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Feedback on the 7th annual E-IMD meeting

The 7th annual E-IMD meeting happened 31st October 2017 in Brussels. 

Website revision

As for the dissemination and website revision, its updating included the publication proposal template, the list of publications and updated list of partners and updated descriptions of the consortium (with thanks to Florian Gleich). Monique Williams and Martin Lindner offered their help to the realisation and sustainability of the newsletter. The question of patient organization involvement was raised. All E-IMD members were asked to contact their national patient organizations and give feedback.

Post Authorization Safety Study for the drug RAVICTI

The E-IMD Consortium is implementing the Post Authorization Safety Study (PASS) for the drug RAVICTI, in collaboration with Horizon Pharma, the Marketing Authorization Holder of RAVICTI. Since the start of the initial negotiations in August 2015 until the acceptance of the Protocol by the PRAC of the European Medicines Agency in December 2016 and the start of patient enrollment in November 2017, the implementation of the partnership faced some delays. However the European Post-Authorization Registry for RAVICTI in Partnership with E-IMD (RRPE) is now fully operational and RAVICTI is successively released in European markets. The tasks ahead are increasing the number of active study centers and achieving the enrollment goals.

The activation process for a RRPE study site consist of the following steps:

  • Signing of the confidentiality agreement (CDA) provided by Horizon Pharma
  • Evaluating the study protocol provided by Horizon Pharma after signing of the CDA
  • Negotiating the clinical trial agreement (CTA), including the amount of reimbursement needed to implement the protocol
  • Submitting the protocol to the local institutional review board
  • Receiving a briefing by the registry management on the correct study procedures

At the moment one E-IMD center is actively enrolling patients on the protocol. The current number of enrolled patients is 10 in the comparator group and 2 in the treatment group. 17 of the 20 E-IMD sites interested in participating have signed the CDA, 8 sites have already evaluated the protocol for feasibility and 7 sites are currently negotiating CTAs with the Marketing Authorization Holder

Publication Rules

A further issue were the publication rules, it was reaffirmed that there should be no honorary and guest author acknowledgments and that each E-IMD member that provides data always should at least be counted as additional contributor and thus listed in PubMed as co-author without any threshold rule regarding patient numbers included.

E-IMD membership

A discussion was held on E-IMD membership criteria which proved to be too ambitious as it yielded no clear consensus on the listed criteria. As for the strategy and working plan, there was consensus that most interested members should be able to fulfil all listed criteria (list forwarded by Johannes and corrected/validated by the SB members). It was a general consensus that the list should be not exclusive and at least tiered into essential criteria (major criteria) that need to be fulfilled (e.g. at least one metabolic specialist, treatment of patients with eligible diagnosis etc.) and non-essential criteria (minor criteria) that should be listed for the sake of documentation (e.g. genetic lab, NBS etc.) but not be compulsive for membership eligibility

Recent publication

Transatlantic combined and comparative data analysis of 1095 patients with urea cycle disorders-a successful strategy for clinical research of rare diseases.

Posset R, Garbade SF, Boy N, Burlina AB, Dionisi-Vici C, Dobbelaere D, Garcia-Cazorla A, de Lonlay P, Teles EL, Vara R, Ah Mew N, Batshaw ML, Baumgartner MR, McCandless S, Seminara J, Summar M, Hoffmann GF, Kölker S, Burgard P; Additional individual contributors of the UCDC and the E-IMD consortium.

J Inherit Metab Dis. 2018 Jul 4. doi: 10.1007/s10545-018-0222-z. [Epub ahead of print]

E-IMD - www.e-imd.net