent years. New medicines, vaccines, and treatments have been developed to tackle some of the leading causes of disease and life-threatening illnesses. It is clear that investment in research and development (R&D) for innovative medicines and treatments is essential for making progress in preventing and treating diseases. Ahead of the legislative process, which should begin by the end of 2022, discussions focus on how Europe can best promote the huge potential benefits of new science and technology within the regulatory framework. The challenges in European healthcare were spelled out by the panellists at the roundtable organised by European Alliance for Personalised Medicine (EAPM). Outcomes from panellists' discussions have been summarized and re-arranged in this paper under five headings: innovation, unmet medical need, access, security of supply, adapting to progress, and efficiency. Some of the conclusions that emerged from the panel are a call for a better overall holistic vision of the future of pharmaceuticals and health in Europe and a collaborative effort among all stakeholders, seeing the delivery of medicines as part of a broader picture of healthcare.

Clinical Development and Initial Approval of Novel Immune Checkpoint Inhibitors in Oncology: Insights From a Global Regulatory Perspective.

Immune checkpoint inhibitors (ICI) have demonstrated meaningful patterns of clinical efficacy across various cancers. During their development, novel regulatory strategies and clinical design approaches were explored. This metrics-based narrative review examines submission strategies and clinical evidence expectations of the US, European, and Japanese drug agencies, as well as their impact on approval and overall development times. Also discussed is the role of emerging clinical science and biomarker evaluation to get the first six ICI initially approved.

Approval probabilities and regulatory review patterns for anticancer drugs in the European Union.

AIM: This article reviews outcomes of marketing authorization applications for anticancer drugs in the EU and outlines factors and hurdles of impact. METHODS: Procedures for initial approval of anticancer and non-cancer drugs were analyzed and compared to anticancer drug approvals in the USA and Japan for the same period. RESULTS: From 2006 to 2011, the regulatory review of 46 marketing authorization applications resulted in 29 new cancer drug approvals. The overall approval probability (63%) lagged behind the probability for non-cancer drugs (73%). Longer median active review times in line with additional clock-stop and EU Commission decision-making times as well as submission delays contribute to the 7.2 months median time-to-market delay [95% CI 4.7-15.0 months] compared to the USA; Japanese patients had to wait an additional 25.1 months [95% CI 6.2-34.1 months]. CONCLUSION: Marketing authorization applications for anticancer drugs in the EU are associated with modest approval success. Patients in the USA get access to new products earlier, fostered by the more frequent use of expedited review procedures. So far, both procedures were used in the EU for applications claiming a major public health interest, characterized by pivotal clinical trial hazard ratios below 0.70.