The international rare cancers initiative.

Keat, Nicola; Law, Kody J. H.; McConnell, Andrea; Calvo, Fabien; Negrouk, Anastassia; Lacombe, Denis; Welch, Jack; Trimble, Edward L.; Gross, Thomas G.; Schaetzen, Gaetan de; Bréchot, J.-M.; Pauporté, Iris; O'Riordan, Maura; Seymour, Matthew

doi:10.1200/jco.2014.32.15_suppl.e17504

Cited by 10 publications

(12 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…22 International collaboration in clinical trials is essential to improve outcomes for rare cancers. To this end, the International Rare Cancers Initiative was established in 2011 as a joint initiative between the NCI, Cancer Research UK, the 23 In addition, in 2014, the National Clinical Trials Network was launched in the United States with a focus on the study of rare cancers. 24 Notably, there has been tremendous success in the advancement of treatment for many childhood cancers, in large part because treatment for children's cancers has been concentrated in specialized centers and the joint research collaborations among these centers.…”

Section: Discussionmentioning

confidence: 99%

The burden of rare cancers in the United States

DeSantis

Kramer

Jemal

2017

CA A Cancer J Clinicians

123

105

View full text Add to dashboard Cite

There are limited published data on the burden of rare cancers in the United States. By using data from the North American Association of Central Cancer Registries and the Surveillance, Epidemiology, and End Results program, the authors provide information on incidence rates, stage at diagnosis, and survival for more than 100 rare cancers (defined as an incidence of fewer than 6 cases per 100,000 individuals per year) in the United States. Overall, approximately 20% of patients with cancer in the United States are diagnosed with a rare cancer. Rare cancers make up a larger proportion of cancers diagnosed in Hispanic (24%) and Asian/Pacific Islander (22%) patients compared with non-Hispanic blacks (20%) and non-Hispanic whites (19%). More than two-thirds (71%) of cancers occurring in children and adolescents are rare cancers compared with less than 20% of cancers diagnosed in patients aged 65 years and older. Among solid tumors, 59% of rare cancers are diagnosed at regional or distant stages compared with 45% of common cancers. In part because of this stage distribution, 5-year relative survival is poorer for patients with a rare cancer compared with those diagnosed with a common cancer among both males (55% vs 75%) and females (60% vs 74%). However, 5-year relative survival is substantially higher for children and adolescents diagnosed with a rare cancer (82%) than for adults (46% for ages 65-79 years). Continued efforts are needed to develop interventions for prevention, early detection, and treatment to reduce the burden of rare cancers. Such discoveries can often advance knowledge for all cancers. CA Cancer J Clin 2017. © 2017 American Cancer Society. CA Cancer J Clin 2017;67:261-272. © 2017 American Cancer Society.

show abstract

Section: Discussionmentioning

confidence: 99%

The burden of rare cancers in the United States

DeSantis

Kramer

Jemal

2017

CA A Cancer J Clinicians

123

105

View full text Add to dashboard Cite

show abstract

“…The International Rare Cancers Initiative (IRCI) was set up in 2011 and comprises organisations involved in trials in rare cancers from several countries, including the UK, USA, Canada, and France [8]. One of the IRCI's objectives is to promote the development of innovative methodologies for research in rare cancers.…”

Section: Discussionmentioning

confidence: 99%

Sample size calculation in randomised phase II selection trials using a margin of practical equivalence

Dehbi

Hackshaw

2020

Trials

View full text Add to dashboard Cite

Background: In rare cancers or subtypes of common cancers, a comparison of multiple promising treatments may be required. The selected treatment can then be assessed against the standard of care (if it exists) or used as a backbone for combinations with new, possibly targeted, agents. There could be different experimental therapies or different doses of the same therapy, and either can be done in combination with standard treatments. A 'pick-thewinner' design is often used, which focuses on efficacy to select the most promising treatment. However, a treatment with a slightly lower efficacy compared to another treatment may actually be preferred if it has a better toxicity or quality of life profile, is easier to administer, or cheaper. Methods: By pre-defining a margin of practical equivalence in order to calculate the sample size, a more flexible assessment can be made of whether the treatments have very different effects or are sufficiently close so that other factors can be used to choose between them. Using exact binomial probabilities, we calculated the sample size for two-and three-arm randomised selection trials including a margin of practical equivalence with a variety of input parameters. Results: We explain conceptually the margin of practical equivalence in this paper, and provide a free user-friendly web application to calculate the required sample size for a variety of input parameters. Conclusion: The web application should help promote the randomised selection design with a margin of practical equivalence, which provides greater flexibility than the 'pick-the-winner' approach in assessing the results of selection trials.

show abstract

“…[1] RARECARE, a European group working to define the incidence of rare cancers in Europe, defines a rare cancer as one with an incidence <6 per 100,000. [2] Based on the definition of a rare disease as one affecting less than 200,000 people, all childhood cancers are considered rare, affecting 12,400 children and young adults yearly in the United States. Of these cancers, This work was authored as part of the Contributor's official duties as an Employee of the United States Government and is therefore a work of the United States Government.…”

Section: Defining Rare Tumorsmentioning

confidence: 99%

“…In Europe, rare cancers account for 22% of all cancer diagnoses with 541,000 new diagnoses every year. [2] According to an analysis of the Cancer in North America database, rare cancers were proportionally more common among young adults. [1] The same analysis also found that people of non-white race, and those of Hispanic ethnicity, were more likely than Caucasians to have a rare cancer diagnosis, compounding the issue of health disparities amongst ethnic groups.…”

Section: Defining Rare Tumorsmentioning

confidence: 99%

“…Specifically, the limited number of patients with a given rare tumor (1) limits the development of expertise in the disease, slowing diagnosis and early intervention, (2) impedes understanding of molecular pathogenesis to identify actionable mutations and (3) increases the time, and thus cost, needed to complete clinical trials or makes successful accrual of patients nearly impossible. These challenges have been partially addressed by changes in regulations governing drug development for rare disease indications and by the founding of national and international initiatives to promote research and clinical development in the field of rare diseases.…”

Section: Expert Opinionmentioning

confidence: 99%

See 1 more Smart Citation