How big is the need for cross-border access to clinical trials? (Guest blog)

Adult and paediatric patients with rare tumours and other rare diseases are particularly in need for treatment options. In some cases, these can be found in other countries were treatments are available to patients who have no access to such treatments in their home country. EU Directive 2011/24/EU “Directive on application of patients’ rights in cross-border healthcare” defines the principles that national legislations had to implement in order to cover healthcare of their citizens provided in other countries. However, this Directive does not clarify the cross-border access of patients to clinical trials. Clinical trials investigating new therapy concepts are of high interest to these patients. Many are prepared to make all efforts to join such clinical trials. Patients and their families even sacrifice their savings to enable the patient’s participation in a clinical trial in another country. But the national conditions for such cross-border access to clinical trials are very different in practical terms, and disadvantageous to the poor where clarification is not provided.

But how big is the need for cross-border access to clinical trials today and how frequently is it really happening? What are the practical hurdles for patients and investigators trying to enable such cross-border patient participation? What are the legal, financial and logistic problems for commercial and academic sponsors of clinical trials attractive to those patients? Is the topic of sufficient practical relevance to seek regulatory clarity on the rules for cross-border access to clinical trials on an EU level or would it be better to provide best practices for enabling direct stakeholder solutions?

EFPIA’s Oncology Platform provided an unrestricted grant to a research consortium consisting of the European Forum for Good Clinical Practice (EFGCP), EORTC, Catholic University of Leuven, and the Patvocates Network to generate concrete information on these questions. The consortium collected information by literature review, structured interviews with 40+ experts from many different disciplines in different EU countries and a very broadly disseminated multi-stakeholder survey that provided results from 400 evaluable responses.
The high-level results were presented at the ECCO Summit mid September in Brussels. Detailed results are currently worked out, will be provided in a comprehensive Final Study Report to EFPIA, disseminated in various publications, discussed in conferences, and presented to the European Commission’s DG Santé.

The consortium came to the conclusions that:
  • the topic is of very high relevance for patients in desperate situations
  • but currently their need for access to a clinical trial is not fulfilled in many cases due to the unclear legal conditions and healthcare coverage, their personal financial situation and the big practical hurdles for all involved parties
  • amending the Directive on cross-border access to treatment or developing a new Directive on cross-border access to clinical trials is deemed to take too much time for patients in need for finding access to treatment now and in the next years
  • developing a guideline on best practices to cross-border access to clinical trials within the current options and already available infrastructure might be the more constructive option. This guideline would aim at:
    • Encouraging EU member states to enable neighbouring country national healthcare solutions like in the Nordic countries or university-based solutions like in the border region between Germany and The Netherlands,
    • Adding this topic to the agenda of European Research Networks established according to the Directive on cross-border access to healthcare
    • Defining the needs for clarifying the patient liability insurance conditions if enrolled patients come from another country than the investigator’s
    • Defining recommendations for the social security and healthcare conditions for long-term follow-up in the patient’s home country
    • Clarifying the role of patients’ home country treating physician as contributor to data collection for the clinical trial
The resulting questions from this investigation will be: is there enough political and resourced will to find and enable solutions for the needs of this particular patient group? Will there be an easily accessible source of information for patients in need of clinical trial options? Will there be a reliable and easy way of identifying patients’ acceptability for a respective clinical trial within their home country? Will physicians in the EU in future be able to comprehensively and systematically provide access to innovative treatments through participation in a clinical trial to severely sick, mostly life-threatened patients wherever they come from? Will there be improved options to bring clinical trials closer to patients’ home through remote, decentralised trial participation?
Solution facets enabled by different stakeholders and technologies are in development but the decision on developing a comprehensive framework is outstanding.

Ingrid Klingmann

Ingrid Klingmann is the Chairman of the European Forum for Good Clinical Practice (EFGCP). 
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