Ferguson United States Chromosome 18 deletion Patients and The mother of a patient developed the Not specified • (2002) families Chromosome 18 Registry and Research Society, [19] uniting three independent organizations Providing guidance on establishing patient organizations Mai United States LFS Patient At a workshop at the National Institutes of Not specified • (2012) organizations Health, patient organizations shared their [57] perspectives and advice on forming support and advocacy groups Establishing international patient organization alliances Woodward International aHUS Patient Representatives from 6 European countries • (2016) organizations (Belgium, France, Italy, Russia, Spain, and the [156] UK) met and agreed to collaborate as the aHUS Alliance. Since then, another 6 organizations have become affiliated with the organization (Australia, Canada, Germany, India, Netherlands, and USA) Boon Netherlands NMDs Patient The VSN helped to establish the International Not specified • (2010) organizations Pompe Association, an alliance of patient [46] organizations United States VHL Patient The VHLA establishes similar alliances around Not specified • organizations the world Alliance (VHLA) (2014) [40] Fundraising to support further patient organization growth United States VHL Patient The majority of funds supporting the VHLA are Not specified • organizations acquired through fundraising initiatives Alliance (VHLA) (2014) [40] Regulatory decision-making Input into the regulatory decision-making process Genetic United Kingdom Rare diseases in general Patients Patient input on acceptable risk should be Not applicable • Alliance UK imbedded into regulatory decision-making (2014) [97] Proposed Pharma Canada Rare diseases in general Patients and As part of the new Orphan Drug Framework in This is a pilot project that will be used by • • Letter families Canada, patients provide input on how the disease Health Canada to assess and refine its approach
Ferguson (2002) [19] United States Chromosome 18 deletion Patients and families • The mother of a patient developed the Chromosome 18 Registry and Research Society, uniting three independent organizations Not specified Providing guidance on establishing patient organizations Mai (2012) [57] United States LFS Patient organizations • At a workshop at the National Institutes of Health, patient organizations shared their perspectives and advice on forming support and advocacy groups Not specified Establishing international patient organization alliances Woodward (2016) [156] International aHUS Patient organizations • Representatives from 6 European countries (Belgium, France, Italy, Russia, Spain, and the UK) met and agreed to collaborate as the aHUS Alliance. Since then, another 6 organizations have become affiliated with the organization (Australia, Canada, Germany, India, Netherlands, and USA) Boon (2010) [46] Netherlands NMDs Patient organizations • The VSN helped to establish the International Pompe Association, an alliance of patient organizations Not specified Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The VHLA establishes similar alliances around the world Not specified Fundraising to support further patient organization growth Alliance (VHLA) (2014) [40] United States VHL Patient organizations • The majority of funds supporting the VHLA are acquired through fundraising initiatives Not specified Regulatory decision-making Input into the regulatory decision-making process Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patient input on acceptable risk should be imbedded into regulatory decision-making Not applicable Pharma Letter Canada Rare diseases in general Patients and families • As part of the new Orphan Drug Framework in Canada, patients provide input on how the disease • This is a pilot project that will be used by Health Canada to assess and refine its approach
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