How to promote the sustainable development of valuable orphan drugs while ensuring access to these medicines for all affected citizens? Access to treatment is not only hampered because of lack of incentives towards research and development, it is also unequal because of differing pricing and reimbursement practices by companies and by national authorities, as well as assessment of added clinical value and awareness and expertise amongst health professionals.
Times of austerity always affect those who are already most in need and vulnerable. For this reason 2010 may become a year with serious setbacks for Rare Disease patients. Already a number of disappointing news confirm this apprehension: Recently we learned that the compromise on the directive on cross boarder health care will not bring [...]
Seriously ill patients who have exhausted all treatment options often seek access to investigational drugs by way of government-sanctioned “expanded access programs”. Recently, the U.S. Food and Drug Administration (FDA) implemented new regulations to clarify and increase patient access to investigational drugs.
However, even with the new rules, this topic is more complex than it may [...]
