In December 2014, the US FDA published a binding guidance document, Guidance for Industry – Providing Regulatory Submissions in Electronic Format – Standardized Study Data, that establishes a requirement for submitting study data (clinical and non-clinical) in electronic format conforming to CDISC standards.
The study data requirement is not retroactive and will only apply to studies initiated on or after the effective date, as is stated in the FDA guidance.
“After the publication of this guidance, all studies with a start date 24 months after the publication date (December 2014) must use the appropriate FDA-supported standards, formats, and terminologies specified in the Catalog for NDA, ANDA, and certain BLA submissions. Study data contained in certain IND submissions must use the specified formats for electronic submission in studies with a start date 36 months after the publication of this guidance.”
To aid the industry in complying with these study data submission requirements, the FDA developed guidance documents germane to each submission type – NDAs, BLAs, ANDAs, and INDs. FDA guidance makes clear the risks of non-compliance.
“The Agency may Refuse to File (RTF) for NDAs and BLAs, or Refuse to Receive (RTR) for ANDAs if an electronic submission does not have study data in conformance to the required standards specified in the Catalog.”
Failure to understand the FDA guidance could result in lengthy submission rework and cause delays in getting the drug to market, in turn resulting in loss of revenue.
All life science companies, particularly early stage companies with limited resources and a limited understanding of the data study requirements, should utilize expertise available at the FDA as well as experienced Contract Research Organizations (CROs) to ensure they’re in compliance at each step of the process prior to submission. Start as early as possible to reduce the risk of encountering roadblocks. Pre-NDA/BLA meetings are almost always too late to start these discussions as rework is time consuming and likely to cause further delay.
Being clear on expectations from the outset of the process is the most successful path to on-time submissions in compliance with the FDA’s new clinical data requirements. It’s also important to tap into expert knowledge, especially as these changes take effect.