50th Orphan Medicine Receives Positive Opinion
The European Union's efforts in combating rare diseases reached a new milestone this October, with the 50th positive opinion on an 'orphan' medicine being adopted by the EMEA's Committee for Medicinal Products for Human Use (CHMP).
50th orphan medicine receives positive opinion
The European Union's efforts in combating rare diseases reached a new milestone this October, with the 50th positive opinion on an 'orphan' medicine being adopted by the EMEA's Committee for Medicinal Products for Human Use (CHMP).
To date, a total of 569 medicines have been awarded orphan-designation status by the European Commission, based on recommendations of the
EMEA's Committee for Orphan Medicinal Products (COMP). Of these, 50 have now been successful in obtaining a positive EMEA opinion on marketing authorisation, with many more positive opinions expected over the coming years.
Developed specifically for the diagnosis, treatment or prevention of rare diseases, these medicines have the potential to offer relief to many thousands of European citizens suffering from chronic and often debilitating diseases who would otherwise have few-if any-treatment options available.
A listing of the 50 orphan medicines to have received a positive CHMP opinion is available here:
http://www.emea.europa.eu/pdfs/human/comp/56357508en.pdf
See also: Orphan drugs and rare diseases at a glance
http://www.emea.europa.eu/pdfs/human/comp/29007207en.pdf
EMEA website section: Orphan medicinal products
http://www.emea.europa.eu/htms/human/orphans/intro.htm
Driving Diversity with the Integrated Research Model
October 16th 2024Ashley Moultrie, CCRP, senior director, DEI & community engagement, Javara discusses current trends and challenges with achieving greater diversity in clinical trials, how integrated research organizations are bringing care directly to patients, and more.
AI in Clinical Trials: A Long, But Promising Road Ahead
May 29th 2024Stephen Pyke, chief clinical data and digital officer, Parexel, discusses how AI can be used in clinical trials to streamline operational processes, the importance of collaboration and data sharing in advancing the use of technology, and more.
The Rise of Predictive Engagement Tools in Clinical Trials
November 22nd 2024Patient attrition can be a significant barrier to the success of a randomized controlled trial (RCT). Today, with the help of AI-powered predictive engagement tools, clinical study managers are finding ways to proactively reduce attrition rates in RCTs, and increase the effectiveness of their trial. In this guide, we look at the role AI-powered patient engagement tools play in clinical research, from the problems they’re being used to solve to the areas and indications in which they’re being deployed.
