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008			211108s2021 sz a s |||| 0|eng d
020			_a9783030786052
024	7		_a10.1007/978-3-030-78605-2 _2doi
040			_aES-MaUEC _bspa _cES-MaUEC _dES-MaUEC
050		4	_aRM301.25 _b2021 EB
245	0	0	_aRare Disease Drug Development . _bClinical, Scientific, Patient, and Caregiver Perspectives _cedited by Raymond A. Huml
250			_aFirst edition 2021
264		1	_aCham _bSpringer International Publishing _c2021
264		4	_c2021
300			_a1 recurso en línea (XXIV, 409 páginas) _b36 ilustraciones
336			_2rdacontent _aTexto _btxt
337			_2rdamedia _aelectrónico _bc
338			_2rdacarrier _arecurso electrónico _bcr
347			_aArchivo de texto _bPDF
520			_aThis book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
988			_aSpringer_Medicine_2021
650		7	_2embne _aFarmacología _xInvestigación _9183058
700	1		_aHuml, Raymond A. _eeditor literario _4edt _4http://id.loc.gov/vocabulary/relators/edt
776	0	8	_iPrinted edition: _z9783030786045
776	0	8	_iPrinted edition: _z9783030786069
776	0	8	_iPrinted edition: _z9783030786076
856	4	0	_uhttps://go.openathens.net/redirector/universidadeuropea.es?url=https://doi.org/10.1007/978-3-030-78605-2 _zAcceso a este recurso digital (usuarios Universidad Europea de Madrid)
942			_2lcc _cLE
998			_b02/2022 _dz _ek _zSI