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Rare diseases (RD) affect approximately 30 million Americans, half of whom are children. This study is the first to comprehensively evaluate their medical device needs via a survey of physicians. The study sought to identify and document the presumed unmet diagnostic and therapeutic device needs for RD management; clarify the magnitude of the potential unmet need; and generate meaningful data to inform medical device stakeholders. A cross-sectional nonprobability survey was conducted. The study population was drawn from the membership files of four groups: FDA Medical Devices Advisory Committee, Pediatric Advisory Committee, Pediatric Device Consortia, and National Institutes of Health (NIH) Rare Diseases Clinical Research Network. Only physician respondents with experience or knowledge regarding RD were eligible. Among eligible respondents, 90% confirmed the need for innovative devices to care for people with RD. Over 850 device needs were identified for 436 RD, with 74% of needs related to children. Pediatric physicians (OR = 2.11, 95% CI 1.01–4.39, P = 0.046) and physicians with more RD experience reflected greater dissatisfaction with existing devices (OR = 4.49, 95% CI 2.25–8.96, P < 0.0001). Creation of entirely new devices is the top recommendation for mitigating needs. This study demonstrates a major public health need for innovative medical devices to care for children and adults with RD. FDA and NIH support and seek opportunities to accelerate device development for these vulnerable patients.

Table of contentsO1 The European Social Preferences Measurement (ESPM) study project: social cost value analysis, budget impact, commercial life cycle revenue management, and the economics of biopharmaceutical Research & Development (R&D)Michael Schlander, Søren Holm, Erik Nord, Jeff Richardson, Silvio Garattini, Peter Kolominsky-Rabas, Deborah Marshall, Ulf Persson, Maarten Postma, Steven Simoens, Oriol de Solà Morales, Keith Tolley, Mondher Toumi, Harry TelserO2 Newborn Screening: the potential and the challengesJames R BonhamO3 Untreatable disease outcomes - how would we measure them?Helmut Hintner, Anja Diem, Martin LaimerO4 Taking Integrated Care Forward: Experiences from Canada to inspire service provision for people living with rare disease in EuropeRéjean HébertO5 Listening to the patient’s voice: social media listening for safety and benefits in rare diseasesNabarun Dasgupta, Carrie E. Pierce, Melissa JordanO6 Via Opta: Mobile apps making visually impaired patients’ lives easier Barbara Bori, Mohanad Fors, Emilie PrazakovaO7 A report of the IRDiRC “Small Population Clinical Trial” Task ForceSimon DayO8 HAE patient identification and diagnosis: An innovative, ‘game changing’ collaborationThomas J. Croce Jr.O9 Co-creating with the community: primary packaging & administration for people with haemophiliaJonas Fransson, Philip WoodO10 Go with Gaucher, taking forward the next generation. How to involve young people to create a new generation of patient advocatesAnne-Grethe Lauridsen, Joanne Higgs, Vesna Stojmirova AleksovskaP1 ODAK – Orphan Drug for Acanthamoeba KeratitisChristina Olsen, Ritchie Head, Antonio Asero, Vincenzo Papa, Christa van Kan, Loic Favennec, Silvana Venturella, Michela Salvador, Alan KrolP5 Rare Navigators help people living with rare diseases to manage the social – and healthcare systems Stephanie J. Nielsen, Birthe B. HolmP6 The eAcademy for Tay-Sachs & Sandhoff disease appDaniel Lewi, Patricia DurãoP10 The role of a patient organisation in driving the research agenda in a rare diseaseHeather Band, Andrea WestP13 Expertise for rare diseases mappedMarinda J.A. Hammann, Marije C. Effing-Boele, Hanka K. DekkerP14 The hidden costs of rare diseases: a feasibility studyAmy Hunter, Amy SimpsonP15 FDA’s new natural history grant program: support to build a solid foundation for development of products for rare diseasesGumei Liu, Katherine Needleman, Debra Lewis, Gayatri RaoP17 Understanding the wider impact of adrenal insufficiency: patient organisation involvement in the TAIN projectAmy Simpson, Amy Hunter, Martin J WhitakerP20 Bridging the gaps between medical and social care for people living with a rare diseaseRaquel Castro

The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration. Orphan drug designation, the granting of special status to drugs or biologics (“drugs”) for the treatment of rare diseases, one of the ODA's key incentive programs, has seen major increases in volume over recent years. The new era of precision medicine and the development of therapies directed toward smaller “orphan” subsets of common diseases have been suggested as being a major driver. We evaluated the basis for orphan drug designations and orphan subsets in relation to the impact of precision medicines. We found that the increasing numbers of orphan drug designation determinations were not driven by precision medicines separating common diseases into orphan subsets and that orphan subsets overall also represented a relatively small proportion of designations.

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Drug administration procedures are very much vital and are considered to be the prime-most criteria during the drug design and its appliance. Scrutinizing this, drugs are designed in such a way, bringing some change in their persona like bioavailability and bioequivalence. One such approach called prodrug, first started in 1950's is still a fertile area of research because of it's insist persona. Prodrugs are the shrouded drugs which are one or two chemical or enzymatic steps away from the active parent drug. Present review delineates about the prodrug action, types of prodrugs, new prodrug therapies, nanotech allied prodrugs.