Factors affecting the feasibility of post-authorisation RCTs for conditionally authorised anticancer medicines: a...

Area of Science:

• Oncology
• Clinical Trial Design
• Regulatory Science

Background:

• Conditional marketing authorisation for anticancer medicines often leads to delayed data collection from post-authorisation trials.
• This delay raises concerns about the feasibility of conducting post-authorisation randomised controlled trials (RCTs) to address outstanding clinical uncertainties.

Purpose of the Study:

• To explore factors influencing the feasibility of post-authorisation RCTs in oncology.
• To gather perspectives from stakeholders involved in the design, ethical approval, and conduct of these trials.

Main Methods:

• Conducted four qualitative focus groups (FGs) in a European oncology context.
• Engaged 28 participants including patients, physicians, medical ethicists, and pharmaceutical industry representatives.
• Discussions were guided by a script informed by 14 semi-structured interviews with relevant stakeholders.

Main Results:

• Identified key feasibility factors across trial design (endpoints, comparators), trial conduct (patient/physician willingness), and external influences (commercial availability, competing trials, equipoise).
• Highlighted the necessity of post-authorisation regulatory interaction for data collection when RCTs appear infeasible.
• Emphasized the importance of addressing factors like medicine availability and clinical equipoise perceptions.

Conclusions:

• Recommend in-depth assessment of patient recruitment and retention during regulatory feasibility evaluations for conditional authorisations.
• Advocate for improved communication by sponsors and regulators to patients and physicians regarding uncertainties and the need for post-authorisation RCTs.
• Suggest that enhanced feasibility evaluations can facilitate the timely completion of post-authorisation RCTs, resolving uncertainties efficiently.