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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Do we need authorized orphan drugs when compounded medications are available?
Journal of Clinical Pharmacy and Therapeutics
|September 15, 2012
Summary
Orphan drug designation may not be necessary for compounded medications with established clinical evidence. A balance is needed between new orphan drugs and existing, evidence-based treatments for rare diseases.
Area of Science:
- Pharmacology
- Drug Development
- Rare Diseases
Background:
- Orphan drugs are vital for diagnosing, preventing, and treating rare diseases.
- Compounded medications have a history of effective use for rare conditions, supported by clinical evidence.
Purpose of the Study:
- To question the necessity of orphan drug designation for compounded medications with a long history of effective use.
- To present case studies illustrating the use of compounded and off-label medications for rare diseases.
Main Methods:
- Review of case studies involving compounded and off-label medications for rare diseases.
- Analysis of the regulatory pathways for orphan drugs versus existing treatments.
Main Results:
- Examples of compounded drugs include amifampridine, ibuprofen, and zinc acetate for specific rare diseases.
- Off-label use of non-orphan drugs like Hydrea and Viagra became designated orphan products for other rare conditions.
Conclusions:
- A better balance is needed between developing new orphan drugs and utilizing compounded or off-label medications.
- Focusing orphan drug development on rare diseases with high unmet medical needs is crucial.
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