Vivek Subbiah: Why Every Rare Cancer Patient Deserves Molecular Profiling?

Vivek Subbiah, Chief of Early-Phase Drug Development at the Sarah Cannon Research Institute, shared a post on X:

“TWEETORIAL/X’torial: Why Every Rare Cancer Patient Deserves Molecular Profiling? 
Here’s a shocking fact: Rare cancers make up 25% of ALL cancer cases, yet most patients never get the molecular testing that could save their lives.
In our policy and practice paper – 20th anniversary of (JCO Oncology Practice) Our position. Comprehensive molecular profiling must be standard care for rare cancers.

Title: Imperative of Comprehensive Molecular Profiling as Standard of Care for Patients With Rare Cancers

Authors: Vivek Subbiah, Razelle Kurzrock

Read the Full Article.

The numbers are staggering: ~200 types of rare/ultra-rare cancers exist Rare = <15 per 100,000 people Ultra-rare = <1 per million/year Yet together they affect millions of patients worldwide. Each deserves a fighting chance.

Here’s the game-changer: The FDA has approved 9 tissue-agnostic therapies that work across cancer types based on molecular features, not where the cancer started. But here’s the catch – you can only access them if your tumor gets molecular profiling (NGS).

Title: The evolving landscape of tissue-agnostic therapies in precision oncology

Authors: Vivek Subbiah, Mohamed A. Gouda, Bettina Ryll, Howard A. Burris III, Razelle Kurzrock

Read the Full Article.

The 9 FDA-approved tissue-agnostic therapies include: Larotrectinib/entrectinib for NTRK fusions. Pembrolizumab for MSI-H tumors
Dabrafenib + trametinib for BRAF V600E
Trastuzumab deruxtecan for HER2+ tumors
Without testing = no access. Read further.

Why are cancers rare? Often because there’s only one molecular pathway driving them (vs common cancers with multiple drivers). This makes rare cancers especially good candidates for precision therapy – if we know what to target.

Title: Designing Clinical Trials for Patients With Rare Cancers: Connecting the Zebras

Authors: Vivek Subbiah, Megan Othus, Jim Palma, Branko Cuglievan, Razelle Kurzrock

Read the Full Article.

The cruel irony: Traditional drug development fails rare cancer patients because: Too few patients for large trials Limited commercial incentive Poor understanding of biology No standard of care for control arms We need a new approach.

The evidence is mounting: Studies like DRUP, SAMBA-101, I-PREDICT show rare cancer patients benefit from molecular matching at the same rates as common cancers. Clinical benefit rates: ~33% for both rare AND common cancers when matched to therapy.

But isn’t molecular testing expensive?” Nope! Costs have plummeted. Studies show precision medicine approaches result in: 22% lower costs 1.3 years longer survival It’s actually more expensive not to test.

Current reality check: Even for lung cancer with 10 known targets, NGS utilization is shockingly low. For rare cancers? Even worse due to: Limited awareness Inconsistent guidelines Access disparities Reimbursement challenges.

Professional guidelines exist but they’re: Complex and inconsistent Leave most rare cancers “orphaned” Create confusion for oncologists We need harmonized, clear guidance: Test all rare cancers.

Success stories matter: Some patients with MSI-H or high TMB tumors achieve long-term complete remissions – essentially cures – even with metastatic disease. These outcomes are only possible with molecular profiling.

What should happen at diagnosis for every rare cancer patient: Comprehensive NGS (mutations + fusions) HER2 testing by IHC Germline testing when indicated Results reviewed by molecular tumor board This should be standard, not optional.

Title: Universal Germline and Tumor Genomic Testing Needed to Win the War Against Cancer: Genomics Is the Diagnosis

Authors: Vivek Subbiah, Razelle Kurzrock,

Read the Full Article.

The bigger picture: Collecting molecular data from rare cancer patients:
Advances our understanding. Drives research for new therapies. Helps future patients. Builds the learning healthcare system we need. Every test matters.

Bottom line: Comprehensive molecular profiling isn’t just “nice to have” – it’s an imperative for rare cancer patients. It’s their pathway to: Accurate diagnosis. Targeted therapies. Clinical trials. Hope for better outcomes.

Call to action: Oncologists: Order NGS for ALL rare cancer patients Payers: Cover comprehensive profiling Patients/families: Advocate for testing Researchers: Continue building the evidence Every rare cancer patient deserves precision medicine.”