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[Orphan drugs]
1Laboratorio di Chimica del Farmaco, Istituto Superiore di Sanità, Roma.
Annali Dell'Istituto Superiore Di Sanita
|January 1, 1991
Summary
Developing new drugs for rare diseases and those prevalent in developing nations is challenging due to high costs. Regulatory bodies like the USA and WHO offer incentives to address this unmet medical need.
Area of Science:
- Drug discovery and development
- Rare diseases
- Global health
Background:
- Over 60% of known diseases are considered rare, lacking satisfactory therapies.
- High drug development costs make treatments for rare and developing country diseases unprofitable.
- Existing drugs, if no longer profitable, can also be classified as orphan drugs.
Purpose of the Study:
- To highlight the challenges in developing new drugs for rare diseases and those in developing countries.
- To discuss the economic factors contributing to the 'orphan drug' designation.
- To present existing strategies addressing the orphan drug problem.
Main Methods:
- Review of existing literature on drug development economics and rare diseases.
- Analysis of regulatory approaches by the USA (Orphan Drug Act) and WHO (Tropical Diseases Research programme).
- Examination of the definition and scope of orphan drugs.
Main Results:
- Drug development is economically unviable for rare diseases and diseases common in developing countries.
- The Orphan Drug Act in the USA and WHO's research programs aim to incentivize orphan drug development.
- Uncommon formulations or discontinued products may also fall under the orphan drug category.
Conclusions:
- Significant unmet medical needs exist for rare diseases and those affecting developing nations.
- Economic and regulatory strategies are crucial for addressing the orphan drug market.
- Continued research and development are necessary to ensure drug availability for all populations.