CLINICAL TRIALS FOR
RARE CANCERS
Rare cancer patients were systematically underserved by conventional drug development for decades. Tumour-agnostic approvals and basket trial design changed the eligibility logic. Comprehensive genomic profiling is how patients find out whether that change applies to them.
analyticsAt a Glance
- check_circleRare cancer trials often use basket or umbrella designs to enrol across multiple tumour types
- check_circleCompassionate use and expanded access may be available before a trial reaches your country
- check_circleInternational trials offer the only pathway for many rare cancer patients
- check_circleCancerFax helps identify active trials and coordinate access to specialist centres globally
How Molecular Targeting Changed Trial Access for Rare Cancers
Tumour-Agnostic Approvals
MSI-H/pembrolizumab (2017). NTRK fusions/larotrectinib and entrectinib (2018-2019). TMB-high/pembrolizumab (2020). RET fusions/selpercatinib (2022). Rare cancer type does not block access. The molecular feature is the eligibility criterion.
Basket Trials
Specifically designed to enrol based on molecular features across cancer types. Some of the original NTRK basket trial cohorts consisted substantially of rare tumour patients who had no other viable treatment at the time.
NCI-MATCH and Platform Programmes
Screen patients tumour molecular profiles and assign them to treatment arms based on findings. Multiple cancer types. Multiple alterations covered. Important source of trial access for uncommon alterations.
FGFR, IDH and Sarcoma-Specific Programmes
FGFR2 fusions in cholangiocarcinoma and beyond. IDH1/IDH2 in glioma, AML, and other rare diagnoses. EWSR1 fusions in Ewing sarcoma, SS18-SSX in synovial sarcoma. Dedicated research programmes alongside broader molecular-matched options.
Frequently Asked Questions
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination — travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
Questions About Clinical Trial Access for Your Rare Cancer?
CancerFax specialises in connecting rare cancer patients with tumour-agnostic treatment options and molecular-matched basket trials worldwide.
This content is for informational purposes only and does not constitute medical advice. Always consult a qualified oncologist before making treatment decisions.