Validating Configurable Interactive Response Technology Solutions for Clinical Studies

There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies, especially for single gene mutation disorders. The US Food and Drug Administration (FDA) has more than 700 active Investigational New Drug Applications (INDs) for gene and cell therapies and in 2017, the FDA approved two cell-based gene therapies and it is anticipated that gene therapy will become a mainstay treatment for many rare diseases.

The science of medicine has allowed us to make incredible advances in diagnosing and treating diseases. But the complexity of human biology is staggering. Every person is unique and in many ways, so are diseases. Yet the digital revolution in healthcare provides new ways to both collect high-quality data from each patient and connect it to data from large pools of other patients for analysis. This enables us to arrive at a deeper understanding of how to treat an individual. Only then can we see what distinguishes each of us as individuals, and translate that into personalized and thus improved care for every person. Real-world evidence, molecular information generated from next-generation sequencing, data from wearable devices and mobile apps and novel clinical trials are transforming the future of care.

Nanomaterial continues to be a hot topic among regulatory agencies globally. The recent ban on TiO2 (E171) as a food additive in Europe has highlighted the need to have a comprehensive understanding of the global regulatory environment related to the presence of nanoparticles in food additives and excipients.

Enabling formulations becomes more and more necessary to obtain adequate exposure of APIs during (pre)-clinical research. Currently, most registered formulations to improve bioavailability are based on lipidic systems, which make use of intrinsic digestion processes within the GI tract, facilitating the absorption of APIs. Aside from a general absorption mechanism, lipidic systems can also promote lymphatic transport of poorly soluble drugs, such as hormones. In addition, many different indications, such as HIV therapy, oncology, immunosuppressant and even arising therapies related to medicinal cannabis (e.g. CBD) can benefit from lipid formulations. These formulations create multiple possibilities for oral administration because they can be manufactured as solutions, suspensions, emulsions, and self-(micro)emulsifying systems.