Dunkle United States Rare diseases in general Patient NORD administers Patient Assistance Programs Not specified • (2010) organizations for uninsured and underinsured patients [47] Dunkle United States Rare diseases in general Patient NORD provides drugs at no charge to eligible Not specified • (2010) organizations patients through its Medication Assistance [47] Programs Dunkle United States Rare diseases in general Patient NORD provides premium and copayment funds Not specified • (2010) organizations for patients with certain disorders, who cannot [47] afford out-of-pocket costs associated with their plans Landy Not specified Rare diseases in general Patient Patient organizations provide financial assistance Not specified • (2012) organizations to patients [55] Support for patients participating in clinical trials Bendixen United States DMD Families Families who have participated in a trial should Not applicable • (2016) provide peer support for those who are just [98] entering trials Proposed Dunkle United States Rare diseases in general Patient Patient organizations provide travel and Not specified • (2010) organizations temporary housing assistance to patients and [47] families who must travel to participate in clinical trials EURODIS Europe Rare diseases in general Patient Patient organizations support patients during a Not specified • (2011) organizations study to reduce the rate of drop-outs and [21] incomplete files Clinical care support for patients Genetic United Kingdom Rare diseases in general Patients Patients, in partnership with their physicians, Not applicable • Alliance UK should decide whether the benefits of a new drug (2014) outweigh its risks. [97] Proposed Pattacini Italy Hemophilia Patients Patients access part of their clinical records Data is extracted in an anonymous format and • • (2009) through a web-based electronic published on a website ‘xl’Emofilia, [50] in order to consult their data Some data is also processed and sent to the outpatients’ record, • and record bleeding events and home infusions Italian Registry of Hemophilia and Allied Patient-entered data is validated by doctors at a Disorders • treating Hemophilia Centre The authors state that this system has • Patients receive training to use these records improved the management of data, facilitated • work, and improved the quality of care for patients
Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD administers Patient Assistance Programs for uninsured and underinsured patients Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides drugs at no charge to eligible patients through its Medication Assistance Programs Not specified Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides premium and copayment funds for patients with certain disorders, who cannot afford out-of-pocket costs associated with their plans Not specified Landy (2012) [55] Not specified Rare diseases in general Patient organizations • Patient organizations provide financial assistance to patients Not specified Support for patients participating in clinical trials Bendixen (2016) [98] Proposed United States DMD Families • Families who have participated in a trial should provide peer support for those who are just entering trials Not applicable Dunkle (2010) [47] United States Rare diseases in general Patient organizations • Patient organizations provide travel and temporary housing assistance to patients and families who must travel to participate in clinical trials Not specified EURODIS (2011) [21] Europe Rare diseases in general Patient organizations • Patient organizations support patients during a study to reduce the rate of drop-outs and incomplete files Not specified Clinical care support for patients Genetic Alliance UK (2014) [97] Proposed United Kingdom Rare diseases in general Patients • Patients, in partnership with their physicians, should decide whether the benefits of a new drug outweigh its risks. Not applicable Pattacini (2009) [50] Italy Hemophilia Patients • Patients access part of their clinical records through ‘xl’Emofilia, a web-based electronic outpatients’ record, in order to consult their data and record bleeding events and home infusions • Patient-entered data is validated by doctors at a treating Hemophilia Centre • Patients receive training to use these records • Data is extracted in an anonymous format and published on a website • Some data is also processed and sent to the Italian Registry of Hemophilia and Allied Disorders • The authors state that this system has improved the management of data, facilitated work, and improved the quality of care for patients
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