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Multi-stakeholder dialogue paving the way to increased understanding of complex clinical trials (Guest blog)

Over 400 participants joined an interactive multistakeholder workshop on "Accelerating Adoption of Complex Clinical Trials in Europe and Beyond" organised by EFPIA with other stakeholders including regulators, ethics committees, HTA Bodies, patient representatives, CROs (Clinical Research Organisations) and clinical trial investigators. The workshop had an ambitious aim to identify solutions to some of the challenges seen today when conducting complex clinical trials (CCTs). The objective for increasing understanding of CCTs across stakeholders was achieved and very useful feedback was given by different stakeholders to potential solutions and next steps.

EFPIA's Sini Eskola opened each day of the two-day workshop along with Jan Geissler representing patients and Peter Arlett from the European Medicines Agency. It was clear from the start that a big focus of the discussion would be how to design CCTs so that patients would be comfortable in joining and remaining in the trial with a change from a drug-centric approach to a more patient-centric approach to designing CCTs. The obvious conclusion was for patients to be involved in the design of such trials and the more complex the design, more collaboration with patients is needed. Rita Magenheim, representing patients, outlined that it is important that patient needs are considered throughout the trial, not just during the development of the initial protocol.

Regulators from both the EU and US outlined some of the work they are doing to help sponsors navigate the regulatory environment to conduct CCTs. Dionne Price from the FDA gave a summary of their complex innovative design (CID) pilot, some of the learnings from the pilot submissions to date and it was agreed that a similar pilot would be useful in EU. Kristof Bonnarens (DG SANTE) and Peter Arlett (EMA) acknowledged the important milestone of CTIS (Clinical Trial Information System) development and EU CTR going live with the aim to harmonise and facilitate the framework for conducting Clinical Trials in Europe. 

All stakeholders recognised the need to collaborate when designing CCTs. This collaboration needs to be done early and often. Collaboration is not just about sponsors seeking advice on the expectations of stakeholders when it comes to CCTs; there is also a need to collaborate so that information about CCTs can be shared across different stakeholders leading to a culture of learning and education. Tony Humphreys from the EMA highlighted a strong need for developing expertise in handling complex designs and that this is a global issue, not just an EU issue. He stated having this culture of sharing knowledge and collaboration is a challenge that requires stakeholders to recognise there is no competitive advantage in keeping the knowledge to themselves.

Niklas Hedberg from the Swedish TLV outlined that from the perspective of HTA Bodies, although they recognise the 'CCT train' has started its journey and we can't reverse it, there is significant 'gap' where the needs of HTA bodies have not been considered. He requested that the HTA needs are carefully considered during the early stages of designing CCTs, there are more joint efforts to develop and validate new collaboration models, and that more information about CCTs is available from 'independent bodies'.

A series of break-out sessions allowed participants to have more in depth interactive discussions on some of the key challenges in designing and conducting CCTs. The sessions included discussions on: designing master protocols; regulatory processes and systems; greater involvement of patients; trials incorporating historical controls or with adaptive features; operational aspects; and ensuring all stakeholders have the right knowledge and training on CCTs.

Although all break-out sessions had conclusions specific to their topic, a theme that was common across several sessions was the need to establish mechanisms via collaboration platforms that would allow stakeholders to share advice on the design and conduct of CCTs and to share experience and learnings from such trials. Several suggestions on how these collaboration platforms could be established were put forward and these ideas will be followed up in subsequent follow up discussions. From the Workshop, it was clear there was the willingness to establish and maintain these types of collaboration platforms.

Summing up a 'highly successful' workshop, Chrissie Fletcher from GSK and Chair of EFPIA's CREG Innovative Clinical Trial pillar group stated that one of her key takeaways from the workshop is we should be designing 'fit-for-purpose' clinical trials with a patient centric focus: start first with the question that the study aims to address, then select a trial design to address that question, and then identify what data will be needed to answer the question. Mireille Muller from Novartis and Anja Schiel from the Norwegian Medicines Agency concluded the workshop by recognising the high level of interaction across the multiple stakeholders and that the workshop has identified various solutions to some of the challenges in designing and conducting CCTs. Participants recognised that this was only the start and that much work still needs to be done but they committed to continue to work together to deliver those solutions.

 

Note: Workshop was recorded and is available here
The workshop report will be
 available soon.

Nick Sykes

Nick Sykes has worked in Regulatory Affairs since 1992 at various companies and joined Pfizer’s regulatory affairs...
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Chrissie Fletcher

Chrissie is the Vice President of Development Statistics at GlaxoSmithKline.  She leads a group of statisticians...
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Mireille Muller

Mireille Muller is currently Executive Regulatory Policy Director at Novartis, focusing on clinical trials, advanced...
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