EORTC: Advancing research and treatment for rare cancers

Rare cancers are a major public health issue that requires urgent attention and action: around one in five cancers is rare: the incidence for rare cancers is less than six individuals per 100,000 a year. Rare cancers include more than 300 different types of cancers and may affect any organ in the body. They represent about 22% of all adult cancer cases, but their mortality rate is 30%. This means that rare cancers are responsible for one in three cancer deaths*.

On 29 February 2024, EORTC is joining other organisations in supporting Rare Disease Day, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease.

As part of its mission, EORTC works to address how to better care for rare and ultra-rare cancer patients, recognising the challenges this patient population faces in accessing new drugs and treatments rarely registered for them. This will be an essential priority in 2024 and beyond in our attempt to solve this unmet need.

How EORTC is making a difference for rare cancer patients: achievements and ongoing activities

EORTC has established and supports several disease-oriented groups that focus on specific rare cancers, such as sarcoma, head and neck, brain, uveal melanoma, and others. These groups design and conduct clinical trials, develop guidelines, and foster collaboration and education among rare cancer experts and stakeholders.

In addition, our organisation has an outstanding track record of rare cancer clinical trials that have transformed the lives of patients & their families and saves healthcare systems millions – without any commercial interest at stake. Some examples:

  • the EORTC CREATE trial looked at the long-term efficacy using crizotinib in patients with advanced, inoperable inflammatory myofibroblastic tumour.
  • EORTC trials significantly influenced the management of gastrointestinal stromal tumours (GIST) by demonstrating the continued benefit of imatinib in metastatic GIST patients, with some individuals surviving up to 10 years after treatment initiation.
  • In sarcoma research, EORTC’s large-scale clinical trials have significantly impacted treatment approaches. For example, our work has influenced the understanding of adjuvant chemotherapy and systemic treatment optimisation for sarcoma patients.

EORTC continues its outstanding work in rare and ultra rare-cancers: some of the EORTC’s ongoing activities in rare cancers are:

  • EORTC 1809 STRASS II trial STRASS II is a randomised phase III study of chemotherapy followed by surgery versus surgery alone to improve disease control and survival in patients with high-risk retroperitoneal sarcoma. Retroperitoneal sarcoma is an aggressive and rare cancer affecting the soft tissues of the body.
  • EORTC has launched and operates the SPECTA platform, a network of clinical and molecular screening platforms that collects and analyses biological samples and clinical data from cancer patients across Europe. The platform enables the identification of genetic alterations in rare cancers, which could be potential targets for therapy. SPECTA also facilitates the enrolment of rare cancer patients into clinical trials that match their molecular profile.
  • EORTC has initiated and leads the OligoRARE trial, a randomised clinical trial that evaluates the benefit of adding stereotactic body radiotherapy to standard of care treatment in patients with oligometastatic rare cancers. Oligometastatic cancer is an intermediate state between localised and widespread metastatic cancer, with a limited number of lesions and organs involved. The trial aims to improve the overall survival and quality of life of these patients, who have limited treatment options and poor prognosis.
  • EORTC 1634 PersoMed-I: PersoMed-I is a multicentre, international Phase II clinical trial on medulloblastoma, a rare type of brain cancer mostly affecting young people. Given that the standard treatment for this cancer has predominantly been tested in randomised trials involving children, our trial aims to identify the most effective treatment options for teenagers and adults.
  • EORTC and EURACAN developed a collaborative clinical research project called “ARCAGEN.” The goal of ARCAGEN is to detect genetic variations in rare cancers.  Over three and a half years, 918 patients from 14 European countries with rare adult solid tumours at an advanced phase received a molecular profile of their cancer. The study shows that molecular profiling for rare cancer patients is feasible, with a low failure rate and a fast turnaround time [14 days on average], bringing new treatment options for patients.
  • EORTC participates and contributes to several European and international networks and consortia that address the challenges and needs of rare cancer research and care, such as EURACAN and others. These collaborations aim to increase the visibility and recognition of rare cancers, harmonise the definition and classification of rare cancers, promote the exchange of knowledge and best practices, and advocate for the rights and interests of rare cancer patients. Read the latest publication: Management of patients with rare adult solid cancers: objectives and evaluation of European reference networks (ERN) EURACAN – The Lancet Regional Health – Europe

And many more! More information on ongoing trials is available on our website.

Join us to celebrate Rare Disease Day: together, we leave no one behind.

* Gatta G, van der Zwan JM, Casali PG, Siesling S, Dei Tos AP, Kunkler I, Otter R, Licitra L, Mallone S, Tavilla A, Trama A, Capocaccia R; RARECARE working group. Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer. 2011 Nov;47(17):2493-511. doi: 10.1016/j.ejca.2011.08.008. Epub 2011 Oct 25. PMID: 22033323.


The EORTC-1809-STBSG – STRASS-2 / STREXIT-2 trial is being funded by Anticancer Fund (ACF), EORTC Soft Tissue and Bone Sarcoma Group, EORTC Cancer Research Fund (ECRF), Climbers Against Cancer, CRUK, Sarcoma UK, Novo Nordisk Fonden and Horizon Europe funding program (project 101103843), Australian Medical Research Future Fund and the Australia and New Zealand Sarcoma Association.

ARCAGEN is a SPECTA platform downstream project. The SPECTA Platform is supported by Alliance Healthcare. Alliance Healthcare will become Cencora. ARCAGEN is a collaboration between EORTC and EURACAN and is funded by F. Hoffman-La Roche.

OligoRare is supported by Anticancer Fund (ACF), Rising Tide Foundation and Kom op tegen Kanker.

PersoMed-I is supported by Gateway for Cancer Research, Deutsche Krebshilfe, Canteen Australia; Cancer Australia, the Dutch Cancer Society, the French Ministry of Health, and the Swiss Brain Tumor Foundation

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