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Upcoming NurOwn Adcomm Highlights Regulatory Role for Patient Advocates

Sep 25, 2023 - biospace.com
Patient advocacy organizations (PAOs) are increasingly influencing the drug approval process, particularly in the neurodegenerative and neuromuscular spaces, leading to debates about the ethics of this influence and calls for greater transparency. These organizations can exert influence through public comments, patient representatives on advisory committees, and direct pressure on the FDA. However, concerns have been raised about potential conflicts of interest, as many PAOs receive funding from the pharmaceutical industry.

Some experts, including Genevieve Kanter, associate professor of public policy at the University of Southern California, are calling for greater transparency in the selection process for patient representatives and the financial support of these organizations. Meanwhile, patient advocates argue that their voices are essential to the regulatory process and that industry funding does not affect their ability to represent people affected by disease. The debate continues as the role of PAOs in the drug approval process becomes more prominent.

Key takeaways:

  • Patient advocacy organizations (PAOs) have a significant influence on the drug approval process, particularly in the neurodegenerative and neuromuscular spaces, leading to debates about the ethics of this influence and calls for greater transparency.
  • PAOs can exert influence through three pathways: the public comment section, the presence of a patient representative on advisory committees, and potentially exerting pressure directly on the FDA.
  • There are concerns about potential conflicts of interest due to the significant funding many PAOs receive from the pharmaceutical industry, although some argue that this does not affect their ability to represent patients.
  • There are calls for greater transparency in the selection process for patient representatives on advisory committees, including information about their affiliations and sources of financial support.
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