BioWorld International Correspondent

BRUSSELS, Belgium - One year after the European Union's new orphan drug promotion program started up, there are already calls for improvements. The program was intended principally to boost biotechnology research. But now, after European drug firms and European drug regulators met in London in April to review the way the new scheme is working, the European Medicines Evaluation Agency, which operates the program, has published a list of recommendations for changes.

At the top of the list for improvements is giving more money to back the program, so that the EMEA can provide enough scientists to advise companies at an early stage on the design of clinical trial protocols, and so that there is no artificial ceiling to the number of orphan drugs that can win the exemption from marketing authorization fees that the scheme promises.

The industry, represented by the three principal European industry groupings involved in biotechnology medicines (the European Federation of Pharmaceutical Industries and Associations, the Emerging Biopharmaceutical Enterprises, and EuropaBio), recognized that the EMEA had tried hard to be accessible and available during the first year of the scheme's operation. But critics wanted more, in terms of speed, quantity of staff, and use of outside scientific experts.

From the side of the regulators, professor Josep Torrent-Farnell, the Spanish academic who chairs the EU committee that designates orphan drugs, also insisted on the importance of developing closer teamwork between his committee and the EMEA and its advisers. In particular, he said, it is important to ensure that the early advice given on protocols is in line with the criteria that the EMEA eventually uses to assess the validity of marketing authorization applications.

In a bid to improve the functioning of the system in the short term, the EMEA is going to set up a dedicated orphan drugs website, to provide public summary reports of positive and negative decisions, to draw up additional guidelines to streamline the putting together of applications for orphan drug designation, to increase the opportunities for face-to-face meetings between industry and regulators, and to set up performance indicators to measure the speed and effectiveness of the procedure.

Over the medium term, the EMEA is going to explore how updated information on designated orphan drugs can be disseminated so that the public can easily access it.

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