Partial Orphan Cancer Drugs : US Food and Drug Administration Approval, Clinical Benefit, Trials, Epidemiology, Price, Beneficiaries, and Spending
Copyright © 2024. Published by Elsevier Inc..
OBJECTIVES: The Orphan Drug Act (ODA) incentivizes drug development for rare diseases with limited sales potential. Partial orphans-drugs used to treat rare and common diseases-frequently turn into multi-billion dollar blockbusters. This study analyzes partial orphan cancer drugs' development, approval, and economics.
METHODS: 170 drugs with US Food and Drug Administration approval for 455 cancer indications were identified (2000-2021). 110 full, 22 partial, and 38 non-orphan drugs were compared regarding their approval, benefits, trials, epidemiology, price, beneficiaries, and spending with data from regulatory documents, Global Burden of Disease study, and Medicare and Medicaid.
RESULTS: Full orphans, relative to partial and non-orphans, were more frequently monotherapies for hematologic cancers supported by smaller single-arm trials treating diseases with a lower incidence and higher severity. The time from first to second indication approval was 1 year shorter for partial than full orphans. Full orphans offered a greater overall survival (median: 4.0 vs 2.8 vs 2.8 months, P < .001) and progression-free survival benefit (median: 5.1 vs 2.5 vs 3.6 months, P < .001). Monthly prices were higher for full and partial than non-orphan drugs (median: $17 177 vs $13 284 vs $12 457, P < .001). Beneficiaries (8790 vs 4390 vs 1730) and spending ($570 vs $305 vs $156 million) per drug were greater for partial than non-and full orphans.
CONCLUSIONS: Although partial orphans' benefits, trials, and economics are more similar to non-than full orphans, they receive all of the ODA's benefits and are swiftly extended to new indications; resulting in greater spending. A maximum ODA revenue/patient threshold could limit expenditure on partial orphans.
Medienart: |
E-Artikel |
---|
Erscheinungsjahr: |
2024 |
---|---|
Erschienen: |
2024 |
Enthalten in: |
Zur Gesamtaufnahme - volume:27 |
---|---|
Enthalten in: |
Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research - 27(2024), 4 vom: 21. Apr., Seite 449-457 |
Sprache: |
Englisch |
---|
Beteiligte Personen: |
Michaeli, Thomas [VerfasserIn] |
---|
Links: |
---|
Anmerkungen: |
Date Completed 05.04.2024 Date Revised 25.04.2024 published: Print-Electronic Citation Status MEDLINE |
---|
doi: |
10.1016/j.jval.2024.01.002 |
---|
funding: |
|
---|---|
Förderinstitution / Projekttitel: |
|
PPN (Katalog-ID): |
NLM367354780 |
---|
LEADER | 01000caa a22002652 4500 | ||
---|---|---|---|
001 | NLM367354780 | ||
003 | DE-627 | ||
005 | 20240425232726.0 | ||
007 | cr uuu---uuuuu | ||
008 | 240121s2024 xx |||||o 00| ||eng c | ||
024 | 7 | |a 10.1016/j.jval.2024.01.002 |2 doi | |
028 | 5 | 2 | |a pubmed24n1386.xml |
035 | |a (DE-627)NLM367354780 | ||
035 | |a (NLM)38244983 | ||
035 | |a (PII)S1098-3015(24)00030-5 | ||
040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
041 | |a eng | ||
100 | 1 | |a Michaeli, Thomas |e verfasserin |4 aut | |
245 | 1 | 0 | |a Partial Orphan Cancer Drugs |b US Food and Drug Administration Approval, Clinical Benefit, Trials, Epidemiology, Price, Beneficiaries, and Spending |
264 | 1 | |c 2024 | |
336 | |a Text |b txt |2 rdacontent | ||
337 | |a ƒaComputermedien |b c |2 rdamedia | ||
338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
500 | |a Date Completed 05.04.2024 | ||
500 | |a Date Revised 25.04.2024 | ||
500 | |a published: Print-Electronic | ||
500 | |a Citation Status MEDLINE | ||
520 | |a Copyright © 2024. Published by Elsevier Inc. | ||
520 | |a OBJECTIVES: The Orphan Drug Act (ODA) incentivizes drug development for rare diseases with limited sales potential. Partial orphans-drugs used to treat rare and common diseases-frequently turn into multi-billion dollar blockbusters. This study analyzes partial orphan cancer drugs' development, approval, and economics | ||
520 | |a METHODS: 170 drugs with US Food and Drug Administration approval for 455 cancer indications were identified (2000-2021). 110 full, 22 partial, and 38 non-orphan drugs were compared regarding their approval, benefits, trials, epidemiology, price, beneficiaries, and spending with data from regulatory documents, Global Burden of Disease study, and Medicare and Medicaid | ||
520 | |a RESULTS: Full orphans, relative to partial and non-orphans, were more frequently monotherapies for hematologic cancers supported by smaller single-arm trials treating diseases with a lower incidence and higher severity. The time from first to second indication approval was 1 year shorter for partial than full orphans. Full orphans offered a greater overall survival (median: 4.0 vs 2.8 vs 2.8 months, P < .001) and progression-free survival benefit (median: 5.1 vs 2.5 vs 3.6 months, P < .001). Monthly prices were higher for full and partial than non-orphan drugs (median: $17 177 vs $13 284 vs $12 457, P < .001). Beneficiaries (8790 vs 4390 vs 1730) and spending ($570 vs $305 vs $156 million) per drug were greater for partial than non-and full orphans | ||
520 | |a CONCLUSIONS: Although partial orphans' benefits, trials, and economics are more similar to non-than full orphans, they receive all of the ODA's benefits and are swiftly extended to new indications; resulting in greater spending. A maximum ODA revenue/patient threshold could limit expenditure on partial orphans | ||
650 | 4 | |a Journal Article | |
650 | 4 | |a Medicaid | |
650 | 4 | |a Medicare | |
650 | 4 | |a cancer drug | |
650 | 4 | |a clinical trial | |
650 | 4 | |a drug development | |
650 | 4 | |a drug spending | |
650 | 4 | |a epidemiology | |
650 | 4 | |a orphan designation | |
650 | 4 | |a overall survival | |
650 | 4 | |a partial orphan | |
650 | 4 | |a price | |
650 | 4 | |a progression-free survival | |
650 | 4 | |a rare diseases | |
650 | 7 | |a Pharmaceutical Preparations |2 NLM | |
650 | 7 | |a Antineoplastic Agents |2 NLM | |
700 | 1 | |a Michaeli, Daniel Tobias |e verfasserin |4 aut | |
773 | 0 | 8 | |i Enthalten in |t Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research |d 1998 |g 27(2024), 4 vom: 21. Apr., Seite 449-457 |w (DE-627)NLM115934545 |x 1524-4733 |7 nnns |
773 | 1 | 8 | |g volume:27 |g year:2024 |g number:4 |g day:21 |g month:04 |g pages:449-457 |
856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.jval.2024.01.002 |3 Volltext |
912 | |a GBV_USEFLAG_A | ||
912 | |a GBV_NLM | ||
951 | |a AR | ||
952 | |d 27 |j 2024 |e 4 |b 21 |c 04 |h 449-457 |