Pfizer, the New Jersey- based biopharmaceutical company, recently experienced a significant disappointment. A trial for the drug rivipansel, a potential treatment for the debilitating vaso-occlusive crises (VOCs) endured by virtually every person with sickle cell disease (SCD), did not meet its study endpoints.
“However, even though the study didn’t turn out as we had hoped, the good news is that the rivipansel program greatly advanced our understanding of SCD and VOCs, which will be important for future SCD research programs and drug development,” said Dr. Kevin Williams, chief medical officer for rare disease at Pfizer in a recent statement. “The one thing that working in SCD and collaborating with the NNPA and SCD-focused patient groups over the last several years has taught me is that this community is strong.” Williams said. “Their strength inspires me and my team every day and fuels our passion for our work in advancing the scientific research of rare diseases like SCD.”
SCD is the most common inherited blood disorder, and it disproportionately affects people of African descent. VOCs, one of the most common complications of SCD, are marked by extreme pain resulting from sickle-shaped red blood cells clumping together in the bloodstream. The standard of care for patients experiencing these pain crises has remained largely unchanged since the first report of SCD in 1910, and is restricted mostly to treating disease symptoms, like the use of opioids for pain management.
“We are in the process of reviewing the trial data closely to ensure our findings can help inform and shape future SCD programs,” Williams said. “I want to stress again that more trials in the rare disease space, particularly for SCD are desperately needed. I am confident in saying our steadfast commitment to this community will continue, as we work tirelessly to look for new ways to continue advancing care.”
A recent poll found that a majority of respondents had positive or neutral attitudes toward SCD clinical trials. Attendees indicated a willingness to participate in them if provided the appropriate information.
“These results are encouraging and suggest people are becoming more inclined to participate in clinical trials for hard-to-treat diseases like SCD,” Williams said.
Williams leads a medical affairs organization of approximately 150 medical colleagues around the globe supporting Pfizer’s efforts and portfolio in rare disease. He joined Pfizer in2004. In addition to his medical degree, Williams has a Master’s in Public Health from the UCLA School of Public Health and a Juris Doctorate from Harvard Law School.
