Recommendations for the use of clinical outcome assessments in rare disease drug development

Olalekan Lee Aiyegbusi, Noleen K. McCorry, Melanie J. Calvert, John Devin Peipert, Samantha Cruz Rivera, Robert Muni-Lofra, Solomon Alexis, Fez Awan, Tom Bailey, Lucinda Billingham, Rosaline Callaghan, Christine Collins, Brooke M. Currie, Mel Dixon, Melanie Duddridge, James Ennis, Linda Fred, Sarah Greenwell, Christian Griffiths, Asha Hareendran, Onyekachukwu A. Illoh, Kerry Leeson-Beevers, Krishna Letchemanan, Steven P. Lister, Christopher McCabe, Molly McFatrich, Amy Jayne McKnight, Fiona McLaughlin, Ramona Moldovan, Lindsey T. Murray, Daniel J. O'Connor, Hafiz Oko-osi, Louise Oni, Kate M. Pritchard, Khadija Rerhou Rantell, Volker Straub, Timothy G. Barrett, David Jones, Michael Clarke, Hayley Comins, Simon Gates, Amber R. Hart, Victoria Hedley, Blánaid Hicks, Martin R. Higgs, Matthew Hosken, Ameeta Retzer, Sarah Scullion, Laura A. Wyatt, Steven J. Blackburn, Catherine Turner

Open source

DOI
10.1016/j.eclinm.2026.104073
Published
2026-08
Container
eClinicalMedicine
Publisher
Elsevier BV
Open access
unknown

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BibTeX

@article{allodium:10.1016/j.eclinm.2026.104073,
  title = {Recommendations for the use of clinical outcome assessments in rare disease drug development},
  author = {Olalekan Lee Aiyegbusi and Noleen K. McCorry and Melanie J. Calvert and John Devin Peipert and Samantha Cruz Rivera and Robert Muni-Lofra and Solomon Alexis and Fez Awan and Tom Bailey and Lucinda Billingham and Rosaline Callaghan and Christine Collins and Brooke M. Currie and Mel Dixon and Melanie Duddridge and James Ennis and Linda Fred and Sarah Greenwell and Christian Griffiths and Asha Hareendran and Onyekachukwu A. Illoh and Kerry Leeson-Beevers and Krishna Letchemanan and Steven P. Lister and Christopher McCabe and Molly McFatrich and Amy Jayne McKnight and Fiona McLaughlin and Ramona Moldovan and Lindsey T. Murray and Daniel J. O'Connor and Hafiz Oko-osi and Louise Oni and Kate M. Pritchard and Khadija Rerhou Rantell and Volker Straub and Timothy G. Barrett and David Jones and Michael Clarke and Hayley Comins and Simon Gates and Amber R. Hart and Victoria Hedley and Blánaid Hicks and Martin R. Higgs and Matthew Hosken and Ameeta Retzer and Sarah Scullion and Laura A. Wyatt and Steven J. Blackburn and Catherine Turner},
  year = {2026},
  journal = {eClinicalMedicine},
  doi = {10.1016/j.eclinm.2026.104073},
  url = {https://doi.org/10.1016/j.eclinm.2026.104073}
}

RIS

TY  - JOUR
TI  - Recommendations for the use of clinical outcome assessments in rare disease drug development
AU  - Olalekan Lee Aiyegbusi
AU  - Noleen K. McCorry
AU  - Melanie J. Calvert
AU  - John Devin Peipert
AU  - Samantha Cruz Rivera
AU  - Robert Muni-Lofra
AU  - Solomon Alexis
AU  - Fez Awan
AU  - Tom Bailey
AU  - Lucinda Billingham
AU  - Rosaline Callaghan
AU  - Christine Collins
AU  - Brooke M. Currie
AU  - Mel Dixon
AU  - Melanie Duddridge
AU  - James Ennis
AU  - Linda Fred
AU  - Sarah Greenwell
AU  - Christian Griffiths
AU  - Asha Hareendran
AU  - Onyekachukwu A. Illoh
AU  - Kerry Leeson-Beevers
AU  - Krishna Letchemanan
AU  - Steven P. Lister
AU  - Christopher McCabe
AU  - Molly McFatrich
AU  - Amy Jayne McKnight
AU  - Fiona McLaughlin
AU  - Ramona Moldovan
AU  - Lindsey T. Murray
AU  - Daniel J. O'Connor
AU  - Hafiz Oko-osi
AU  - Louise Oni
AU  - Kate M. Pritchard
AU  - Khadija Rerhou Rantell
AU  - Volker Straub
AU  - Timothy G. Barrett
AU  - David Jones
AU  - Michael Clarke
AU  - Hayley Comins
AU  - Simon Gates
AU  - Amber R. Hart
AU  - Victoria Hedley
AU  - Blánaid Hicks
AU  - Martin R. Higgs
AU  - Matthew Hosken
AU  - Ameeta Retzer
AU  - Sarah Scullion
AU  - Laura A. Wyatt
AU  - Steven J. Blackburn
AU  - Catherine Turner
PY  - 2026
JO  - eClinicalMedicine
DO  - 10.1016/j.eclinm.2026.104073
UR  - https://doi.org/10.1016/j.eclinm.2026.104073
ER  - 

APA

Aiyegbusi, O. L., McCorry, N. K., Calvert, M. J., Peipert, J. D., Rivera, S. C., Muni-Lofra, R., Alexis, S., Awan, F., Bailey, T., Billingham, L., Callaghan, R., Collins, C., Currie, B. M., Dixon, M., Duddridge, M., Ennis, J., Fred, L., Greenwell, S., Griffiths, C., Hareendran, A., Illoh, O. A., Leeson-Beevers, K., Letchemanan, K., Lister, S. P., McCabe, C., McFatrich, M., McKnight, A. J., McLaughlin, F., Moldovan, R., Murray, L. T., O'Connor, D. J., Oko-osi, H., Oni, L., Pritchard, K. M., Rantell, K. R., Straub, V., Barrett, T. G., Jones, D., Clarke, M., Comins, H., Gates, S., Hart, A. R., Hedley, V., Hicks, B., Higgs, M. R., Hosken, M., Retzer, A., Scullion, S., Wyatt, L. A., Blackburn, S. J., & Turner, C. (2026). Recommendations for the use of clinical outcome assessments in rare disease drug development. eClinicalMedicine. https://doi.org/10.1016/j.eclinm.2026.104073

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