Designs and challenges for personalized medicine studies in oncology: focus on the SHIVA trial

Area of Science:

• Oncology
• Genomics
• Translational Medicine

Background:

• Personalized medicine uses genetic, protein, and environmental data for disease management.
• In oncology, personalized medicine emerged with molecularly targeted agents, prescribed based on tumor location and histology.
• Current predictive biomarkers are validated only for specific tumor types.

Purpose of the Study:

• To evaluate the relevance of prescribing molecularly targeted therapies based on tumor molecular abnormalities, irrespective of primary tumor location and histology.
• To review study designs for evaluating personalized medicine approaches in oncology.
• To detail the SHIVA trial design, a randomized proof-of-concept phase II trial.

Main Methods:

• Review of observational cohorts, nonrandomized, and randomized clinical trials for personalized medicine evaluation.
• Detailed design of the SHIVA trial: a randomized phase II trial comparing molecular profiling-guided therapy versus conventional therapy in refractory cancer patients.
• Discussion of challenges and future perspectives for personalized medicine implementation.

Main Results:

• The study reviews various designs for evaluating personalized medicine, discussing their pros and cons.
• The SHIVA trial design is presented as a proof-of-concept for this approach.
• Challenges in implementing personalized medicine in oncology are discussed.

Conclusions:

• Personalized medicine in oncology, guided by molecular profiling, needs prospective validation.
• Different clinical trial designs are proposed to evaluate this novel therapeutic strategy.
• Overcoming implementation challenges is crucial for advancing personalized medicine in cancer care.