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Updated: Jun 23, 2026

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
Accepting risk in the acceleration of drug development for rare cancers
David Ashley1, David Thomas2, Lia Gore3
1School of Medicine, Deakin University, Geelong, VIC, Australia; Andrew Love Cancer Centre, Barwon Health, Geelong, VIC, Australia.
Abstract:
Rare cancers collectively contribute a disproportionate fraction of the total burden of cancer. The oncology community is increasingly facing small numbers of patients with each cancer subtype, requiring cooperation and collaboration to complete multicentre trials that advance knowledge and patient care. At the same time, new insights into the biology of rare cancers have led to an explosion in knowledge and development of targeted agents. These insights and techniques are set to revolutionise the care of patients with cancer. However, drug development strategies and the availability of new agents for rare cancers are at risk of stalling owing to the ever-increasing complexity and costs of clinical trials. Finding solutions to these problems is imperative to the future of cancer care. We propose that a greater degree of risk sharing is needed than is currently accepted to enable the use of new methods with confidence, and to keep pace with scientific advancement.
Insights
Rare cancers pose challenges for clinical trials due to small patient numbers. Increased risk-sharing is crucial for developing targeted therapies and advancing rare cancer care.
Area of Science:
- Oncology
- Rare Cancers
- Clinical Trials
Background:
- Rare cancers represent a significant cancer burden.
- Small patient cohorts per subtype necessitate multicenter collaborations for clinical trials.
- Advances in rare cancer biology are driving the development of targeted agents.
Purpose of the Study:
- To address the challenges in rare cancer drug development.
- To propose solutions for the increasing complexity and costs of clinical trials.
- To advocate for enhanced risk-sharing models in rare cancer research.
Main Methods:
- Review of current challenges in rare cancer clinical trials.
- Analysis of the impact of biological insights on targeted agent development.
- Proposal for a revised risk-sharing framework for drug development.
Main Results:
- Current drug development strategies are hindered by trial complexities and costs.
- New biological insights offer revolutionary potential for rare cancer treatment.
- Existing risk-sharing models are insufficient for the pace of scientific advancement.
Conclusions:
- Greater risk-sharing is imperative for rare cancer drug development.
- Implementing new methods requires confidence and adaptation to scientific progress.
- Addressing these issues is vital for the future of oncology care for rare cancer patients.
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