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200 10$aRare Disease Drug Development $eClinical, Scientific, Patient, and Caregiver Perspectives /$fedited by Raymond A. Huml
205   $a1st ed. 2021.
210  1$aCham :$cSpringer International Publishing :$cImprint: Springer,$d2021.
215   $a1 online resource (417 pages)
311 08$aPrint version: Huml, Raymond A. Rare Disease Drug Development Cham : Springer International Publishing AG,c2021 9783030786045 
327   $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 -- .
330   $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.
606   $aMedicine
606   $aClinical medicine$xResearch
606   $aPharmacology
606   $aImmunology
606   $aTherapeutics
606   $aPharmacovigilance
606   $aClinical Medicine
606   $aClinical Research
606   $aPharmacology
606   $aImmunology
606   $aTherapeutics
606   $aDrug Safety and Pharmacovigilance
606   $aMalalties rares$2thub
606   $aDesenvolupament de medicaments$2thub
606   $aQuimioteràpia$2thub
608   $aLlibres electrònics$2thub
615  0$aMedicine.
615  0$aClinical medicine$xResearch.
615  0$aPharmacology.
615  0$aImmunology.
615  0$aTherapeutics.
615  0$aPharmacovigilance.
615 14$aClinical Medicine.
615 24$aClinical Research.
615 24$aPharmacology.
615 24$aImmunology.
615 24$aTherapeutics.
615 24$aDrug Safety and Pharmacovigilance.
615  7$aMalalties rares
615  7$aDesenvolupament de medicaments
615  7$aQuimioteràpia
676   $a615.19
702   $aHuml$b Raymond A.
801  0$bMiAaPQ
801  1$bMiAaPQ
801  2$bMiAaPQ
906   $aBOOK
912   $a9910508447303321
996   $aRare Disease Drug Development$92587208
997   $aUNINA