Comment from Bower, Kaylin
AnonymousSupportIndividual
Summary: Dr. Luciana Borio, a medical professional, provides a citation from a scientific dispute resolution appeal to illustrate the potential risks of patient-focused drug development. She argues that the FDA must maintain objectivity and data-driven standards to ensure that products are not approved based on "hope" or "theoretical constructs" rather than meaningful clinical benefit.
This comment is for docket: FDA-2026-N-3947: "Impacts of Patient-Focused Drug Development Meetings; Establishment of a Public Docket; Request for Information and Comments":
From the "Scientific Dispute Resolution Appeal regarding Eteplirsen" from Luciana Borio, M.D., Acting Chief Scientist to Robert Califf, M.D., Commissioner of Food and Drugs, dated August 8, 2016:
I am sharing this citation as an example of what patient-focused drug development is NOT supposed to be!:
"FDA must remain steadfast in its commitment to alleviating pain and suffering, approach the most challenging problems with absolute determination, and apply maximum flexibility to facilitate the development and availability of effective treatments. The agency's value centers on its ability to do all of the above while maintaining objectivity, even in the face of political pressure. FDA should never mislead patients by granting even accelerated approval to products that are not shown to offer the prospect of meaningful benefit to patients under the appropriate regulatory and scientific standard.
I acknowledge that there are currently no specific drugs available to treat patients with DMD and that issuance of a complete response letter would cast uncertainty on whether eteplirsen would continue to be developed, based on business and financial decisions that are external to FDA. However, approving products based on hope, on subjective clinical judgment, or on theoretical constructs that are not anchored in data leads to irreparable damage to patients. Approval at this time could deter others from pursuing the development of truly effective treatments, both for DMD and other serious, life-threatening conditions. Granting accelerated approval here on the basis of the data submitted could make matters worse for patients with no existing meaningful therapies-both by discouraging others from developing effective therapies for DMD and by encouraging other developments to seek approval for serious conditions before they have invested the time and research necessary to establish whether a product is likely to confer clinical benefit" (page 26)