334: Pharma silence on RFK Jr. & parents trying to develop gene therapies for their children
Dec 12, 2024
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Jason Mast, a colleague from STAT, dives into the intricate world of gene therapy, spotlighting a father's inspiring quest for solutions to help his child. They discuss the significant hurdles in this field, from financial challenges to unique fundraising efforts by families. Meanwhile, the conversation touches on why pharmaceutical companies remain silent on Robert F. Kennedy Jr.'s criticisms, exploring the broader implications for health policy. The emotional journey of families navigating rare conditions sheds light on the urgent need for effective therapies.
The pharmaceutical industry's silence on Robert F. Kennedy Jr.'s nomination suggests a strategic shift in its lobbying approach under the Biden administration.
Despite advances in gene therapy, many families are left to navigate high costs and limited development for rare genetic treatments, exemplified by individual advocates like Terry Pirvilakis.
Deep dives
Pharma's Silence on RFK Jr.
Robert F. Kennedy Jr. has criticized the pharmaceutical industry for alleged corruption and public health harms, yet the industry remains silent regarding his nomination for HHS secretary. Despite his long-standing negative rhetoric about drug makers and their influence on government, current reports indicate that pharmaceutical companies are not actively lobbying against his confirmation. Analysts suggest that the industry may be assessing the broader implications of his potential policies or prioritizing other regulatory concerns related to the FDA. This situation reflects a possible shift in the pharmaceutical industry's approach to lobbying under the Biden administration, possibly indicating weakened influence.
Gene Therapy Innovations and Challenges
Recent advancements in genetics have enabled the diagnosis and potential treatment of rare genetic disorders, yet the development and commercialization of these therapies remain limited. High costs, small market sizes, and lengthy development timelines deter many pharmaceutical companies from pursuing these promising treatments, leaving families with few options. This has led individuals like Terry Pirvilakis to take matters into their own hands, advocating for research and therapy options for their children suffering from rare conditions, often relying on personal fundraising efforts. Despite challenges in scaling these solutions, their dedication highlights a critical gap in the industry’s focus on rare genetic diseases.
GLP-1 Drugs and Addiction Research
Emerging research suggests that GLP-1 drugs, known for managing obesity, may also have potential applications in treating addiction to substances like alcohol and drugs. These medications work on brain circuits related to desire, which could help in suppressing cravings beyond food-related issues. Recently, Eli Lilly announced plans to initiate large studies to evaluate the efficacy of GLP-1 treatments for addiction, marking a significant step in expanding the use of these drugs. This initiative responds to a growing call for increased investment in addiction treatment options, a significant public health challenge.
Washington correspondent John Wilkerson joins to discuss why drugmakers have not lobbied against Robert F. Kennedy Jr.'s nomination to lead the Department of Health and Human Services, despite his repeated criticisms of the pharmaceutical industry. We also bring on our colleague Jason Mast to talk about the major hurdles in the field of gene therapy and how one father is trying to overcome them.
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