Tagged with Orphan Drugs,

Rare Diseases In the run-up to Rare Disease Day 2019, David H. Crean, PhD, Managing Director for Objective Capital Partners, reviews the investment and deal activity for orphan drugs in 2018 and delves into future forecasts for the rare disease space.   With drugs currently available for only about 5% of rare diseases,…

China It has been announced by China’s State Council that as of March 1st 2019, the government will reduce the VAT on a “first batch” of 21 rare disease drugs and four active pharmaceutical ingredients (API’s) by more than 80% in a bid to get new treatments to market faster. Historically, China has…

USA 80 orphan drugs (those drugs intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions) were approved in the USA in 2017, a record year and more than double the 40 approved in 2016.   Orphan drug spending grew from 5 to 10%…

Europe 125 medicines for rare diseases were approved by the EMA between 2004 and 2014, of which 71 were granted orphan drug status. This chart shows the availability of 112 of these drugs across European countries. The largest number of medicines for rare diseases were in continuous use in Germany and…

Korea Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment of acute myeloid leukaemia (AML).   The endorsement of FDA’s Orphan Drug Designation (ODD) — invented to smooth the development of therapies for life-threatening…

Innovation Patient groups oftentimes serve as the authority on a specific disease when it comes to advancing research.   “Without patient perspective, there is no good medical product development,” Rachel Sherman, the FDA’s principal deputy commissioner proclaimed during her keynote at last week’s NORD’s Rare Disease & Orphan Products Breakthrough Summit.…

Pharma Ad Schuurman of the National Health Care Institute of the Netherlands (“Zorginstituut”) explains the main missions and mandates of this organization with regards to the basic insurance package covering all Dutch citizens, as well as its perception of pay-for-performance models, pan-European joint-reimbursement initiatives, and Zorginstituut’s policies on orphan drugs reimbursement and quality…

orphan drugs Gabriela Pittis discusses the Argentinian healthcare system’s ability to diagnose, treat, and regulate rare diseases, and the country’s progress in further developing what is already the region’s most comprehensive system in this regard.   Is the Argentinian system able to reliably diagnose and treat rare diseases? “Rare diseases” is a…

Government Regulation As the only country in the G8 without an orphan drugs act, the President of Médunik, a company focused on bringing orphan drugs into the country, talks about the desperate need for better legislation in this area for the benefit of patients, and the misconceptions of treating Canada as a…