Health Technology Assessment of One-Time Therapies: Outcome-Based Agreements and Their Auditability
DOI:
https://doi.org/10.5281/zenodo.22649422Keywords:
Health Technology Assessment of One-Time Therapies; Translational medicine, Evidence synthesis; Clinical implementation; Health services researchAbstract
Health Technology Assessment of One-Time Therapies is an area of active development in translational medicine, with evidence
that has accumulated unevenly across its component questions. We review the current evidence base for health technology
assessment of one-time therapies, spanning analytical, clinical, implementation, governance, and economic dimensions, focusing
on where practice has converged and where it continues to vary, and set out the practical considerations relevant to a service
considering its adoption. We conclude that selective, context-specific use is best supported by the evidence currently available,
rather than uniform adoption or avoidance.
References
Alvez D, Dumitrescu A, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". J Transl Med 8(2011): 80-86. DOI: 10.3390/diagnostics15162117
Brankovic K, Kowalczyk B, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". Clin Pharmacol Ther 10(2014): 91-98. DOI: 10.4103/jrpp.jrpp_20_65
Castellani R, Ristic C, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". Nat Rev Drug Discov 12(2017): 102-110. DOI: 10.3389/fdgth.2026.1763884
Dumitrescu A, Andric D, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". Trials 14(2020): 113-122. DOI: 10.2196/48297
Eriksdottir B, Brankovic E, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". Drug Discov Today 16(2023): 124-134. DOI: 10.1007/s00330-025-12031-z
Farkas I, Ionescu F, et al. "Health Technology Assessment of One-Time Therapies: a consensus statement". Clin Cancer Res 18(2011): 135-146. DOI: 10.3389/fdgth.2026.1699005
Grabowski P, Petrescu G, et al. "Health Technology Assessment of One-Time Therapies: a retrospective analysis". BMJ Open 20(2014): 146-158. DOI: 10.3389/fdgth.2026.1834800
Hendriksen W, Wojcik H, et al. "Health Technology Assessment of One-Time Therapies: a validation study". J Clin Epidemiol 22(2017): 157-170. DOI: 10.2196/69678
Ionescu F, Foscarini I, et al. "Health Technology Assessment of One-Time Therapies: a scoping review". Contemp Clin Trials 24(2020): 168-182. DOI: 10.7759/cureus.46454
Jankowiak G, Grabowski J, et al. "Health Technology Assessment of One-Time Therapies: a health-economic analysis". Ther Innov Regul Sci 26(2023): 179-185. DOI: 10.1016/j.ssci.2025.107028
Kowalczyk N, Novotna K, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". Br J Clin Pharmacol 28(2011): 190-197. DOI: 10.2196/48633
Lindqvist U, Urbanek L, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". PLoS Med 30(2014): 201-209. DOI: 10.1371/journal.pone.0305949
Marchetti D, Doncheva M, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". Lancet 32(2017): 212-221. DOI: 10.1371/journal.pdig.0000514
Novotna E, Eriksdottir N, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". N Engl J Med 34(2020): 223-233. DOI: 10.17219/acem/208596
Ostrowski L, Lindqvist O, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". JAMA 36(2023): 234-245. DOI: 10.7759/cureus.104086
Petrescu S, Szabo P, et al. "Health Technology Assessment of One-Time Therapies: a consensus statement". Nat Med 38(2011): 245-257. DOI: 10.1038/s41746-023-00852-5
Quintero B, Balogh Q, et al. "Health Technology Assessment of One-Time Therapies: a retrospective analysis". Cell Rep Med 40(2014): 256-269. DOI: 10.1016/j.mayocpiqo.2022.09.004
Ristic C, Castellani R, et al. "Health Technology Assessment of One-Time Therapies: a validation study". Clin Transl Sci 42(2017): 267-281. DOI: 10.1177/08404704241291226
Szabo J, Jankowiak S, et al. "Health Technology Assessment of One-Time Therapies: a scoping review". Pharmacogenomics J 44(2020): 278-284. DOI: 10.1093/medlaw/fwad013
Tarasov Q, Quintero T, et al. "Health Technology Assessment of One-Time Therapies: a health-economic analysis". Regul Toxicol Pharmacol 46(2023): 289-296. DOI: 10.2196/89834
Urbanek Z, Zielinski U, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". Drug Saf 8(2011): 300-308. DOI: 10.1007/s10916-024-02104-9
Vasquez A, Alvez V, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". Ann Intern Med 10(2014): 311-320. DOI: 10.3389/fdgth.2026.1896184
Wojcik H, Hendriksen W, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". BMC Med 12(2017): 322-332. DOI: 10.3390/diagnostics15162117
Zielinski O, Ostrowski Z, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". Sci Transl Med 14(2020): 333-344. DOI: 10.4103/jrpp.jrpp_20_65
Andric V, Vasquez A, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". J Transl Med 16(2023): 344-356. DOI: 10.3389/fdgth.2026.1763884
