(2014) affects their ability to manage their day-to-day to collecting patient input, facilitating this [111] lives, what treatments are currently available, what process in future orphan drug authorizations therapeutic benefits are most important to them, Pilot project and their risk tolerance for new treatments EMA European Union Rare diseases in general Patient Patient organizations are consulted on Not specified • (2012) organizations requests by the European Medicines [69] scientific committees and working parties Agency’ [Website] They take part in discussions on the development • and authorization of medicines EMA European Union Rare diseases in general Patient Patient organizations review written information Not specified • (2012) organizations on medicines prepared by the EMA [69] [Website] EMA European Union Rare diseases in general Patient Patient organizations are involved in the Not specified • (2012) organizations preparation of EMA guidelines [69] [Website] EMA European Union Rare diseases in general Patient Patient organizations take part in EMA Not specified • (2012) organizations conferences and workshops [69] [Website] FDA United States Rare diseases in general Patients and Patients and families attend public meetings Not specified • (2017) families hosted by the FDA where they have the chance to [159] discuss their disease and its impact on their daily lives, the types of treatment benefits that matter to them most, and their perspectives on the adequacy of current therapies FDA United States Rare diseases in general Patients and As part of the Patient Representative Not specified • FDA’s (2017) families Program, patients and families provide input on [159] disease-specific issues relating to medical products in various stages of development, review and approval Black United States SMEI Patient The IDEA League contributed data used in the Stiripentol received orphan drug designation in • • (2011) organizations orphan drug designation application for stiripentol the United States [22] in the United States Dunkle United States Rare diseases in general Patient NORD provides input into FDA processes, such Not specified • (2010) organizations as reviewing applications and standards for [47] drug and device testing Membership on a regulatory decision-making or advisory committee
(2014) [111] Pilot project affects their ability to manage their day-to-day lives, what treatments are currently available, what therapeutic benefits are most important to them, and their risk tolerance for new treatments to collecting patient input, facilitating this process in future orphan drug authorizations EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations are consulted on requests by the European Medicines Agency’ scientific committees and working parties • They take part in discussions on the development and authorization of medicines Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations review written information on medicines prepared by the EMA Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations are involved in the preparation of EMA guidelines Not specified EMA (2012) [69] [Website] European Union Rare diseases in general Patient organizations • Patient organizations take part in EMA conferences and workshops Not specified FDA (2017) [159] United States Rare diseases in general Patients and families • Patients and families attend public meetings hosted by the FDA where they have the chance to discuss their disease and its impact on their daily lives, the types of treatment benefits that matter to them most, and their perspectives on the adequacy of current therapies Not specified FDA (2017) [159] United States Rare diseases in general Patients and families • As part of the FDA’s Patient Representative Program, patients and families provide input on disease-specific issues relating to medical products in various stages of development, review and approval Not specified Black (2011) [22] United States SMEI Patient organizations • The IDEA League contributed data used in the orphan drug designation application for stiripentol in the United States • Stiripentol received orphan drug designation in the United States Dunkle (2010) [47] United States Rare diseases in general Patient organizations • NORD provides input into FDA processes, such as reviewing applications and standards for drug and device testing Not specified Membership on a regulatory decision-making or advisory committee
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