Importance of selecting technology specifically designed to address the needs of post-approval research
Over the last decade, across the globe, the regulatory landscape has changed considerably, with greater focus and awareness on pharmaceutical products post-approval. Legislation, coupled with public concerns about the safety of marketed drugs and the need to evaluate safety and effectiveness of the marketed products in a real-world setting is forcing many companies to embark on post-approval research.
While post-marketing research is evolving rapidly, and these studies are becoming far more complex, our knowledge in implementing and conducting such studies has not evolved with the same pace. Therefore, sponsors and Clinical Research Organizations (CROs) often want to apply a cookie-cutter approach, including technology, to post-approval research that mimics pre-marketing Randomized Controlled Trials (RCT). This is a mistake for several reasons.
First, a common misconception in post-approval research is that "one size fits all" when it comes to Standard Operating Procedures (SOPs). By mimicking practices, procedures and technology associated with registration studies, you can unknowingly decrease overall study efficiencies. This typically is a direct result of not having post-approval specific SOPs.
When teams utilize SOPs that govern RCTs across multiple functions, they are infusing processes with unnecessary stringency that is heavily geared toward meeting the regulatory requirements associated with registration trials. This translates into increased resources inflating overall study cost. Therefore, it is critical to have a unique set of post-approval specific SOPs to successfully conduct post-approval research. Through the implementation of post-approval specific SOPs, you will increase study efficiencies, decrease resource burn and reduce overall cost.
A second challenge of post-approval research is the size and scope of these studies. They are typically conducted in large and geographically diverse populations. Due to the large number of patients enrolled, and the large number of sites, it is important to have post-approval specific technology that can support the scale of the research and the large amounts of data. Technology deployed in post-approval research must not be developed using the same amount of rigor needed in RCT since the regulations are vastly different post-approval.
The next logical question is, "What are the important elements of a technology platform used to support post-approval research?" First, it is the ability to be scalable to adjust to individual study design, sponsor and regulatory requirements, as well as to be flexible to accommodate the changes in the life cycle of the study. The requirements across the various types of post-approval research will vary from large, simple post-approval studies to large, complex safety surveillance studies. Post-approval technology should be able to accommodate all types of study designs. By having the necessary flexibility and scalability, you are able to alleviate unnecessary steps associated with pre-market RCT's technology and databases, which can decrease overall study costs by 10-15%.
Second is the ability to have programmed analytics and associated reports that can be easily modified to address the changing needs throughout the life of the study. It is necessary to have the ability to share information in a discrete manner for each unique stakeholder involved. The power of data and analysis in decision making does not need to be limited to interim analysis or periodic reports to regulatory agencies. Providing the sponsor, physician, patient, CRO, and vendors access to real-time metrics and reports allows all stakeholders to be held accountable and remain invested and engaged in the study; this can produce powerful results.
Teams often find it difficult to truly have a finger on the pulse of all aspects of the study and monitor overall study progress on a continual basis in studies with long-term follow-up. Often times, the studies go on "auto-pilot" during follow-up once enrollment has been met. This is the wrong approach.
Long-term follow-up requires continual evaluation of processes and procedures to protect the sponsor's return on investment (ROI). For example, it would be a mistake to assume that the strategies deployed to successfully capture data and follow-up on patients should remain the same and not be adapted depending on where the project is in its life-cycle.
Due to the long-term nature and associated change in demographics, it is important to be dynamic versus static in evaluations and subsequent approaches. If you truly have an understanding of the metrics and progress, you can easily adapt and implement the necessary changes to keep the success of the study going. You also have the ability to understand when the study is truly at risk and needs intervention to protect the sponsor's investment and commitment to regulatory agencies. But how do you monitor the progress, easily identify trends and shift your approach throughout the course of a post-approval study?
Our experience at Bioclinica Post-Approval Research is that you must have technology specifically designed to address the needs of post-approval research. This technology should be both robust and a fully-integrated platform that acts as a central repository for multiple datasets including eCRF, Patient Reported Outcomes (PRO), safety data, third party vendor data, and more.
Often, in clinical research, we function in discrete silos ensuring that the proper expertise is overseeing individual components and data of a trial. For example, data managers are closely working with sites and integrity of the data collected while the pharmacovigilance group is ensuring that all Adverse Events are reported per regulations, and project managers are watching budget, time and scope.
Teams report updates to the relevant stakeholders, but it is difficult to put all of the "pieces of the puzzle" together to see a holistic view of the study progress and identify if the sponsor will be successful or is at risk. Data and associated analyses should not be done sequentially or in silos in post-approval research, but rather in real-time and simultaneously.
By having all data loaded into a central repository, the stakeholders are able to view discrete datasets as well as have access to analytics across datasets in real-time to survey and monitor progress on a routine basis. Stakeholders will now have access to the holistic picture of the study and the power to implement necessary strategies to meet protocol and regulatory requirements and protect investments.
Considering the orders of magnitude of post-approval studies, it is desirable to have technology drive the study as opposed to many resources drive the study. An equally important component of this integrated technology is the ability to be informed and make data-driven decisions as this allows stakeholders to demonstrate the necessary due diligence to defend the study conduct and results of post-approval research to help protect all parties involved.
In summary, post-approval research is unique and a specialized area of expertise in clinical research. To effectively conduct post-approval research, you truly need to have a different mindset than RCT, and the ability to understand the unique needs of post-approval research.
Integrated post-approval specific technology plays a critical role in managing long-term, large-scale, and costly studies that should be supported by deep domain knowledge and operational expertise. This combination will allow all stakeholders to be successful in the planning, execution, and conduct of their post-approval research whether it is to fulfill regulatory commitments or protect investments in marketed drugs/devices.