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 Orphan Drugs: completing a European dream
EuropaBio proposes the following actions to the EU Commission in order to fully apply the legislative framework for rare disease treatments, as established by the Orphan Medicinal Products (OMP) Regulation EC/141/2000:
1
Undertake educational programmes to build awareness about rare diseases in Europe, at European as well as at national level;
2
Establish an EU-wide network for diagnostic testing for rare diseases to ensure timely intervention for patients;
3
Promote a Europe-wide compassionate use system for the supply of orphan medicines to patients;
4
Increase understanding of the EU Regulation in EU Member States in order to eliminate conflicts with national legislation;
5
Review the incentives for OMP development in Member States; eliminate the confusion around the 10-year market exclusivity;
6
Review disincentives to orphan drug development at national level, e.g. additional requirements for clinical and cost-effectiveness data;
7
Facilitate clinical trials in the field of rare diseases, under the EU “Clinical Trials Directive” and review cost implications of post-marketing
7
commitments; and
8
Coordinate and streamline EU rare disease research and therapy development within the EU Commission and with the EMEA and the FDA.
EuropaBio Actions
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Input
into Commission review of cross-border
healthcare issues to ensure orphan drugs
are on the top of the Agenda.
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Developing and advocating biotech industry response to European Commission proposed guideline for market exclusivity.
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Advocate for appropriate incentives at both Commission and Member State level for the development of orphan drugs.
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Input into survey on current availability of approved orphan drugs in Member States.
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Completion and publication of first study on economic impact of the European Union’s Regulation on Orphan Medicinal Products, which came into force in 2000.
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Involvement in EMEA’s COMP Working Group with Interested Parties, ensuring effective industry participation.
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Support of EPPOSI Annual Conference on Rare Diseases.
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Advocate for fair and balanced views of health technology assessment in orphan drugs.
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