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Enrollment in early-phase clinical trials in pediatric oncology: The experience at Institut Curie
Aurore Surun1, Marie-Églantine Dujaric2, Isabelle Aerts1
1Department of Pediatric Oncology, Oncology Center SIREDO (Care, Innovation, Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, Paris, France.
Insights
The European Paediatric Regulation improved pediatric oncology trial access. However, barriers like trial design and parental concerns limit participation, necessitating improvements for better medicine accessibility.
Area of Science:
- Pediatric Oncology
- Clinical Trial Accessibility
- Regulatory Science
Background:
- The European Paediatric Regulation (2007) aimed to enhance pediatric access to new medicines.
- This study investigated early-phase trial accessibility in pediatric oncology under this regulation.
- Key factors explored included reasons for non-invitation, parental refusal, and inclusion failures.
Purpose of the Study:
- To evaluate the accessibility of early-phase clinical trials for pediatric oncology patients.
- To identify barriers preventing patient participation in these trials.
- To inform strategies for improving access to novel pediatric cancer therapies.
Main Methods:
- Retrospective chart review of pediatric cancer patients at Institut Curie, Paris (July 2010 - December 2013).
- Analysis focused on patients in the palliative phase with progressive disease.
- Data collected on trial invitations, parental decisions, and reasons for inclusion failure.
Main Results:
- Of 100 palliative-phase patients, 52 were invited to early-phase trials.
- Parental refusal occurred in 20 cases, citing quality of life and trial constraints.
- Inclusion failed in 14 patients post-approval due to rapid deterioration; 5 received no invitations due to trial unavailability.
Conclusions:
- The Paediatric Regulation has increased early-phase trial availability for children.
- Optimizing trial proposal timing, trial design, and patient/parent information is crucial.
- Reducing waiting lists and addressing logistical/psychological barriers can improve pediatric access to new medicines.
Background:
The European Paediatric Regulation was introduced in 2007 to facilitate access to new medicines for children. Our study explored accessibility of early-phase trials in pediatric oncology, in line with the European Paediatric Regulation, to identify the reasons for not inviting patients to participate, parents' refusal, or inclusion failure.
Procedure:
We conducted a retrospective chart review at Institut Curie, Paris, for all pediatric patients whose cancer progressed despite known effective treatments between July 2010 and December 2013.
Results:
Out of 100 patients in the palliative phase, 52 received one or more invitations to participate in early-phase trials. Twenty parents declined the invitation, mainly prioritizing quality of life or fearing constraints. Fourteen inclusions failed despite parental approval, mostly due to rapid clinical deterioration. Five patients received no invitations because no early-phase trials were available. Major reasons for noninclusion in the 43 remaining patients were presence of exclusion criteria or other physical factors, preference for conventional treatment, constraints, psychological factors, and follow-up in another hospital after moving.
Conclusions:
The Paediatric Regulation has led to increased availability of early-phase trials. Better timing of the proposal, designing less constraining early-phase trials, reducing waiting lists, and improving information for parents and children would facilitate pediatric access to new medicines.
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