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IND, NDA and BLA submissions containing real-world data and real-world evidence (RWD/RWE) should highlight that info in the cover letter to that submission, according to the latest FDA guidance on best practices for the using such data to support claims in an application. While the guidance may seem mundane to an untrained eye, this highlights … Read more

Last year, the most common FDA 483 finding from inspections of drug manufacturing sites was non-compliant handling of deviation investigations and inadequate quality unit oversight. Inadequate CAPA, data integrity issues, and deficiencies in training of personnel wrapped up the top 5 major findings by FDA inspectors. The annual report on the GMP compliance in the … Read more

An independent study found that more than 76% of the drugs approved under the accelerated approval pathway (AAP) in the US did not demonstrate any additional therapeutic benefits compared to the previously available treatment options for the same indication. Also, the US FDA approved nearly three times as many drugs under this pathway compared to … Read more

This week’s court ruling to deregulate the most common form of autologous stem cell therapies would allow clinics to openly offer these treatments without the fear of FDA. The court, however, only ruled in favor of the medical practice but not the marketing claims made by the providers to lure their patients to opt for … Read more

In a stunning decision against the FDA, the United States District Court for the Central District of California ruled this week that autologous fat stem cell products cannot be regulated as new drugs. The ruling contains several comments by the court that upend about two decades of FDA’s regulatory decisions and several guidance documents, that … Read more

Medical devices not approved for sale in the US can be exported to other countries provided FDA determines that such exportation is not contrary to the public health and safety, and the device has the approval of the foreign country to which it is being exported. The manufacturers of such devices need to obtain an … Read more

Clinical trial sponsors with limited resources are permitted to charge patients for participating in their trial. However, there are rules and strict limitations for the cost that can be recovered from patients, and FDA’s permission must be obtained prior to charging patients. Sponsors have been allowed to charge the cost of manufacturing the investigational drug … Read more

Conducting clinical trials in children is extremely challenging so very few drugs are tested and approved based on pediatric clinical trials. Pediatricians frequently guesstimate dosing for their patients based on subjective assessment of the dosing data approved for adults. However, there might be an FDA-acceptable statistical methodology for formal extrapolation of adult doses to children … Read more

At $2.8 million per patient, the recently approved gene therapy treatment from Bluebird Bio is one of the most expensive treatments approved by the FDA. While developers may justify the cost of such treatments, is there an appetite with the payors for covering such treatments? Without coverage practically no patient can afford to pay for … Read more

Safety report for serious adverse events (SAE) observed in bioequivalence/bioavailability (BA/BE) studies are submitted as FDA MedWatch Forms (FDA 3500A Form) to the FDA. Going forward, FDA requires that sponsors of BA/BE studies submit SAE reports electronically via the ESG or the Safety Reporting Portal (SRP) only. The purpose of such reporting is to make … Read more