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Quantifying emerging drugs for very rare conditions
K A Miles1, C Packer, A Stevens
1Department of Public Health and Epidemiology, University of Birmingham, Birmingham, B15 2TT, UK. k.miles@bham.ac.uk
QJM : Monthly Journal of the Association of Physicians
|April 26, 2007
Summary
Pharmaceutical companies are developing 113 drugs for rare conditions, with many progressing through late-stage clinical trials. Future discussions must address drug affordability and cost-effectiveness for rare disease treatments.
Area of Science:
- Pharmaceutical development
- Rare disease research
- Health economics
Background:
- European Union legislation promotes the development of drugs for rare conditions.
- High costs and long-term use of rare disease drugs raise affordability and cost-effectiveness concerns.
Purpose of the Study:
- To quantify the number of drugs in development for very rare conditions.
Main Methods:
- Defined very rare conditions as <1:50,000 prevalence.
- Identified drugs in Phase II, Phase III, or pre-registration using commercial databases.
Main Results:
- 113 drugs are in development for 42 very rare conditions.
- 16 drugs are pre-registration, 29 in Phase III, and 65 in Phase II.
- Some drugs target multiple indications or are in combined trial phases.
Conclusions:
- A significant number of these drugs are expected to reach the market.
- National policies and clear decision-making frameworks are needed for drug affordability and cost-effectiveness assessment.
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