001440893 000__ 04719cam\a2200541\a\4500
001440893 001__ 1440893
001440893 003__ OCoLC
001440893 005__ 20230309004708.0
001440893 006__ m\\\\\o\\d\\\\\\\\
001440893 007__ cr\un\nnnunnun
001440893 008__ 211115s2021\\\\sz\\\\\\o\\\\\001\0\eng\d
001440893 019__ $$a1285162888$$a1285167076$$a1285238723$$a1285253297$$a1285781995$$a1292517551$$a1294357270
001440893 020__ $$a9783030786052$$q(electronic bk.)
001440893 020__ $$a3030786056$$q(electronic bk.)
001440893 020__ $$z3030786048
001440893 020__ $$z9783030786045
001440893 0247_ $$a10.1007/978-3-030-78605-2$$2doi
001440893 035__ $$aSP(OCoLC)1285274795
001440893 040__ $$aYDX$$beng$$epn$$cYDX$$dGW5XE$$dEBLCP$$dOCLCF$$dDKU$$dOCLCO$$dDCT$$dOCLCQ$$dCOM$$dOCLCO$$dUKAHL$$dOCLCQ$$dN$T$$dBNG
001440893 049__ $$aISEA
001440893 050_4 $$aRM301.25$$b.R37 2021
001440893 08204 $$a615.1/9$$223
001440893 24500 $$aRare disease drug development :$$bclinical, scientific, patient, and caregiver perspectives /$$cRaymond A. Huml, editor.
001440893 260__ $$aCham, Switzerland :$$bSpringer,$$c2021.
001440893 300__ $$a1 online resource
001440893 336__ $$atext$$btxt$$2rdacontent
001440893 337__ $$acomputer$$bc$$2rdamedia
001440893 338__ $$aonline resource$$bcr$$2rdacarrier
001440893 347__ $$atext file
001440893 347__ $$bPDF
001440893 500__ $$aIncludes index.
001440893 5050_ $$aIntroduction to Rare Diseases and Market Overview -- The Patient Perspective -- Select Patient Narratives -- The Caregiver Perspective -- The Critical, Multidimensional Role of Patient Advocacy Groups in Rare Disease -- A Mental Health Perspective -- Investment Decisions Related to Rare Disease Drug Development -- Optimizing Rare Disease Registries and Natural History Studies -- Novel Approaches to Clinical Trials in Rare Diseases -- Patient Benefits from Innovative Designs in Rare Diseases -- Central Nervous System Rare Disease Drug Development -- Oncologic Rare Disease Drug Development -- Hematologic Rare Disease Drug Development -- Lessons From Rare Disease and Gene Therapy Clinical Studies in Ophthalmology -- Rare Diseases in the Pediatric Population -- Cell and Gene Therapy in Rare Diseases -- The Feasibility Assessment -- The Evolving Regulatory Space and the Advent of Patient-Focused Drug Development -- Operational Aspects of Rare Disease Drug Development -- Accelerating Rare Disease Drug Development -- Select Rare Disease Drug Approvals: Lessons Learned -- A Rapid Market Access Strategy for Orphan Medicinal Products (OMPs) with Highlights Regarding the Pricing and Reimbursement Process and Barriers to Patient Use -- Integrated Life Cycle Management for Rare and Orphan Products -- The Case for Real-World Data and Real-World Evidence Generation in Rare and Orphan Medicinal Drug Development -- Closing Remarks --
001440893 506__ $$aAccess limited to authorized users.
001440893 520__ $$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.
001440893 588__ $$aOnline resource; title from PDF title page (SpringerLink, viewed November 23, 2021).
001440893 650_0 $$aDrug development.
001440893 650_0 $$aRare diseases$$xChemotherapy.
001440893 650_6 $$aMédicaments$$xDéveloppement.
001440893 650_6 $$aMaladies rares$$xChimiothérapie.
001440893 655_0 $$aElectronic books.
001440893 7001_ $$aHuml, Raymond A.,$$eeditor.
001440893 77608 $$iPrint version:$$tRare disease drug development.$$dCham, Switzerland : Springer, 2021$$z3030786048$$z9783030786045$$w(OCoLC)1250512598
001440893 852__ $$bebk
001440893 85640 $$3Springer Nature$$uhttps://univsouthin.idm.oclc.org/login?url=https://link.springer.com/10.1007/978-3-030-78605-2$$zOnline Access$$91397441.1
001440893 909CO $$ooai:library.usi.edu:1440893$$pGLOBAL_SET
001440893 980__ $$aBIB
001440893 980__ $$aEBOOK
001440893 982__ $$aEbook
001440893 983__ $$aOnline
001440893 994__ $$a92$$bISE