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Mini oral session - Policy and preventive strategies

1696MO - Partial orphan cancer drugs: FDA approval, clinical benefit, trials, epidemiology, price, beneficiaries, and spending


23 Oct 2023


Mini oral session - Policy and preventive strategies


Clinical Research;  Global Cancer Statistics;  Molecular Oncology;  Cancer Care Equity Principles and Health Economics;  Rare Cancers;  Therapy

Tumour Site


Daniel Michaeli


Annals of Oncology (2023) 34 (suppl_2): S925-S953. 10.1016/S0923-7534(23)01945-2


D.T. Michaeli1, C.T. Michaeli2

Author affiliations

  • 1 Third Department Of Medicine, Medizinische Fakultät Mannheim der Universität Heidelberg, 68167 - Mannheim/DE
  • 2 Abteilung Für Personalisierte Onkologie Mit Schwerpunkt Lungenkarzinom, UMM - Universitaetsklinikum Mannheim, 68167 - Mannheim/DE


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Abstract 1696MO


The Orphan Drug Act (ODA) incentivizes drug development for rare diseases with limited sales potential. Yet, orphan drugs frequently turn into multi-billion-dollar blockbusters, particularly those used to treat rare and common diseases: partial orphans. This study analyses the development, US Food and Drug Administration (FDA) approval, epidemiology, and economics of full, partial, and non-orphan cancer drugs.


170 drugs with FDA approval for 455 cancer indications were identified between 2000-2022. Full, partial, and non-orphan drugs were compared regarding their regulatory approval, clinical benefit, trials, epidemiology, price, beneficiaries, and spending with data extracted from FDA documents, Global Burden of Disease study, and Medicare & Medicaid.


We identified 110 full, 22 partial, and 38 non-orphan cancer drugs. Time from first to second FDA approval was shorter for partial than non-and full orphans (median 1.3 vs 1.6 vs 2.3 years). Full orphans, relative to partial and non-orphans, were more frequently third-line (17% vs 8 vs 7, p=.025) monotherapies (74% vs 58 vs 58, p=.002) for hematologic cancers (66% vs 4 vs 0, p<.001) supported by smaller (median 154 patients vs 416 vs 536, p<.001) open-label (56% vs 64 vs 87, p<.001) single-arm trials (19% vs 23 vs 50, p<.001). Disease incidence was lower and disease severity higher for full than partial and non-orphans. Full orphans offered a significantly greater overall survival (median: 4.0 vs 2.8 vs 2.8, p<.001) and progression-free survival (median: 5.1 vs 2.5 vs 3.6, p<.001). Monthly prices were higher for full and partial than non-orphans (median $17177 vs 13284 vs 12457, 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.


The benefit, trials, and epidemiology of partial orphan cancer drugs are more similar to non-orphans than full orphans. Yet, partial orphans receive all of the ODA’s incentives and are swiftly extended to new indications, resulting in greater prices, beneficiaries, and spending. Establishing a maximum revenue threshold for the ODA’s benefits alongside indication-specific pricing could reduce expenditure on partial orphans.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.


Has not received any funding.


All authors have declared no conflicts of interest.

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