Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice
Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-worl...
| Main Authors: | , , , , , , , , , , , |
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| Other Authors: | |
| Format: | Article |
| Language: | English |
| Published: |
Wiley
2020
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| Online Access: | https://hdl.handle.net/1721.1/128557 |
| _version_ | 1826191823478980608 |
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| author | Selker, Harry P. Eichler, Hans‐Georg Stockbridge, Norman L. McElwee, Newell E. Dere, Willard H. Cohen, Theodora Erban, John K. Seyfert‐Margolis, Vicki L. Honig, Peter K. Kaitin, Kenneth I. Oye, Kenneth A D'Agostino, Ralph B. |
| author2 | Massachusetts Institute of Technology. Department of Political Science |
| author_facet | Massachusetts Institute of Technology. Department of Political Science Selker, Harry P. Eichler, Hans‐Georg Stockbridge, Norman L. McElwee, Newell E. Dere, Willard H. Cohen, Theodora Erban, John K. Seyfert‐Margolis, Vicki L. Honig, Peter K. Kaitin, Kenneth I. Oye, Kenneth A D'Agostino, Ralph B. |
| author_sort | Selker, Harry P. |
| collection | MIT |
| description | Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-world conditions with longer follow-up periods. In “efficacy-to-effectiveness (E2E) trials,” if the initial efficacy trial component is positive, the trial seamlessly transitions to an effectiveness trial component to efficiently yield both types of evidence. Yet more time could be saved by simultaneously addressing efficacy and effectiveness in an “efficacy and effectiveness too (EE2) trial.” Additionally, hybrids of the E2E and EE2 approaches with differing degrees of overlap of the two components could allow flexibility for specific drug development needs. In planning EE2 trials, each stakeholder's current and future needs, incentives, and perspective must be considered. Although challenging, the ultimate benefits to stakeholders, the health system, and the public should justify this effort. |
| first_indexed | 2024-09-23T09:01:51Z |
| format | Article |
| id | mit-1721.1/128557 |
| institution | Massachusetts Institute of Technology |
| language | English |
| last_indexed | 2024-09-23T09:01:51Z |
| publishDate | 2020 |
| publisher | Wiley |
| record_format | dspace |
| spelling | mit-1721.1/1285572022-09-26T09:59:26Z Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice Selker, Harry P. Eichler, Hans‐Georg Stockbridge, Norman L. McElwee, Newell E. Dere, Willard H. Cohen, Theodora Erban, John K. Seyfert‐Margolis, Vicki L. Honig, Peter K. Kaitin, Kenneth I. Oye, Kenneth A D'Agostino, Ralph B. Massachusetts Institute of Technology. Department of Political Science Massachusetts Institute of Technology. Center for Biomedical Innovation Efficacy trials, designed to gain regulatory marketing approval, evaluate drugs in optimally selected patients under advantageous conditions for relatively short time periods. Effectiveness trials, designed to evaluate use in usual practice, assess treatments among more typical patients in real-world conditions with longer follow-up periods. In “efficacy-to-effectiveness (E2E) trials,” if the initial efficacy trial component is positive, the trial seamlessly transitions to an effectiveness trial component to efficiently yield both types of evidence. Yet more time could be saved by simultaneously addressing efficacy and effectiveness in an “efficacy and effectiveness too (EE2) trial.” Additionally, hybrids of the E2E and EE2 approaches with differing degrees of overlap of the two components could allow flexibility for specific drug development needs. In planning EE2 trials, each stakeholder's current and future needs, incentives, and perspective must be considered. Although challenging, the ultimate benefits to stakeholders, the health system, and the public should justify this effort. National Center for Advancing Translational Sciences (Grant U24TR001609) 2020-11-20T22:55:48Z 2020-11-20T22:55:48Z 2019-02 2018-10 2020-06-12T15:47:55Z Article http://purl.org/eprint/type/JournalArticle 0009-9236 1532-6535 https://hdl.handle.net/1721.1/128557 Selker, Harry P. et al. "Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice." Clinical Pharmacology and Therapeutics 105, 4 (February 2019): 857-866 © 2019 The Authors en http://dx.doi.org/10.1002/cpt.1347 Clinical Pharmacology and Therapeutics Creative Commons Attribution NonCommercial License 4.0 https://creativecommons.org/licenses/by-nc/4.0/ application/pdf Wiley Wiley |
| spellingShingle | Selker, Harry P. Eichler, Hans‐Georg Stockbridge, Norman L. McElwee, Newell E. Dere, Willard H. Cohen, Theodora Erban, John K. Seyfert‐Margolis, Vicki L. Honig, Peter K. Kaitin, Kenneth I. Oye, Kenneth A D'Agostino, Ralph B. Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title_full | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title_fullStr | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title_full_unstemmed | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title_short | Efficacy and Effectiveness Too Trials: Clinical Trial Designs to Generate Evidence on Efficacy and on Effectiveness in Wide Practice |
| title_sort | efficacy and effectiveness too trials clinical trial designs to generate evidence on efficacy and on effectiveness in wide practice |
| url | https://hdl.handle.net/1721.1/128557 |
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