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FDA is Out of Touch With Patients’ Needs: A Former HHS Employee
(Thursday, March 2, 2023) A heartrending article by a former assistant secretary of public affairs and deputy chief of staff at the Department of Health and Human Services, highlights the disconnect between FDA’s requirements for critical drug approval and patients’ need. This is not the only case of its kind where patients and physicians complain of the opacity of the FDA’s processes and the delays it causes in the availability of direly needed products, and the ultimate cost of these treatments to the patients. The case in point is a rare pediatric disease, Juvenile Batten Disease or CLN3, that can be treated with a drug, Miglustat, approved for other indications. Miglustat has shown promise in off-label uses for CLN3 but since that’s not the approved use, it is not covered by the child’s insurance providers. Its out-of-pocket cost of $24,000 per month makes it impossible for practically any parent to afford private treatment. The worst case is that FDA’s Office of Rare Diseases and Medical Genetics has required a placebo-controlled clinical trial for the approval of Miglustat for CLN3. If using a placebo for a patient population that has limited time to live, and in whom delay is equivalent to harm, seems unreasonable and unethical, you are not alone. FDA occasionally takes stands about approval requirements for such diseases that are out in line from the patients and physicians’ perspectives and interest. It is FDA’s job to make unbiased, scientific and unemotional decisions about the drugs put forward to it for review, but if there is a case for flexibility, this fits the bill. The drug’s safety has already been evaluated and accepted by the FDA so any additional questions about its safety can be easily addressed. The off-label data that shows promised should be sufficient to allow an open-label, externally controlled clinical trial in CLN3 patients, who are all very young children with few years to live at most. There are numerous examples of even new chemical entities approved based on open-label clinical data. Even if we assume that the developers of Miglustat have not proposed external controls, FDA should be taking the lead on suggesting potential clinical development pathways for the rare disease. Drugs for rare disease need out-of-the-box thinking using regulatory flexibility and patient-centric thinking, so “caring for patients” does not just become a hollow promise, as it seems in this case. 
AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: [email protected] Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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