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. 2024 Jun 5:11:1404922.
doi: 10.3389/fmed.2024.1404922. eCollection 2024.

Clinical development and marketing application review times for novel orphan-designated drugs

Ebru Demirci  1 , Jennifer Knicley  1 , Lori Fiorentino  1
Affiliations

Clinical development and marketing application review times for novel orphan-designated drugs

Ebru Demirci et al. Front Med (Lausanne). .

Abstract

Development of an orphan-designated drug has been more challenging and financially less attractive than that of other drugs due to low prevalence of the condition, poorly defined biomarkers and lack of experience of healthcare providers in diagnosing and treating the condition. Guidance and incentives in some countries support the sponsors in developing orphan-designated drugs despite the challenges. Expedited regulatory programs as offered by the United States (US) Food and Drug Administration (FDA) and the European Medicines Agency (EMA) support the development of drugs, provide shorter marketing application review times or provide preliminary approval. In this study, we analyze marketing application review times in the US and in the European Union (EU) and clinical development times for novel, i.e., containing new molecular entity, orphan-designated drugs that were approved in the US between 1 June 2020 and 31 May 2023, and their correlation with expedited regulatory programs. Seventy-three marketing applications for novel orphan-designated drugs were approved by the FDA, and 39 also received a positive opinion from the EMA. The marketing application review time by the FDA for the 73 novel orphan-designated drugs approved in the US was 244 days (n = 73, median), and the marketing application review time by the EMA for the 39 drugs that were also approved in the EU was 353 days (n = 39, median). The typical clinical development time for a novel orphan-designated drug was 7.2 years (n = 72).

Keywords: European Medicines Agency (EMA); US Food and Drug Administration (FDA); clinical development; clinical development time; marketing application review time; novel drugs; orphan drugs; orphan-designated drugs.

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Conflict of interest statement

ED, JK, and LF were employed by Pharming Group N.V. The paper was created based on data from various sources but is neither a legal interpretation nor a statement reflecting any policy, view, or opinion of contributing data sources. The views expressed in the paper are those of the authors alone, and do not reflect the view or opinion of the data suppliers.

Figures

Figure 1
Figure 1
(A) Marketing application review time, and application type in the US (new drug application (NDA) and biologics license application (BLA)) or type of product in the EU. Outliers are represented as dots, except for terlipressin (4,884 days in categories US all drugs and US NDA). Data source: Cortellis Regulatory Intelligence. (B) Clinical development time and application type in the US. Center lines show the medians. Whiskers extend to minimum and maximum values. Outliers are represented as dots. Number of data points is displayed for each category. Data source: Cortellis Clinical Trials Intelligence.
Figure 2
Figure 2
Clinical development time (median) and expedited regulatory programs provided by the FDA for 72 drugs that went or did not go through breakthrough therapy designation (BTD) or fast track designation (FTD). Number of data points is displayed for each category. Data source: Cortellis Clinical Trials Intelligence.
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