Immuno-Oncology Trials: Breakthrough Drugs Present Unique Clinical Challenges

Immuno-oncology treatments have rapidly emerged as a prime area for cancer innovation, but they also present unique challenges small to mid-size oncology companies should consider as they move toward clinical trials.

Joy Yucaitis, senior director of oncology strategy and I recently had the opportunity to weigh in on these challenges and provide some strategic guidance in a series of articles on the topic.

Changing Endpoints

Traditional oncology trials often use tumor burden as a key metric for cancer progression. This criterion works well for chemotherapy trials because a decrease in tumor size indicates chemotherapy is working. However, effective immunotherapy treatments may result in a temporary increase in tumor mass because t-cells are infiltrating and attacking the targeted tumors. This is why it’s important to ensure the CRAs on your study have immuno-oncology experience.

In addition to accounting for the differences in biology behind a tumor’s response to immunotherapy compared to chemotherapy, the lifesaving potential of immuno-oncology treatments has increased the importance of surrogate endpoints that predict overall survival. Promising trial results with surrogate endpoints, such as overall response rate and progression-free survival, can often lead to expedited review and approval by the FDA, even out of Phase 1 clinical trials. You can read more about these changes in clinical endpoints in a recent Genetic Engineering & Biotechnology News article.

Rapidly Changing Standard of Care

Checkpoint inhibitors, an exciting new type of immuno-oncology treatment, unblind a patient’s t-cells from recognizing tumor cells. The promise of checkpoint inhibitors has led to a quickening pace and number of drug approvals, which, ironically, has introduced new challenges for developers entering clinical trials and raises numerous questions. Among the multiple checkpoint inhibitors competing for market share, which one do you choose in a forward-looking protocol? How do you design your protocol to account for these rapidly changing standards of care spurred by this new treatment? When can you be aggressive, and when should you adhere to traditional standards?

You can learn more about the impact of rapidly evolving standards of care on immuno-oncology clinical development in my interview with PharmaTimes.

Top Considerations for Designing Immuno-Oncology Clinical Trials

Considering the various nuances immuno-oncology clinical trials present, it’s important to prioritize areas of focus when designing a study protocol.

In addition to establishing the specific endpoints described above, study designs should address the precise identification of patients, determining a clinically effective dose, and managing patient safety. Addressing these issues early in study design will help speed a trial to a successful conclusion.

Read more about our prescriptions in Joy’s CenterWatch Monthly article.

To learn more about best practices in designing clinical trials for immuno-oncology treatments, read our white paper “Mastering Oncology Immunotherapy Clinical Trials.”

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