Knowledge of a disease’s natural history is essential to building the scientific foundation for an effective clinical development program. That’s especially the case when developing drugs to treat rare diseases, which by their very nature are poorly understood. Natural history studies don’t just track the course of diseases over time, but identify demographic, genetic, environmental,...
Designing and conducting psoriasis clinical trials is tricky. After all, psoriasis is a highly variable group of conditions that produce a range of debilitating and often subjective symptoms. So how do researchers assess efficacy in novel psoriasis treatments? Read on for the answer. Considerations for Psoriasis Trials In clinical practice, broad global assessments are used...
When it comes to analgesic drug development, it’s important to optimize design of early phase trials. The main objectives of studies at this stage are to: Explore a range of doses Obtain data on onset, peak effect, and duration Assess relative efficacy in comparison to established analgesics To accurately evaluate their product, sponsors need to...
Patient registry and natural history studies are often thought of as interchangeable, but there are major differences in these approaches to clinical drug development. Understanding the role of each is important, as well as understanding the distinctions on how these study designs are used in pursuing treatments for rare diseases. Patient Registries A patient registry...
Psoriasis is caused by dysregulated interactions of the innate and adaptive immune system in skin epithelium and connective tissue, including the activation and proliferation of T helper cells. As an autoimmune disorder causing widespread inflammation, it is unsurprising that psoriasis is associated with a number of non-dermatological conditions. Continued psoriasis research may give insight into the etiology...
Psoriasis is a dermatological disorder that is often paired with underlying systemic disease and multiple comorbidities. While a common condition, its wide range of severity and causes, along with several clinical subtypes, make it challenging to treat. Global Impact of Psoriasis The World Health Organization estimates at least 100 million individuals globally live with psoriasis,...
The effects of rigorous patient advocacy are easily seen today throughout the pharmaceutical industry. Patients who suffer the debilitating effects of rare diseases are banding together to be heard in an attempt to have their conditions studied to further the development of treatments and cures. However, the influence of patient advocacy groups has not always...
Translational research has the potential to help all patients, but the principles behind bench-to-bedside research hold special promise for patients with rare diseases — many of which have no standard treatments. Here’s how changes to the way rare oncology studies are performed are getting treatments to the patients who need them at a faster rate. What Makes...
In December 2016, the United States passed an important piece of regulatory legislation called the 21st Century Cures Act. These FDA regulations cover multiple types of clinical research but include some specific provisions for medical device development. In contrast to the regulatory reform seen in the EU — which includes a much-needed increased emphasis on...
Good clinical practice (GCP) guidelines govern important aspects of clinical trials to ensure patient safety and scientific integrity. “GCP” is a quality standard which refers to the document put forth by the International Council on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). The ICH is a group made up of...