advanced cancer

Cancer clinical trials represent the largest single category of recruiting studies worldwide, encompassing everything from early-phase safety studies of novel oncology drugs to large Phase 3 comparisons against current standard-of-care chemotherapy regimens. The field has accelerated dramatically with the rise of immunotherapy, targeted therapies, and CAR-T cell treatments that have transformed outcomes for previously untreatable malignancies.

Trials include checkpoint inhibitors (PD-1/PD-L1 agents), KRAS inhibitors, ADC (antibody-drug conjugates), combination chemotherapy regimens, radiation protocols, and surgical technique comparisons. Many trials stratify by biomarker status (PD-L1 expression, TMB, MSI-H) to identify patients most likely to benefit.

The NCI, pharmaceutical sponsors, and academic cancer centers collectively fund thousands of oncology trials annually across every cancer type.

Disease Burden & Epidemiology

Cancer is a leading cause of death globally, with approximately 19.3 million new cases and 10 million deaths recorded in 2020 according to the Global Cancer Observatory. In the United States alone, the American Cancer Society estimates roughly 1.9 million new cancer diagnoses annually. The lifetime risk of developing invasive cancer is approximately 40% for men and 39% for women in the US. While survival rates have improved substantially β€” the five-year survival rate across all cancers has risen from 49% in the 1970s to approximately 68% today β€” the disease remains the second leading cause of death in high-income countries. Lung, colorectal, breast, and prostate cancers collectively account for the highest incidence and mortality globally, while rare malignancies such as pancreatic and glioblastoma continue to carry the poorest prognoses. Geographic variation is substantial: low- and middle-income countries bear a disproportionate burden due to limited screening infrastructure, delayed diagnosis, and restricted access to systemic therapy. Clinical trials are essential to improving these outcomes at a population level.

Key Research Trends & Landmark Studies

The past decade has produced transformative trial results that have fundamentally changed cancer treatment standards. The KEYNOTE-024 trial established pembrolizumab as first-line standard of care for high PD-L1 non-small cell lung cancer, replacing chemotherapy in a defined biomarker population. The CheckMate 067 trial demonstrated durable 10-year survival in advanced melanoma patients receiving nivolumab plus ipilimumab combination immunotherapy. The DESTINY-Breast03 trial validated trastuzumab deruxtecan (T-DXd) as superior to standard HER2-targeted therapy in HER2-positive breast cancer, accelerating the ADC class across tumor types. In hematology, the TRANSCEND trial led to axicabtagene ciloleucel approval for relapsed DLBCL, establishing CAR-T cell therapy as standard salvage in B-cell lymphoma. The SOLO-1 trial confirmed olaparib (PARP inhibitor) benefit in BRCA-mutated ovarian cancer maintenance, validating germline testing as a standard oncology workflow. Currently active platform trials β€” including NCI-MATCH, TAPUR, and ASCO TARGET β€” use basket and umbrella designs to match patients to experimental therapies based on molecular tumor profiling rather than histological site of origin.

Patient Guide: How to Find & Join a Trial

If you or a family member has been diagnosed with cancer, discussing clinical trial eligibility with your oncologist at every treatment decision point is strongly recommended β€” not just at relapse. Academic cancer centers affiliated with the NCI Cancer Center Program (51 designated centers) typically offer the widest trial portfolio and dedicated clinical trials navigation services. To search independently, filter by your cancer type, treatment history, and location on this page or directly on ClinicalTrials.gov. When reviewing a trial, focus on the eligibility criteria section: prior treatment requirements, performance status (ECOG 0-2 is most common), biomarker requirements, and organ function thresholds are the most frequent barriers to enrollment. Ask your care team about tissue and blood banking β€” many trials require archival biopsy material for biomarker testing before enrollment. Most Phase 2 and 3 oncology trials cover experimental treatment costs; standard-of-care costs may or may not be covered by insurance.

Frequently Asked Questions — advanced cancer Clinical Trials

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