During the annual ASH meeting, investigators issue new clinical development guidelines for sickle cell disease therapies.
During the American Society of Hematology (ASH) 2019 annual meeting, investigators released a comprehensive new set of recommendations that establish uniformity and global standards for clinical trial endpoints to evaluate sickle cell disease therapies.
The recommendations are the result of 7 experts and patient led panels convened by ASH and US Food and Drug Administration (FDA) to improve the design of clinical trials for sickle cell disease, including promoting broader use of patient reported outcomes and biomarkers as clinical endpoints.
Sickle cell disease is the most common inherited red blood cell disorder in the US, affecting millions of people worldwide. For people suffering from sickle cell disease, red blood cells become crescent or sickle-shaped, instead of round, contributing to the vaso-occlusive crises this patient population experiences.
Some of physical complications include acute pain crises, joint and organ damage, impaired cognitive function, and a reduced life expectancy.
Currently there are only 4 FDA approved treatments for sickle cell. Bone marrow transplants are also a cure for a portion of sickle cell patients, but not everyone.
There are other therapies being worked on in the pipeline, including potentially curative gene therapies.
“There are a number of investigational drugs in development that target different manifestations of SCD,” Julie Panepinto, MD, MSPH, Professor of Pediatric Hematology, Medical College of Wisconsin/Children's Wisconsin, co-chair of ASH's Guideline Oversight Committee, and co-chair of the workshop, said in a statement. “However, there are no clear standardized endpoints for evaluating the effect of therapies on clinical outcomes and patient well-being.”
Panepinto said clinical research should generally incorporate endpoints that are both measurable and directly beneficial to the patient based on their preferences and experiences.
“What's happening in SCD is really exciting and many of us feel we are on the cusp of identifying multiple disease-modifying therapies,” Panepinto said. “The field is exploding, so we want to be sure we are measuring relevant endpoints for researchers, clinicians, and patients because that helps us advance the field and get new therapies approved.”
During a 2-day workshop at ASH led by Panepinto and Ann Farrell, MD, Director of the FDA's Division of Hematology Products and co-chair of the workshop, a 7-person panel conducted extensive literature reviews, assessed available evidence, and used expert judgement to identify existing endpoints that can be incorporated into clinical trials.
The panels focused on the following areas: