Cargando…
Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium
New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in d...
Autores principales: | , , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8670024/ https://www.ncbi.nlm.nih.gov/pubmed/34906169 http://dx.doi.org/10.1186/s13023-021-02137-0 |
_version_ | 1784614894865219584 |
---|---|
author | Rudebeck, Mattias Scott, Ciarán Rhodes, Nicholas P. van Kan, Christa Olsson, Birgitta Al-sbou, Mohammed Hall, Anthony K. Sireau, Nicolas Ranganath, Lakshminarayan R. |
author_facet | Rudebeck, Mattias Scott, Ciarán Rhodes, Nicholas P. van Kan, Christa Olsson, Birgitta Al-sbou, Mohammed Hall, Anthony K. Sireau, Nicolas Ranganath, Lakshminarayan R. |
author_sort | Rudebeck, Mattias |
collection | PubMed |
description | New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. Our unique consortium model incorporated several of the identified developments under one project, DevelopAKUre, truly bringing together academia, industry and patient organisations in clinical drug development. We found that the equal partnership between all parties in our consortium was a key success factor creating a momentum based on a strong organisational culture where all partners had high engagement and taking ownership of the entire programme. With an agreed mutual objective, this provided synergies through connecting the strengths of the individual parties. Another key success factor was the central role of the patient organisation within the management team, and their unique study participants’ advocacy role securing the understanding and meeting the needs of the clinical study participants in real-time. This resulted in an accelerated enrolment into the clinical studies with a high retention rate allowing for delivery of the programme with significantly improved timelines. Our project was partly funded through an external EU research grant, enabling our model with equal partnership. Further attention within the community should be given to establishing a functional framework where sustainable funding and risk sharing between private and public organisations allow for our model to be replicated. |
format | Online Article Text |
id | pubmed-8670024 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-86700242021-12-15 Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium Rudebeck, Mattias Scott, Ciarán Rhodes, Nicholas P. van Kan, Christa Olsson, Birgitta Al-sbou, Mohammed Hall, Anthony K. Sireau, Nicolas Ranganath, Lakshminarayan R. Orphanet J Rare Dis Position Statement New opportunities have arisen for development of therapies for rare diseases with the increased focus and progress in the field. However, standardised framework integrating individual initiatives has not been formed. We present lessons learned and best practice from a collaborative success case in developing a treatment for a rare genetic disease. Our unique consortium model incorporated several of the identified developments under one project, DevelopAKUre, truly bringing together academia, industry and patient organisations in clinical drug development. We found that the equal partnership between all parties in our consortium was a key success factor creating a momentum based on a strong organisational culture where all partners had high engagement and taking ownership of the entire programme. With an agreed mutual objective, this provided synergies through connecting the strengths of the individual parties. Another key success factor was the central role of the patient organisation within the management team, and their unique study participants’ advocacy role securing the understanding and meeting the needs of the clinical study participants in real-time. This resulted in an accelerated enrolment into the clinical studies with a high retention rate allowing for delivery of the programme with significantly improved timelines. Our project was partly funded through an external EU research grant, enabling our model with equal partnership. Further attention within the community should be given to establishing a functional framework where sustainable funding and risk sharing between private and public organisations allow for our model to be replicated. BioMed Central 2021-12-14 /pmc/articles/PMC8670024/ /pubmed/34906169 http://dx.doi.org/10.1186/s13023-021-02137-0 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Position Statement Rudebeck, Mattias Scott, Ciarán Rhodes, Nicholas P. van Kan, Christa Olsson, Birgitta Al-sbou, Mohammed Hall, Anthony K. Sireau, Nicolas Ranganath, Lakshminarayan R. Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title | Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title_full | Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title_fullStr | Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title_full_unstemmed | Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title_short | Clinical development innovation in rare diseases: lessons learned and best practices from the DevelopAKUre consortium |
title_sort | clinical development innovation in rare diseases: lessons learned and best practices from the developakure consortium |
topic | Position Statement |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8670024/ https://www.ncbi.nlm.nih.gov/pubmed/34906169 http://dx.doi.org/10.1186/s13023-021-02137-0 |
work_keys_str_mv | AT rudebeckmattias clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT scottciaran clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT rhodesnicholasp clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT vankanchrista clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT olssonbirgitta clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT alsboumohammed clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT hallanthonyk clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT sireaunicolas clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium AT ranganathlakshminarayanr clinicaldevelopmentinnovationinrarediseaseslessonslearnedandbestpracticesfromthedevelopakureconsortium |