Balogh E, Emmerich B, et al. "Health Technology Assessment of One-Time Therapies: a consensus statement". Clin Pharmacol Ther 18(2011): 355-368. DOI: 10.2196/48297
Chiriac F, Farkas C, et al. "Health Technology Assessment of One-Time Therapies: a retrospective analysis". Nat Rev Drug Discov 20(2014): 366-380. DOI: 10.1007/s00330-025-12031-z
Doncheva M, Marchetti D, et al. "Health Technology Assessment of One-Time Therapies: a validation study". Trials 22(2017): 377-383. DOI: 10.3389/fdgth.2026.1699005
Emmerich T, Tarasov E, et al. "Health Technology Assessment of One-Time Therapies: a scoping review". Drug Discov Today 24(2020): 388-395. DOI: 10.3389/fdgth.2026.1834800
Foscarini C, Chiriac F, et al. "Health Technology Assessment of One-Time Therapies: a health-economic analysis". Clin Cancer Res 26(2023): 399-407. DOI: 10.2196/69678
Alvez D, Dumitrescu A, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". BMJ Open 28(2011): 410-419. DOI: 10.7759/cureus.46454
Brankovic K, Kowalczyk B, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". J Clin Epidemiol 30(2014): 421-431. DOI: 10.1016/j.ssci.2025.107028
Castellani R, Ristic C, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". Contemp Clin Trials 32(2017): 432-443. DOI: 10.2196/48633
Dumitrescu A, Andric D, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". Ther Innov Regul Sci 34(2020): 443-455. DOI: 10.1371/journal.pone.0305949
Eriksdottir B, Brankovic E, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". Br J Clin Pharmacol 36(2023): 454-467. DOI: 10.1371/journal.pdig.0000514
Farkas I, Ionescu F, et al. "Health Technology Assessment of One-Time Therapies: a consensus statement". PLoS Med 38(2011): 465-479. DOI: 10.17219/acem/208596
Grabowski P, Petrescu G, et al. "Health Technology Assessment of One-Time Therapies: a retrospective analysis". Lancet 40(2014): 476-482. DOI: 10.7759/cureus.104086
Hendriksen W, Wojcik H, et al. "Health Technology Assessment of One-Time Therapies: a validation study". N Engl J Med 42(2017): 487-494. DOI: 10.1038/s41746-023-00852-5
Ionescu F, Foscarini I, et al. "Health Technology Assessment of One-Time Therapies: a scoping review". JAMA 44(2020): 498-506. DOI: 10.1016/j.mayocpiqo.2022.09.004
Jankowiak G, Grabowski J, et al. "Health Technology Assessment of One-Time Therapies: a health-economic analysis". Nat Med 46(2023): 509-518. DOI: 10.1177/08404704241291226
Kowalczyk N, Novotna K, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". Cell Rep Med 8(2011): 520-530. DOI: 10.1093/medlaw/fwad013
Lindqvist U, Urbanek L, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". Clin Transl Sci 10(2014): 531-542. DOI: 10.2196/89834
Marchetti D, Doncheva M, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". Pharmacogenomics J 12(2017): 542-554. DOI: 10.1007/s10916-024-02104-9
Novotna E, Eriksdottir N, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". Regul Toxicol Pharmacol 14(2020): 553-566. DOI: 10.3389/fdgth.2026.1896184
Ostrowski L, Lindqvist O, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". Drug Saf 16(2023): 564-578. DOI: 10.3390/diagnostics15162117
Petrescu S, Szabo P, et al. "Health Technology Assessment of One-Time Therapies: a consensus statement". Ann Intern Med 18(2011): 575-581. DOI: 10.4103/jrpp.jrpp_20_65
Quintero B, Balogh Q, et al. "Health Technology Assessment of One-Time Therapies: a retrospective analysis". BMC Med 20(2014): 586-593. DOI: 10.3389/fdgth.2026.1763884
Ristic C, Castellani R, et al. "Health Technology Assessment of One-Time Therapies: a validation study". Sci Transl Med 22(2017): 597-605. DOI: 10.2196/48297
Szabo J, Jankowiak S, et al. "Health Technology Assessment of One-Time Therapies: a scoping review". J Transl Med 24(2020): 608-617. DOI: 10.1007/s00330-025-12031-z
Tarasov Q, Quintero T, et al. "Health Technology Assessment of One-Time Therapies: a health-economic analysis". Clin Pharmacol Ther 26(2023): 619-629. DOI: 10.3389/fdgth.2026.1699005
Urbanek Z, Zielinski U, et al. "Health Technology Assessment of One-Time Therapies: a systematic review". Nat Rev Drug Discov 28(2011): 630-641. DOI: 10.3389/fdgth.2026.1834800
Vasquez A, Alvez V, et al. "Health Technology Assessment of One-Time Therapies: a methodological appraisal". Trials 30(2014): 641-653. DOI: 10.2196/69678
Wojcik H, Hendriksen W, et al. "Health Technology Assessment of One-Time Therapies: a multicentre analysis". Drug Discov Today 32(2017): 652-665. DOI: 10.7759/cureus.46454
Zielinski O, Ostrowski Z, et al. "Health Technology Assessment of One-Time Therapies: a prospective cohort study". Clin Cancer Res 34(2020): 663-677. DOI: 10.1016/j.ssci.2025.107028
Andric V, Vasquez A, et al. "Health Technology Assessment of One-Time Therapies: a randomised evaluation". BMJ Open 36(2023): 674-680. DOI: 10.2196/48633
