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Rare Disease Drug Development : Clinical, Scientific, Patient, and Caregiver Perspectives / / edited by Raymond A. Huml
| Rare Disease Drug Development : Clinical, Scientific, Patient, and Caregiver Perspectives / / edited by Raymond A. Huml |
| Edizione | [1st ed. 2021.] |
| Pubbl/distr/stampa | Cham : , : Springer International Publishing : , : Imprint : Springer, , 2021 |
| Descrizione fisica | 1 online resource (417 pages) |
| Disciplina | 615.19 |
| Soggetto topico |
Medicine
Clinical medicine - Research Pharmacology Immunology Therapeutics Pharmacovigilance Clinical Medicine Clinical Research Drug Safety and Pharmacovigilance Malalties rares Desenvolupament de medicaments Quimioteràpia |
| Soggetto genere / forma | Llibres electrònics |
| ISBN | 3-030-78605-6 |
| Formato | Materiale a stampa  |
| Livello bibliografico | Monografia |
| Lingua di pubblicazione | eng |
| Nota di contenuto | Introduction 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 -- . |
| Record Nr. | UNINA-9910508447303321 |
| Cham : , : Springer International Publishing : , : Imprint : Springer, , 2021 | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
Translational science of rare diseases
| Translational science of rare diseases |
| Pubbl/distr/stampa | Amsterdam, : IOS Press, [2016]- |
| Descrizione fisica | Online-Ressource |
| Disciplina | 610 |
| Soggetto topico | Malalties rares |
| Soggetto genere / forma |
Revistes electròniques.
Zeitschrift |
| ISSN | 2214-6512 |
| Formato | Materiale a stampa |
| Livello bibliografico | Periodico |
| Lingua di pubblicazione | eng |
| Record Nr. | UNINA-9910894438903321 |
| Amsterdam, : IOS Press, [2016]- | ||
| Materiale a stampa | ||
| Lo trovi qui: Univ. Federico II | ||
| ||
