How to balance valuable innovation with affordable access to medicines in Belgium?

Background: Countries are struggling to provide affordable access to medicines while supporting the market entry of innovative, expensive products. This Perspective aims to discuss challenges and avenues for balancing health care system objectives of access, affordability and innovation related to medicines in Belgium (and in other countries). Methods: This Perspective focuses on the R&D, regulatory approval and market access phases, with particular attention to oncology medicines, precision medicines, orphan medicines, advanced therapies, repurposed medicines, generics and biosimilars. The authors conducted a narrative review of the peer-reviewed literature, of the grey literature (such as policy documents and reports of consultancy agencies), and of their own research. Results: Health care stakeholders need to consider various initiatives for balancing innovation with access to medicines, which relate to clinical and non-clinical outcomes (e.g. supporting the conduct of pragmatic clinical trials, treatment optimisation and patient preference studies, optimising the use of real-world evidence in market access decision making), value assessment (e.g. increasing the transparency of the reimbursement system and criteria, tailoring the design of managed entry agreements to specific types of uncertainty), affordability (e.g. harnessing the role of generics and biosimilars in encouraging price competition, maximising opportunities for personalising and repurposing medicines) and access mechanisms (e.g. promoting collaboration and early dialogue between stakeholders including patients). Conclusion: Although there is no silver bullet that can balance valuable innovation with affordable access to medicines, (Belgian) policy and decision makers should continue to explore initiatives that exploit the potential of both the on-patent and off-patent pharmaceutical markets.

Cross-Border Access to Clinical Trials in the EU: Exploratory Study on Needs and Reality.

Objectives: To analyze the current situation of cross-border access to clinical trials in the EU with an overview of stakeholders' real-life experience, and to identify the needs, challenges, and potential for facilitation of cross-border access. Methods: We employed a mixed methods design. Semi-structured interviews and an online survey were conducted with a wide range of stakeholders: patient representatives, investigators/physicians, policy and regulatory experts, academic and commercial sponsor representatives, ethics committee members. Interviews underwent a framework analysis. The survey was analyzed descriptively. Results: Three hundred ninety six individuals responded to the survey. The majority were investigators/physicians (46%) and patient representatives (33%). Thirty eight individuals were interviewed. The majority were investigators/physicians (29%) and patient representatives (29%). All European regions were represented in the study. The highest response rate was received from residents of Western European countries (38% of survey respondents, 45% of interviewees), the lowest from Eastern Europe (9% of survey respondents, 5% of interviewees). The study suggested that cross-border participation in clinical trials occurs in practice, however very rarely. Ninety two percentage of survey respondents and the majority of interviewees perceived as needed the possibility to access clinical trials abroad. However, most interviewees also opined that patients ideally should not have to travel in order to access experimental treatment. The lack of access to treatment in the home country of the patient was described as the main motivation to participate in a clinical trial in another country. The logistical and financial burden for patients was perceived as the biggest challenge. Different stakeholders expressed diverging opinions regarding the allocation of financial and organizational responsibility for enabling cross-border access to clinical trials. Participants provided a number of proposals for improving the current system, which were carefully evaluated by the research team and informed future recommendations. Conclusions: Participation in clinical trials abroad is happening rarely but should be facilitated. There was a consensus on the need for reliable and accessible information regarding practical aspects, as well as multi-stakeholder, multi-national recommendations on existing options and best practice on cross-border access to clinical trials. Broader interdisciplinary research is recommended before discussing options in the EU legislative framework to enable clearly defined conditions for cross-border access to clinical trials.

Conducting Non-COVID-19 Clinical Trials during the Pandemic: Can Today's Learning Impact Framework Efficiency?

The COVID-19 pandemic has severely disrupted non-coronavirus clinical trials. In the case of life-threatening diseases, such as cancer, this is particularly dangerous, as treatment cannot simply be stopped. In the EU, guidelines for the management of ongoing studies were issued; however, national coordination is still lacking. This article aims to raise awareness on the struggle of managing ongoing clinical trials in the EU during the pandemic. The goals are to bring attention, from a legal and regulatory point of view to the difficulties faced by those involved in clinical research, and to critically position the current hurdles against the backdrop of the existing legal and ethical framework. We investigated the EU guidance and the national approaches of all EU/EEA Member States, and critically discussed selected issues. We argue that the crisis may be an opportunity to foresee meaningful changes in the EU clinical trials framework post-COVID-19.