one of the largest HMOs in Israel Advocating for legislation Dunkle United States Rare diseases in general Patient NORD representatives testify before The passing of the Orphan Drug Act was due • • (2010) organizations Congressional committees on a regular basis in part to the advocacy efforts of the coalition [47] efforts of NORD have focused on: rare that would eventual become NORD •Advocacy disease and orphan drug legislation; ensuring adequate funding for the FDA, the NIH, and the SSA; extended patent period on orphan products; the removal of lifetime caps from health insurance policies; and for legislation to improve patient access to clinical trials Lasker United States PBC Patient The PBCers Organization advocates for those Not specified • (2005) organization with PBC [49] Landy Not specified Rare diseases in general Patient Patient organizations lobby policy makers Not specified • (2012) organizations [55] Awareness campaigns Black United States SMEI Patient The IDEA League participates in national Not specified • (2011) organizations awareness campaigns [22] Dunkle United States Rare diseases in general Patient Patient organizations created Rare Disease Day Not specified • (2010) organizations [47] Conferences and Workshops Participating in conferences and workshops Molster Australia Rare diseases in general Patients, Patients, families, and patient organizations Participants identified a range of key issues for • • (2012) families and participated in workshops at the Australian Rare consideration, with associated goals and actions [95] patient Diseases Symposium discussing the issues, goals, There was agreement that the • organizations and actions relevant to the development of a recommendations drawn out from the outcomes national plan of the workshops should be used to inform and frame the development of a national plan Hosting conferences and workshops De Blieck United States JNCL Patient The BDRSA hosts an Annual Conference The conference provides a venue for the • • (2013) organizations URBC to enroll families in the contact registry, [35] the natural history and disease-specific rating scale study, and/or neuropsychological investigations Lasker United States PBC Patient The PBCers Organization hosts conferences with Not specified •
one of the largest HMOs in Israel Advocating for legislation Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD representatives testify before Congressional committees on a regular basis •Advocacy efforts of NORD have focused on: rare disease and orphan drug legislation; ensuring adequate funding for the FDA, the NIH, and the SSA; extended patent period on orphan products; the removal of lifetime caps from health insurance policies; and for legislation to improve patient access to clinical trials • The passing of the Orphan Drug Act was due in part to the advocacy efforts of the coalition that would eventual become NORD Lasker (2005) [49] United States PBC Patient organization • The PBCers Organization advocates for those with PBC Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations lobby policy makers Not specified Awareness campaigns Black (2011) [22] United States SMEI Patient organizations • The IDEA League participates in national awareness campaigns Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • Patient organizations created Rare Disease Day Not specified Conferences and Workshops Participating in conferences and workshops Molster (2012) [95] Australia Rare diseases in general Patients, families and patient organizations • Patients, families, and patient organizations participated in workshops at the Australian Rare Diseases Symposium discussing the issues, goals, and actions relevant to the development of a national plan • Participants identified a range of key issues for consideration, with associated goals and actions • There was agreement that the recommendations drawn out from the outcomes of the workshops should be used to inform and frame the development of a national plan Hosting conferences and workshops De Blieck (2013) [35] United States JNCL Patient organizations • The BDRSA hosts an Annual Conference • The conference provides a venue for the URBC to enroll families in the contact registry, the natural history and disease-specific rating scale study, and/or neuropsychological investigations Lasker United States PBC Patient • The PBCers Organization hosts conferences with Not specified
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