Called the Critical Path for Sickle Cell Disease (CP-SCD), the initiative will facilitate collaborations between researchers, develop community consensus, and engage with regulatory agencies to pinpoint and address regulatory issues that limit the approval of new therapies.
“The sharing and integration of knowledge, data and collaboration across different organizations is key in our efforts to generate tools that will accelerate medical product development for sickle cell disease,” Klaus Romero, MD, C-Path’s chief science officer, said in a press release.
“C-Path is uniquely positioned to lead CP-SCD as it has extensive experience in accessing and leveraging data from people living with rare diseases to support regulatory-grade solutions for unmet needs in medical product development,” Romero added.
Despite recent advances in sickle cell disease research, which has led to the development of many potential therapeutics, as well as therapy approvals, regulatory approval of these new therapies is limited by a lack of understanding of the natural history of the disease and how to accurately measure therapeutic effects.
CP-SCD was set up to further an understanding of these issues and develop tools to overcome them. Specifically, it will work to identify tools such as disease progression models, computer-based trial simulators, clinical outcome measures including patient-reported outcomes, efficacy and safety biomarkers, and seek out regulatory endorsements for the use of these tools.
Furthermore, the consortium will develop new clinical trial designs such as defining trial entry criteria, endpoint selection, optimizing control arms, and standardizing data collection and standards.
“A goal of this consortium is to establish an international forum that engages regulatory agencies around the globe to identify and address issues that impact the development and approval of new therapies for sickle cell disease,” said Jane Larkindale, PhD, CP-SCD’s executive director.
September was National Sickle Cell Disease Awareness Month, which helped focus attention on the need for research and treatment of sickle cell disease, an inherited condition that causes red blood cells to become deformed or sickled. As a result, a person can become anemic, in addition to obstructing blood flow, causing pain and organ damage.
While there are some approved therapies, none have the potential to cure the condition, except for stem cell transplants, which take blood stem cells from a healthy donor to provide a source of functional red blood cells. However, few patients find a matching donor.
“We are excited to … continue to work towards making a difference in the sickle cell community,” Larkindale said.
C-Path is a nonprofit, independent organization established in 2005 as a private and public partnership. It has organized numerous global consortia, including more than 1,600 scientists from academia, disease foundations, patient organizations, government and regulatory agencies, and dozens of biotech and pharmaceutical companies.