distribution of this data. Dunkle United States Rare diseases in general Patient NORD collaborates with industry, allowing Not specified • (2010) organizations companies to participate on its Corporate Council [47] Dunkle United States Rare diseases in general Patient Medical Advisory Committee allows Not specified • NORD’s (2010) organizations for the incorporation of the view of health care [47] professionals into their advocacy process Dunkle United States Rare diseases in general Patient NORD and EURODIS signed a Memorandum of Not specified • (2010) organizations Understanding and are collaborating on several [47] initiatives aiming to increase global awareness, promote R&D on new treatments, and advocating for more compassionate public policies Ferguson United States GIST Patient The LRG established a Science Team, which Not specified • (2002) organizations reviews medical literature and communicates with [19] leading GIST specialists Groft United States Rare diseases in general Patient Patient organizations establish global Not specified • (2013) organizations partnerships as part of the RDCRN [23] United States VHL Patient The VHLA establishes collaborations with The International VHL Alliance allows the • • organizations various organizations (e.g., Genetic Alliance, VHLA to connect with 90% of all diagnosed Alliance National Organization for Rare Disorders) and acts VHL patients around the world (VHLA) as the home for the International VHL Alliance, an (2014) international network of VHL Affiliates [40] Education Informational resources Patient International Rare diseases in general Patient The Patient Partner project, a collaboration Not specified • Partner organizations involving patient organization alliances (e.g., [20] Genetic Alliance UK), established a set of guidelines for the involvement of patient organizations and patient representatives in clinical research The guidelines describe clinical trials and discuss • how patients and patient organizations can get involved, who they should become involved with, and ethical considerations for partnerships Scarpa Europe MPS II Patient Patient organizations provide verbal and written Not specified • (2011) organizations information on issues like education, grants, [45] equipment, care plans, independent living, pre-and
distribution of this data. Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD collaborates with industry, allowing companies to participate on its Corporate Council Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD’s Medical Advisory Committee allows for the incorporation of the view of health care professionals into their advocacy process Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD and EURODIS signed a Memorandum of Understanding and are collaborating on several initiatives aiming to increase global awareness, promote R&D on new treatments, and advocating for more compassionate public policies Not specified Ferguson (2002) [19] United States GIST Patient organizations • The LRG established a Science Team, which reviews medical literature and communicates with leading GIST specialists Not specified Groft (2013) [23] United States Rare diseases in general Patient organizations • Patient organizations establish global partnerships as part of the RDCRN Not specified Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The VHLA establishes collaborations with various organizations (e.g., Genetic Alliance, National Organization for Rare Disorders) and acts as the home for the International VHL Alliance, an international network of VHL Affiliates • The International VHL Alliance allows the VHLA to connect with 90% of all diagnosed VHL patients around the world Education Informational resources Patient Partner [20] International Rare diseases in general Patient organizations • The Patient Partner project, a collaboration involving patient organization alliances (e.g., Genetic Alliance UK), established a set of guidelines for the involvement of patient organizations and patient representatives in clinical research • The guidelines describe clinical trials and discuss how patients and patient organizations can get involved, who they should become involved with, and ethical considerations for partnerships Not specified Scarpa (2011) [45] Europe MPS II Patient organizations • Patient organizations provide verbal and written information on issues like education, grants, equipment, care plans, independent living, pre-and Not specified