# Orphan drug

Source: https://onco.cc/terms/orphan-drug/  
OnCo record `orphan-drug` (Term). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A drug for a rare disease (in the US, fewer than 200,000 patients) that gets extra incentives, tax credits, fee waivers and seven years of market exclusivity, to make development worthwhile. Most cancers qualify, so most cancer drugs are orphans.

## Summary

The US Orphan Drug Act of 1983 and its EU equivalent in 2000 were designed for neglected diseases, but because cancer is subdivided into molecularly and anatomically defined groups, each individually rare, around 40% of new cancer drugs now carry orphan status, some of them blockbusters. The incentives have clearly accelerated drugs for rare cancers such as GIST, CML and mesothelioma, and critics argue they are also gamed by 'salami-slicing' indications and contribute to high prices. Orphan status is separate from the accelerated, breakthrough and priority pathways that speed review.

## Fields

- Kind: Term
- Last checked: 2026-09-09
- Also known as: orphan drugs; orphan designation; orphan drug designation; orphan status; orphan indication; rare disease; rare diseases

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Orphan_drug
- Wikipedia: https://en.wikipedia.org/wiki/Orphan_drug

## Connected records

- terms: [Accelerated approval](https://onco.cc/terms/accelerated-approval/), [Breakthrough Therapy / Priority Review / Priority Voucher](https://onco.cc/terms/breakthrough-designation/), [India New Drugs and Clinical Trials Rules 2019](https://onco.cc/terms/india-new-drugs-rules-2019/), [Off-label](https://onco.cc/terms/off-label/), [Orphan Drug Act 1983](https://onco.cc/terms/orphan-drug-act/), [RACE for Children Act](https://onco.cc/terms/race-for-children-act/), [Rare cancers](https://onco.cc/terms/rare-cancers/), [Regulation (EC) No 141/2000: EU orphan medicines](https://onco.cc/terms/eu-orphan-regulation/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

---
JSON: https://onco.cc/api/v1/entities/orphan-drug.json