Collaboration for rare disease drug discovery research.

Rare disease research has reached a tipping point, with the confluence of scientific and technologic developments that if appropriately harnessed, could lead to key breakthroughs and treatments for this set of devastating disorders. Industry-wide trends have revealed that the traditional drug discovery research and development (R&D) model is no longer viable, and drug companies are evolving their approach. Rather than only pursue blockbuster therapeutics for heterogeneous, common diseases, drug companies have increasingly begun to shift their focus to rare diseases.

The role of brain tumor advocacy groups.

The brain tumor community is a unique, underserved population that has not seen significant improvements in survival over the last four decades. In the search for effective treatments for brain tumors, nonprofit patient advocacy organizations identify and fill the gaps that the for-profit sector and the government have not addressed or cannot address. Although many articles have been written on the roles of patient advocacy groups in general, or in targeted areas such as clinical trial recruitment, none have looked at the brain tumor community specifically.