There may be as many as 7,000 rare diseases. The European Union defines a disease as rare when it affects less than one in 2,000 people. According to the Orphan Drug Act, a rare disease affects less than 200,000 people in the United States. Support from FDA, EMA and other regulatory agencies, combined with technological and scientific advances, have made it more feasible for pharmaceutical companies to give dedicated attention to orphan drug development. In doing so, they're developing medications for rare diseases that often have no cure and no available treatment. To ensure novel therapies advance from the lab to regulatory approval, a blockbuster-drug approach won't work. Managing rare disease studies to completion requires innovative thinking in nearly every phase of program development. Those willing to become trailblazers have the highest odds of success.
To execute precision medicine clinical trials, sponsors have adopted a range of new approaches, especially in early-stage studies. Many of these models allow sponsors to recruit more patients and bring drugs to market faster— goals drug developers in all therapeutic areas work toward.
As clinical trials become more complex and costly, the need for skilled project managers to oversee them is more important than ever. Project management methodologies drive success for enterprise organizations in all sectors. These same principles can help pharmaceutical and biotech companies run more efficient, successful clinical trials.
Gaps in dermatological research present potentially lucrative market opportunities for pharmaceutical companies. Patients that suffer from skin conditions and diseases also benefit. Effective treatments relieve irritating, chronic, and often painful symptoms, which improves their quality of life and lifts the psychological and social burden of a visible dermatological issue.
3 Measures to Overcome Operational Failures Every year, tens of thousands of clinical trials are initiated. Every year, thousands of those trials falter, and, if corrective [...]
Advanced Analytics for the Monitoring of Clinical Trials What if there was a way to harness all this data we’re collecting and turn it into a [...]
The CDISC set of standards revolutionized data management in clinical trials by introducing a standardized model for data interoperability. The standard has been shown to decrease study data resource requirements by 60% overall, and 70-90% in start-up stages when implemented early. This results in dramatic decreases in cost, but also increases data accuracy and time to lock, to say nothing of the tremendous benefits having a standard dataset available in the course of the study. Download our whitepaper and find out how to make early CDISC work for your trial.
Operating a Pharmacovigilance Group in a Multicultural Global Environment The way an individual interprets language is influenced by many factors, including culture, background, education, training, and [...]
Optimizing Ophthalmic CE ANDA Trials Clinical Endpoint ANDA Program Optimization White Paper Series Download White Paper
Navigating through the Clinical Trial Authorization Process in Russia Clinical Endpoint ANDA Program Optimization White Paper Series Download White Paper