orphan drug
Uncommon disease medication
An orphan drug is a pharmaceutical developed to treat a rare disease—one affecting so few patients that commercial development wouldn't otherwise be profitable.
Government incentives like tax breaks and extended patent protection encourage companies to produce these medications.
The FDA granted orphan drug status to the new treatment for the genetic disorder.
Infrequent medical conditions
- genetic disordersConditions caused by mutations
- cystic fibrosisGenetic disorder thickening mucus
- Huntington's diseaseInherited fatal neurodegenerative condition
- muscular dystrophyGenetic muscle-wasting condition
hemophilia · sickle cell anemia · progeria · amyloidosis · narcolepsy · acromegaly
Controlled health research experiments
- biotechApplied biological engineering
- research grantsMoney awarded for studies
- patient advocacySupporting medical care rights
- medical innovationNew development in healing
Care for an ailment
- biologicLiving-organism-derived drug
- gene therapyMedical DNA modification treatment
- monoclonal antibodyEngineered protein from cloned cells
- cell therapyTreatment using transplanted cells
precision medicine · targeted therapy · disease-modifying · pharmacology
Financial assistance from government
- niche marketFocused commercial segment
- premium pricingCharging above competitors deliberately
- unprofitableProducing no financial gain
- development costsSpending to create something
research funding · underserved · cost-effectiveness · reimbursement · return on investment
Official drug safety certification
- fast trackAccelerated advancement path
- compassionate usePre-approval medical treatment allowance
- voucher programFunded alternatives through certificates
- tax creditsReductions in amount owed
rare diseases
Infrequent medical conditions
- genetic disordersConditions caused by mutations
- cystic fibrosisGenetic disorder thickening mucus
- Huntington's diseaseInherited fatal neurodegenerative condition
muscular dystrophy · hemophilia · sickle cell anemia · progeria · amyloidosis · narcolepsy · acromegaly
clinical trials
Controlled health research experiments
- biotechApplied biological engineering
- research grantsMoney awarded for studies
- patient advocacySupporting medical care rights
medical innovation · proof of concept · preclinical · investigational · pipeline
treatment
Care for an ailment
- biologicLiving-organism-derived drug
- gene therapyMedical DNA modification treatment
- monoclonal antibodyEngineered protein from cloned cells
cell therapy · precision medicine · targeted therapy · disease-modifying · pharmacology
subsidy
Financial assistance from government
- niche marketFocused commercial segment
- premium pricingCharging above competitors deliberately
- unprofitableProducing no financial gain
development costs · research funding · underserved · cost-effectiveness · reimbursement · return on investment
FDA approval
Official drug safety certification
- fast trackAccelerated advancement path
- compassionate usePre-approval medical treatment allowance
- voucher programFunded alternatives through certificates
orphan drugblockbuster drug
Exceptionally high-selling medication
Lipitor · Viagra · Prozac · over-the-counter drugs · aspirin
orphan drugover-the-counter drug
Medicine available without prescription
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