Comment from Anonymous

Thank you for opening this comment period. Currently, the regulatory framework of the FDA is insufficient to meaningfully encourage the scope of supplemental new drug applications necessary to meet patient demands. Whether through incentive programs, reducing evidentiary requirements, or addressing agency inflexibility and rigidity in reviewing clinical data, additional action is needed. The FDA should work with Congress to propose and introduce new legislation to better empower the FDA to, in collaboration with the National Institutes of Health, meet increasing patient needs. Significant financial resources exist for this work, so long as our country's spending is reallocated from investing in the military towards investing in developing the most effective and efficient drug approval infrastructure of any country. At the moment, we fall quite short of this goal, prioritizing military spending over the health of the public. While national security is critical, we have diverted an immense amount of funds to increasing our military excess, at the expense of patients. I would urge you to evaluate what means the agency currently has to revise its own regulatory framework to reduce the burdens imposed on sponsors to achieve supplemental approval. I argue that this can be achieved through several mechanisms: - developing an evidentiary standard for SNDAs that allows for greater reliance on existing published literature about unapproved, yet safe and effective uses of a drug (such as off-label prescribing practices, which have increasingly absorbed the burden imposed by the FDA's insufficient regulatory framework); - developing an evidentiary standard for SNDAs that draws on mechanistic, population-based, and external-control-generated data; - further developing and expanding regulatory frameworks for use of real-world data to demonstrate effectiveness and safety; - developing new regulatory opportunities to expand what conditions may constitute as an "orphan condition"; -