The marketplace for orphan drugs is growing, and changes in the regulatory landscape are providing favorable conditions for collaboration in the area of drug development in rare diseases. Understanding the regulatory and operational nuances of orphan drug development can help sponsors position their promising compounds for clinical and commercial success. Today more than ever, researchers...
Inadequate funding and burdensome regulatory barriers in the United States are two challenges that must be tackled if clinical researchers are to answer important public health questions about the pros and cons of cannabis and cannabinoid use, one of the world’s leading scientific bodies has concluded. These are among nearly 100 conclusions and recommendations reached...
According to a Council of Foreign Relations report, ensuring a safe and secure healthcare marketplace that can provide innovative therapies is no longer an undertaking for a single nation. The report concludes there is no way a single government regulator, including the gold-standard U.S. Food and Drug Administration (FDA), can support innovation and regulate products...
On June 25, 2018, the U.S. Food and Drug Administration (FDA) approved Epidiolex (cannabidiol, or CBD) oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. In addition to ushering in the first-ever approved drug for Dravet syndrome, the greenlighting of Epidiolex marks the...
In May 2012, the leaders of 30 medicine regulatory bodies from around the world gathered at the 65th World Health Assembly in Geneva, collectively puzzling over how to improve cooperation among them. Their answer came later that year: form the International Coalition of Medicines Regulatory Authorities (ICMRA), a voluntary group of Heads of Agency (HoA)...
DURHAM, N.C., JULY 30, 2018 — Premier Research, a global provider of clinical development services to biopharmaceutical product innovators, has acquired Regulatory Professionals, Inc. (RPI), a California-based company that provides regulatory expertise and solutions to biotechnology, pharmaceutical, and medical device companies. This partnership will form a new Regulatory Services Division of Premier Research, and Donna...
In a clinical trial landscape where global, multi-center studies have become increasingly common, trial monitoring has become more complex. Regulations require that sponsors of medical device studies perform site monitoring to ensure that investigators are compliant with federal regulations, sponsor agreements, the investigational plan and the requirements set forth in the Investigational Review Board (IRB)...
While clinical investigations of medical devices and investigational drugs have their differences, what they do have in common is the goal of safeguarding the rights and welfare of study participants while bringing safe and effective products to market as quickly and efficiently as possible. Not all medical devices require a clinical trial, depending on their...
To achieve operational excellence in gene therapy trials, sponsors must understand – and overcome – obstacles ranging from start-up regulations and site selection to patient recruitment and retention. Gene therapy holds promise for treating a wide range of diseases, from cancer and diabetes to rare genetic disorders. It has also sparked great interest because it...
Like any new medical product, gene therapies must undergo rigorous approval processes. However, there are a number of additional regulations for gene therapies that don’t apply to conventional pharmaceuticals. This is especially true within the European Union. (It’s important to note that while most gene therapy studies are based in the U.S. and Europe — which...