BOSTON, August, 7, 2019 — Yesterday afternoon, the FDA announced that Novartis informed the agency in June of a data manipulation issue that affects the accuracy of certain animal testing data related to onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), a gene therapy approved in May to treat certain individuals with spinal muscular atrophy (SMA). In its announcement, the FDA clarified that “these data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program.”
The Institute for Clinical and Economic Review (ICER) is deeply concerned about issues related to data integrity, and we are monitoring this situation closely. The animal data identified by the FDA did not factor into ICER’s assessment of the comparative clinical effectiveness and economic value of Zolgensma. However, if additional investigations reveal that data from the human trials also were compromised in any way, ICER will issue an update to our earlier assessment.
The FDA is encouraging any parents of children with SMA who have questions or concerns to contact the agency at (800) 835-4709.
The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.
ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.