Translating Academic Drug Discovery Into Clinical Development: A Survey of the Awareness of Regulatory Support and Requirements Among Stakeholders in Europe

Abstract

Important discoveries by academic drug developers hold the promise of bringing innovative treatments that address unmet medical needs to the market. However, the drug development process has proved to be challenging and demanding for academic researchers, and regulatory challenges are an important barrier to implementing academic findings in clinical practice. European regulators offer varying degrees of support services to help drug developers meet regulatory standards and requirements. "Strengthening Training of Academia in Regulatory Sciences and Supporting Regulatory Scientific Advice" (STARS) is a European Commission-funded consortium aiming to strengthen the training of academics in regulatory science and requirements. Here, we report the results of four surveys that investigated the awareness and utilization of support tools offered by European regulators and identified the regulatory challenges and support needs of researchers. The surveys targeted four main European stakeholders in academic medicines research: academic research groups (706 respondents), academic research centers (99), funding organizations (49), and regulators (22). The results show that while European regulators provide various regulatory support tools, less than half of the responding academic researchers were aware of these tools and many experienced challenges in reaching a sufficient level of regulatory knowledge. There was a general lack of understanding of the regulatory environment that was aggravated by poor communication between stakeholders. The results of this study form a foundation for an improved European medicines regulatory network, in which regulatory challenges faced by academia are tackled.

Figure 1

STARS (a) surveys' respondent numbers and (b) response rates by country. Darker color represents a higher participation rate. EMA, European Medicines Agency; NCAs, national competent authorities; STARS, Strengthening Training of Academia in Regulatory Sciences and Supporting Regulatory Scientific Advice.

Figure 2

Research activities in the respondent research groups and research centers. The left side shows percentages of the research groups that were working on a designated research area or topic. On the right side is shown percentages of the research centers that considered a designated research area or topic to be very significant or significant. *Single‐arm, two‐arm, and multiarm studies and basket or umbrella trials. **Observational, registry, and case‐control studies. ***Preclinical studies, for example on pathways, biological mechanisms, or pathophysiology. A total of 684 research groups and 91 research centers answered this question. e/m health, electronic or mobile health solutions (e.g., digital health application/service R&D); MCC, manufacturing, characterization, and control; R&D, research and development.

Figure 3

The awareness and use rates of NCAs' and EMA's support services by the research groups and research centers. (a) Percentage of the research groups and research centers that were aware of the designated regulatory support type. (b) Percentage research groups and research centers that had used the NCAs' and EMA's support services. A total of 498 research groups and 89 research centers answered this question. EMA, European Medicines Agency; NCAs, national competent authorities; PRIME, the EMA's primary medicines scheme; SME, small and medium‐sized enterprises.

Figure 4

Regulatory challenges experienced by the respondent research groups. The graph shows percentages of the research groups who considered that a designated topic had been a “very big” or “big” challenge for the research team. A total of 455 research groups answered this question.

Figure 5

Regulatory support requested in different research topics. The black bars indicate the percentage of research groups that had sought regulatory support on a designated topic from NCAs. The gray bars show the percentage of research centers from which the research groups had sought regulatory support on the designated topics. A total of 498 research groups and 89 research centers answered this question. ATMP, advanced therapy medicinal product; e/m health, electronic or mobile health solutions (e.g., digital health application/service R&D); HTA, health technology assessment; MCC, manufacturing, characterization, and control; NCA, national competent authority; PIP, pediatric investigation plan; R&D, research and development.

Figure 6

Distribution of open‐ended answers to the question, “What is the most critical gap or deficiency in the current regulatory system?” in 11 response categories, shown as percentages of the total responses. The research groups and research centers gave 272 and 101 open answers, respectively. GDPR, general data protection regulation.