STAT+: Pharmalittle: We’re reading about FDA and a ‘smear campaign,’ Cigna buying a large pharmacy, and more

FDA Commissioner Marty Makary defended the agency's recent rejections of drugs aimed at treating rare diseases during an interview with CNBC. His comments come amid scrutiny regarding the FDA's decision-making process for approving medications for these conditions. The rejections have sparked discussions about the agency's criteria and the implications for patients with rare diseases who may be in need of new treatment options. In addition to addressing the rejections, Makary also responded to remarks made by Vinay Prasad, a top official at the FDA. Prasad's comments have drawn attention and may have contributed to the ongoing debate about the agency's approach to drug approvals. The FDA's decisions have raised questions about the balance between ensuring drug safety and efficacy while also considering the urgent needs of patients suffering from rare diseases. The context of these discussions highlights the challenges faced by regulatory bodies like the FDA in navigating the complexities of drug approval processes, particularly for conditions that affect a smaller patient population. The agency's stance has implications not only for pharmaceutical companies but also for patients and healthcare providers who rely on access to effective treatments. Overall, the FDA's recent actions and the ensuing dialogue among its leadership underscore the critical nature of drug approval processes and the ongoing challenges in addressing the needs of patients with rare diseases.