Clinical research for rare cancers: Is it a reality in the global regulatory landscape? The international rare cancer initiative

Anastassia Negrouk, Denis Lacombe, Edward L. Trimble, Matthew Seymour

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Introduction: Rare cancers together account for about 22% of new cancer diagnoses each year, which is more than any single common cancer. They constitute a serious public health problem with distinctive challenges. Compared with common cancers, less is known about their etiology and molecular pathology, and therapeutic options are mostly poorly evidence based with outcomes that are on average inferior to those of more common cancers. There is, therefore, a pressing need for well-organized clinical research to address these deficiencies. The International rare cancer initiative (IRCI) was formed in 2011 with the aim of stimulating and facilitating the development of international clinical trials for patients with rare cancers, and so helping to establish better, more evidence-based treatments. As of October 2013, 8 clinical trials are in design, in set up or recruiting, in seven rare cancer types, giving IRCI a good understanding of the current research conditions for rare cancer. Areas covered: This paper sets the scene of clinical research in rare cancers and presents an overview of organizational, methodological, regulatory and financial challenges facing researchers in this field. It also proposes possible steps to improve the research environment for the future. Expert opinion: Based on our observations, we believe that measures should be taken to improve the integration of research across international boundaries, to improve the funding of academic research and to better integrate the research capacity of industry with the public sector for the greater benefits of patients and society.

Original languageEnglish (US)
Pages (from-to)433-440
Number of pages8
JournalExpert Opinion on Orphan Drugs
Issue number5
StatePublished - May 2014
Externally publishedYes


  • Clinical trials
  • International rare cancer initiative
  • Rare cancers
  • Regulatory

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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