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Small to medium size pharma companies have low adoption rates when it comes to implementing CDISC standards. Is this due to lack of awareness or understanding? How does a company that is new to standards see the wood through the trees in the new regulatory environment? Let us explore a number of approaches in how to best establish data standards between sponsors and CROs.
Though we cannot imagine a daily life without standards, the pharmaceutical world has been a very slow adopter. Standards are still very much seen as something that FDA or PMDA want. Moreover, standards sound complex, and one should leave it to the experts, right? In the below article, we will describe some benefits to having standards early (or rather, the risks of not having standards), and show some approaches to implementing standards that are not overly complex and will not burn the clinical development budget of even a small biotech company with a very limited portfolio.
APPROACHES TO IMPLEMENTING A LIBRARY
While undoubtedly there are many more models and variants of models than mentioned in this article, we will consider models that can work for small to midsize bio-techs, pharmaceutical, and device companies and academia. There is an assumption that the company either has in-house data standards expertise or has access to expertise through a third party (CRO, technology partner or consultant). The approaches presented below, gradually increase in complexity and associated cost, but also with increasing benefits when implemented well. The article assumes that CRF builds and data management work is outsourced to a CRO, but each of the models can work with an in-house team as well.
APPROACH 1: THE CRO IS THE EXPERT, LET THEM DECIDE
Most small biotech companies, with a successful “first in human” trial need to start thinking about their Compound Development Strategy, and probably their company growth strategy. At a time when costs are high and Return on Investment is still a distant goal, a company may find it wise to hire a biostatistician, chances are they will not prioritize on hiring a statistical programmer, data manager, let alone a data standards steward. Nevertheless, many have heard that if they want to submit their data to the FDA or PMDA, they will have to adhere to CDISC standards. So, when writing the RFP for the next trial, CDISC deliverables are required. All CROs confirm that their deliverables are CDISC compliant, so why does everyone say that standards are complicated?
For two parties (Sponsor and CRO) to “speak the same language”, they need to agree on what is captured (the CRF, or in CDISC terminology – CDASH) and how it is represented (SDTM). While the “light” approach, where the Biotech company relies on the CRO to define the expectations, presents its challenges, there are situations where this model can work. If a Biotech company is committed to a single CRO, where this CRO is really a biometrics extension of the Biotech company, then this is a cheap and effective solution. Most likely the CRO will have invested in their own set of standards and the Biotech company profits from this investment.
APPROACH 2: THE SPONSOR OWNS A BASIC LIBRARY
At a minimum, a “simple” library should contain a CRF layout or other form of data visualization and associated SDTM annotations. This will ensure the consistency of data collection and data reporting. In this case, the Sponsor will communicate with the CRO through a study set up package. This package contains a protocol, a (sub)set of CRFs and preferably data specifications.
The data specifications are particularly useful when there are multiple ways of collecting data, and the protocol does not provide the level of detail that a CRO may need (refer to the example of the medical history of substance abuse). A data specification package may be as simple as a list of the unique name and version of CRFs to be used. How confident can you be that the CRO delivers what you expect? The advantage of standardization is that there are tools on the market that facilitate the review of your SDTM data structure. While they will not check the content of the data, these tools are instrumental to checking your data for compliance and will give you some level of confidence that the data is conformant to FDA or PMDA expectations. When would this be a good model? It is a sustainable option for a small to midsize company, that has a focus on quality and consistency, may plan to bring a portfolio into full development (phase II/III), but has a limited budget for infrastructure. In other words, a library would be a standalone tool, in its most elementary form a versioned word document or excel spreadsheet.
APPROACH 3: THE SPONSOR OWNS AN INTEGRATED LIBRARY
Once standards are implemented, the options for automation, thus building efficiency and quality into your process, are almost endless. An integrated library not only contains a data visualization and SDTM mapping but will also have a metadata library. Additional information captured in this library includes code lists, computational methods, value level metadata, etc. Certainly, when your library is still small or managed by a limited number of users, it is still possible to maintain the library in Excel. Careful design of your metadata library will allow a very simple extraction routine for study builds and generation of define.xml according to the latest standards. Once the Integrated Library is in place, you can start implementing automation, including, but not limited to automated EDC build and automated validation of your data structure.
It is clear that clinical data standards are here to stay, and the sooner in your process, you build in the standardization, the greater the benefit, sponsors will still have to make decisions on the level of standardization. This will greatly depend on the company’s operational model, staff and budget. There is no one size fits all solution, but there is a solution for everyone. Key for success is to look at the long-term goals and processes and implement stepwise. Even a minimal investment can make a huge difference in how CROs and Sponsors communicate and manage expectations.
At MaxisIT, we clearly understand strategic priorities within clinical R&D, and we can resonate that well with our similar experiences of implementing solutions for improving Clinical Development Portfolio via an integrated platform-based approach; which delivers timely access to study specific as well as standardized and aggregated clinical trial operations as well as patient data, allows efficient trial oversight via remote monitoring, statistically assessed controls, data quality management, clinical reviews, and statistical computing.
Moreover, it provides capabilities for planned vs. actual trending, optimization, as well as for fraud detection and risk-based monitoring. MaxisIT’s Integrated Technology Platform is a purpose-built solution, which helps Pharmaceutical & Life sciences industry by “Empowering Business Stakeholders with Integrated Computing, and Self-service Dashboards in the strategically externalized enterprise environment with major focus on the core clinical operations data as well as clinical information assets; which allows improved control over externalized, CROs and partners driven, clinical ecosystem; and enable in-time decision support, continuous monitoring over regulatory compliance, and greater operational efficiency at a measurable rate”.