Reframing risks in rare diseases: economics of networks, spillovers, and scale

被引:0
|
作者
Runge, Carlisle Ford [1 ]
Campbell, James [2 ]
Runge, Carlisle P. [3 ]
机构
[1] Univ Minnesota, Dept Appl Econ, St Paul, MN 55108 USA
[2] Univ Minnesota, Sch Publ Hlth, Div Hlth Policy & Management, Minneapolis, MN USA
[3] Binera Inc, US Dept HHS, Adm Strateg Preparedness & Response, Biomed Adv Res & Dev Author BARDA, Washington, DC USA
关键词
rare diseases; economics; networks; spillovers; scale; clustering; drug repurposing; basket trials;
D O I
10.3389/fphar.2024.1516725
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Rare diseases affect over three hundred million individuals globally. Investment in research and development remains incommensurate with the challenges rare diseases pose. Further investment in information sharing platforms to promote common and standardized network technologies for rare disease is needed. Rare disease R&D generates information and assets that spill over in other ways, providing benefits that may not be apparent to investors ex ante. Analytical and computational methods recently applied at scale are promising. One important way of achieving efficiencies of scale in R&D is clustering rare diseases into groups with similar traits.
引用
收藏
页数:3
相关论文
共 50 条
  • [31] Disease Management Programs for Adults with Often Diseases and Competence Networks for Children and Adolescents with Rare Diseases
    Goebel, U.
    Gortner, L.
    KLINISCHE PADIATRIE, 2011, 223 (01): : 1 - 3
  • [32] The European Reference Networks for rare and complex diseases respond to the Ukrainian crisis
    Dollfus, Helene
    Arzimanoglou, Alexis
    Scarpa, Maurizio
    LANCET REGIONAL HEALTH-EUROPE, 2022, 19
  • [33] The Tuscany Regional Network for rare diseases: from European Reference Networks' experience to registry based organisation and management model for rare diseases
    Pieroni, Federica
    Marrucci, Sonia
    Di Pietro, Linda
    Berni, Cecilia
    Scaletti, Cristina
    ORPHANET JOURNAL OF RARE DISEASES, 2023, 18 (01)
  • [34] Centers of Competence and Primary Care Networks for Children and Adolescents with rare Diseases
    Waldhauser, Franz
    Deutsch, Johann
    Gobara, Sonia
    PADIATRIE UND PADOLOGIE, 2015, 50 : S25 - S26
  • [35] The Tuscany Regional Network for rare diseases: from European Reference Networks’ experience to registry based organisation and management model for rare diseases
    Federica Pieroni
    Sonia Marrucci
    Linda Di Pietro
    Cecilia Berni
    Cristina Scaletti
    Orphanet Journal of Rare Diseases, 18
  • [36] The risks of therapeutic misconception and individual patient (n=1) "trials" in rare diseases such as Duchenne dystrophy
    Aartsma-Rus, Annemieke
    NEUROMUSCULAR DISORDERS, 2011, 21 (01) : 13 - 15
  • [37] Large-scale computational drug repositioning to find treatments for rare diseases
    Rajiv Gandhi Govindaraj
    Misagh Naderi
    Manali Singha
    Jeffrey Lemoine
    Michal Brylinski
    npj Systems Biology and Applications, 4
  • [38] Large-scale computational drug repositioning to find treatments for rare diseases
    Govindaraj, Rajiv Gandhi
    Naderi, Misagh
    Singha, Manali
    Lemoine, Jeffrey
    Brylinski, Michal
    NPJ SYSTEMS BIOLOGY AND APPLICATIONS, 2018, 4
  • [39] Mechanisms to decrease the diseases spreading on generalized scale-free networks
    Galiceanu, Mircea
    Mendes, Carlos F. O.
    Maciel, Cassio M.
    Beims, Marcus W.
    CHAOS, 2021, 31 (03)
  • [40] The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference Networks
    Teresinha Evangelista
    Victoria Hedley
    Antonio Atalaia
    Matt Johnson
    Stephen Lynn
    Yann Le Cam
    Kate Bushby
    Orphanet Journal of Rare Diseases, 11