Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem
In recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on c...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Springer Nature
2017-09-01
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| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.15252/emmm.201708203 |
| _version_ | 1797280495625568256 |
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| author | Annette C Bakker Salvatore La Rosa |
| author_facet | Annette C Bakker Salvatore La Rosa |
| author_sort | Annette C Bakker |
| collection | DOAJ |
| description | In recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on creating specific tools for accelerating the identification and development of effective treatments. Here, we describe a new model, developed by the Children's Tumor Foundation (CTF), by which foundations may play a role in the rare disease ecosystem. On the one hand, the CTF uses its position to build bridges between academic scientists, biotech and pharmaceutical companies, and patients, to accelerate the development of treatments that really matter most to patients. On the other hand, it acts as a niche investor to fund an integrated platform for critical R&D endeavors—including elements such as a patient registry, biobank, and open data platforms—which smoothen the transition from basic discovery to clinical benefit. Currently, the Children's Tumor Foundation is launching a call to top finance experts to collaborate on building an innovative model that will guarantee long‐term sustainability of this integrated platform (Fig ). |
| first_indexed | 2024-03-07T16:41:06Z |
| format | Article |
| id | doaj.art-2d04e9da60004555893d47d3c2bf232a |
| institution | Directory Open Access Journal |
| issn | 1757-4676 1757-4684 |
| language | English |
| last_indexed | 2024-03-07T16:41:06Z |
| publishDate | 2017-09-01 |
| publisher | Springer Nature |
| record_format | Article |
| series | EMBO Molecular Medicine |
| spelling | doaj.art-2d04e9da60004555893d47d3c2bf232a2024-03-03T08:12:08ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842017-09-01991179118210.15252/emmm.201708203Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystemAnnette C Bakker0Salvatore La Rosa1Children's Tumor Foundation New York NY USAChildren's Tumor Foundation New York NY USAIn recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on creating specific tools for accelerating the identification and development of effective treatments. Here, we describe a new model, developed by the Children's Tumor Foundation (CTF), by which foundations may play a role in the rare disease ecosystem. On the one hand, the CTF uses its position to build bridges between academic scientists, biotech and pharmaceutical companies, and patients, to accelerate the development of treatments that really matter most to patients. On the other hand, it acts as a niche investor to fund an integrated platform for critical R&D endeavors—including elements such as a patient registry, biobank, and open data platforms—which smoothen the transition from basic discovery to clinical benefit. Currently, the Children's Tumor Foundation is launching a call to top finance experts to collaborate on building an innovative model that will guarantee long‐term sustainability of this integrated platform (Fig ).https://doi.org/10.15252/emmm.201708203 |
| spellingShingle | Annette C Bakker Salvatore La Rosa Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem EMBO Molecular Medicine |
| title | Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem |
| title_full | Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem |
| title_fullStr | Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem |
| title_full_unstemmed | Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem |
| title_short | Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem |
| title_sort | rethinking the nonprofit foundation an emerging niche in the rare disease ecosystem |
| url | https://doi.org/10.15252/emmm.201708203 |
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