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Orphan drug development is progressing too slowly.
Roberta Joppi1, Vittorio Bertele, Silvio Garattini
1Mario Negri Institute for Pharmacological Research, Milan, Italy.
British Journal of Clinical Pharmacology
|February 21, 2006
Summary
Few orphan medicinal products (OMPs) are approved due to methodological limitations in dossiers and limited incentives. Improving documentation and support is crucial for addressing the unmet needs in rare diseases.
Area of Science:
- Pharmacoeconomics
- Regulatory Science
- Drug Development
Background:
- Orphan medicinal products (OMPs) are vital for treating rare diseases.
- Regulatory pathways, like the European Union's, aim to incentivize OMP development.
- Assessing the success rate and quality of OMP applications is critical for understanding development barriers.
Purpose of the Study:
- To evaluate the methodological quality of submitted OMP dossiers.
- To identify factors contributing to the low number of approved OMPs in Europe.
Main Methods:
- Data on OMP designations and approvals were sourced from the European Commission's database.
- European Public Assessment Reports (EPARs) were reviewed for methodological details.
- Analysis included dossier quality assessment and approval statistics.
Main Results:
- Only 18 out of 255 (7.1%) designated OMPs received marketing authorization.
- Common methodological deficiencies included suboptimal clinical trial design and lack of active comparators.
- The use of surrogate endpoints was also frequently noted as a limitation.
Conclusions:
- Limited European incentives for manufacturers may hinder OMP development.
- Insufficient methodological rigor in OMP dossiers appears to be a significant barrier to approval.
- Addressing these issues is essential given the large number of rare diseases lacking effective therapies.