Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review

[1] Douglas C(2015)Why orphan drug coverage reimbursement decision-making needs patient and public involvement Health Policy 119 588-596

[2] Wilcox E(2014)An orphan drug framework (ODF) for Canada J Popul Ther Clin Pharmacol 21 e42-e46

[3] Burgess M(2013)Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries Orphanet J Rare Dis 8 198-664

[4] Lynd L(2012)Orphan drug development: an economically viable strategy for biopharma R&D Drug Discov Today 17 660-476

[5] Lee D(2011)Accelerating access to treatments for rare diseases Nat Rev Drug Discov 10 475-39

[6] Wong B(2014)Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a rare opportunity? Patient 8 29-32

[7] Morel T(2005)Scoping studies: towards a methodological framework Int J Soc Res Methodol 8 19-243

[8] Arickx F(2010)Scoping studies: advancing the methodology Implement Sci 5 69-104

[9] Befrits G(2012)Involvement of patient organisations in research and development of orphan drugs for rare diseases in Europe Mol Syndromol 3 237-1021

[10] Siviero P(2011)The impact of parent advocacy groups, the internet, and social networking on rare diseases: the IDEA league and IDEA league United Kingdom example Epilepsia 52 102-105

[1] Douglas C(2015)Why orphan drug coverage reimbursement decision-making needs patient and public involvement Health Policy 119 588-596

[2] Wilcox E(2014)An orphan drug framework (ODF) for Canada J Popul Ther Clin Pharmacol 21 e42-e46

[3] Burgess M(2013)Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries Orphanet J Rare Dis 8 198-664

[4] Lynd L(2012)Orphan drug development: an economically viable strategy for biopharma R&D Drug Discov Today 17 660-476

[5] Lee D(2011)Accelerating access to treatments for rare diseases Nat Rev Drug Discov 10 475-39

[6] Wong B(2014)Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a rare opportunity? Patient 8 29-32

[7] Morel T(2005)Scoping studies: towards a methodological framework Int J Soc Res Methodol 8 19-243

[8] Arickx F(2010)Scoping studies: advancing the methodology Implement Sci 5 69-104

[9] Befrits G(2012)Involvement of patient organisations in research and development of orphan drugs for rare diseases in Europe Mol Syndromol 3 237-1021

[10] Siviero P(2011)The impact of parent advocacy groups, the internet, and social networking on rare diseases: the IDEA league and IDEA league United Kingdom example Epilepsia 52 102-105