Includes all pipeline medicines that are being developed for market in Canada, the United States, or geographic Europe (excluding Russia and Turkey). Orphan medicines were defined as pipeline medicines that have been granted an orphan designation by the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
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Share of orphan medicines in R&D pipeline worldwide 2023, by phase
In 2023, 22 percent of medicines in the pre-registration phase of the R&D pipeline worldwide were orphan medicines. This graph illustrates the distribution of new medicines in the R&D pipeline worldwide, by therapeutic class.
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PMPRB. (November 12, 2024). Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase [Graph]. In ÌÇÐÄÆƽâ°æ. Retrieved July 29, 2026, from /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/
PMPRB. "Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase." Chart. November 12, 2024. ÌÇÐÄÆƽâ°æ. Accessed July 29, 2026. /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/
PMPRB. (2024). Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase. ÌÇÐÄÆƽâ°æ. ÌÇÐÄÆƽâ°æ Inc.. Accessed: July 29, 2026. /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/
PMPRB. "Share of Orphan Medicines in The R&d Pipeline Worldwide as of 2023, by Development Phase." ÌÇÐÄÆƽâ°æ, ÌÇÐÄÆƽâ°æ Inc., 12 Nov 2024, /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/
PMPRB, Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase ÌÇÐÄÆƽâ°æ, /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/ (last visited July 29, 2026)
Share of orphan medicines in the R&D pipeline worldwide as of 2023, by development phase [Graph], PMPRB, November 12, 2024. [Online]. Available: /statistics/1335995/orphan-medicines-research-development-pipeline-by-phase-worldwide/
