APHON Letter to FDA Regarding Partial Clinical Hold for ALL Trials
March 1st, 2023
Dr. Robert M. Califf
Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Re: Partial clinical hold on AALL1731 and AALL1821
Dear Dr. Califf:
On behalf of the Association of Pediatric Hematology/Oncology Nurses (APHON), representing approximately 4,000 pediatric nurses who are dedicated to promoting optimal nursing care for children, adolescents, and young adults with cancer and blood disorders, and their families, we write to express our concerns about the U.S. Food and Drug Administration’s (FDA) notice of a partial clinical hold for trials AALL1731 and AALL1821 conducted by the Children’s Oncology Group (COG), both of which include the delivery of blinatumomab.
In the United States, in 2023, it is estimated that 15,190 children and adolescents between the ages of 0 to 19 will be diagnosed with cancer. Approximately 1 in 263 children are diagnosed with cancer before they turn 20 years old. As you know, cancer is the leading disease-related cause of death among both children and adolescents. However, the death rate for cancer has declined by more than half from 1970 to 2020 in both children and adolescents, largely due to improvements in treatment and high participation in clinical trials for the most common cancers, especially among children.
Through clinical trials, patients are offered the opportunity to receive cutting-edge treatments. These trials simultaneously advance the care for our patients while allowing the cancer community to learn more about the cancer and how treatments impacts patients. The current successes that have happened in the treatment of pediatric cancer is the direct result of this evaluation and will also lead to additional and less toxic treatments moving forward.
It has come to our attention that the FDA has disallowed the delivery of 72- and 96-hour IV bags of blinatumomab to newly diagnosed patients enrolling in AALL1731 and AALL1821 due to safety concerns.
These are two critical trials that the COG is conducting for standard risk and relapsed acute lymphoblastic leukemia (ALL). We agree that the safety of children enrolled in these studies is of the upmost importance, and we are concerned that this decision could lead to new safety risks for children involved in these studies.
We also believe this decision could place an undue burden on families receiving this treatment. In the absence of 72- and 96-hour IV bag options, families choosing to participate in AALL1731 or AALL1821 will either need to be hospitalized for the entirety of the 28-day infusion of blinatumomab or will need to return to the clinic every 1-2 days for an IV bag change, both of which carry their own health and safety risks. The logistics required to return to the clinic every 1-2 days – from time off of work to travel time and lodging requirements to additional childcare needs – could make trial participation no longer possible for a significant portion of children with ALL, particularly those with limited resources. We urge the FDA to work expeditiously with trial sponsors in considering the impact this clinical hold could have on trial participation and accessibility in addition to weighing any safety concerns.
As pediatric oncology nurses, we have firsthand understanding of the importance of ongoing clinical trials for both children and adolescents with cancer and their families and hope the FDA will take our comments into consideration. If you have any questions, please feel free to contact Wendy Chill, Manager of Health Policy and Advocacy at wchill@aphon.org.
Sincerely,
Janice Nuuhiwa, MSN, RN, NPD-BC, CPHON
President, Association of Pediatric Hematology/Oncology Nurses
8735 W. Higgins Road Ste. 300 • Chicago, IL 60631 • Phone: 855.202.9760 (US Residents Only) | 847.375.4724
Fax: 847.375.6436 • info@aphon.org • www.aphon.org