Pediatric Hematologist-Oncologist and Oakland IRB Chair Jennifer Michlitsch, MD, is a specialist in pediatric blood diseases who is also passionate about increasing access to care and clinical trials for all children and their families. This passion for providing care for all patients also influences her work on the IRB to ensure the integrity of research while upholding ethical standards.
We spoke with Dr. Michlitsch about her work on the IRB, the special challenges associated with approving pediatric studies, and the importance of improving access to pediatric clinical trials.
Can you share a bit about your background and your role at UCSF?
I am a pediatric oncologist at UCSF, having completed my fellowship in 2009 at what was then Children's Hospital Oakland. I was drawn to Children's Hospital Oakland because of its unique culture, which combines academic excellence with a mission to provide care for all patients, regardless of their background, primary language, insurance status, or ability to pay.
My work, both clinical and research, focuses on ensuring access to standard therapies and clinical trials for children, adolescents, and young adults from diverse backgrounds. Historically, Phase 1 clinical trials in oncology have been limited to a few centers across the country, creating access barriers, especially for families who cannot travel. To address this, I strive to open clinical trials in Oakland, allowing us to treat patients with relapsed, refractory, and hard-to-treat cancers who might otherwise lack access to emerging therapies.
What are some of the special challenges in reviewing and approving pediatric studies?
Pediatric studies involve unique challenges because decisions are made by parents or legal guardians on behalf of children. One challenge is determining the appropriate age cutoffs for obtaining various levels of assent from children. As clinical trials become more complex, especially with gene and immunotherapies, it is crucial to explain studies at an appropriate level for both children and adults. For example, obtaining verbal assent from children aged 7 to 12 requires careful consideration of how much detail to provide. This requires case-by-case deliberation by the IRB and the study team to ensure that we adequately communicate the details of a clinical trial in a way that is understandable to children and their families.
What other considerations are important in pediatric studies, especially those involving rare diseases?
Pediatric studies have unique considerations such as the risk of sedation for procedures like MRIs, which often require general anesthesia for young children. This adds another layer of risk compared to adult studies. We also have strict limits on how much blood can be drawn for research based on the patient's body weight to ensure safety. These factors necessitate careful planning and review by the IRB to balance research needs with patient safety. Additionally, including diverse populations in studies is crucial as we need to understand how new therapies work across different ethnic groups.
What systems or resources must be put in place to increase access to clinical trials for both parents and children?
Language is a significant barrier to access. Our policy requires that consent and assent documents be available in the patient's native language if more than a few non-English speaking patients are to be enrolled. While large pharmaceutical companies can typically fund these translations, smaller research groups often lack the resources. I've obtained small grants to help translate documents, but funding can be limited. This inequity in access means some families have historically been excluded based on language, which is unacceptable. We have been able to provide support for translations through our RAGE Program, but we need more resources to ensure that all patients can participate in clinical trials, which is essential for studying the efficacy of new interventions across diverse populations.
The IRB has created a DEIA working group to make progress on this and other initiatives to increase diversity, equity, inclusion, and access.
What have you found most fulfilling or impactful about serving on the IRB?
Helping investigators navigate the process of getting their studies approved is incredibly fulfilling. Our goal is always to enable studies to open while ensuring patient rights are protected and informed consent is obtained. Open communication between the IRB and study investigators is key. In Oakland, we invite PIs to present new studies, which allows for direct dialogue and clarification of any concerns. This practice, now adopted by other UCSF IRBs, as part of the Office of Research’s Clinical Trial Excellence Campaign, fosters understanding and collaboration, ultimately facilitating the opening of important clinical trials.
How would you describe the importance of the IRB within the UCSF community?
The IRB is critical to UCSF's mission of conducting responsible research. It ensures that the rights and interests of study participants are always the top priority. The IRB's role in protecting participants is essential for maintaining the integrity of our research and advancing medical knowledge while upholding ethical standards.
Is there anything else you would like to add?
The field of pediatric oncology has made significant advancements in recent years, with many cancers now having high cure rates due to clinical trials. It's crucial to continue opening and enrolling patients in studies to further improve outcomes. My passion for this work drives me to ensure that all children, regardless of background, have access to the latest therapies and research opportunities.