Trend of clinical trials of new drugs for rare diseases in China in recent 10 years

[1] Dawkins HJS(2018)Progress in Rare Diseases Research 2010–2016: an IRDiRC perspective Clin Transl Sci 11 11-20

[2] Chang X(2022)A survey of registered pharmacological clinical trials on rare neurological diseases in children in 2010–2020 Front Pediatr 10 963601-43

[3] Burton A(2021)Drug Discovery and Development in Rare Diseases: taking a closer look at the Tafamidis Story Drug Des Devel Ther 15 1225-4

[4] Mellerio JE(2022)The challenges of clinical trials in rare diseases Br J Dermatol 187 453-92

[5] Ferreira CR(2019)The burden of rare diseases Am J Med Genet A 179 885-14

[6] Stoller JK(2018)The challenge of Rare Diseases Chest 153 1309-111

[7] Tambuyzer E(2020)Therapies for rare diseases: therapeutic modalities, progress and challenges ahead Nat Rev Drug Discovery 19 93-2

[8] Rudebeck M(2021)Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium Orphanet J Rare Dis 16 510-10

[9] Musters A(2020)Room for improvement in clinical trials for rare diseases Nat Rev Rheumatol 16 131-208

[10] Tas SW(2022)Trends in innovative drug development in China Nat Rev Drug Discov 21 709-40

[1] Dawkins HJS(2018)Progress in Rare Diseases Research 2010–2016: an IRDiRC perspective Clin Transl Sci 11 11-20

[2] Chang X(2022)A survey of registered pharmacological clinical trials on rare neurological diseases in children in 2010–2020 Front Pediatr 10 963601-43

[3] Burton A(2021)Drug Discovery and Development in Rare Diseases: taking a closer look at the Tafamidis Story Drug Des Devel Ther 15 1225-4

[4] Mellerio JE(2022)The challenges of clinical trials in rare diseases Br J Dermatol 187 453-92

[5] Ferreira CR(2019)The burden of rare diseases Am J Med Genet A 179 885-14

[6] Stoller JK(2018)The challenge of Rare Diseases Chest 153 1309-111

[7] Tambuyzer E(2020)Therapies for rare diseases: therapeutic modalities, progress and challenges ahead Nat Rev Drug Discovery 19 93-2

[8] Rudebeck M(2021)Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium Orphanet J Rare Dis 16 510-10

[9] Musters A(2020)Room for improvement in clinical trials for rare diseases Nat Rev Rheumatol 16 131-208

[10] Tas SW(2022)Trends in innovative drug development in China Nat Rev Drug Discov 21 709-40