To CDISC or not to CDISC? or: a time and place for everything.

The Clinical Data Interchange Standards Consortium (CDISC) is a non-profit organisation devoted to the development data standards for use in broader medical and especially in clinical research. Both the US FDA and the Japanese PMDA require the use of specific CDISC standards in submissions, but notably the EMA in Europe does not have equivalent requirements. 

As with any well-thought-out standard, implementation of CDISC standards offers significant advantages and increases in efficiency, especially when multiple organisations are involved – which in drug development is invariably the case with sponsor companies typically working with various service providers before finally submitting to regulatory authorities. 

In the 2000s (CDISC was started in 1997 and became international in 2001), it was thus my naive belief that CDISC implementation would rapidly become an industry-wide fact in pharma and biotech and across all stages of development without exceptions, especially after the FDA started to “recommend” its use in 2006 (it took another ten years, until 2016, for the US regulator to finally require CDISC in submissions). 

In the intervening 20 years, not only have I witnessed that this rapid and complete adoption did indeed not happen, I also learned to look past my own enthusiasm for CDISC standards and their ongoing development and understand circumstances in which adoption might actually offer little or even no tangible benefit to a company – circumstances that are more likely to apply to start-ups than to established companies. 

The first question to consider is a question of place. As already pointed out, the US FDA requires the use of CDISC in submissions, as does the Japanese PMDA. The EMA in Europe does not. Accordingly, depending on where one wishes to submit, CDISC may or may not be required. If you want to go to the US (or Japan) you must have CDISC. If you want to go to Europe, it is up to you whether to use CDISC or not. 

The second question to consider is a question of time. Again, the requirement for CDISC pertains to submission. Before submission, CDISC is not required. Especially in the early stages of clinical development (First in Human and other Phase I studies and moving into Phase II), any potential later need for CDISC is thus going to be well in the future. Depending on whether you even plan to do pivotal studies and/ or handle any submissions yourself, this need for CDISC may even come up only after an in-development asset has been acquired by another company. 

The final question then is to assess the pros and cons of going with or without CDISC in those circumstances where or when you do not need it, and this boils down to a question of costs (direct and indirect). The equation is simple: Deciding to go with CDISC brings the already mentioned increases in efficiency (and interoperability with other users), but the implementation of the standards requires you to draw from a limited expert pool for recruiting (for your internal processes including building those processes in the first place – alternatively, you can of course instead invest resources in training your existing staff) as well as from a limited pool of vendors (for outsourced aspects) already fluent in CDISC; deciding to go without CDISC will broaden the pools you can draw on (both internally and externally) and will allow you to focus your selection criteria on other areas than CDISC knowledge/ compliance – this may very well be cost-efficient in the short-term, but you should remain aware that once you are (re-)orienting towards a US submission, conversion to CDISC standards will become necessary. The efforts you will need to expend at that point will depend on the amount, structure and quality of the data to be converted – and while conversion will always be possible, sometimes more work will be required (e.g., when data structure differs significantly between individual studies). 

CDISC homepage: 
https://www.cdisc.org